113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 646 / Drugs : 471 - (DrugBank : 105) / Drug target genes : 59 - Drug target pathways : 170
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT05532813 (ClinicalTrials.gov) | June 2023 | 6/9/2022 | Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease) | Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease). A Phase III, Prospective, Multicentre, Randomized, Double-blind Controlled Study | Steinert's Disease;Myotonic Dystrophy 1;Metformin | Drug: Treatment taken | Assistance Publique - Hôpitaux de Paris | NULL | Not yet recruiting | 18 Years | 70 Years | All | 142 | Phase 3 | France |
| 2 | NCT05689164 (ClinicalTrials.gov) | April 14, 2023 | 9/1/2023 | A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy. | Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study | Duchenne Muscular Dystrophy | Biological: fordadistrogene movaparvovec | Pfizer | NULL | Not yet recruiting | 0 Years | N/A | Male | 250 | Phase 3 | NULL |
| 3 | NCT05230459 (ClinicalTrials.gov) | March 12, 2023 | 27/1/2022 | A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) | A Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Safety of LION-101 Gene Therapy in Adult Subjects (18-65 Years) With Genetic Confirmation of Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) - Part 1 | Limb Girdle Muscular Dystrophy;Limb-Girdle Muscular Dystrophy Type 2;LGMD2I;Muscular Dystrophy;LGMD2;LGMD;FKRP;FKRP Mutation;Fukutin Related Protein | Genetic: LION-101 dose level 1;Genetic: LION-101 dose level 2;Other: Placebo | Asklepios Biopharmaceutical, Inc. | NULL | Recruiting | 18 Years | 65 Years | All | 10 | Phase 1/Phase 2 | United States |
| 4 | NCT05753462 (ClinicalTrials.gov) | March 2023 | 6/2/2023 | Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy | Phase 1/2a, Monocentric, Open Label Study to Evaluate the Safety, PK and PD of SQY51 in Paediatric and Adult Patients With a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Phase 1, SQY51;Drug: Phase 2a, SQY51 (cohort 1);Drug: Phase 2a, SQY51 (cohort 2);Drug: Phase 2a, SQY51 (cohort 3) | Sqy Therapeutics | Biotrial | Recruiting | 6 Years | N/A | Male | 12 | Phase 1/Phase 2 | France |
| 5 | NCT05548556 (ClinicalTrials.gov) | February 7, 2023 | 16/9/2022 | A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy | A Phase II, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Evaluate the Pharmacodynamics, Safety, Tolerability, Pharmacokinetics, and Efficacy of RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy (FSHD) | Drug: Placebo;Drug: RO7204239 | Hoffmann-La Roche | NULL | Recruiting | 18 Years | 65 Years | All | 48 | Phase 2 | Denmark |
| 6 | NCT05747924 (ClinicalTrials.gov) | February 2023 | 17/2/2023 | Phase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) | A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) | Facio-Scapulo-Humeral Dystrophy;Atrophy, Facioscapulohumeral;Atrophies, Facioscapulohumeral;Facioscapulohumeral Atrophy;Muscular Dystrophies;Muscular Dystrophy, Facioscapulohumeral;FSH Muscular Dystrophy;Landouzy Dejerine Dystrophy;Landouzy-Dejerine Muscular Dystrophy;Dystrophies, Landouzy-Dejerine;Dystrophy, Landouzy-Dejerine;Landouzy-Dejerine Syndrome;Muscular Dystrophy, Landouzy Dejerine;Progressive Muscular Dystrophy;FSH;Facioscapulohumeral Muscular Dystrophy 2;FSHD;FSHD1;FSHD2;FMD;FMD2;Fascioscapulohumeral Muscular Dystrophy;Fascioscapulohumeral Muscular Dystrophy Type 1;Fascioscapulohumeral Muscular Dystrophy Type 2;Dystrophies, Facioscapulohumeral Muscular;Dystrophy, Facioscapulohumeral Muscular;Facioscapulohumeral Muscular Dystrophy 1 | Drug: AOC 1020;Drug: Placebo | Avidity Biosciences, Inc. | NULL | Not yet recruiting | 18 Years | 65 Years | All | 72 | Phase 1/Phase 2 | NULL |
| 7 | NCT05693142 (ClinicalTrials.gov) | January 4, 2023 | 4/1/2023 | AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) | A Phase 1/2 Open-label, Dose Escalation and Dose Expansion Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Genetic: RGX-202 | REGENXBIO Inc. | NULL | Recruiting | 4 Years | 11 Years | Male | 18 | Phase 1/Phase 2 | United States |
| 8 | NCT05670730 (ClinicalTrials.gov) | November 9, 2022 | 6/12/2022 | Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping | A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping | Duchenne Muscular Dystrophy;Exon 44 | Drug: AOC 1044;Drug: Placebo | Avidity Biosciences, Inc. | NULL | Recruiting | 7 Years | 45 Years | Male | 64 | Phase 1/Phase 2 | United States |
| 9 | NCT05558813 (ClinicalTrials.gov) | November 2022 | 15/4/2022 | Natural History of Duchenne Muscular Dystrophy Cardiomyopathy (DMD-CMP) | Prospective Cardiac Magnetic Resonance Imaging Study in Duchenne Muscular Dystrophy (DMD-CMP) | Duchenne Muscular Dystrophy | Diagnostic Test: Cardiac MRI;Biological: Blood assays | Assistance Publique - Hôpitaux de Paris | NULL | Not yet recruiting | 6 Years | N/A | Male | 150 | N/A | France |
| 10 | NCT05540860 (ClinicalTrials.gov) | October 24, 2022 | 6/9/2022 | A Study of EDG-5506 in Children With Duchenne Muscular Dystrophy | A 2-part Phase 2 Study of Safety, Pharmacokinetics and Biomarkers in Children With Duchenne Muscular Dystrophy Including a Randomized, Double-Blind, Placebo-Controlled Part A, Followed by an Open-Label Part B | Duchenne Muscular Dystrophy | Drug: EDG-5506 Dose 1;Drug: EDG-5506 Dose 2;Drug: EDG-5506 Dose 3;Drug: Placebo | Edgewise Therapeutics, Inc. | NULL | Recruiting | 4 Years | 9 Years | Male | 27 | Phase 2 | United States |
| 11 | NCT05481879 (ClinicalTrials.gov) | September 5, 2022 | 28/7/2022 | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1 | A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants With Myotonic Dystrophy Type 1 | Myotonic Dystrophy Type 1 (DM1) | Drug: DYNE-101;Drug: Placebo | Dyne Therapeutics | NULL | Recruiting | 18 Years | 49 Years | All | 64 | Phase 1/Phase 2 | France;Netherlands;New Zealand;United Kingdom |
| 12 | NCT05514249 (ClinicalTrials.gov) | August 31, 2022 | 11/8/2022 | Treatment of a Single Patient With CRD-TMH-001 | Treatment of a Single Patient With CRD-TMH-001 | Duchenne Muscular Dystrophy | Drug: CRD-TMH-001 | Cure Rare Disease, Inc | University of Massachusetts, Worcester | Active, not recruiting | 18 Years | 28 Years | Male | 1 | Phase 1 | United States |
| 13 | NCT05166109 (ClinicalTrials.gov) | August 19, 2022 | 8/12/2021 | A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD) | A Phase II Pilot Trial of Vamorolone vs. Placebo for the Treatment of Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Vamorolone;Drug: Placebo | ReveraGen BioPharma, Inc. | Santhera Pharmaceuticals | Recruiting | 18 Years | 64 Years | Male | 39 | Phase 2 | United States |
| 14 | NCT05524883 (ClinicalTrials.gov) | August 12, 2022 | 30/8/2022 | Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy (DMD) | Drug: DYNE-251;Drug: Placebo | Dyne Therapeutics | NULL | Recruiting | 4 Years | 16 Years | Male | 46 | Phase 1/Phase 2 | United States;Belgium;Spain |
| 15 | NCT05429372 (ClinicalTrials.gov) | August 8, 2022 | 8/10/2021 | Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy | A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY | Muscular Dystrophy, Duchenne | Genetic: PF-06939926 | Pfizer | NULL | Recruiting | 2 Years | 3 Years | Male | 10 | Phase 2 | United States;Australia |
| 16 | NCT05479981 (ClinicalTrials.gov) | August 4, 2022 | 27/7/2022 | Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients | A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients | DM1;Muscular Dystrophies;Myotonic Dystrophy;Myotonic Dystrophy 1;Myotonic Disorders;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, Inborn;Heredodegenerative Disorders, Nervous System;Neurodegenerative Diseases | Drug: Placebo;Drug: AOC 1001 | Avidity Biosciences, Inc. | NULL | Enrolling by invitation | 18 Years | 66 Years | All | 44 | Phase 2 | United States |
| 17 | EUCTR2022-000389-16-ES (EUCTR) | 14/07/2022 | 15/07/2022 | A Phase 3 study to determine the Efficacy and Safety of Losmapimod inTreating Patients with Facioscapulohumeral Muscular Dystrophy | A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients with Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Losmapimod Product Code: FTX-1821 INN or Proposed INN: Losmapimod | Fulcrum Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 230 | Phase 3 | France;United States;Canada;Spain;Denmark;Germany;Netherlands;United Kingdom;Italy | ||
| 18 | NCT05291091 (ClinicalTrials.gov) | July 6, 2022 | 14/3/2022 | Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (CANYON) | A Phase 2 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents With Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: EDG-5506 Dose 1;Drug: EDG-5506 Dose 2;Drug: EDG-5506 Dose 3;Drug: EDG-5506 Dose 4;Drug: EDG-5506 Dose 5;Drug: Placebo | Edgewise Therapeutics, Inc. | Medpace, Inc. | Recruiting | 12 Years | 50 Years | Male | 66 | Phase 2 | United States;Netherlands;United Kingdom |
| 19 | EUCTR2018-001762-42-SI (EUCTR) | 01/07/2022 | 05/05/2022 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 20 | EUCTR2015-002069-52-NL (EUCTR) | 01/07/2022 | 09/06/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Greece;Spain;Ireland;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Denmark;Bulgaria;Norway;Netherlands;Germany;Sweden | ||
| 21 | EUCTR2019-003374-91-BE (EUCTR) | 30/06/2022 | 15/10/2021 | Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: SRP-9001 INN or Proposed INN: Not available Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 3 | France;United States;Taiwan;Hong Kong;Spain;Belgium;Germany;United Kingdom;Italy;Japan | ||
| 22 | NCT05126758 (ClinicalTrials.gov) | June 22, 2022 | 4/11/2021 | A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy | Muscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neuromuscular Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn;Nervous System Diseases | Biological: CAP-1002;Drug: Placebo | Capricor Inc. | NULL | Recruiting | 10 Years | N/A | Male | 68 | Phase 3 | United States |
| 23 | NCT05397470 (ClinicalTrials.gov) | June 16, 2022 | 4/5/2022 | Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (Reach) | A Phase 3 Global, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) (Reach) | Facioscapulohumeral Muscular Dystrophy (FSHD) | Drug: Losmapimod;Drug: Placebo oral tablet | Fulcrum Therapeutics | NULL | Recruiting | 18 Years | 65 Years | All | 230 | Phase 3 | United States;Canada;Denmark;France;Germany;Italy;Netherlands;Spain;United Kingdom |
| 24 | EUCTR2018-001762-42-CZ (EUCTR) | 06/04/2022 | 22/12/2021 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 25 | EUCTR2018-001762-42-HU (EUCTR) | 28/03/2022 | 13/12/2021 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 26 | NCT05185622 (ClinicalTrials.gov) | March 21, 2022 | 9/11/2021 | A Study to Assess Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-Label, Multiple Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys Ages 2 to <4 Years and 7 to <18 Years With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone | ReveraGen BioPharma, Inc. | Santhera Pharmaceuticals | Recruiting | 2 Years | 17 Years | Male | 44 | Phase 2 | Canada |
| 27 | EUCTR2018-001762-42-NO (EUCTR) | 04/03/2022 | 02/12/2021 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 28 | EUCTR2019-003374-91-ES (EUCTR) | 21/02/2022 | 20/10/2021 | Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: SRP-9001 INN or Proposed INN: Not available Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 3 | France;United States;Taiwan;Hong Kong;Belgium;Spain;Germany;United Kingdom;Italy;Japan | ||
| 29 | EUCTR2021-004276-33-DK (EUCTR) | 11/02/2022 | 01/10/2021 | GNT0006 gene therapy trial in patients with limb-girdle muscular dystrophy | A phase 1-2 multicenter study (2 stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMD R9, formerly LGMD2I) | FKRP-related limb-girdle muscular dystrophy (LGMD R9) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rAAV9-hFKRPco_miR-208a Product Code: GNT0006 INN or Proposed INN: not yet available Other descriptive name: Adeno-associated virus serotype 9 carrying the human fukutin-related protein and target sequence of the miR-208a | Atamyo Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 39 | Phase 1;Phase 2 | France;United States;Canada;Denmark;Norway;Germany;United Kingdom;Sweden | ||
| 30 | EUCTR2018-001762-42-GR (EUCTR) | 01/02/2022 | 01/12/2021 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 31 | NCT05338099 (ClinicalTrials.gov) | January 18, 2022 | 6/1/2022 | Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) | Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients With Duchenne Muscular Dystrophy(DMD) | Duchenne Muscular Dystrophy | Drug: EN001 | ENCell | NULL | Completed | 2 Years | 18 Years | Male | 7 | Phase 1 | Korea, Republic of |
| 32 | NCT04336826 (ClinicalTrials.gov) | December 29, 2021 | 3/4/2020 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy | Nonsene Mutation Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Recruiting | 6 Months | 2 Years | Male | 10 | Phase 2 | United States |
| 33 | NCT05160415 (ClinicalTrials.gov) | December 28, 2021 | 2/12/2021 | A Study of EDG-5506 in Adult Males With Becker Muscular Dystrophy | A Phase 1b, Open-label Study of the Safety and Pharmacokinetics of EDG-5506 in Adults With Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: EDG-5506 | Edgewise Therapeutics, Inc. | Medpace, Inc. | Active, not recruiting | 18 Years | 55 Years | Male | 12 | Phase 1 | United States |
| 34 | NCT05195775 (ClinicalTrials.gov) | December 14, 2021 | 4/1/2022 | Tadalafil as Adjuvant Therapy for DMD | Tadalafil as an Adjuvant to Therapy for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Tadalafil | University of Florida | NULL | Recruiting | 7 Years | 13 Years | Male | 25 | Phase 2/Phase 3 | United States |
| 35 | EUCTR2021-000122-10-GR (EUCTR) | 25/11/2021 | 13/10/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 36 | NCT05027269 (ClinicalTrials.gov) | October 28, 2021 | 24/8/2021 | Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients | A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients | DM1;Myotonic Dystrophy 1;Myotonic Dystrophy;Myotonic Dystrophy Type 1 (DM1);Dystrophy Myotonic;Myotonic Disorders;Steinert Disease;Myotonic Muscular Dystrophy | Drug: AOC 1001;Drug: Placebo | Avidity Biosciences, Inc. | NULL | Active, not recruiting | 18 Years | 65 Years | All | 44 | Phase 1/Phase 2 | United States |
| 37 | NCT05096221 (ClinicalTrials.gov) | October 27, 2021 | 14/10/2021 | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy | Genetic: SRP-9001;Genetic: Placebo | Sarepta Therapeutics, Inc. | Hoffmann-La Roche | Active, not recruiting | 4 Years | 7 Years | Male | 126 | Phase 3 | United States;Belgium;Germany;Hong Kong;Italy;Japan;Spain;Taiwan;United Kingdom |
| 38 | EUCTR2019-002076-13-NO (EUCTR) | 08/09/2021 | 18/02/2021 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 39 | JPRN-jRCT2031210252 | 06/09/2021 | 18/08/2021 | A phase 1 study in patients with Fukuyama-type congenital muscular dystrophy | A multicenter phase 1 study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy | Fukuyama-type congenital muscular dystrophy | This study consists of the following 4 cohorts. The study will start with cohort 1 and only D-mannitol will be administered once during the premedication phase, followed by 12 simultaneous doses of NS-035 and D-mannitol once weekly during the treatment phase. The dose of D-mannitol was fixed at 500 mg / kg in all cohorts, and NS-035 was gradually increased from cohort 1 to cohort 4 (1.6 mg/kg, 6.0 mg/kg, 20 mg/kg and 40 mg/kg, respectively). | Toda Tatsushi | NULL | Recruiting | >= 5age old | <= 10age old | Both | 12 | Phase 1 | Japan |
| 40 | NCT04624750 (ClinicalTrials.gov) | September 3, 2021 | 20/10/2020 | Open Label Study in Adolescents and Children With Myotonic Disorders | An Open-label, Non-Comparative Study to Evaluate the Steady-State Pharmacokinetics, Safety, and Efficacy of Mexiletine in Adolescents and Children With Myotonic Disorders | Myotonic Dystrophy | Drug: Mexiletine | Lupin Ltd. | NULL | Recruiting | 6 Years | 18 Years | All | 14 | Phase 3 | France |
| 41 | ChiCTR2100050052 | 2021-09-01 | 2021-08-16 | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Observational study of Lonafarnib in Chinese patients with progeria and progeroid laminopathy | Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies | Experimental group:Treatment with Lonafarnib; | The Children's Hospital Zhejiang University School of Medicine | NULL | Pending | 1 | 99 | Both | Experimental group:25; | N/A | China |
| 42 | NCT05004129 (ClinicalTrials.gov) | August 23, 2021 | 5/8/2021 | Safety and Efficacy of Tideglusib in Congenital Myotonic Dystrophy | A 52-Week, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X) | Congenital Myotonic Dystrophy | Drug: Tideglusib | AMO Pharma Limited | NULL | Enrolling by invitation | 6 Years | 17 Years | All | 56 | Phase 2/Phase 3 | United States;Australia;Canada;New Zealand |
| 43 | EUCTR2019-000601-77-DE (EUCTR) | 17/08/2021 | 31/03/2020 | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 44 | EUCTR2020-003653-30-ES (EUCTR) | 26/07/2021 | 15/12/2021 | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;Italy;China | ||
| 45 | NCT04700046 (ClinicalTrials.gov) | July 6, 2021 | 5/1/2021 | Study to Investigate the Efficacy and Safety of Mexiletine in Patients With Myotonic Dystrophy Type 1 and Type 2 | A Randomized, Double-blind, Placebo-controlled, Multi-center Study to Investigate the Efficacy and Safety of Mexiletine During 26 Weeks of Treatment in Patients With Myotonic Dystrophy Type 1 and Type 2 [The MIND Study] | Myotonic Dystrophy Type 1 and Type 2 | Drug: Mexiletine 167 mg;Drug: Placebo | Lupin Ltd. | NULL | Not yet recruiting | 18 Years | N/A | All | 158 | Phase 3 | NULL |
| 46 | EUCTR2021-000122-10-NL (EUCTR) | 30/06/2021 | 08/04/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 47 | EUCTR2020-000699-39-NL (EUCTR) | 28/06/2021 | 09/02/2021 | Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected by Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) | Ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Phase 3 | United States;France;Canada;Spain;Belgium;Austria;Australia;Netherlands;United Kingdom;Italy | ||
| 48 | NCT04886518 (ClinicalTrials.gov) | June 28, 2021 | 10/5/2021 | Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 | A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1, Followed by an Open-Label Extension | Myotonic Dystrophy 1;Excessive Daytime Sleepiness | Drug: Pitolisant Oral Tablet;Drug: Placebo oral tablet | Harmony Biosciences, LLC | NULL | Recruiting | 18 Years | 65 Years | All | 78 | Phase 2 | United States;Canada |
| 49 | NCT05135663 (ClinicalTrials.gov) | June 23, 2021 | 5/10/2021 | Extension Study of NS-089/NCNP-02 in DMD | A Phase II, Open-Label, Extension Study of NS-089/NCNP-02 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: NS-089/NCNP-02 | Nippon Shinyaku Co., Ltd. | NULL | Active, not recruiting | N/A | N/A | Male | 6 | Phase 2 | Japan |
| 50 | EUCTR2019-002921-31-BE (EUCTR) | 18/06/2021 | 07/09/2020 | Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY | DUCHENNE MUSCULAR DYSTROPHY (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PF-06939926 Product Code: PF-06939926 INN or Proposed INN: PF-06939926 Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of | ||
| 51 | EUCTR2021-000122-10-ES (EUCTR) | 15/06/2021 | 30/07/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 52 | NCT04687020 (ClinicalTrials.gov) | June 10, 2021 | 16/12/2020 | Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) | Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502) | Duchenne Muscular Dystrophy | Drug: Viltolarsen | NS Pharma, Inc. | NULL | Active, not recruiting | N/A | N/A | Male | 9 | Phase 4 | United States;Canada |
| 53 | EUCTR2020-003653-30-IT (EUCTR) | 08/06/2021 | 08/06/2021 | This is an Open-label Study to evaluate the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys with Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls - Galactic-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: [NS-065/NCNP-01] INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 2 | United States;Spain;Turkey;Russian Federation;China;Italy | ||
| 54 | EUCTR2020-004901-29-PL (EUCTR) | 01/06/2021 | 20/01/2021 | Metoprolol Safety and Efficacy in the Prevention of Cardiomyopathy in Patients with Duchenne Muscular Dystrophy (DMD). | The efficacy and safety of Metoprolol as add-on treatment to standard of care in preventing cardiomyopathy in patients with Duchenne Muscular Dystrophy aged 8-16 years. A randomized, double-blind, placebo controlledstudy. - MeDMD | Duchenne muscular dystrophyCardiomyopathyTachycardia MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Betaloc ZOK 25, 23,75 mg INN or Proposed INN: Metoprolol Other descriptive name: METOPROLOL SUCCINATE Trade Name: Betaloc ZOK 100, 95 mg INN or Proposed INN: Metoprolol Other descriptive name: METOPROLOL SUCCINATE | Medical University of Gdansk | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 144 | Phase 3 | Poland | ||
| 55 | NCT05412394 (ClinicalTrials.gov) | April 30, 2021 | 2/2/2022 | Once Weekly Infant Corticosteroid Trial for DMD | Phase-2 Trial of 5mg/kg/Week Prednisolone in Young Boys With DMD | Duchenne Muscular Dystrophy | Drug: Prednisolone | Anne M. Connolly | Ann & Robert H Lurie Children's Hospital of Chicago;Children's Hospital Medical Center, Cincinnati | Recruiting | 1 Month | 30 Months | Male | 26 | Phase 4 | United States |
| 56 | EUCTR2020-000699-39-AT (EUCTR) | 26/04/2021 | 29/01/2021 | Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) | Ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Phase 3 | United States;France;Canada;Spain;Belgium;Australia;Austria;Netherlands;United Kingdom;Italy | ||
| 57 | EUCTR2021-000122-10-IT (EUCTR) | 26/04/2021 | 08/06/2021 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: [NS-065/NCNP-01] INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | ||
| 58 | EUCTR2020-000699-39-IT (EUCTR) | 19/04/2021 | 04/06/2021 | Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected by Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) - na | Ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: [FG-3019] INN or Proposed INN: PAMREVLUMAB | FIBROGEN | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Phase 3 | France;United States;Czechia;Canada;Spain;Belgium;Austria;Australia;Netherlands;United Kingdom;Switzerland;Italy | ||
| 59 | NCT04768062 (ClinicalTrials.gov) | April 13, 2021 | 19/2/2021 | Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Viltolarsen | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd. | Enrolling by invitation | N/A | N/A | Male | 74 | Phase 3 | Australia;Greece;Italy;Japan;Korea, Republic of;Netherlands;New Zealand;Russian Federation;Spain;Turkey |
| 60 | NCT04821063 (ClinicalTrials.gov) | April 13, 2021 | 26/3/2021 | Placebo-Corrected Effects of Therapeutic Dose (100 mg) and Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval | A Randomized, Partially Double-Blind, Four-Period, Four-Treatment, Crossover Study Investigating the Placebo-Corrected Effects of a Therapeutic Dose (100 mg) and a Supratherapeutic Dose (300 mg) of ITF2357 (Givinostat) and Moxifloxacin on QT/QTC Interval in Healthy Male and Female Subjects | Duchenne and Becker Muscular Dystrophy;Polycytemia Vera | Drug: ITF2357 10 mg/mL;Drug: Placebo;Drug: Moxifloxacin Hydrochloride | Italfarmaco | NULL | Completed | 18 Years | 55 Years | All | 31 | Phase 1 | Canada |
| 61 | EUCTR2019-002921-31-FR (EUCTR) | 15/03/2021 | 07/09/2020 | Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY | DUCHENNE MUSCULAR DYSTROPHY (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PF-06939926 Product Code: PF-06939926 INN or Proposed INN: PF-06939926 Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of | ||
| 62 | NCT03692312 (ClinicalTrials.gov) | March 3, 2021 | 16/3/2018 | Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM) | Congenital Myotonic Dystrophy | Drug: Tideglusib;Drug: Placebo | AMO Pharma Limited | NULL | Active, not recruiting | 6 Years | 16 Years | All | 56 | Phase 2/Phase 3 | United States;Australia;Canada;New Zealand;United Kingdom |
| 63 | NCT04184882 (ClinicalTrials.gov) | February 24, 2021 | 2/12/2019 | A Study to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Participants With Duchenne Muscular Dystrophy (DMD) | A Randomized, Double Blind, Placebo-Controlled Phase 1b Study With Open-Label Extension to Assess the Safety, Tolerability and Preliminary Efficacy of ASP0367 (MA-0211) in Pediatric Male Patients With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) | Drug: Bocidelpar;Drug: Placebo | Astellas Pharma Inc | NULL | Terminated | 8 Years | 16 Years | Male | 8 | Phase 1 | United States |
| 64 | NCT04800874 (ClinicalTrials.gov) | February 18, 2021 | 25/1/2021 | Open Label Phase 2 Study of BBP-418 in Patients With LGMD2I | An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003) | LGMD2I | Drug: BBP-418 | ML Bio Solutions, Inc. | NULL | Enrolling by invitation | 12 Years | 55 Years | All | 16 | Phase 2 | United States |
| 65 | EUCTR2020-000698-26-AT (EUCTR) | 02/02/2021 | 17/08/2020 | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 96 | Phase 3 | United States;Czechia;Spain;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands | ||
| 66 | EUCTR2020-000698-26-NL (EUCTR) | 20/01/2021 | 08/09/2020 | Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 90 | Phase 3 | United States;Czechia;Spain;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands | ||
| 67 | JPRN-jRCT2041200087 | 07/01/2021 | 25/12/2020 | An Open-label, Long-Term study of Ataluren in Nonsense Mutation Duchenne Muscular Dystrophy | An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD | Nonsense Mutation Duchenne Muscular Dystrophy | Subjects will receive ataluren tid as an oral suspension at respective morning, mid-day, and evening doses of 10 mg/kg, 10 mg/kg, and 20 mg/kg. | Penematsa Vinay | NULL | Recruiting | >= 5age old | Not applicable | Male | 25 | Phase 3 | China;Japan |
| 68 | EUCTR2020-000698-26-IT (EUCTR) | 28/12/2020 | 06/11/2020 | Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) - na | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: [FG-3019] INN or Proposed INN: PAMREVLUMAB | FIBROGEN | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 90 | Phase 3 | United States;Czechia;Slovakia;Spain;Turkey;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands | ||
| 69 | NCT04616807 (ClinicalTrials.gov) | December 17, 2020 | 23/10/2020 | An Observational Study in Adult Patients With Non-dystrophic Myotonic Disorders | An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders | Myotonic Dystrophy | Drug: Mexiletine | Lupin Ltd. | NULL | Active, not recruiting | 18 Years | N/A | All | 53 | France;Germany;United Kingdom | |
| 70 | EUCTR2020-000698-26-GB (EUCTR) | 16/12/2020 | 14/10/2020 | Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 90 | Phase 3 | United States;Slovakia;Spain;Turkey;Austria;Israel;United Kingdom;Italy;Switzerland;France;Czech Republic;Canada;Belgium;Australia;Netherlands | ||
| 71 | EUCTR2020-000698-26-CZ (EUCTR) | 15/12/2020 | 19/08/2020 | Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 92 | Phase 3 | France;United States;Czechia;Czech Republic;Belgium;Spain;Austria;Netherlands;United Kingdom;Italy;Switzerland;China | ||
| 72 | NCT04632940 (ClinicalTrials.gov) | December 11, 2020 | 12/11/2020 | Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Pamrevlumab;Drug: Placebo;Drug: Corticosteroids | FibroGen | NULL | Active, not recruiting | 6 Years | 11 Years | Male | 70 | Phase 3 | United States;Australia;Austria;Belgium;Canada;China;France;Italy;Netherlands;Spain;United Kingdom;Switzerland |
| 73 | EUCTR2015-002069-52-IE (EUCTR) | 11/12/2020 | 21/05/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 74 | EUCTR2019-002921-31-IT (EUCTR) | 04/12/2020 | 06/11/2020 | Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY - - | DUCHENNE MUSCULAR DYSTROPHY (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PF-06939926 Product Code: [PF-06939926] INN or Proposed INN: PF-06939926 | PFIZER INC | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of | ||
| 75 | EUCTR2017-004625-32-BG (EUCTR) | 03/12/2020 | 28/10/2020 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | ||
| 76 | EUCTR2020-002093-27-FR (EUCTR) | 30/11/2020 | 24/06/2020 | A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. The study will start with finding the proper treatment dose (part 1).After that, a comparative study versus placebo will start to assess the safety and the effectiveness of the proper dose of this therapy (part 2).In the end, a follow up period will continue to investigate the treatment safety and efficacy over longer time (part 3). | Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular DystrophyA phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys. - Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: rAAV8-hMD1 Product Code: GNT0004 INN or Proposed INN: To be determined Other descriptive name: Adeno-associated viral vector serotype 8 containing the human MD1 gene | Genethon | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 51 | Phase 1;Phase 2;Phase 3 | United States;France;Israel;United Kingdom | ||
| 77 | NCT04626674 (ClinicalTrials.gov) | November 23, 2020 | 6/11/2020 | A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) | An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR) | Muscular Dystrophy, Duchenne | Genetic: SRP-9001 | Sarepta Therapeutics, Inc. | Hoffmann-La Roche | Enrolling by invitation | 3 Years | N/A | Male | 46 | Phase 1 | United States |
| 78 | EUCTR2019-002076-13-GR (EUCTR) | 12/11/2020 | 24/09/2020 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 79 | NCT04634682 (ClinicalTrials.gov) | November 9, 2020 | 25/9/2020 | Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 | Assessing the Effect of the MYODM Food Supplement on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 | Myotonic Dystrophy 1 | Dietary Supplement: MYODM | Myogem Health Company, S.L. | NULL | Completed | 18 Years | N/A | All | 30 | N/A | Spain |
| 80 | NCT04281485 (ClinicalTrials.gov) | November 5, 2020 | 11/2/2020 | Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Genetic: PF-06939926;Other: Placebo | Pfizer | NULL | Recruiting | 4 Years | 7 Years | Male | 99 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Japan;Korea, Republic of;Russian Federation;Spain;Switzerland;Taiwan;United Kingdom |
| 81 | NCT04708314 (ClinicalTrials.gov) | October 31, 2020 | 21/9/2020 | An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy | An Open-Label Study to Evaluate the Safety of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Golodirsen 50 MG/1 ML Intravenous Solution [VYONDYS 53] | Rare Disease Research, LLC | Sarepta Therapeutics, Inc. | Terminated | 7 Years | N/A | Male | 2 | Phase 4 | United States |
| 82 | EUCTR2018-001762-42-DE (EUCTR) | 29/10/2020 | 01/10/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Poland;Belgium;Romania;Germany;Norway;New Zealand;Sweden | ||
| 83 | NCT03340675 (ClinicalTrials.gov) | October 19, 2020 | 3/11/2017 | Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study With an Open-Label Extension to Determine the Safety, Pharmacokinetics and Efficacy of Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy Cardiomyopathy;Cardiomyopathy, Dilated | Drug: Ifetroban;Drug: Placebo | Cumberland Pharmaceuticals | Vanderbilt University Medical Center | Recruiting | 7 Years | N/A | Male | 48 | Phase 2 | United States |
| 84 | EUCTR2019-002921-31-GB (EUCTR) | 29/09/2020 | 02/07/2020 | Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY | DUCHENNE MUSCULAR DYSTROPHY (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PF-06939926 Product Code: PF-06939926 INN or Proposed INN: PF-06939926 Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene | Pfizer Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Italy;Switzerland;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of | ||
| 85 | NCT04698551 (ClinicalTrials.gov) | September 1, 2020 | 1/9/2020 | NIPD on cffDNA for Triplet Repeat Diseases | Comparison of Two NGS Phasing Techniques of Parental Haplotypes for NIPD of Triplet Expansion Diseases | Myotonic Dystrophy 1;Huntington Disease;Fragile X Syndrome | Genetic: Non invasive prenatal diagnosis | University Hospital, Montpellier | Agence de La Biomédecine | Active, not recruiting | 18 Months | N/A | All | 36 | France | |
| 86 | NCT04371666 (ClinicalTrials.gov) | July 30, 2020 | 29/4/2020 | Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory DMD | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Subjects With Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Pamrevlumab;Drug: Placebo;Drug: Corticosteroids | FibroGen | NULL | Active, not recruiting | 12 Years | N/A | Male | 92 | Phase 3 | United States;Australia;Austria;Belgium;Canada;China;Czechia;France;Israel;Italy;Netherlands;Spain;Switzerland;United Kingdom |
| 87 | NCT04322357 (ClinicalTrials.gov) | July 30, 2020 | 24/3/2020 | Weekend Steroids and Exercise as Therapy for DMD | Impact and Interplay of Corticosteroid Regimen and Exercise Training on DMD Muscle Function | Duchenne Muscular Dystrophy (DMD) | Drug: Prednisone;Behavioral: In-home Exercise Training;Drug: Prednisone with daily edasalonexent;Drug: Prednisone plus exercise | University of Florida | U.S. Army Medical Research and Development Command;Catabasis Pharmaceuticals | Recruiting | 5 Years | 8 Years | Male | 89 | Phase 2 | United States |
| 88 | EUCTR2018-001762-42-NL (EUCTR) | 20/07/2020 | 21/07/2020 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne MuscularDystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Poland;Belgium;Romania;Germany;Norway;New Zealand;Sweden | ||
| 89 | NCT04428476 (ClinicalTrials.gov) | July 20, 2020 | 9/6/2020 | Open-label Extension of the HOPE-2 Trial | Open-Label Extension of the HOPE-2 Duchenne Muscular Dystrophy Trial | Duchenne Muscular Dystrophy | Biological: CAP-1002 | Capricor Inc. | NULL | Active, not recruiting | 10 Years | N/A | All | 13 | Phase 2 | United States |
| 90 | NCT04386304 (ClinicalTrials.gov) | July 13, 2020 | 1/5/2020 | Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy | A Phase 1, Open-label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of Orally Administered (+)-Epicatechin in Patients With Becker or Becker-like Muscular Dystrophy With Continued Ambulation Past 16 Years of Age | Becker Muscular Dystrophy | Drug: (+)-Epicatechin | Epirium Bio Inc. | NULL | Completed | 16 Years | 59 Years | Male | 22 | Phase 1 | United States |
| 91 | NCT03992430 (ClinicalTrials.gov) | July 13, 2020 | 18/6/2019 | A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON) | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Muscular Dystrophy, Duchenne | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Recruiting | 4 Years | 13 Years | Male | 154 | Phase 3 | United States;Canada;Colombia;Czechia;Denmark;France;Germany;Greece;Hungary;India;Ireland;Korea, Republic of;Mexico;Netherlands;New Zealand;Norway;Poland;Romania;Slovenia;Spain;Taiwan;Turkey;United Kingdom |
| 92 | EUCTR2019-004426-24-IT (EUCTR) | 06/07/2020 | 15/06/2021 | Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers. | Deflazacort TREATment in LMNA related congenital muscular dystrophy: study of clinical effectiveness and search for reliable biomarkers. - TREAT-LMNA | Congenital Muscolar distrophy LMNA related MedDRA version: 20.0;Level: LLT;Classification code 10003718;Term: Atrophy skeletal muscle;System Organ Class: 100000004859;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Deflazacort Product Code: [Deflazacort] INN or Proposed INN: deflazacort Trade Name: Lucen Product Name: Lucen Product Code: [-] INN or Proposed INN: esomeprazolo Other descriptive name: esomeprazole | AZIENDA OSPEDALIERO-UNIVERSITARIA PISANA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | Italy | ||
| 93 | NCT04461561 (ClinicalTrials.gov) | July 1, 2020 | 28/6/2020 | Using NPT to Evaluate Providing PPC as ELNEC-PPC WBT for Nurses | Using Normalization Process Theory to Evaluate Providing Pediatric Palliative Care at End-of-Life as Web-Based Training Intervention for Nurses: | Cancer;Cardiac Anomaly;Cystic Fibrosis;Muscular Dystrophy;HIV/AIDS;Batten's Disease;Cerebral Palsy | Genetic: end-of-life nursing education consortium-pediatric palliative care as web based-training plus usual care | Altoosi University College | Babylon University | Recruiting | 20 Years | 65 Years | All | 172 | N/A | Iraq |
| 94 | NCT04433234 (ClinicalTrials.gov) | June 30, 2020 | 12/6/2020 | Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy | A Phase II, Long-term, Extension Study of DS-5141b in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: DS-5141b | Daiichi Sankyo Co., Ltd. | NULL | Active, not recruiting | 5 Years | N/A | Male | 8 | Phase 2 | Japan |
| 95 | EUCTR2019-003563-22-IE (EUCTR) | 23/06/2020 | 21/01/2020 | A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy | An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT | Catabasis Pharmaceuticals Inc. | NULL | Not Recruiting | Female: no Male: yes | 140 | Phase 3 | United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden | ||
| 96 | EUCTR2017-004625-32-SE (EUCTR) | 17/06/2020 | 09/08/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | ||
| 97 | NCT03895528 (ClinicalTrials.gov) | June 8, 2020 | 27/3/2019 | Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy | A Treatment IND (Investigational New Drug) Protocol for EAP (Expanded Access Program) for the Use of Lonafarnib in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) or Progeroid Laminopathy | Progeria;HGPS | Drug: Lonafarnib | Eiger BioPharmaceuticals | NULL | Approved for marketing | 12 Months | N/A | All | NULL | ||
| 98 | ChiCTR2000032525 | 2020-05-01 | 2020-05-01 | Early intervention for subclinical myocardial prevention of ACEI and beta-blocker in patients with Duchenne muscular dystrophy: a prospective multicenter randomized controlled clinical trial | Early intervention for subclinical myocardial prevention of ACEI and beta-blocker in patients with Duchenne muscular dystrophy: a prospective multicenter randomized controlled clinical trial | Duchenne muscular dystrophy | Group A:Perindopril;Group B:metoprolol;Group C:Perindopril and metoprolol; | West China Second University Hospital, Sichuan University | NULL | Recruiting | 1 | 18 | Male | Group A:106;Group B:106;Group C:106; | Phase 4 | China |
| 99 | EUCTR2018-001762-42-SE (EUCTR) | 27/04/2020 | 26/09/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 152 | Phase 3 | United States;Taiwan;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Canada;Argentina;Belgium;Peru;Australia;Denmark;Netherlands;Germany;New Zealand;Sweden;Korea, Republic of | ||
| 100 | EUCTR2019-003563-22-GB (EUCTR) | 23/04/2020 | 13/01/2020 | A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy | An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy - An Open-Label Extension Study of Edasalonexent in DMD Patients | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT | Catabasis Pharmaceuticals Inc. | NULL | Not Recruiting | Female: no Male: yes | 140 | Phase 3 | United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden | ||
| 101 | EUCTR2019-003563-22-DE (EUCTR) | 16/04/2020 | 31/01/2020 | A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy | An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT | Catabasis Pharmaceuticals Inc. | NULL | Not Recruiting | Female: no Male: yes | 140 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | ||
| 102 | EUCTR2019-003563-22-SE (EUCTR) | 15/04/2020 | 03/02/2020 | A study to provide Edasalonexent to Pediatric Patients with Duchenne Muscular Dystrophy | An Open-Label Extension Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT | Catabasis Pharmaceuticals Inc. | NULL | Not Recruiting | Female: no Male: yes | 140 | Phase 3 | United States;Canada;Ireland;Australia;Germany;United Kingdom;Sweden | ||
| 103 | NCT04060199 (ClinicalTrials.gov) | April 14, 2020 | 15/8/2019 | Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Viltolarsen;Drug: Placebo | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd. | Recruiting | 4 Years | 7 Years | Male | 74 | Phase 3 | United States;Australia;Canada;Chile;Greece;Hong Kong;Italy;Japan;Korea, Republic of;Mexico;Netherlands;New Zealand;Norway;Russian Federation;Spain;Taiwan;Turkey;Ukraine;United Kingdom;Sweden |
| 104 | EUCTR2019-000601-77-NL (EUCTR) | 10/04/2020 | 25/11/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy | ||
| 105 | EUCTR2015-002069-52-NO (EUCTR) | 07/04/2020 | 30/09/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Germany;Norway;Sweden | ||
| 106 | EUCTR2018-001762-42-IE (EUCTR) | 03/04/2020 | 01/07/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 107 | EUCTR2019-004602-94-BE (EUCTR) | 27/03/2020 | 03/02/2020 | NA | A 3-part, Randomized, Double Blind, Adaptive Seamless Phase 1-3 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO101 in Non-Ambulatory Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy and Evidence of Respiratory Deterioration - Adaptive Seamless Phase 1-3 Study of Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO | Duchenne Muscular Dystrophy regardless the genotype MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: BIO101 INN or Proposed INN: BIO101 Other descriptive name: BIO101 Product Code: BIO101 INN or Proposed INN: BIO101 Other descriptive name: BIO101 Product Code: BIO101 INN or Proposed INN: BIO101 Other descriptive name: BIO101 | Biophytis | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 1 | Belgium | ||
| 108 | EUCTR2017-004554-42-FR (EUCTR) | 26/03/2020 | 17/08/2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 100 | Phase 3 | France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | ||
| 109 | EUCTR2016-000602-10-BG (EUCTR) | 10/03/2020 | 07/01/2020 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 110 | EUCTR2019-002076-13-NL (EUCTR) | 03/03/2020 | 25/11/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Hong Kong;Taiwan;Greece;Spain;Ukraine;Turkey;Chile;Russian Federation;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Sweden;Korea, Republic of | ||
| 111 | EUCTR2019-002076-13-GB (EUCTR) | 02/03/2020 | 16/09/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 112 | NCT04179409 (ClinicalTrials.gov) | February 18, 2020 | 12/8/2019 | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of Casimersen, Eteplirsen and Golodirsen in Subjects With Duchenne Muscular Dystrophy Carrying Eligible DMD Duplications | Duchenne Muscular Dystrophy | Drug: Casimersen;Drug: Eteplirsen;Drug: Golodirsen | Kevin Flanigan | Sarepta Therapeutics, Inc. | Active, not recruiting | 6 Months | N/A | Male | 3 | Phase 2 | United States |
| 113 | NCT04264442 (ClinicalTrials.gov) | February 13, 2020 | 30/1/2020 | Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE) | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE) | Facioscapulohumeral Muscular Dystrophy (FSHD) | Drug: Losmapimod | Fulcrum Therapeutics | NULL | Active, not recruiting | 18 Years | 65 Years | All | 76 | Phase 2 | United States;Canada;France;Spain;Germany |
| 114 | EUCTR2019-002076-13-IT (EUCTR) | 27/01/2020 | 05/01/2021 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: [NS-065/NCNP-01] INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Greece;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Brazil;Belgium;Australia;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 115 | EUCTR2017-004279-30-NL (EUCTR) | 23/01/2020 | 16/05/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Germany;Netherlands;United Kingdom;Switzerland;Italy;Sweden | ||
| 116 | EUCTR2017-000397-10-GB (EUCTR) | 20/01/2020 | 15/03/2019 | Study in which all the patients take the same investigational drug with theaim of evaluate the long-term safety, tolerability, and efficacy ofGIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophywho have been already treated in one of the GIVINOSTAT studies in thepast | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies. - Givinostat DMD long term study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GIVINOSTAT (hyrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 185 | Phase 3 | Canada;Netherlands;Belgium;United States;United Kingdom;Italy;Germany;Spain;France | ||
| 117 | NCT04240314 (ClinicalTrials.gov) | January 15, 2020 | 22/1/2020 | AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications. | Phase I/IIa Systemic Gene Delivery Clinical Trial of scAAV9.U7.ACCA for Exon 2 Duplication-Associated Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: scAAV9.U7.ACCA | Megan Waldrop | Audentes Therapeutics | Active, not recruiting | 6 Months | 13 Years | Male | 3 | Phase 1/Phase 2 | United States |
| 118 | EUCTR2019-000601-77-ES (EUCTR) | 15/01/2020 | 05/07/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | United States;Canada;Belgium;Spain;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 119 | EUCTR2018-001762-42-DK (EUCTR) | 18/12/2019 | 30/10/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 154 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Colombia;Italy;Switzerland;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;Slovenia;Turkey;United Kingdom;Hungary;Mexico;Canada;Belgium;Poland;Romania;Germany;Norway;New Zealand;Sweden | ||
| 120 | EUCTR2019-002076-13-ES (EUCTR) | 16/12/2019 | 06/11/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 74 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Taiwan;Spain;Turkey;Russian Federation;Chile;United Kingdom;Italy;France;Canada;Poland;Belgium;Brazil;Australia;Netherlands;Japan;Sweden;Korea, Republic of | ||
| 121 | EUCTR2019-000601-77-IE (EUCTR) | 11/12/2019 | 26/06/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;United Kingdom;Italy | ||
| 122 | NCT04129294 (ClinicalTrials.gov) | December 2, 2019 | 15/10/2019 | Exploratory Study of NS-089/NCNP-02 in DMD | Exploratory Study of NS-089/NCNP-02 in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: NS-089/NCNP-02 | National Center of Neurology and Psychiatry, Japan | Nippon Shinyaku Co., Ltd. | Completed | 4 Years | 17 Years | Male | 6 | Phase 1/Phase 2 | Japan |
| 123 | EUCTR2015-002069-52-GR (EUCTR) | 29/11/2019 | 11/09/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 124 | EUCTR2015-002069-52-DK (EUCTR) | 26/11/2019 | 25/07/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;United States;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 125 | NCT02878694 (ClinicalTrials.gov) | November 14, 2019 | 8/3/2016 | Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft | Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft | Muscular Dystrophy, Oculopharyngeal;Ptosis | Biological: Myoblast autologous graft | University Hospital, Caen | University Hospital, Rouen;Rennes University Hospital;Centre Hospitalier Universitaire, Amiens;University Hospital, Lille;University Hospital, Brest;Groupe Hospitalier Pitie-Salpetriere | Terminated | 18 Years | 75 Years | All | 1 | Phase 2/Phase 3 | France |
| 126 | EUCTR2019-000601-77-GB (EUCTR) | 04/11/2019 | 12/07/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Netherlands;Germany;Italy;United Kingdom | ||
| 127 | NCT03863119 (ClinicalTrials.gov) | November 1, 2019 | 21/2/2019 | Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy | An Open-Label, Expanded Access Protocol for Boys With Duchenne Muscular Dystrophy Who Have Completed the Long-Term Extension (VBP15-LTE) or VBP15-004 Studies | Duchenne Muscular Dystrophy | Drug: Vamorolone | ReveraGen BioPharma, Inc. | NULL | Available | N/A | N/A | Male | United States;Canada;Israel | ||
| 128 | EUCTR2018-001762-42-ES (EUCTR) | 29/10/2019 | 09/08/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 152 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;Korea, Republic of | ||
| 129 | EUCTR2018-004740-36-DK (EUCTR) | 29/10/2019 | 25/06/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;France;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 130 | EUCTR2019-001181-15-ES (EUCTR) | 29/10/2019 | 29/10/2019 | Safety and Efficacy of Losmapimod in Patients with FSHD | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Losmapimod Product Code: FTX-1821 INN or Proposed INN: LOSMAPIMOD | Fulcrum Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Spain;Germany | ||
| 131 | EUCTR2018-004740-36-DE (EUCTR) | 14/10/2019 | 29/04/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | France;United States;Canada;Denmark;Russian Federation;Norway;Germany;Sweden | ||
| 132 | EUCTR2017-002704-27-NL (EUCTR) | 10/10/2019 | 25/02/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 120 | Phase 2 | United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden | ||
| 133 | EUCTR2017-000397-10-NL (EUCTR) | 01/10/2019 | 06/11/2019 | Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | France;United States;Serbia;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy | ||
| 134 | EUCTR2018-004009-22-CZ (EUCTR) | 25/09/2019 | 26/06/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | United States;Japan;United Kingdom;Spain;Canada;Czech Republic;Netherlands;Sweden;Turkey;Belgium;Ireland;Poland;Italy;Australia;France;Germany | ||
| 135 | EUCTR2019-001825-28-GB (EUCTR) | 24/09/2019 | 29/07/2019 | Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy: a phase I/IIa study / DMD04 | Mesoangioblast-mediated exon 51 skipping for genetic correction of dystrophin, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non randomized, open label, phase I/IIa study. - Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Lentiviral Vector Trans-Skip gene modified Autologous Mesoangioblasts | The University of Manchester | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 136 | EUCTR2019-001181-15-FR (EUCTR) | 17/09/2019 | 29/05/2019 | Safety and Efficacy of Losmapimod in Patients with FSHD | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Losmapimod Product Code: FTX-1821 INN or Proposed INN: LOSMAPIMOD | Fulcrum Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 66 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Spain;Germany | ||
| 137 | EUCTR2018-001762-42-GB (EUCTR) | 17/09/2019 | 04/07/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 122 | Phase 3 | United States;Czechia;Taiwan;Spain;Ireland;Turkey;Russian Federation;Colombia;Switzerland;United Kingdom;France;Mexico;Canada;Poland;Belgium;Australia;Denmark;Norway;Germany;Netherlands;New Zealand;Sweden;Korea, Republic of | ||
| 138 | NCT03907072 (ClinicalTrials.gov) | September 4, 2019 | 5/4/2019 | Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy (DYSTANCE 51) | Duchenne Muscular Dystrophy | Drug: WVE-210201 (suvodirsen);Drug: Placebo | Wave Life Sciences Ltd. | NULL | Terminated | 5 Years | 12 Years | Male | 6 | Phase 2/Phase 3 | United States;Belgium;Canada;Czechia;France;Italy;Sweden;United Kingdom |
| 139 | NCT04004000 (ClinicalTrials.gov) | August 23, 2019 | 25/6/2019 | Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension | An Open-Label Pilot Study of Losmapimod to Evaluate the Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments in Subjects With Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) With Extension | Facioscapulohumeral Muscular Dystrophy 1 | Drug: Losmapimod | Fulcrum Therapeutics | NULL | Active, not recruiting | 18 Years | 65 Years | All | 14 | Phase 2 | Netherlands |
| 140 | EUCTR2018-004740-36-FR (EUCTR) | 19/08/2019 | 04/06/2019 | A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I | A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I | Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0;Level: PT;Classification code 10028356;Term: Muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) INN or Proposed INN: Deflazacort Other descriptive name: DEFLAZACORT | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 3 | United States;France;Canada;Denmark;Norway;Germany;Sweden | ||
| 141 | EUCTR2017-002704-27-ES (EUCTR) | 13/08/2019 | 11/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 142 | NCT04003974 (ClinicalTrials.gov) | August 9, 2019 | 25/6/2019 | Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy (FSHD) | Drug: Losmapimod;Drug: Placebo oral tablet | Fulcrum Therapeutics | NULL | Completed | 18 Years | 65 Years | All | 80 | Phase 2 | United States;Canada;France;Spain;Germany |
| 143 | EUCTR2017-002704-27-GR (EUCTR) | 01/08/2019 | 10/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- andActive-controlled Study with Double-Blind Extension to Assess the Efficacyand Safety of Vamorolone in Ambulant Boys with Duchenne MuscularDystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc | NULL | Not Recruiting | Female: no Male: yes | 120 | Phase 2 | Czech Republic;Canada;Greece;Spain;Belgium;Australia;Israel;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 144 | EUCTR2018-004009-22-SE (EUCTR) | 20/07/2019 | 05/04/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden | ||
| 145 | EUCTR2018-000692-32-IT (EUCTR) | 17/07/2019 | 14/01/2020 | Trial to evaluate the efficacy of the drug metformin on the muscles in patients with myotonic distrophy type 1 | EFFICACY OF METFORMIN ON MOTILITY AND STRENGTH IN MYOTONIC DYSTROPHY TYPE 1. A randomized, double blind, placebo-controlled, multicenter clinical trial. - MetMyd | Myotonic Distrophy type 1, confirmed by genetic testing, with a CTG expansion size >100 MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Level: LLT;Classification code 10013987;Term: Dystrophia myotonica;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: METFORMINA - 500 30 COMPRESSE 500 MG Product Name: Metformina INN or Proposed INN: Metformina | DIP. MEDICINA DEI SISTEMI UNIVERSITà DEGLI STUDI DI ROMA TOR VERGATA | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 194 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Italy | ||
| 146 | EUCTR2018-004009-22-IT (EUCTR) | 09/07/2019 | 05/11/2020 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy - - | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: [WVE-210201] Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | United States;Czechia;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Netherlands;Japan;Sweden | ||
| 147 | EUCTR2017-002704-27-BE (EUCTR) | 06/07/2019 | 21/02/2018 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 120 | Phase 2 | United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 148 | NCT04054375 (ClinicalTrials.gov) | July 1, 2019 | 7/8/2019 | Weekly Steroids in Muscular Dystrophy | Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy | Limb-girdle Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Prednisone | Northwestern University | NULL | Active, not recruiting | 18 Years | 65 Years | All | 20 | Phase 2 | United States |
| 149 | EUCTR2019-000337-39-GB (EUCTR) | 28/06/2019 | 23/04/2019 | A research study of a new investigational medicinal product for thetreatment of Duchenne Muscular Dystrophy patients who have completedstudy 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen inPatients with Duchenne Muscular Dystrophy Who Have Completed Study4658-102 | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||
| 150 | EUCTR2018-004009-22-GB (EUCTR) | 27/06/2019 | 28/03/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden | ||
| 151 | NCT03985878 (ClinicalTrials.gov) | June 26, 2019 | 11/6/2019 | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 | Duchenne Muscular Dystrophy | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Terminated | 2 Years | 5 Years | Male | 15 | Phase 2 | Belgium;France;Italy;United Kingdom |
| 152 | NCT04004065 (ClinicalTrials.gov) | June 26, 2019 | 27/6/2019 | Two-Part Study for Dose Determination of SRP-5051 (Vesleteplirsen) (Part A), Then Dose Expansion (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | NULL | Recruiting | 7 Years | 21 Years | Male | 60 | Phase 2 | United States;Belgium;Canada;Germany;Italy;Netherlands;Spain;United Kingdom |
| 153 | EUCTR2017-002704-27-CZ (EUCTR) | 19/06/2019 | 22/03/2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 154 | NCT03959189 (ClinicalTrials.gov) | June 17, 2019 | 16/5/2019 | Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 | Double-Blind, Placebo-Controlled, Dose-Range-Finding, Crossover Trial of Single Day Administration of ERX-963 in Adults With Myotonic Dystrophy Type 1 | Myotonic Dystrophy, Type 1 (DM1);Myotonic Dystrophy | Drug: ERX-963;Drug: Placebo | Expansion Therapeutics, Inc. | NULL | Completed | 18 Years | 65 Years | All | 12 | Phase 1 | United States |
| 155 | EUCTR2019-000337-39-IT (EUCTR) | 13/06/2019 | 01/02/2021 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 - Not applicable | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: [AVI-4658] INN or Proposed INN: ETEPLIRSEN | SAREPTA THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom;Italy | ||
| 156 | EUCTR2017-000397-10-BE (EUCTR) | 12/06/2019 | 12/02/2019 | Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 185 | Phase 3 | United States;France;Canada;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy | ||
| 157 | EUCTR2018-004009-22-BE (EUCTR) | 07/06/2019 | 21/05/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Netherlands;Japan;Sweden | ||
| 158 | NCT03789734 (ClinicalTrials.gov) | June 4, 2019 | 12/12/2018 | Safety Study of BLS-M22 in Healthy Volunteers | A Dose Block-randomized, Double-blind, Placebo-controlled and Dose-escalation Phase I Clinical Trial to Evaluate Safety of BLS-M22 Following Single/Multiple Oral Administration in Healthy Adult Volunteers | Muscular Dystrophy, Duchenne | Biological: BLS-M22;Other: Placebo | BioLeaders Corporation | NULL | Completed | 19 Years | 55 Years | All | 37 | Phase 1 | Korea, Republic of |
| 159 | EUCTR2019-000337-39-BE (EUCTR) | 03/06/2019 | 08/04/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;United Kingdom | ||
| 160 | EUCTR2017-000397-10-ES (EUCTR) | 16/05/2019 | 11/04/2019 | Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD study long term | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 185 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom | ||
| 161 | NCT03783923 (ClinicalTrials.gov) | May 15, 2019 | 19/12/2018 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | A Multicenter Open Label Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | Limb-Girdle Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 18 Years | N/A | All | 30 | Phase 3 | United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden |
| 162 | EUCTR2019-000305-79-ES (EUCTR) | 14/05/2019 | 12/04/2019 | An extension study to the A083-02 and A083-03 studies that evaluate the long-term effect of the ACE-083 investigational product in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) previously participated in study A083-02 and also in patients with Charcot-Marie Tooth (CMT) disease type 1 and X who previously participated in study A083-03. | An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients with Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03 | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ACE-083 Product Code: ACE-083 INN or Proposed INN: pending Other descriptive name: ACE-083 | Acceleron Pharma Inc. | NULL | Not Recruiting | Female: yes Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain | ||
| 163 | NCT03943290 (ClinicalTrials.gov) | May 10, 2019 | 17/4/2019 | Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX) | An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) Previously Enrolled in Study A083-02 and in Patients With Charcot-Marie Tooth (CMT) Disease Types 1 and X Previously Enrolled in Study A083-03 | Facioscapulohumeral Muscular Dystrophy;Charcot-Marie-Tooth Disease | Drug: ACE-083 | Acceleron Pharma, Inc. | NULL | Terminated | 18 Years | N/A | All | 62 | Phase 2 | United States;Canada;Spain |
| 164 | NCT03936894 (ClinicalTrials.gov) | May 1, 2019 | 29/4/2019 | Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy | A Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Canakinumab Injection [Ilaris] | Children's National Research Institute | Foundation to Eradicate Duchenne | Recruiting | 2 Years | N/A | Male | 6 | Phase 1/Phase 2 | United States |
| 165 | NCT03796637 (ClinicalTrials.gov) | April 11, 2019 | 4/1/2019 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren | Phase 2, Non-Interventional, Clinical Study to Assess Dystrophin Levels in Subjects With Nonsense Mutation Duchenne Muscular Dystrophy Who Have Been Treated With Ataluren for =9 Months | Duchenne Muscular Dystrophy | Other: Dystrophin levels;Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 6 | Phase 2 | United States |
| 166 | NCT04120168 (ClinicalTrials.gov) | April 1, 2019 | 7/10/2019 | Study Determining the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease | Multicenter Non-Drug Screening Study to Determine the Frequency of Duchenne Muscular Dystrophy and Late-onset Pompe Disease in Children With Unexplained Transaminase Elevation | Duchenne Muscular Dystrophy;Pompe Disease (Late-onset) | Genetic: Laboratory Tests | Turkish Society of Pediatric Gastroenterology, Hepatology and Nutrition | NULL | Completed | 3 Months | 18 Years | All | 590 | Turkey | |
| 167 | NCT03917719 (ClinicalTrials.gov) | March 14, 2019 | 12/4/2019 | An Open-Label Extension Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy | An Open-Label Extension Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Edasalonexent | Catabasis Pharmaceuticals | NULL | Terminated | 4 Years | 12 Years | Male | 130 | Phase 3 | United States;Australia;Canada;Germany;Sweden;United Kingdom |
| 168 | EUCTR2015-001192-48-FR (EUCTR) | 04/03/2019 | 11/09/2015 | Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes. | Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes. - TTT-PT-DOP | Patients atteints d’une DMOP et présentant un ptosis MedDRA version: 18.0;Level: LLT;Classification code 10010498;Term: Congenital hereditary muscular dystrophy;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Myoblastes autologues | CHU CAEN | NULL | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | France | ||
| 169 | NCT02592941 (ClinicalTrials.gov) | March 1, 2019 | 29/10/2015 | Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy | An Open Label, Expanded Access Protocol Intended to Provide Treatment With MP-104 (Deflazacort) to U.S. Children, Adolescents, and/or Adults With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | Parexel;Dohmen Life Science Services | Approved for marketing | 5 Years | N/A | All | United States | ||
| 170 | EUCTR2017-004279-30-AT (EUCTR) | 15/02/2019 | 04/01/2019 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 171 | EUCTR2018-000975-34-NL (EUCTR) | 06/02/2019 | 16/08/2018 | A Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 1 | United States;France;Canada;Belgium;Netherlands;United Kingdom;Italy | ||
| 172 | EUCTR2017-004625-32-FR (EUCTR) | 05/02/2019 | 23/07/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;France;Australia;Bulgaria;Germany | ||
| 173 | EUCTR2018-000464-29-DE (EUCTR) | 31/01/2019 | 24/09/2018 | A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: EDASALONEXENT | Catabasis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: no Male: yes | 126 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | ||
| 174 | EUCTR2017-004554-42-GB (EUCTR) | 29/01/2019 | 03/08/2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open labelextension of a multicentre, randomised, double-blind, placebo-controlled,phase 3 safety and efficacy trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 100 | Phase 3 | France;Belgium;Spain;Netherlands;Germany;Switzerland;United Kingdom | ||
| 175 | EUCTR2017-004554-42-ES (EUCTR) | 22/01/2019 | 17/09/2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMD A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: TAMOX INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | ||
| 176 | NCT03779646 (ClinicalTrials.gov) | January 16, 2019 | 14/12/2018 | Bisoprolol in DMD Early Cardiomyopathy | Bisoprolol for Early Cardiomyopathy in Duchenne Muscular Dystrophy: a Randomized, Controlled Trial | Duchenne Muscular Dystrophy;Cardiomyopathy, Dilated | Drug: Bisoprolol Fumarate | Peking Union Medical College Hospital | National Natural Science Foundation of China;Chinese Academy of Medical Sciences | Recruiting | 7 Years | N/A | Male | 42 | Phase 2/Phase 3 | China |
| 177 | EUCTR2018-000464-29-IE (EUCTR) | 16/01/2019 | 02/10/2018 | A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy. | A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OFEDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: Edasalonexent | Catabasis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: no Male: yes | 126 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | ||
| 178 | EUCTR2017-004554-42-NL (EUCTR) | 16/01/2019 | 02/10/2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: TAMOXIFEN INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 100 | Phase 3 | France;Belgium;Spain;Turkey;Germany;Netherlands;United Kingdom;Switzerland | ||
| 179 | EUCTR2017-002213-60-GB (EUCTR) | 08/01/2019 | 11/12/2018 | A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophy | A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SGT-001 Product Code: rAAV9-CK8-h-µD5 INN or Proposed INN: SGT-001 Other descriptive name: rAAV9-CK8-h-µD5 | Solid Biosciences Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 32 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 180 | NCT03648827 (ClinicalTrials.gov) | December 21, 2018 | 24/8/2018 | A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 7 Years | Male | 20 | Phase 2 | United States |
| 181 | EUCTR2017-004279-30-ES (EUCTR) | 21/12/2018 | 16/11/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Belgium;Spain;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 182 | EUCTR2017-004625-32-BE (EUCTR) | 20/12/2018 | 10/09/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
| 183 | JPRN-jRCTs031180038 | 18/12/2018 | 12/11/2018 | Tranilast-MD | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD | muscular dystrophy muscular dystrophy ,heart failure ,tranilast;muscular dystrophies | Tranilast 300 mg / day is administered three times per minute. Treatment for 28 weeks (in principle, outpatient administration). As of the 28th week, reconfirmation of consent regarding continuation of administration is confirmed, and if confirmation is obtained, further treatment for 116 weeks is carried out. | Matsumura Tsuyoshi | NULL | Not Recruiting | >= 13age old | Not applicable | Both | 20 | N/A | Japan |
| 184 | EUCTR2016-000602-10-HU (EUCTR) | 18/12/2018 | 05/11/2018 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 185 | JPRN-UMIN000031965 | 2018/12/14 | 30/03/2018 | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD | Heart failure patients with muscular dystrophy who showed brain natriuretic peptide (BNP) of 100 pg/mL or more in spite of myocardial protection treatment | Tranilast 300 mg/day is administered three times per day as the study treatment. The start date of study treatment is defined as the medication start date, and 28 weeks of study treatment will be performed (in principle, by outpatient administration). We reconfirm consent for continuation of administration at 28 weeks, and if consent is obtained, further treatment for 116 weeks will be carried out. As long as there are no particular problems, the outpatient visit during the observation period is in 4 weeks (21-35 days), 12 weeks (77-91 days), 20 weeks (134-147 days), 24 weeks (161-175 days), and 28 weeks (190-203 days) after starting medication. A prescription until the next outpatient visit will be issued at the consultation. The outpatient visit after consent reconfirmation is done at 12-weeks intervals after 36 weeks, and the prescription until the next outpatient visit will be issued until the next 144 weeks after starting medication at the consultation. Evaluation of various examination findings including clinical findings, cardiac functions, respiratory functions, motor function, QOL Questionnaire (MDQoL-60, SF-12), and adverse events at the designated timing. In order to confirm TRPV2 inhibitory effect by tranilast and to assess its effectiveness as a biomarker, central laboratory tests for the TRPV2 expression analysis will be performed. | National Hospital Organization Toneyama National Hospital | NULL | Complete: follow-up continuing | 13years-old | Not applicable | Male and Female | 20 | Not selected | Japan |
| 186 | EUCTR2015-002069-52-PL (EUCTR) | 11/12/2018 | 13/09/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Russian Federation;Hungary;United States;United Kingdom;Portugal;Spain;Greece;Canada;Czech Republic;Sweden;Netherlands;Belgium;Norway;Ireland;Poland;Denmark;Italy;Israel;Australia;Bulgaria;France;Serbia;Germany | ||
| 187 | EUCTR2018-000464-29-SE (EUCTR) | 11/12/2018 | 24/09/2018 | A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy. | A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OFEDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 INN or Proposed INN: Edasalonexent | Catabasis Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: no Male: yes | 126 | Phase 3 | Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden;United States | ||
| 188 | NCT03777319 (ClinicalTrials.gov) | December 5, 2018 | 10/12/2018 | Spironolactone Versus Prednisolone in DMD | A Randomized Open Label Trial of Spironolactone Versus Prednisolone in Corticosteroid-naïve Boys With DMD | Muscular Dystrophy, Duchenne | Drug: Spironolactone;Drug: Prednisolone | Kevin Flanigan | Muscular Dystrophy Association | Terminated | 4 Years | 7 Years | Male | 2 | Phase 1 | United States |
| 189 | EUCTR2016-004623-23-GB (EUCTR) | 19/11/2018 | 08/02/2018 | A Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy - Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy | Treatment of child and adolescent congenital myotonic dystrophy. MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib INN or Proposed INN: Tideglusib Other descriptive name: 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione | AMO Pharma Ltd | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 56 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Canada;United Kingdom | ||
| 190 | EUCTR2017-001629-41-NL (EUCTR) | 15/11/2018 | 31/05/2018 | Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy | A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy ofgivinostat in patients with Becker Muscular Dystrophy - NA | Distrofia Muscolare di Becker (DMB) MedDRA version: 20.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.P.A. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | Netherlands;Italy | ||
| 191 | EUCTR2018-000975-34-BE (EUCTR) | 13/11/2018 | 21/09/2018 | A Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 | A Multicenter, Open-Label Extension Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 1 | Canada;Netherlands;Belgium;United States;Italy;United Kingdom;France | ||
| 192 | NCT03642145 (ClinicalTrials.gov) | October 31, 2018 | 10/7/2018 | A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD) | A 52-Week Phase 3B Randomized Open-Label Study Evaluating the Safety and Pharmacokinetics of Emflaza® (Deflazacort) Compared to a Comparable Natural History Control Group in Males Aged =2 to <5 Years With Duchenne Muscular Dystrophy (DMD) Followed by a 52-Week Extension Period | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Withdrawn | 2 Years | 4 Years | Male | 0 | Phase 3 | United States |
| 193 | NCT03652259 (ClinicalTrials.gov) | October 27, 2018 | 28/8/2018 | Gene Delivery Clinical Trial of SRP-9003 (Bidridistrogene Xeboparvovec) for Participants With Limb-Girdle Muscular Dystrophy, Type 2E (LGMD2E) (Beta-Sarcoglycan Deficiency) | A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (ß-Sarcoglycan Deficiency) | Limb-Girdle Muscular Dystrophy, Type 2E | Genetic: SRP-9003 | Sarepta Therapeutics, Inc. | NULL | Active, not recruiting | 4 Years | 15 Years | All | 6 | Phase 1/Phase 2 | United States |
| 194 | EUCTR2017-004279-30-GB (EUCTR) | 26/10/2018 | 09/05/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;Switzerland;Italy;United Kingdom;Sweden | ||
| 195 | EUCTR2018-000975-34-GB (EUCTR) | 25/10/2018 | 26/06/2018 | A Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 | A Multicenter, Open-Label Extension Study of WVE-210201 in Patients Previously Enrolled in WVE-DMDX51-001 | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 1 | France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom | ||
| 196 | NCT03703882 (ClinicalTrials.gov) | October 2, 2018 | 8/10/2018 | Phase III Study of Edasalonexent in Boys With Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients With Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Edasalonexent;Drug: Placebo | Catabasis Pharmaceuticals | NULL | Completed | 4 Years | 7 Years | Male | 131 | Phase 3 | United States;Australia;Canada;Germany;Ireland;Israel;Sweden;United Kingdom |
| 197 | EUCTR2017-002704-27-GB (EUCTR) | 21/09/2018 | 25/06/2019 | A Study to Assess the efficacy and the safety of Vamorolone in comparison to Prednisone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 198 | ChiCTR1800018340 | 2018-09-12 | 2018-09-12 | Clinical study for cardiac MRI in evaluating early myocardial lesions in patients with muscular dystrophy | Clinical study for cardiac MRI in evaluating early myocardial lesions in patients with muscular dystrophy | Muscular dystrophy | Gold Standard:Genomic sequencing, muscle biopsy;Index test:Myocardial strain, T1/T2mapping value, late delayed enhancement (LGE), myocardial first pass perfusion; | Department of Radiology, West China Second Hospital, Sichuan University | NULL | Recruiting | 4 | 18 | Male | Target condition:200;Difficult condition:30 | China | |
| 199 | EUCTR2017-004625-32-ES (EUCTR) | 07/09/2018 | 10/09/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 150 | Phase 3 | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | ||
| 200 | EUCTR2017-004139-35-IT (EUCTR) | 31/08/2018 | 17/06/2021 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy - Clinical Trial in Male Subjects with Muscular Disease | Duchenne Muscular Distrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosintropina acetato Product Code: [MNK-1411] INN or Proposed INN: TETRACOSACTIDE ESACETATO Other descriptive name: Cosyntropin acetate Trade Name: Synacthen Ampoules 250 mcg Product Name: Synacthen Ampoules 250 microgrammi Product Code: [Synacthen Ampoules 250 microgrammi] INN or Proposed INN: TETRACOSACTIDE | MALLINCKRODT ARD INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 132 | Phase 2 | Serbia;United States;Spain;Costa Rica;Ukraine;Ireland;Turkey;Israel;Russian Federation;United Kingdom;Italy;India;Mexico;Canada;Puerto Rico;Poland;Bulgaria | ||
| 201 | EUCTR2017-004279-30-IT (EUCTR) | 29/08/2018 | 22/01/2021 | A clinical study to assess the long-term safety and efficacy of idebenonetreatment in patients with Duchenne Muscular Dystrophy (DMD) whocompleted the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safetyand Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy(DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone Product Code: [Idebenone] INN or Proposed INN: IDEBENONE | SANTHERA PHARMACEUTICALS | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | France;United States;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 202 | EUCTR2017-004139-35-BG (EUCTR) | 28/08/2018 | 28/06/2018 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Duchenne Muscular Distrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosyntropin acetate Product Code: MNK-1411 INN or Proposed INN: TETRACOSACTIDE HEXAACETATE Other descriptive name: Cosyntropin acetate | Mallinckrodt ARD Inc. | NULL | Not Recruiting | Female: no Male: yes | 132 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Serbia;United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Bulgaria;Germany;Netherlands | ||
| 203 | EUCTR2017-002704-27-SE (EUCTR) | 15/08/2018 | 12/03/2018 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: VAMOROLONE Product Name: Prednisone INN or Proposed INN: PREDNISONE Other descriptive name: PREDNISONE | ReveraGen BioPharma, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 2 | United States;Czechia;Greece;Spain;Israel;Italy;United Kingdom;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||
| 204 | NCT03558958 (ClinicalTrials.gov) | August 8, 2018 | 5/6/2018 | Safety and Efficacy of P-188 NF in DMD Patients | An Exploratory, Open-label Study to Assess the Effect of P-188 NF (Carmeseal-MD) on Safety, on Respiratory and Cardiac Dysfunction and on Upper Limb Strength in Non-ambulatory Patients With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: P-188 NF | Phrixus Pharmaceuticals, Inc. | Charley's Fund | Terminated | 12 Years | 25 Years | Male | 2 | Phase 2 | United States |
| 205 | EUCTR2015-002069-52-BG (EUCTR) | 06/08/2018 | 11/06/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 206 | EUCTR2017-004625-32-GB (EUCTR) | 26/07/2018 | 23/01/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Phase 3 | United States;Finland;Spain;Ireland;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Poland;Australia;Germany;Sweden | ||
| 207 | NCT03400852 (ClinicalTrials.gov) | July 17, 2018 | 9/1/2018 | A Study to Assess the Efficacy and Safety of MNK-1411 in Duchenne Muscular Dystrophy | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Participants 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Cosyntropin;Other: Placebo | Mallinckrodt ARD LLC | NULL | Terminated | 4 Years | 8 Years | Male | 44 | Phase 2 | United States;Bulgaria;Israel;Italy;Mexico;Serbia;Spain;Turkey |
| 208 | EUCTR2016-001615-21-BG (EUCTR) | 06/07/2018 | 12/04/2018 | An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 years | A Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom | ||
| 209 | NCT03603288 (ClinicalTrials.gov) | July 4, 2018 | 31/5/2018 | Phase III Study With Idebenone in Patients With Duchenne Muscular Dystrophy (SIDEROS-E) | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Who Completed the SIDEROS Study | Duchenne Muscular Dystrophy | Drug: idebenone 150 mg film-coated tablets | Santhera Pharmaceuticals | NULL | Terminated | 11 Years | N/A | Male | 161 | Phase 3 | United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom |
| 210 | NCT03439670 (ClinicalTrials.gov) | June 29, 2018 | 9/1/2018 | A Study to Assess the Efficacy and Safety of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study With Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone;Drug: Prednisone;Other: Placebo | ReveraGen BioPharma, Inc. | European Union;Cooperative International Neuromuscular Research Group;Newcastle University;University of Pittsburgh | Completed | 4 Years | 7 Years | Male | 121 | Phase 2 | United States;Australia;Belgium;Canada;Czechia;Greece;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy |
| 211 | EUCTR2017-002686-21-NL (EUCTR) | 26/06/2018 | 19/06/2018 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 32 | Phase 1 | France;United States;Czech Republic;Belgium;Spain;Ireland;Denmark;Netherlands;Italy;United Kingdom | ||
| 212 | EUCTR2017-004139-35-BE (EUCTR) | 18/06/2018 | 03/04/2018 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Duchenne Muscular Distrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosyntropin acetate Product Code: MNK-1411 INN or Proposed INN: TETRACOSACTIDE HEXAACETATE Other descriptive name: Cosyntropin acetate | Mallinckrodt ARD Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 132 | Phase 2 | United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands | ||
| 213 | EUCTR2017-004279-30-BE (EUCTR) | 14/06/2018 | 04/05/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 214 | NCT03354039 (ClinicalTrials.gov) | June 12, 2018 | 10/10/2017 | Tamoxifen in Duchenne Muscular Dystrophy | Tamoxifen in Duchenne Muscular Dystrophy: A Multicenter, Randomised, Double-blind, Placebo-controlled, Phase 3 Safety and Efficacy 48-week Trial | Duchenne Muscular Dystrophy | Drug: Tamoxifen;Drug: Matching placebo | University Hospital, Basel, Switzerland | NULL | Completed | 78 Months | 16 Years | Male | 93 | Phase 3 | France;Germany;Netherlands;Spain;Switzerland;United Kingdom |
| 215 | EUCTR2017-004139-35-ES (EUCTR) | 12/06/2018 | 19/03/2018 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Duchenne Muscular Distrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosyntropin acetate Product Code: MNK-1411 INN or Proposed INN: TETRACOSACTIDE HEXAACETATE Other descriptive name: Cosyntropin acetate | Mallinckrodt ARD Inc. | NULL | Not Recruiting | Female: no Male: yes | 132 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Bulgaria;Germany;Netherlands | ||
| 216 | NCT03424460 (ClinicalTrials.gov) | June 11, 2018 | 7/12/2017 | Venous Thromboembolism in Myotonic Dystrophy Type 1 | Venous Thromboembolism in Myotonic Dystrophy Type 1 | Myotonic Dystrophy 1 | Biological: Haemostasis tests;Biological: Monocytes and megacaryocytes culture and RNA extraction;Genetic: RNA extraction | Assistance Publique - Hôpitaux de Paris | AFM-Téléthon (Funding);Recherche Clinique Paris Descartes Necker Cochin Sainte Anne | Recruiting | 18 Years | N/A | All | 130 | N/A | France |
| 217 | EUCTR2017-002686-21-BE (EUCTR) | 28/05/2018 | 12/03/2018 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 32 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom | ||
| 218 | EUCTR2017-002686-21-IT (EUCTR) | 25/05/2018 | 05/11/2020 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy - - | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: [WVE-210201] INN or Proposed INN: WVE-210201 | WAVE LIFE SCIENCES USA INC | NULL | Not Recruiting | Female: no Male: yes | 32 | Phase 1 | United States;France;Canada;Belgium;Netherlands;United Kingdom;Italy | ||
| 219 | EUCTR2016-003257-15-ES (EUCTR) | 12/04/2018 | 04/01/2018 | A Phase 2 clinical trial that is randomized and controlled by a placebo (similar to the product under investigation but does not have any therapeutic effect) of ACE-083 in Patients with muscular dystrophy (on face, around shoulder blades, in upper arms). The study is a double-blind study, this means that neither you nor your doctor will know if you receive study drug or placebo | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients with Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ACE-083 Product Code: ACE-083 INN or Proposed INN: pending Other descriptive name: ACE-083 | Acceleron Pharma Inc. | NULL | Not Recruiting | Female: yes Male: yes | 92 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Spain | ||
| 220 | EUCTR2017-002686-21-GB (EUCTR) | 11/04/2018 | 27/12/2017 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 32 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom | ||
| 221 | NCT03406780 (ClinicalTrials.gov) | March 4, 2018 | 15/1/2018 | A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy | A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Biological: CAP-1002;Drug: Placebo | Capricor Inc. | NULL | Completed | 10 Years | N/A | Male | 18 | Phase 2 | United States |
| 222 | NCT03508947 (ClinicalTrials.gov) | January 24, 2018 | 16/4/2018 | Safety and Tolerability of WVE-210201 in Patients With Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: WVE-210201;Drug: Placebo | Wave Life Sciences Ltd. | NULL | Completed | 5 Years | 18 Years | Male | 36 | Phase 1 | United States;Belgium;Canada;France;Italy;Netherlands;United Kingdom |
| 223 | NCT03362502 (ClinicalTrials.gov) | January 23, 2018 | 29/11/2017 | A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy | A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Genetic: PF-06939926 | Pfizer | NULL | Active, not recruiting | 4 Years | N/A | Male | 23 | Phase 1 | United States |
| 224 | EUCTR2017-001223-49-BG (EUCTR) | 16/01/2018 | 11/10/2017 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 340 | Phase 3 | United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Brazil;Malaysia;Poland;Australia;Bulgaria;China;Japan;Korea, Republic of | ||
| 225 | EUCTR2017-003568-10-GB (EUCTR) | 05/01/2018 | 17/05/2018 | An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study toAssess the Long-term Safety and Efficacy of Vamorolone in Boys withDuchenne Muscular Dystrophy (DMD) - VBP15-LTE | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 Product Name: Vamorolone INN or Proposed INN: Vamorolone Other descriptive name: EV substance code: SUB188638 | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 226 | NCT03123913 (ClinicalTrials.gov) | December 18, 2017 | 12/4/2017 | Study of Testosterone and rHGH in FSHD | Study of Testosterone and rHGH in FSHD (STARFISH): A Proof-of-Concept Study | Facioscapulohumeral Muscular Dystrophy | Drug: Testosterone Enanthate;Drug: Somatropin | University of Rochester | NULL | Completed | 18 Years | 65 Years | Male | 20 | Phase 1 | United States |
| 227 | NCT03238235 (ClinicalTrials.gov) | December 12, 2017 | 12/7/2017 | Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Becker Muscular Dystrophy | A Randomised, Double Blind, Placebo Controlled Study to Evaluate the Micro-macroscopic Effects on Muscles, the Safety and Tolerability, and the Efficacy of Givinostat in Patients With Becker Muscular Dystrophy (BMD) | Becker Muscular Dystrophy | Drug: givinostat;Drug: placebo | Italfarmaco | NULL | Active, not recruiting | 18 Years | 65 Years | Male | 51 | Phase 2 | Italy;Netherlands |
| 228 | NCT03368742 (ClinicalTrials.gov) | December 6, 2017 | 5/12/2017 | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Genetic: SGT-001 | Solid Biosciences, LLC | NULL | Active, not recruiting | 4 Years | 17 Years | Male | 16 | Phase 1/Phase 2 | United States |
| 229 | EUCTR2017-003568-10-SE (EUCTR) | 23/11/2017 | 05/10/2017 | An Extension Study to Assess the Long-term Safety and Efficacy ofVamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE Product Name: Vamorolone INN or Proposed INN: VAMOROLONE | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 230 | EUCTR2016-001654-18-DE (EUCTR) | 13/11/2017 | 23/06/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 159 | Phase 3 | United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 231 | EUCTR2016-001654-18-FR (EUCTR) | 10/11/2017 | 30/10/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | Bristol-Myers Squibb International Corporation | NULL | Not Recruiting | Female: no Male: yes | 160 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 232 | EUCTR2016-001654-18-BE (EUCTR) | 10/11/2017 | 26/06/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy | A randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 (BMS-986089) in ambulatory boys with Duchenne Muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMS-986089-01 (RO7239361) Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 (BMS-986089) INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01 (RO7239361) , anti-myostatin Product Name: BMS-986089-01 (RO7239361) Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 (BMS-986089) INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01 (RO7239361), anti-myostatin Product Name: BMS-986089-01 (RO7239361) Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 (BMS-986089) INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01 (RO7239361), anti-myostatin Product Name: BMS-986089-01 (RO7239361) Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: RO7239361 (BMS-986089) INN or Proposed INN: Anti-myostati | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 159 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 233 | EUCTR2016-001654-18-GB (EUCTR) | 01/11/2017 | 27/06/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, Anti-myostatin Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 159 | Phase 3 | United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 234 | NCT02653833 (ClinicalTrials.gov) | November 1, 2017 | 14/12/2015 | The Study of Skeletal Muscle Blood Flow in Becker Muscular Dystrophy | Skeletal Muscle Blood Flow in Becker Muscular Dystrophy | Muscular Dystrophy | Drug: Tadalafil 20 MG;Other: beetroot juice extract | Cedars-Sinai Medical Center | NULL | Terminated | 18 Years | 45 Years | Male | 6 | Early Phase 1 | United States |
| 235 | EUCTR2016-001654-18-NL (EUCTR) | 26/10/2017 | 12/07/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, Anti-myostatin Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 159 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Netherlands;Germany;Japan;Sweden | ||
| 236 | NCT03373968 (ClinicalTrials.gov) | October 24, 2017 | 4/12/2017 | Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study | Open Label, Long-term Safety, Tolerability, and Efficacy Study of GIVINOSTAT in All DMD Patients Who Have Been Previously Treated in One of the GIVINOSTAT Studies | Duchenne Muscular Dystrophy | Drug: Givinostat | Italfarmaco | Cromsource | Enrolling by invitation | 7 Years | N/A | Male | 206 | Phase 2/Phase 3 | United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;United Kingdom |
| 237 | EUCTR2016-001654-18-IT (EUCTR) | 19/09/2017 | 07/02/2018 | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy - - | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy - - | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION | NULL | Not Recruiting | Female: no Male: yes | 160 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 238 | EUCTR2015-002069-52-CZ (EUCTR) | 11/09/2017 | 14/03/2017 | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;Korea, Republic of;Czechia;United Kingdom;Hungary;Czech Republic;Mexico;Canada;Argentina;Belgium;Poland;Bulgaria;Norway;Germany;Sweden | ||
| 239 | EUCTR2017-001629-41-IT (EUCTR) | 08/09/2017 | 15/01/2021 | Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy. | A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy - NA | Becker Muscular Dystrophy (BMD) MedDRA version: 20.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.P.A. | NULL | Not Recruiting | Female: no Male: yes | 55 | Phase 2 | Netherlands;Italy | ||
| 240 | EUCTR2016-001654-18-ES (EUCTR) | 04/09/2017 | 04/07/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of BMS-986089 in Ambulatory Boys with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMS-986089-01 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: BMS-986089-01 Injections, 35 mg/Syringe (50 mg/mL) Product Code: BMS-986089 INN or Proposed INN: Anti-Myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | Bristol-Myers Squibb International Corporation | NULL | Not Recruiting | Female: no Male: yes | 160 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 241 | NCT03218995 (ClinicalTrials.gov) | August 16, 2017 | 9/7/2017 | Study of Eteplirsen in Young Patients With DMD Amenable to Exon 51 Skipping | An Open-label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 6 Months | 48 Months | Male | 15 | Phase 2 | Belgium;France;Italy;United Kingdom;Germany |
| 242 | EUCTR2016-001654-18-SE (EUCTR) | 12/08/2017 | 15/06/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, Anti-myostatin Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin Product Name: RO7239361 Injections, 50 mg/Syringe (71.4 mg/mL) Product Code: RO7239361 INN or Proposed INN: Anti-myostatin Adnectin Other descriptive name: BMS-986089-01, anti-myostatin | F. Hoffmann-La Roche Ltd | NULL | Not Recruiting | Female: no Male: yes | 159 | Phase 3 | United States;Spain;Italy;United Kingdom;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | ||
| 243 | EUCTR2017-000397-10-IT (EUCTR) | 31/07/2017 | 30/01/2018 | Studio nel quale tutti i pazienti prendono lo stesso farmaco sperimentale con lo scopo di valutare a lungo termine la sicurezza, la tollerabilità e l'efficacia del GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in uno studio con GIVINOSTAT. | Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in studio con GIVINOSTAT. - Studio con Givinostat in DMD a lungo termine | Distrofia Muscolare di Duchenne (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (idrocloruro monoidrato) Product Code: ITF2357 INN or Proposed INN: Givinostat (idrocloruro monoidrato) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy | ||
| 244 | EUCTR2016-000951-29-DE (EUCTR) | 13/07/2017 | 13/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 12 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Belgium;Germany;Italy;United Kingdom | ||
| 245 | NCT03167255 (ClinicalTrials.gov) | July 6, 2017 | 22/5/2017 | Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II, Open-Label, Extension Study to Assess the Safety and Efficacy of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01 | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS) | Completed | 4 Years | 10 Years | Male | 16 | Phase 2 | United States;Canada |
| 246 | NCT03039686 (ClinicalTrials.gov) | July 6, 2017 | 27/1/2017 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: RO7239361;Drug: Placebo for RO7239361 | Hoffmann-La Roche | NULL | Terminated | 6 Years | 11 Years | Male | 166 | Phase 2/Phase 3 | United States;Argentina;Australia;Belgium;Canada;France;Germany;Italy;Japan;Netherlands;Spain;Sweden;United Kingdom |
| 247 | NCT03179631 (ClinicalTrials.gov) | July 6, 2017 | 1/6/2017 | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Disease;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren;Drug: PLACEBO | PTC Therapeutics | NULL | Active, not recruiting | 5 Years | N/A | Male | 360 | Phase 3 | United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Taiwan;Thailand;Turkey;Argentina;Chile;Jordan;Sri Lanka |
| 248 | EUCTR2016-000401-36-NL (EUCTR) | 28/06/2017 | 14/12/2016 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Not Recruiting | Female: no Male: yes | 169 | Phase 3 | France;United States;Serbia;Canada;Spain;Belgium;Israel;Germany;Netherlands;United Kingdom;Italy | ||
| 249 | EUCTR2015-002069-52-SE (EUCTR) | 26/06/2017 | 20/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 222 | Phase 3 | Portugal;Serbia;United States;Greece;Spain;Ukraine;Ireland;Chile;Israel;Russian Federation;Italy;France;Denmark;Australia;Netherlands;United Kingdom;Hungary;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Norway;Sweden | ||
| 250 | EUCTR2016-000951-29-GB (EUCTR) | 22/06/2017 | 04/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 15 | Phase 2 | France;Belgium;Germany;Italy;United Kingdom | ||
| 251 | NCT04226924 (ClinicalTrials.gov) | June 15, 2017 | 15/7/2019 | Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose | A Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Trial of Trehalose for the Treatment of | Oculopharyngeal Muscular Dystrophy | Drug: Trehalose | Bioblast Pharma Ltd. | NULL | Withdrawn | 50 Years | 70 Years | All | 0 | Phase 2 | Canada |
| 252 | EUCTR2016-000951-29-IT (EUCTR) | 13/06/2017 | 07/02/2018 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping - N/A | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | SAREPTA THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 12 | Phase 2 | France;Belgium;Germany;United Kingdom;Italy | ||
| 253 | NCT02851797 (ClinicalTrials.gov) | June 1, 2017 | 27/7/2016 | Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy | Randomised, Double Blind, Placebo Controlled, Multicentre Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: givinostat;Drug: placebo | Italfarmaco | Syneos Health | Completed | 6 Years | 17 Years | Male | 179 | Phase 3 | United States;Belgium;Canada;France;Germany;Israel;Italy;Netherlands;Serbia;Spain;United Kingdom |
| 254 | EUCTR2016-000951-29-FR (EUCTR) | 01/06/2017 | 17/07/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1 | France;Belgium;Germany;Italy;United Kingdom | ||
| 255 | EUCTR2016-000951-29-BE (EUCTR) | 29/05/2017 | 11/04/2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: eteplirsen Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 15 | Phase 2 | France;Belgium;Germany;Italy;United Kingdom | ||
| 256 | EUCTR2016-000401-36-IT (EUCTR) | 24/05/2017 | 08/02/2017 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 192 | Phase 3 | France;United States;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 257 | EUCTR2016-000401-36-DE (EUCTR) | 15/05/2017 | 28/10/2016 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Not Recruiting | Female: no Male: yes | 169 | Phase 3 | United States;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 258 | EUCTR2015-002069-52-ES (EUCTR) | 11/05/2017 | 10/03/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | United States;Canada;Spain;Israel;Germany;United Kingdom | ||
| 259 | EUCTR2015-002069-52-BE (EUCTR) | 28/04/2017 | 05/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Greece;Spain;Ukraine;Ireland;Russian Federation;Israel;Italy;France;Denmark;Australia;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Brazil;Belgium;Poland;Bulgaria;Georgia;Norway;Germany;Sweden | ||
| 260 | EUCTR2016-000602-10-NL (EUCTR) | 26/04/2017 | 20/09/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden | ||
| 261 | EUCTR2016-000401-36-GB (EUCTR) | 07/03/2017 | 26/10/2016 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 169 | Phase 3 | United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 262 | EUCTR2016-004262-26-GB (EUCTR) | 16/02/2017 | 21/12/2020 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability,Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne MuscularDystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 263 | EUCTR2016-004462-26-GB (EUCTR) | 16/02/2017 | 23/12/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Australia;Israel;United Kingdom | ||
| 264 | EUCTR2016-004263-38-SE (EUCTR) | 13/02/2017 | 16/12/2016 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy(DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 265 | EUCTR2016-004262-26-SE (EUCTR) | 13/02/2017 | 16/12/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIa, Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone Product Name: Vamorolone INN or Proposed INN: vamorolone | ReveraGen BioPharma Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 266 | EUCTR2016-004263-38-GB (EUCTR) | 10/02/2017 | 08/02/2019 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Longterm Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone Product Name: Vamorolone Product Code: VBP15 INN or Proposed INN: vamorolone | ReveraGen BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Australia;Israel;United Kingdom;Sweden | ||
| 267 | EUCTR2016-000401-36-ES (EUCTR) | 10/02/2017 | 10/02/2017 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 192 | Phase 3 | France;United States;Canada;Belgium;Spain;Israel;Netherlands;Germany;Italy;United Kingdom | ||
| 268 | EUCTR2015-002069-52-DE (EUCTR) | 07/02/2017 | 24/10/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Casimersen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Golodirsen Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | United States;Serbia;Taiwan;Greece;Spain;Ukraine;Ireland;Israel;Russian Federation;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Czechia;United Kingdom;Czech Republic;Hungary;Mexico;Canada;Argentina;Poland;Belgium;Bulgaria;Norway;Germany;Sweden | ||
| 269 | EUCTR2016-001615-21-IT (EUCTR) | 06/02/2017 | 09/01/2017 | An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 years | A Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 95 | Phase 2 | United States;Canada;Poland;Australia;Bulgaria;United Kingdom;Japan;Italy | ||
| 270 | NCT03038399 (ClinicalTrials.gov) | February 2, 2017 | 30/1/2017 | Long-term Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A 24-month Phase II Open-label, Multicenter Long-term Extension Study to Assess the Long-Term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;Cooperative International Neuromuscular Research Group | Completed | 4 Years | 7 Years | Male | 46 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 271 | EUCTR2016-000401-36-BE (EUCTR) | 27/01/2017 | 27/10/2016 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. - EPIDYS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Not Recruiting | Female: no Male: yes | 242 | Phase 3 | United States;Serbia;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | ||
| 272 | EUCTR2015-002069-52-GB (EUCTR) | 23/01/2017 | 05/09/2016 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | Serbia;Portugal;United States;Greece;Spain;Ireland;Russian Federation;Israel;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Poland;Belgium;Australia;Denmark;Bulgaria;Norway;Germany;Sweden | ||
| 273 | EUCTR2015-002530-50-IT (EUCTR) | 19/01/2017 | 22/08/2018 | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of rimeporide in patients with Duchenne Muscular Dystrophy (DMD) | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of rimeporide in patients with Duchenne Muscular Dystrophy (DMD) - RIM4DMD | Distrofia Muscolare di Duchenne (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: rimeporide Product Code: EMD 87580 | ESPERARE FOUNDATION | NULL | Not Recruiting | Female: no Male: yes | 20 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;United Kingdom;Italy | ||
| 274 | EUCTR2015-002069-52-IT (EUCTR) | 19/01/2017 | 07/02/2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 45 Product Name: SRP-4053 Product Code: SRP-4053 Other descriptive name: Oligomero morfolino fosforodiamidato per skipping dell’esone 53 | SAREPTA THERAPEUTICS, INC. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 99 | Phase 3 | France;Czech Republic;Spain;Belgium;Netherlands;Germany;United Kingdom;Italy;Sweden | ||
| 275 | EUCTR2014-002072-92-BG (EUCTR) | 29/12/2016 | 18/10/2016 | A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy | A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 105 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom | ||
| 276 | EUCTR2016-000602-10-GB (EUCTR) | 28/12/2016 | 23/09/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;United Kingdom;Italy;Switzerland;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden | ||
| 277 | EUCTR2016-000602-10-AT (EUCTR) | 21/12/2016 | 01/12/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 278 | EUCTR2016-000602-10-ES (EUCTR) | 20/12/2016 | 18/11/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 279 | EUCTR2016-001615-21-GB (EUCTR) | 12/12/2016 | 21/10/2016 | An extension to Protocol B5161002 to evaluate the long-term safety of as well as to collect data regarding maintenance of effect up to 4 years | A Multicenter, Open-Label Extension Study to Evaluate the Long Term Safety of PF-06252616 in Boys with Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. | NULL | Not Recruiting | Female: no Male: yes | 105 | Phase 2 | United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom | ||
| 280 | NCT02740972 (ClinicalTrials.gov) | December 2016 | 23/3/2016 | Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01;Drug: Placebo | NS Pharma, Inc. | Nippon Shinyaku Co., Ltd.;Cooperative International Neuromuscular Research Group;Therapeutic Research in Neuromuscular Disorders Solutions | Completed | 4 Years | 9 Years | Male | 16 | Phase 2 | United States;Canada | |
| 281 | EUCTR2016-000602-10-FR (EUCTR) | 25/11/2016 | 12/01/2017 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 282 | EUCTR2016-000602-10-DE (EUCTR) | 17/11/2016 | 30/05/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 283 | NCT03236662 (ClinicalTrials.gov) | November 2016 | 7/11/2016 | (-)- Epicatechin Becker Muscular Dystrophy | UCD0115B: An Open-label Extension Study of Purified Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: (-)-Epicatechin | Craig McDonald, MD | Cardero Therapeutics, Inc. | Completed | 18 Years | 70 Years | Male | 2 | Phase 2 | United States |
| 284 | NCT02964377 (ClinicalTrials.gov) | November 2016 | 7/11/2016 | Plus Epicatechin Duchenne Muscular Dystrophy in Non-ambulatory Adolescents | A Single Center Dose Ranging Pilot Study of (+)-Epicatechin in Non-ambulatory Adolescents With Duchenne Muscular Dystrophy and Pre-symptomatic Cardiac Dysfunction | Duchenne Muscular Dystrophy | Drug: (+)- Epicatechin | Craig McDonald, MD | Cardero Therapeutics, Inc. | Completed | 8 Years | 17 Years | Male | 15 | Phase 1/Phase 2 | United States |
| 285 | NCT02927080 (ClinicalTrials.gov) | November 2016 | 5/10/2016 | Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) | A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy | Drug: ACE-083;Drug: ACE-083 or placebo | Acceleron Pharma, Inc. | NULL | Terminated | 18 Years | N/A | All | 95 | Phase 2 | United States;Canada;Spain |
| 286 | EUCTR2016-000602-10-IT (EUCTR) | 20/10/2016 | 06/02/2018 | A clinical study to assess how effective and safe is idebenone treatment inpatients with Duchenne Muscular Dystrophy (DMD) who are currentlyreceiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Studyassessing the Efficacy, Safety and Tolerability of Idebenone inPatients with Duchenne Muscular Dystrophy Receiving GlucocorticoidSteroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: RAXONE - 150 MG- COMPRESSE RIVESTITE CON FILM- USO ORALE- FLACONE (HDPE)- 180 COMPRESSE INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | SANTHERA PHARMACEUTICALS | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 266 | Phase 3 | France;United States;Belgium;Spain;Austria;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | ||
| 287 | EUCTR2013-005489-20-FR (EUCTR) | 18/10/2016 | 18/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 288 | NCT02907619 (ClinicalTrials.gov) | October 13, 2016 | 14/9/2016 | An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy | A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Biological: PF-06252616 | Pfizer | NULL | Terminated | 6 Years | 18 Years | Male | 59 | Phase 2 | United States;Canada;Italy;Japan;United Kingdom |
| 289 | NCT02500381 (ClinicalTrials.gov) | September 28, 2016 | 14/7/2015 | Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) | A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: SRP-4045;Drug: SRP-4053;Drug: Placebo | Sarepta Therapeutics, Inc. | NULL | Active, not recruiting | 6 Years | 13 Years | Male | 229 | Phase 3 | United States;Argentina;Australia;Belgium;Bulgaria;Canada;Czechia;Denmark;France;Germany;Greece;Hungary;India;Ireland;Israel;Italy;Korea, Republic of;Mexico;Poland;Russian Federation;Serbia;Spain;Sweden;United Kingdom |
| 290 | EUCTR2016-000602-10-BE (EUCTR) | 02/09/2016 | 19/07/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | ||
| 291 | NCT02814019 (ClinicalTrials.gov) | September 2016 | 17/6/2016 | A Phase III Double-blind Study With Idebenone in Patients With Duchenne Muscular Dystrophy (DMD) Taking Glucocorticoid Steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study Assessing the Efficacy, Safety and Tolerability of Idebenone in Patients With Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids | Duchenne Muscular Dystrophy (DMD) | Drug: Idebenone 150 mg film-coated tablets;Drug: placebo | Santhera Pharmaceuticals | NULL | Terminated | 10 Years | N/A | Male | 255 | Phase 3 | United States;Austria;Belgium;Bulgaria;France;Germany;Hungary;Ireland;Israel;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom |
| 292 | EUCTR2014-002072-92-PL (EUCTR) | 10/08/2016 | 14/07/2016 | A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy | A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 120 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom | ||
| 293 | NCT02760277 (ClinicalTrials.gov) | July 28, 2016 | 28/4/2016 | An Extension Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/day/day;Drug: Vamorolone 0.75 mg/day/day;Drug: Vamorolone 2.0 mg/day/day;Drug: Vamorolone 6.0 mg/day/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research Group;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) | Completed | 4 Years | 7 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 294 | EUCTR2016-000601-36-IT (EUCTR) | 26/07/2016 | 08/06/2021 | Clinical trial on the efficacy of non invasive ventilation and modafinil on excessive daytime sleepiness in Myotonic Dystrophy type 1 | CLINICAL EFFICACY OF NIV AND MODAFINIL ON EXCESSIVE DAYTIME SLEEPINESS: AMULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED CLINICAL TRIAL IN DM1 - ModaNIV | Myotonic Dystrophy type 1 MedDRA version: 20.0;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: PROVIGIL - 100 MG COMPRESSE 30 COMPRESSE | FONDAZIONE SERENA ONLUS | NULL | Not Recruiting | Female: yes Male: yes | 80 | Phase 3 | Italy | ||
| 295 | NCT02858908 (ClinicalTrials.gov) | July 20, 2016 | 4/8/2016 | Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy | A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy | Myotonic Dystrophy 1 | Drug: Tideglusib | AMO Pharma Limited | NULL | Completed | 12 Years | 45 Years | All | 16 | Phase 2 | United Kingdom |
| 296 | NCT02836418 (ClinicalTrials.gov) | July 12, 2016 | 30/6/2016 | Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy;Limb Girdle Muscular Dystrophy | Drug: ATYR1940 | aTyr Pharma, Inc. | NULL | Completed | 16 Years | 25 Years | All | 8 | Phase 1/Phase 2 | United States;Denmark;Italy |
| 297 | EUCTR2014-002008-25-FR (EUCTR) | 06/07/2016 | 11/01/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Italy;United Kingdom | ||
| 298 | EUCTR2016-000624-25-DK (EUCTR) | 22/06/2016 | 19/04/2016 | An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy | Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 INN or Proposed INN: Not available yet Other descriptive name: ATYR1940 | aTyr Pharma, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Denmark;Italy | ||
| 299 | NCT02819557 (ClinicalTrials.gov) | June 9, 2016 | 16/6/2016 | Study of Ataluren in =2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy | A Phase 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged =2 to <5 Years Old With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | 2 Years | 5 Years | Male | 14 | Phase 2 | United States |
| 300 | NCT02760264 (ClinicalTrials.gov) | June 2016 | 28/4/2016 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy | Drug: Vamorolone 0.25 mg/kg/day;Drug: Vamorolone 0.75 mg/kg/day;Drug: Vamorolone 2.0 mg/kg/day;Drug: Vamorolone 6.0 mg/kg/day | ReveraGen BioPharma, Inc. | University of Pittsburgh;National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);National Institute of Neurological Disorders and Stroke (NINDS);Cooperative International Neuromuscular Research Group | Completed | 4 Years | 6 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 301 | NCT02808585 (ClinicalTrials.gov) | June 2016 | 8/6/2016 | Study to Assess the Safety, Tolerability and PK Response and Explore the PD Response Following 4 Weekly SC Injections of PB1046 in Subjects With Stable Heart Failure With Reduced Ejection Fraction (HFrEF) | Phase 2a, Randomized, Double-blind, Placebo-controlled, Multiple-dose, 2-Part Study to Assess the Safety, Tolerability and Pharmacokinetic Response and Explore the Pharmacodynamic Response Following 4 Weeks of Once Weekly Subcutaneous Injections of PB1046 in Adult Subjects With Stable Heart Failure With Reduced Ejection Fraction (HFrEF) (Part 1) and in Subjects With Cardiac Dysfunction Secondary to Duchenne Muscular Dystrophy (Part 2) | Heart Failure | Drug: PB1046 Injection;Drug: Placebo Injection | PhaseBio Pharmaceuticals Inc. | NULL | Completed | 18 Years | N/A | All | 29 | Phase 2 | United States |
| 302 | NCT02972580 (ClinicalTrials.gov) | June 2016 | 25/7/2016 | Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD | Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Genetic: Genetic characterization | Nationwide Children's Hospital | Parent Project Muscular Dystrophy | Active, not recruiting | 18 Years | N/A | Female | 250 | United States | |
| 303 | NCT02858362 (ClinicalTrials.gov) | June 2016 | 27/7/2016 | Proof of Concept Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With Duchenne Muscular Dystrophy (DMD) | Phaseout DMD: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With Ezutromid in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy (SMT C11005) | Duchenne Muscular Dystrophy | Drug: Ezutromid | Summit Therapeutics | NULL | Terminated | 5 Years | N/A | Male | 43 | Phase 2 | United States;United Kingdom |
| 304 | EUCTR2014-005296-81-BE (EUCTR) | 02/05/2016 | 19/11/2015 | A clinical study to provide drisapersen (study medication) to patients withDuchenne disease (muscular disease) and to assess the efficacy, safety and tolerability. | A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: Drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 104 | Phase 3 | United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden | ||
| 305 | NCT02752048 (ClinicalTrials.gov) | May 2016 | 6/4/2016 | A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy | A Randomized Phase IIa Study of TAS-205 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: TAS-205;Drug: Placebo | Taiho Pharmaceutical Co., Ltd. | NULL | Completed | 5 Years | N/A | Male | 36 | Phase 2 | Japan |
| 306 | EUCTR2016-000067-16-GB (EUCTR) | 20/04/2016 | 13/05/2016 | A clinical trial to investigate how safe and effective tideglusib is, as treatment for adolescents and adults with myotonic dystrophy diagnosed before they were 12 years old. | A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy | Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy MedDRA version: 19.1;Level: PT;Classification code 10068871;Term: Myotonic dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib INN or Proposed INN: Tideglusib Other descriptive name: 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione | AMO Pharma Ltd. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom | ||
| 307 | NCT02958202 (ClinicalTrials.gov) | April 2016 | 27/10/2016 | Extension Study of BMN 044 in Duchenne Muscular Dystrophy (DMD) | A Multi Center, Multi National, Open Label, Extension Study to Evaluate the Long-term Efficacy and Safety of BMN 044 (PRO044) in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: BMN 044 IV 6 mg/kg;Drug: BMN 044 IV 9 mg/kg;Drug: BMN 044 SC 6 mg/kg | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | N/A | Male | 7 | Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 308 | NCT02704325 (ClinicalTrials.gov) | April 2016 | 23/12/2015 | Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 | Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 | Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.GALGT2;Other: PLACEBO (Saline) | Kevin Flanigan | NULL | Withdrawn | 9 Years | N/A | Male | 0 | Phase 1/Phase 2 | United States |
| 309 | NCT02439216 (ClinicalTrials.gov) | April 2016 | 29/4/2015 | Phase 1/2 Study in Boys With Duchenne Muscular Dystrophy | A Phase 1/2 Study of Edasalonexent (CAT-1004) in Pediatric Patients With Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Edasalonexent;Drug: Placebo | Catabasis Pharmaceuticals | NULL | Completed | 4 Years | 7 Years | Male | 31 | Phase 1/Phase 2 | United States |
| 310 | NCT02603562 (ClinicalTrials.gov) | March 30, 2016 | 5/11/2015 | Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy | An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy (FSHD) | Biological: ATYR1940;Biological: Placebo | aTyr Pharma, Inc. | NULL | Completed | 12 Years | 25 Years | All | 8 | Phase 1/Phase 2 | United States;France;Italy |
| 311 | NCT02710591 (ClinicalTrials.gov) | March 2016 | 26/1/2016 | Rimeporide in Patients With Duchenne Muscular Dystrophy | A Phase Ib, Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Oral Doses of Rimeporide in Patients With Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Rimeporide | EspeRare Foundation | NULL | Completed | 6 Years | 14 Years | Male | 20 | Phase 1 | France;Italy;Spain;United Kingdom |
| 312 | EUCTR2015-004333-27-GB (EUCTR) | 19/01/2016 | 04/11/2015 | A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular Dystrophy | A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 INN or Proposed INN: Pending | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 40 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 313 | JPRN-UMIN000020580 | 2016/01/15 | 20/01/2016 | Pilot study of tranilast for cardiomyopathy of muscular dystrophy | Pilot study of tranilast for cardiomyopathy of muscular dystrophy - Pilot study of tranilast for cardiomyopathy of muscular dystrophy | muscular dystrophy | Tranilast 300mg/day for three months (oral intake) | National Hospital Organization Toneyama National Hospital | National Cerebral and Cardiovascular Center | Complete: follow-up complete | 20years-old | Not applicable | Male and Female | 2 | Not applicable | Japan |
| 314 | EUCTR2015-001955-54-DE (EUCTR) | 13/01/2016 | 18/08/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan | ||
| 315 | EUCTR2015-003681-87-BE (EUCTR) | 12/01/2016 | 16/11/2015 | A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study) | A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 50 | Phase 2 | United States;Belgium;Netherlands;Italy;Sweden | ||
| 316 | EUCTR2015-001955-54-BE (EUCTR) | 06/01/2016 | 21/09/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;Belarus;United States;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan | ||
| 317 | NCT02485938 (ClinicalTrials.gov) | January 2016 | 19/6/2015 | HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) | A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy;Cardiomyopathy | Drug: Allogeneic Cardiosphere-Derived Cells (CAP-1002) | Capricor Inc. | NULL | Completed | 12 Years | N/A | Male | 25 | Phase 1/Phase 2 | United States |
| 318 | EUCTR2015-002530-50-FR (EUCTR) | 18/12/2015 | 11/09/2015 | Rimeporide in patients with Duchenne Muscular Dystrophy | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Rimeporide Product Code: EMD 87580 INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate Other descriptive name: Rimeporide INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate Other descriptive name: Rimeporide | EspeRare | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 1 | France;Spain;Italy;United Kingdom;Switzerland | ||
| 319 | EUCTR2015-001910-88-DK (EUCTR) | 17/12/2015 | 30/06/2015 | A Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies | An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies | Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 INN or Proposed INN: Not available yet | aTyr Pharma, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 1;Phase 2 | France;United States;Denmark;Italy | ||
| 320 | EUCTR2015-001955-54-NL (EUCTR) | 08/12/2015 | 09/07/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen | BioMarin Nederland BV | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;United States;Belarus;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan;Sweden | ||
| 321 | NCT02515669 (ClinicalTrials.gov) | December 2, 2015 | 29/7/2015 | Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD | A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy | Muscular Dystrophy (DMD) | Drug: RO7239361;Drug: Placebo | Hoffmann-La Roche | NULL | Terminated | 5 Years | 10 Years | Male | 43 | Phase 1/Phase 2 | United States;Canada |
| 322 | NCT02606136 (ClinicalTrials.gov) | November 30, 2015 | 4/11/2015 | Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) | Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: pamrevlumab (FG-3019) | FibroGen | NULL | Active, not recruiting | 12 Years | N/A | Male | 21 | Phase 2 | United States |
| 323 | NCT02636686 (ClinicalTrials.gov) | November 27, 2015 | 9/12/2015 | Extension Study of Drisapersen in DMD Subjects | An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in Subjects With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | No longer available | 5 Years | 80 Years | Male | United States;Argentina;Australia;Belgium;Bulgaria;Czechia;France;Germany;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom;Czech Republic | ||
| 324 | EUCTR2015-001955-54-ES (EUCTR) | 24/11/2015 | 05/10/2015 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen INN or Proposed INN: drisapersen Other descriptive name: DRISAPERSEN SODIUM | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Belarus;Portugal;Taiwan;Spain;Ukraine;Chile;Israel;Russian Federation;Italy;Switzerland;France;Denmark;Australia;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Brazil;Belgium;Poland;Bulgaria;Germany;Norway;Japan | ||
| 325 | EUCTR2015-003195-68-GB (EUCTR) | 11/11/2015 | 26/10/2015 | Testosterone therapy in DMD | Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy. - Testosterone therapy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Sustanon 250 Product Name: Sustanon 250 Product Code: PRD2175434 INN or Proposed INN: testosterone propionate INN or Proposed INN: testosterone phenylpropionate INN or Proposed INN: testosterone isocaproate INN or Proposed INN: testosterone decanoate | The Newcastle upon Tyne NHS Hospitals Foundation Trust | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 3 | United Kingdom | ||
| 326 | NCT02579239 (ClinicalTrials.gov) | November 5, 2015 | 28/9/2015 | The Safety and Biological Activity of ATYR1940 in Patients With Limb Girdle or Facioscapulohumeral Muscular Dystrophies | An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophies | Limb-Girdle Muscular Dystrophies;Facioscapulohumeral Muscular Dystrophy | Biological: ATYR1940;Biological: Placebo | aTyr Pharma, Inc. | NULL | Completed | 18 Years | 75 Years | All | 18 | Phase 1/Phase 2 | United States;Denmark;France;Italy |
| 327 | EUCTR2015-002530-50-ES (EUCTR) | 04/11/2015 | 25/08/2015 | Rimeporide in patients with Duchenne Muscular Dystrophy | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy. - Rim4DMD | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Rimeporide Product Code: EMD 87580 INN or Proposed INN: N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate Other descriptive name: Rimeporide | EspeRare | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 1 | France;Spain;Italy;United Kingdom;Switzerland | ||
| 328 | NCT02571205 (ClinicalTrials.gov) | November 2015 | 24/8/2015 | Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy | Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Sustanon (testosterone) | Newcastle-upon-Tyne Hospitals NHS Trust | NULL | Completed | 12 Years | 17 Years | Male | 15 | United Kingdom | |
| 329 | EUCTR2015-002530-50-GB (EUCTR) | 29/10/2015 | 07/09/2015 | Rimeporide in patients with Duchenne Muscular Dystrophy | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy - RIM4DMD | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Rimeporide Product Code: EMD 87580 INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate Other descriptive name: Rimeporide INN or Proposed INN: N-(4,5-Bis methanesulfonyl-2-methyl-benzoyl)guanidine hydrochloride monohydrate Other descriptive name: Rimeporide | EspeRare | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 1 | France;Spain;Italy;Switzerland;United Kingdom | ||
| 330 | NCT02530905 (ClinicalTrials.gov) | October 14, 2015 | 10/8/2015 | Dose-Titration and Open-label Extension Study of SRP-4045 in Advanced Stage Duchenne Muscular Dystrophy (DMD) Patients | A Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study Followed by an Open-Label Safety and Efficacy Evaluation of SRP-4045 in Advanced-Stage Patients With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping | Duchenne Muscular Dystrophy | Drug: SRP-4045;Drug: Placebo | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 21 Years | Male | 12 | Phase 1 | United States |
| 331 | NCT02667483 (ClinicalTrials.gov) | October 2015 | 26/1/2016 | Study of DS-5141b in Patients With Duchenne Muscular Dystrophy | Phase I/II Study of DS-5141b: Open-label Study of DS-5141b in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: DS-5141b | Daiichi Sankyo Co., Ltd. | Orphan Disease Treatment Institute Co., Ltd. | Active, not recruiting | 5 Years | 10 Years | Male | 7 | Phase 1/Phase 2 | Japan |
| 332 | NCT02531217 (ClinicalTrials.gov) | September 2015 | 25/6/2015 | Safety, Tolerability, PK, and Activity of ATYR1940 in Patients With Muscular Dystrophy - Study Extension | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients With Fascioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy | Biological: ATYR1940 | aTyr Pharma, Inc. | NULL | Completed | 18 Years | 65 Years | All | 9 | Phase 1/Phase 2 | United States;Italy;Netherlands |
| 333 | NCT03067831 (ClinicalTrials.gov) | September 2015 | 25/2/2017 | Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy | Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Biological: Stem Cells | Stem Cells Arabia | NULL | Recruiting | 4 Years | 25 Years | All | 20 | Phase 1/Phase 2 | Jordan |
| 334 | EUCTR2013-005489-20-BG (EUCTR) | 25/08/2015 | 26/06/2015 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 335 | EUCTR2015-001912-36-NL (EUCTR) | 04/08/2015 | 22/06/2015 | A Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Exposure of ATYR1940 in Adult Patients with Genetic Myopathy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral muscular dystrophy MedDRA version: 19.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 INN or Proposed INN: Not available yet Other descriptive name: ATYR1940 | aTyr Pharma, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Netherlands;Italy | ||
| 336 | EUCTR2015-001967-38-GB (EUCTR) | 03/08/2015 | 20/07/2015 | A research project for boys with Duchenne muscular dystrophy aged 5-9 years. It aims to find out how safe and well tolerated different doses of the experimental medicine are, when given by mouth after breakfast and evening meal. | A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD) - SMTC11004 - Phase 1 study in healthy volunteers and DMD patients | Duchenne muscular dystrophy (DMD) MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SMT C1100 (F5; nanosuspension) Product Code: SMT C1100 (F5) Product Name: SMT C1100 (F6; Powder for Oral Suspension) Product Code: SMT C1100 (F6) | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 24 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 337 | NCT02420379 (ClinicalTrials.gov) | June 30, 2015 | 10/4/2015 | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 4 Years | 6 Years | Male | 33 | Phase 2 | United States |
| 338 | NCT02434627 (ClinicalTrials.gov) | June 2015 | 28/4/2015 | Sodium Nitrate for Muscular Dystrophy | Sodium Nitrate for Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Sodium Nitrate | Cedars-Sinai Medical Center | NULL | Completed | 15 Years | 45 Years | Male | 5 | Phase 1 | United States |
| 339 | NCT02484560 (ClinicalTrials.gov) | June 2015 | 16/6/2015 | Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2 | Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2 | Duchenne Muscular Dystrophy | Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell | University of Gaziantep | Istinye University, Cukurova University, Yildirim Beyazit University, Gaziantep Deva Hospital, Gaziantep Public Hospital | Active, not recruiting | 8 Years | 14 Years | Male | 10 | Phase 1 | Turkey |
| 340 | NCT02525302 (ClinicalTrials.gov) | May 2015 | 18/7/2015 | HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02 | HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02 | Duchenne Muscular Dystrophy | Drug: HT-100 | Akashi Therapeutics | NULL | Terminated | 6 Years | 20 Years | Male | 10 | Phase 2 | United States |
| 341 | EUCTR2013-003605-26-NL (EUCTR) | 13/04/2015 | 19/11/2014 | A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01) | A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 | Prosensa Therapeutics B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 342 | NCT02354352 (ClinicalTrials.gov) | March 20, 2015 | 27/1/2015 | Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy | Therapeutic Potential for Aldosterone Inhibition in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Eplerenone;Drug: Spironolactone | Ohio State University | University of California, Los Angeles;University of Utah;University of Colorado, Denver;University of Kansas Medical Center;Vanderbilt University Medical Center | Completed | 7 Years | N/A | Male | 52 | Phase 3 | United States |
| 343 | EUCTR2014-002072-92-IT (EUCTR) | 05/03/2015 | 12/01/2015 | A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy | A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: - Other descriptive name: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 105 | Phase 2 | United States;Canada;Poland;Bulgaria;United Kingdom;Japan;Italy | ||
| 344 | NCT02376816 (ClinicalTrials.gov) | March 2015 | 26/2/2015 | Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy | Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.Micro-dystrophin | Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.micro-Dystrophin | Jerry R. Mendell | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Completed | 7 Years | N/A | Male | 2 | Phase 1 | United States |
| 345 | NCT01976091 (ClinicalTrials.gov) | February 1, 2015 | 24/7/2013 | A Gene Transfer Therapy Study to Evaluate the Safety of SRP-9004 in Participants With Limb-Girdle Muscular Dystrophy, Type 2D (LGMD2D) | Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA | Limb-Girdle Muscular Dystrophy, Type 2D | Genetic: SRP-9004 | Sarepta Therapeutics, Inc. | Nationwide Children's Hospital | Completed | 7 Years | N/A | All | 6 | Phase 1/Phase 2 | United States |
| 346 | NCT02383511 (ClinicalTrials.gov) | February 2015 | 4/2/2015 | Modified Diet Trial: A Study of SMT C1100 in Paediatric Patients With DMD Who Follow a Balanced Diet | A Phase 1b Placebo-controlled, Multi-centre, Randomized, Double-blind Dose Escalation Study to Evaluate the Pharmacokinetics (PK) and Safety of SMT C1100 in Patients With Duchenne Muscular Dystrophy (DMD) Who Follow a Balanced Diet | Muscular Dystrophy, Duchenne | Drug: SMT C1100 | Summit Therapeutics | NULL | Completed | 5 Years | 13 Years | Male | 12 | Phase 1 | United Kingdom |
| 347 | EUCTR2013-003605-26-BE (EUCTR) | 23/01/2015 | 04/12/2014 | A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01) | A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 | Prosensa Therapeutics B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 348 | NCT02310906 (ClinicalTrials.gov) | January 13, 2015 | 3/12/2014 | Phase I/II Study of SRP-4053 in DMD Patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping | Duchenne Muscular Dystrophy | Drug: Placebo;Drug: SRP-4053 | Sarepta Therapeutics, Inc. | Institut de Myologie, France;Consultants for Research in Imaging and Spectroscopy;Great Ormond Street Hospital for Children NHS Foundation Trust;Catholic University of the Sacred Heart;Royal Holloway University;SYSNAV;University College, London;University of Newcastle Upon-Tyne | Completed | 6 Years | 15 Years | Male | 39 | Phase 1/Phase 2 | United States;France;Italy;United Kingdom |
| 349 | EUCTR2013-005489-20-CZ (EUCTR) | 08/01/2015 | 24/10/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 350 | NCT02354781 (ClinicalTrials.gov) | January 2015 | 26/1/2015 | Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy | Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: rAAV1.CMV.huFollistin344 | Jerry R. Mendell | Duchenne Alliance;Milo Therapeutics | Completed | 7 Years | N/A | Male | 3 | Phase 1/Phase 2 | United States |
| 351 | NCT02328482 (ClinicalTrials.gov) | January 2015 | 25/12/2014 | Continuation Protocol to Protocol BBCO-001 | A Pivotal, Multicenter, Open-label, Randomized Withdrawal, Non-Treatment Concurrent Control Study to Assess the Safety, Tolerability, and Efficacy of Cabaletta® in OPMD Patients Who Participated in Study BBCO-001 | Muscular Dystrophy, Oculopharyngeal (OPMD) | Drug: Tehalose 30gr | Bioblast Pharma Ltd. | NULL | Completed | 18 Years | 80 Years | All | 9 | Phase 3 | Canada |
| 352 | JPRN-UMIN000016092 | 2014/12/31 | 31/12/2014 | Analysis of the effects of animal fat free diet and aspirin for prostaglandins in patients with myotonic dystrophy | Analysis of the effects of animal fat free diet and aspirin for prostaglandins in patients with myotonic dystrophy - Animal fat free diet and aspirin in patients with myotonic dystrophy | myotonic dystrophy | 1st stage (1w): animal fat free diet 2nd stage (1w): animal fat free diet and aspirin (330mg/day) | National Hospital Organization Toneyama National Hospital | Graduate school of engineering Osaka University | Complete: follow-up complete | Not applicable | Not applicable | Male and Female | 10 | Not applicable | Japan |
| 353 | EUCTR2014-002072-92-GB (EUCTR) | 30/12/2014 | 16/09/2014 | A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy | A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 19.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 INN or Proposed INN: PF-06252616 Product Code: PF-06252616 INN or Proposed INN: PF-06252616 | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | NULL | Not Recruiting | Female: no Male: yes | 105 | Phase 2 | United States;Canada;Poland;Australia;Bulgaria;Japan;Italy;United Kingdom | ||
| 354 | EUCTR2013-003605-26-IT (EUCTR) | 02/12/2014 | 11/09/2014 | A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01) | A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 | Prosensa Therapeutics B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Italy;Sweden | ||
| 355 | EUCTR2014-003100-78-GB (EUCTR) | 01/12/2014 | 19/09/2014 | A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet. | SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet. | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 INN or Proposed INN: Not available Other descriptive name: SMT C1100 | Summit (Oxford) Limited | NULL | Not Recruiting | Female: no Male: yes | 12 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 356 | NCT02251600 (ClinicalTrials.gov) | December 2014 | 22/9/2014 | A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | A Multi-center Study to Evaluate the Pharmacokinetics of 21-Desacetyldeflazacort and the Safety of Deflazacort After Oral Administration of Deflazacort Tablets to Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | 16 Years | Male | 24 | Phase 1 | United States |
| 357 | NCT02312011 (ClinicalTrials.gov) | December 2014 | 4/12/2014 | A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 | A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients With Myotonic Dystrophy Type 1 | Myotonic Dystrophy Type 1 | Drug: IONIS-DMPKRx;Drug: Placebo | Ionis Pharmaceuticals, Inc. | NULL | Completed | 20 Years | 55 Years | All | 48 | Phase 1/Phase 2 | United States |
| 358 | NCT01805024 (ClinicalTrials.gov) | December 2014 | 4/3/2013 | Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO) | Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO) | Congenital Muscular Dystrophy | Drug: Omigapil | Santhera Pharmaceuticals | NULL | Completed | 5 Years | 16 Years | All | 20 | Phase 1 | United States |
| 359 | NCT02295748 (ClinicalTrials.gov) | December 2014 | 18/11/2014 | An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort | An Open-Label, Multi-Center, Long-Term Extension Study to Evaluate the Safety and Tolerability of Orally Administrated Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Deflazacort | PTC Therapeutics | NULL | Completed | 4 Years | N/A | Male | 24 | Phase 1 | United States |
| 360 | NCT02329769 (ClinicalTrials.gov) | December 2014 | 22/12/2014 | Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC 6 mg/kg;Drug: PRO044 IV 6 mg/kg;Drug: PRO044 IV 9 mg/kg | BioMarin Pharmaceutical | NULL | Terminated | 9 Years | 20 Years | Male | 15 | Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 361 | EUCTR2013-003605-26-SE (EUCTR) | 26/11/2014 | 26/09/2014 | A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01) | A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: PS188 (company code) Other descriptive name: PS188 | BioMarin Nederland B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | ||
| 362 | NCT02310763 (ClinicalTrials.gov) | November 24, 2014 | 4/11/2014 | A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy | A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Biological: PF-06252616;Drug: Placebo | Pfizer | NULL | Terminated | 6 Years | 15 Years | Male | 121 | Phase 2 | United States;Australia;Bulgaria;Canada;Italy;Japan;Poland;United Kingdom |
| 363 | NCT02255552 (ClinicalTrials.gov) | November 17, 2014 | 25/9/2014 | Study of Eteplirsen in DMD Patients | An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 16 Years | Male | 109 | Phase 3 | United States |
| 364 | EUCTR2014-002008-25-IT (EUCTR) | 13/11/2014 | 10/09/2014 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Phase 1;Phase 2 | France;United Kingdom;Italy | ||
| 365 | NCT02286947 (ClinicalTrials.gov) | November 2014 | 30/10/2014 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Eteplirsen | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 21 Years | Male | 24 | Phase 2 | United States |
| 366 | NCT02036463 (ClinicalTrials.gov) | November 2014 | 6/1/2014 | A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy | CINRG0513: A Trial of Chronotherapy of Corticosteroids in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: Prednisone;Drug: Placebo | Ann & Robert H Lurie Children's Hospital of Chicago | Children's Research Institute | Withdrawn | 3 Years | 6 Years | Male | 0 | Phase 2 | United States |
| 367 | EUCTR2014-002210-23-DK (EUCTR) | 14/10/2014 | 02/06/2014 | Can local botulinum toxin improve swallowing difficulties in persons with msucle disease? | Can local intramuscular botulinum toxin improve dysphagia in patients with myopathic dysphagia and constriction of the cricoid muscle? | Oculopharyngesl muscle dystrophy, inclusion body myositis MedDRA version: 18.1;Level: LLT;Classification code 10019897;Term: Hereditary progressive muscular dystrophy;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Xeomin Product Name: Xeomin Other descriptive name: BOTULINUM TOXIN TYPE A | Rigshospitalet | NULL | Not Recruiting | Female: yes Male: yes | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | Denmark | ||
| 368 | EUCTR2013-004427-37-IT (EUCTR) | 01/10/2014 | 04/09/2014 | Efficacy and tolerability of ibuprofen and isosorbide dinitrate (20 mg, 40 mg, 60 mg, 80 mg) | Multicentre, randomised, double-blind, dose titration design in patients with Duchenne muscular dystrophy to evaluate the efficacy and the tolerability of the combinations of Ibuprofen (200 mg bid) and Isosorbide Dinitrate (20 mg, 40 mg, 60 mg, 80 mg) - ESPP001 | Restrictive respiratory syndrome with frequent pulmonary infections and cardiomyopathy.wasting of skeletal muscle, severe local inflammation and, at least initially, muscle regeneration. MedDRA version: 17.0;Level: PT;Classification code 10064571;Term: Gene mutation;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Isosorbide Dinitrate 20 mg Product Name: Isosorbide Dinitrate INN or Proposed INN: ISOSORBIDE DINITRATE Trade Name: IBUPROFEN 200MG INN or Proposed INN: IBUPROFEN Trade Name: Isosorbide Dinitrate 40 mg Product Name: Isosorbide Dinitrate INN or Proposed INN: ISOSORBIDE DINITRATE Trade Name: Isosorbide Dinitrate 60 mg Product Name: Isosorbide Dinitrate INN or Proposed INN: ISOSORBIDE DINITRATE Trade Name: Isosorbide Dinitrate 80 mg Product Name: Isosorbide Dinitrate INN or Proposed INN: ISOSORBIDE DINITRATE | PARENT PROJECT ONLUS | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 188 | Italy | |||
| 369 | EUCTR2013-005489-20-DE (EUCTR) | 29/09/2014 | 28/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 370 | EUCTR2014-001753-17-NL (EUCTR) | 19/09/2014 | 03/06/2014 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic Myopathy | A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies | Facioscapulohumeral muscular dystrophy MedDRA version: 17.1;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 INN or Proposed INN: N/A | aTyr Pharma, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;France;Netherlands;Italy | ||
| 371 | EUCTR2014-002008-25-GB (EUCTR) | 10/09/2014 | 08/09/2014 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not Available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 48 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Italy;United Kingdom | ||
| 372 | EUCTR2013-005489-20-IT (EUCTR) | 10/09/2014 | 30/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 373 | EUCTR2013-005489-20-ES (EUCTR) | 01/09/2014 | 06/08/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 374 | NCT01834066 (ClinicalTrials.gov) | September 2014 | 26/2/2013 | Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. | Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial | Muscular Dystrophy;Duchenne Muscular Dystrophy, | Biological: Stem Cell | Chaitanya Hospital, Pune | NULL | Recruiting | 6 Years | 25 Years | Both | 25 | Phase 1/Phase 2 | India |
| 375 | NCT02246478 (ClinicalTrials.gov) | September 2014 | 9/9/2014 | A Study of TAS-205 for Duchenne Muscular Dystrophy | A Phase I Study of Single and Multiple Doses of TAS-205 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: TAS-205;Drug: Placebo | Taiho Pharmaceutical Co., Ltd. | NULL | Completed | 5 Years | 15 Years | Male | 21 | Phase 1 | Japan |
| 376 | NCT02235844 (ClinicalTrials.gov) | September 2014 | 8/9/2014 | Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) | Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) | Duchenne's Muscular Dystrophy | Biological: Umbilical Cord Mesenchymal Stem Cells | Allergy and Asthma Consultants, Wichita, Kansas | Aidan Foundation;Neil H. Riordan PhD | Completed | 28 Years | 31 Years | Male | 1 | Phase 1 | United States |
| 377 | EUCTR2013-005489-20-BE (EUCTR) | 28/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 378 | EUCTR2013-005489-20-SE (EUCTR) | 22/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Sweden;Korea, Republic of | ||
| 379 | EUCTR2013-005489-20-GB (EUCTR) | 22/08/2014 | 11/07/2014 | Extension study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Brazil;Belgium;Australia;Bulgaria;Germany;Korea, Republic of;Sweden | ||
| 380 | NCT02239224 (ClinicalTrials.gov) | August 2014 | 7/9/2014 | Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients With Muscular Dystrophy | A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular Dystrophies | Facioscapulohumeral Muscular Dystrophy (FSHD) | Biological: Placebo;Biological: ATYR1940 | aTyr Pharma, Inc. | NULL | Completed | 18 Years | 65 Years | All | 20 | Phase 1/Phase 2 | United States;France;Italy;Netherlands |
| 381 | NCT02251457 (ClinicalTrials.gov) | August 2014 | 25/9/2014 | Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1 | Open Label Trial of Ranolazine in Myotonia Congenita, Paramyotonia Congenita, & Myotonic Dystrophy Type 1 | Myotonia Congenita;Paramyotonia Congenita;Myotonic Dystrophy 1 | Drug: Ranolazine | Ohio State University | Gilead Sciences | Completed | 18 Years | 100 Years | All | 35 | Phase 1 | United States |
| 382 | EUCTR2014-001753-17-IT (EUCTR) | 30/07/2014 | 13/06/2014 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic Myopathy | A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies | Facioscapulohumeral muscular dystrophy MedDRA version: 17.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 INN or Proposed INN: N/A | aTyr Pharma, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 44 | Phase 1;Phase 2 | United States;France;Netherlands;Italy | ||
| 383 | JPRN-UMIN000014836 | 2014/05/01 | 12/08/2014 | A pilot study to evaluate efficacy and safety of resveratrol for muscular dystrophy. | A pilot study to evaluate efficacy and safety of resveratrol for muscular dystrophy. - A pilot study of resveratrol for muscular dystrophy. | Duchenne muscular dystrophy, Becker muscular dystrophy, Fukuyama congenital muscular dystrophy | 500mg resveratrol is administrated daily for 8 weeks. Subsequently, resveratrol is increased to 1500mg by 500mg every 8 weeks. | Sapporo Medical University | NULL | Complete: follow-up complete | 12years-old | Not applicable | Male and Female | 10 | Not selected | Japan |
| 384 | NCT02196467 (ClinicalTrials.gov) | May 2014 | 9/5/2014 | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy | Biological: Myoblast transplantation;Procedure: Saline injection | CHU de Quebec-Universite Laval | NULL | Recruiting | 16 Years | N/A | Male | 10 | Phase 1/Phase 2 | Canada |
| 385 | NCT02167217 (ClinicalTrials.gov) | April 17, 2014 | 3/2/2014 | Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy | Phase 2 Historically Controlled Trial of Corticosteroids in Young Boys With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Prednisolone | Washington University School of Medicine | Nationwide Children's Hospital;Feinberg School of Medicine, Northwestern University;University of Texas Southwestern Medical Center;University of California, Davis;Nemours Hospital, Orlando, FL | Completed | 1 Month | 30 Months | Male | 25 | Phase 2 | United States |
| 386 | NCT02090959 (ClinicalTrials.gov) | March 20, 2014 | 17/3/2014 | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy | A Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Dystrophinopathy | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren | PTC Therapeutics | NULL | Terminated | 7 Years | 15 Years | Male | 219 | Phase 3 | United States;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Czech Republic |
| 387 | EUCTR2012-004527-20-PL (EUCTR) | 12/02/2014 | 27/11/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 388 | NCT02015481 (ClinicalTrials.gov) | February 2014 | 8/12/2013 | Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients | Multi-Center, Dose-Escalation Study, to Assess Safety, Tolerability and Efficacy of Intravenous Cabaletta® in OPMD Patients | Oculopharyngeal Muscular Dystrophy | Drug: Cabaletta | Bioblast Pharma Ltd. | NULL | Completed | 18 Years | 80 Years | All | 25 | Phase 2 | United States;Canada;Israel |
| 389 | NCT01890798 (ClinicalTrials.gov) | January 2014 | 27/6/2013 | Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol | A Continued Access Protocol for Eligible US Subjects With Duchenne Muscular Dystrophy Who Previously Participated in an Approved Drisapersen Study | Muscular Dystrophies | Drug: Drisapersen | GlaxoSmithKline | NULL | Withdrawn | 5 Years | N/A | Male | 0 | Phase 3 | NULL |
| 390 | EUCTR2013-001194-25-NL (EUCTR) | 16/12/2013 | 17/07/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Eli Lilly and Company | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 391 | NCT02285673 (ClinicalTrials.gov) | November 2013 | 5/11/2014 | Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy | Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study | Duchenne Muscular Dystrophy | Biological: Umbilical Cord Mesenchymal Stem Cell | Acibadem University | NULL | Recruiting | 7 Years | 20 Years | Male | 10 | Phase 1/Phase 2 | Turkey |
| 392 | NCT02056808 (ClinicalTrials.gov) | November 2013 | 21/11/2013 | A Phase 1b Study of SMT C1100 in Subjects With Duchenne Muscular Dystrophy (DMD) | SMT C1100 - A Phase 1b, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: SMT C1100 | Summit Corporation Plc. | NULL | Completed | 5 Years | 11 Years | Male | 12 | Phase 1 | United Kingdom |
| 393 | NCT02847975 (ClinicalTrials.gov) | October 2013 | 30/4/2014 | Sodium Nitrate to Improve Blood Flow | Sodium Nitrate to Improve Blood Flow | Becker Muscular Dystrophy | Dietary Supplement: Sodium nitrate | Cedars-Sinai Medical Center | NULL | Completed | 15 Years | 55 Years | Male | 11 | Phase 1 | United States |
| 394 | NCT01995032 (ClinicalTrials.gov) | October 2013 | 20/11/2013 | L-citrulline and Metformin in Duchenne's Muscular Dystrophy | A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne's Muscular Dystrophy | Duchenne's Muscular Dystrophy (DMD) | Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.;Drug: Placebo | University Hospital, Basel, Switzerland | NULL | Completed | 78 Months | 10 Years | All | 47 | Phase 3 | Switzerland |
| 395 | NCT01970735 (ClinicalTrials.gov) | October 2013 | 23/10/2013 | Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2 | Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2 | Muscular Dystrophy, Facioscapulohumeral | Biological: Blood test | Centre Hospitalier Universitaire de Nice | NULL | Recruiting | 18 Years | 75 Years | Both | 100 | N/A | France |
| 396 | NCT01978366 (ClinicalTrials.gov) | October 2013 | 31/10/2013 | Open Label Extension Study of HT-100 in Patients With DMD | An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01 | Duchenne Muscular Dystrophy | Drug: HT-100 | Processa Pharmaceuticals | NULL | Terminated | 6 Years | 20 Years | Male | 17 | Phase 2 | United States |
| 397 | NCT02147639 (ClinicalTrials.gov) | October 2013 | 25/3/2014 | Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Dietary Supplement: Sodium Nitrate;Dietary Supplement: Sodium Nitrate - double dose;Dietary Supplement: Placebo;Procedure: Increased exercise intensity | Cedars-Sinai Medical Center | NULL | Completed | 15 Years | 45 Years | Male | 19 | Phase 2/Phase 3 | United States | |
| 398 | EUCTR2013-001194-25-GB (EUCTR) | 25/09/2013 | 05/06/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Eli Lilly and Company | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 399 | EUCTR2013-001194-25-BE (EUCTR) | 19/09/2013 | 28/06/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Eli Lilly and Company | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 400 | EUCTR2013-001732-21-FR (EUCTR) | 06/09/2013 | 18/02/2014 | A phase II study of metformin in myotonic dystrophy type 1 patients | A randomized, double blind, placebo-controlled phase II study of metformin in myotonic dystrophy type 1 patients - Myomet | Myotonic dystrophy type 1 (DM1) also known as Steinert disease;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: metformin 500mg INN or Proposed INN: METFORMIN | Centre d'Etude des Cellules Souches (CECS) | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France | ||
| 401 | EUCTR2013-001194-25-DE (EUCTR) | 03/09/2013 | 13/06/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Eli Lilly and Company | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of | ||
| 402 | NCT01865084 (ClinicalTrials.gov) | September 2013 | 24/5/2013 | A Study of Tadalafil for Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Tadalafil;Drug: Placebo | Eli Lilly and Company | NULL | Terminated | 7 Years | 14 Years | Male | 331 | Phase 3 | United States;Argentina;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Puerto Rico;Russian Federation;Spain;Taiwan;Turkey;United Kingdom |
| 403 | EUCTR2012-004527-20-SE (EUCTR) | 30/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Sweden;Korea, Republic of | ||
| 404 | EUCTR2012-004527-20-CZ (EUCTR) | 30/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 405 | EUCTR2013-001194-25-ES (EUCTR) | 27/08/2013 | 26/06/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Lilly S.A. | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 406 | EUCTR2011-005042-35-GB (EUCTR) | 23/08/2013 | 18/03/2013 | A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 053 Product Code: BMN 053 INN or Proposed INN: PS524 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 45 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 407 | EUCTR2013-002115-99-GB (EUCTR) | 23/08/2013 | 31/07/2013 | A Clinical Trial to investigate if the drug SMT C1100 is safe and well tolerated in children with DMD when given orally one to three times a day. | SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: SMT C1100 INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole | Summit Corporation plc | NULL | Not Recruiting | Female: no Male: yes | Phase 1 | United Kingdom | |||
| 408 | EUCTR2012-004527-20-DE (EUCTR) | 20/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 409 | JPRN-JMA-IIA00134 | 14/08/2013 | 05/08/2013 | Phase II study of nonsense readthrough compound NPC-14 (Arbekacin sulfate) to explore safety, tolerability, and efficacy in Duchenne muscular dystrophy patients (NORTH POLE DMD study) | Phase II study of nonsense readthrough compound NPC-14 (Arbekacin sulfate) to explore safety, tolerability, and efficacy in Duchenne muscular dystrophy patients (NORTH POLE DMD study) | Duchenne muscular dystrophy | Intervention type:DRUG. Intervention1:NPC-14, Dose form:INJECTION, Route of administration:INTRAVENOUS DRIP, intended dose regimen:Not applicable. Control intervention1:Placebo, Dose form:INJECTION, Route of administration:INTRAVENOUS DRIP, Intended dose regimen:Not applicable. | Yasuhiro Takeshima, MD, PhDHyogo College of Medicine Hospital, department of pediatrics | Not applicable | Completed | >=4 YEARS | No Limit | Male | 21 | Phase 2 | Japan |
| 410 | EUCTR2013-001194-25-IT (EUCTR) | 09/08/2013 | 11/06/2013 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 5 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 10 mg film-coated tablets INN or Proposed INN: TADALAFIL Trade Name: Cialis 20 mg film-coated tablets INN or Proposed INN: TADALAFIL | Eli Lilly and Company | NULL | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of | ||
| 411 | EUCTR2012-004527-20-GB (EUCTR) | 08/08/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 412 | NCT01921374 (ClinicalTrials.gov) | August 2013 | 8/8/2013 | Mother-caregivers of Children With Duchenne Muscular Dystrophy | Profile of Mother-caregivers of Children With Duchenne Muscular Dystrophy | Other Diseases or Conditions | Biological: sleep parameters;Biological: Hormonal profile;Biological: Inflammatory profile;Biological: Cardiovascular profile;Biological: Metabolic profile | Monica Levy Andersen | NULL | Completed | 25 Years | 65 Years | Female | 60 | N/A | Brazil |
| 413 | NCT01918384 (ClinicalTrials.gov) | August 2013 | 1/8/2013 | Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy | Phase II Study of Nonsense Readthrough Compound NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy Patients (NORTH POLE DMD Study) | Muscular Dystrophy, Duchenne | Drug: NPC-14;Drug: Placebo | Kobe University | Japan Medical Association;Nobelpharma | Active, not recruiting | 4 Years | N/A | Male | 21 | Phase 2 | Japan |
| 414 | EUCTR2012-004527-20-BE (EUCTR) | 30/07/2013 | 24/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.0;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;Switzerland;United Kingdom;Czech Republic;Canada;Poland;Belgium;Brazil;Australia;Germany;Korea, Republic of;Sweden | ||
| 415 | EUCTR2012-004527-20-ES (EUCTR) | 17/07/2013 | 12/07/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 416 | EUCTR2012-004527-20-IT (EUCTR) | 01/07/2013 | 27/06/2013 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 16.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | United States;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Switzerland;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | ||
| 417 | EUCTR2011-005042-35-BE (EUCTR) | 26/06/2013 | 21/03/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN053 Product Code: BMN053 INN or Proposed INN: PS524 | BioMarin Nederland B.V. | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 418 | EUCTR2011-005042-35-IT (EUCTR) | 20/06/2013 | 22/03/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 15.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO053 Product Code: PRO053 INN or Proposed INN: PS524 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 419 | EUCTR2011-005042-35-NL (EUCTR) | 13/06/2013 | 15/08/2013 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO053 Product Code: PRO053 INN or Proposed INN: PS524 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Israel;Chile;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | ||
| 420 | NCT02081625 (ClinicalTrials.gov) | June 2013 | 5/3/2014 | Exploratory Study of NS-065/NCNP-01 in DMD | Exploratory Study of NS-065/NCNP-01 in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: NS-065/NCNP-01 | National Center of Neurology and Psychiatry, Japan | Nippon Shinyaku Co., Ltd. | Completed | 5 Years | 18 Years | Male | 10 | Phase 1 | Japan |
| 421 | NCT01957059 (ClinicalTrials.gov) | June 2013 | 2/7/2013 | A Phase I/II Study of BMN053 in Subjects With Duchenne Muscular Dystrophy (DMD) | A Phase I/II, Open-label, Dose Escalating With 48 Week Treatment Study to Assess the Safety and Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of BMN053 (Previously Known as PRO053) in Subjects With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Drug: Regimen Selection Phase Group 2;Drug: Regimen Selection Phase Group 3;Drug: Treatment Phase Group 4;Drug: Regimen Selection Phase Group 1 (COMPLETED);Drug: Dosing Extension | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 18 Years | Male | 9 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom |
| 422 | NCT02018731 (ClinicalTrials.gov) | June 2013 | 17/12/2013 | L-citrulline and Metformin in Becker's Muscular Dystrophy | Pilot Study to Assess the Efficacy of L-Citrulline and Metformin in Adults With Becker's Muscular Dystrophy | Becker's Muscular Dystrophy (BMD) | Drug: Metformin and Metformin & L-Citrulline;Drug: L-Citrulline and Metformin & L-Citrulline | University Hospital, Basel, Switzerland | NULL | Completed | 18 Years | N/A | Both | 20 | Phase 2 | Switzerland |
| 423 | EUCTR2011-005040-10-IT (EUCTR) | 16/05/2013 | 27/02/2013 | A study to test if multiple injections of PRO045 are safe and effective in people who suffer from Duchenne muscular dystrophy | A phase I/II, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of PRO045 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO045 Product Code: PRO045 INN or Proposed INN: PS220 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | Belgium;United Kingdom;Italy | ||
| 424 | NCT01803412 (ClinicalTrials.gov) | May 1, 2013 | 28/2/2013 | A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects | An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of Drisapersen in US and Canadian Subjects With Duchenne Muscular Dystrophy. | Muscular Dystrophies | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | N/A | Male | 53 | Phase 3 | United States;Canada |
| 425 | NCT01847573 (ClinicalTrials.gov) | May 2013 | 2/5/2013 | Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of HT-100 in Duchenne Muscular Dystrophy | A Phase 1b Open Label, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HT-100 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: HT-100 | Processa Pharmaceuticals | NULL | Terminated | 6 Years | 20 Years | Male | 17 | Phase 1/Phase 2 | United States |
| 426 | NCT01856868 (ClinicalTrials.gov) | May 2013 | 9/5/2013 | Use of (-)-Epicatechin in the Treatment of Becker Muscular Dystrophy (Pilot Study) | An Open-label Pilot Study of Purified Tea-derived Epicatechin to Improve Mitochondrial Function, Strength and Skeletal Muscle Exercise Response in Becker Muscular Dystrophy. | Becker Muscular Dystrophy | Drug: (-)-epicatechin | Craig McDonald, MD | Cardero Therapeutics, Inc. | Completed | 18 Years | 60 Years | Male | 7 | Phase 1/Phase 2 | United States |
| 427 | EUCTR2011-005040-10-GB (EUCTR) | 22/04/2013 | 10/10/2012 | A study to test if multiple injections of BMN 045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophy | A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of BMN 045 (previously known as PRO045) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by BMN 045-induced DMD exon 45 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 045 Product Code: BMN 045 INN or Proposed INN: PS220 | BioMarin Pharmaceutical Inc. | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | France;Belgium;Italy;United Kingdom | ||
| 428 | NCT01761292 (ClinicalTrials.gov) | April 2013 | 20/12/2012 | A Study to Assess Safety/Tolerability, pk, Effects on Histology, Clinical Parameters of Givinostat in Children With DMD | A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: Givinostat | Italfarmaco | NULL | Completed | 7 Years | 11 Years | Male | 20 | Phase 1/Phase 2 | Italy |
| 429 | NCT01826487 (ClinicalTrials.gov) | March 26, 2013 | 26/3/2013 | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | A Phase 3 Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Dystrophinopathy | Muscular Dystrophy, Duchenne;Muscular Dystrophies;Muscular Disorders, Atrophic;Muscular Diseases;Musculoskeletal Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | NULL | Completed | 7 Years | 16 Years | Male | 230 | Phase 3 | United States;Australia;Belgium;Brazil;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Switzerland;Turkey;United Kingdom;Bulgaria;Czech Republic |
| 430 | NCT02814110 (ClinicalTrials.gov) | March 1, 2013 | 23/6/2016 | Efficacy Safety of Granulocyte Colony-stimulating Factor Treatment Children and Adolescents With Muscular Dystrophy | Efficacy and the Safety of Granulocyte Colony-stimulating Factor Treatment in Children and Adolescents With Muscular Dystrophy: An Open Study | Increase Muscle Strength in Patients With Muscular Dystrophy | Drug: Granulocyte colony-stimulating factor (Filgrastim) | Medical University of Bialystok | NULL | Recruiting | 5 Years | 15 Years | All | 27 | Phase 1 | Poland |
| 431 | NCT01826422 (ClinicalTrials.gov) | March 2013 | 4/4/2013 | Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients | Effect of Eicosapentaenoic Fatty Acid (EPA) and Docosahexaenoic Fatty Acids (DHA) Supplementation on the Inflammation State and Metabolic Disorders in Patients With Duchenne Muscular Dystrophy or Becker Muscular Dystrophy | Muscular Dystrophy, Duchenne | Dietary Supplement: EPA and DHA;Dietary Supplement: Placebo Comparator | Coordinación de Investigación en Salud, Mexico | Instituto Nacional de Rehabilitacion | Completed | 6 Years | 18 Years | Male | 40 | N/A | Mexico |
| 432 | EUCTR2010-023744-33-IT (EUCTR) | 26/02/2013 | 28/12/2012 | Duchenne muscular dystrophy: a clinical trial to find the optimum steroid regimen. | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen. - FOR-DMD | Duchenne muscular dystrophy boys between 4 and 8 years of age, able to rise from the floor without support, not previously treated with steroids. MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DELTACORTENE*20CPR 5MG INN or Proposed INN: PREDNISONE Trade Name: DEFLAN*10CPR 6MG INN or Proposed INN: DEFLAZACORT | AZIENDA OSPEDALIERA DI PADOVA | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;United Kingdom;Italy | |||
| 433 | EUCTR2012-002566-12-IT (EUCTR) | 04/02/2013 | 17/10/2012 | A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy | A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy - DSC | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: SOC;Classification code 10028395;Term: Musculoskeletal and connective tissue disorders;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat Product Code: ITF2357 INN or Proposed INN: Givinostat Other descriptive name: givinostat Product Name: Givinostat Product Code: ITF2357 INN or Proposed INN: Givinostat Other descriptive name: Givinostat | ITALFARMACO | NULL | Not Recruiting | Female: no Male: yes | 20 | Phase 2 | Italy | ||
| 434 | NCT03433807 (ClinicalTrials.gov) | January 29, 2013 | 8/2/2018 | Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) | Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Idebenone | Santhera Pharmaceuticals | NULL | Temporarily not available | 8 Years | N/A | All | United States | ||
| 435 | NCT01826474 (ClinicalTrials.gov) | January 2013 | 20/3/2013 | Phase IIb Study of PRO045 in Subjects With Duchenne Muscular Dystrophy | A Phase IIb, Open-label Study to Assess the Efficacy, Safety, Pharmacodynamics and Pharmacokinetics of Multiple Subcutaneous Doses of PRO045 in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO045, 0.15 mg/kg/week;Drug: PRO045, 1.0 mg/kg/week;Drug: PRO045, 3.0 mg/kg/week;Drug: PRO045, 6.0 mg/kg/week;Drug: PRO045, 9.0 mg/kg/week;Drug: PRO045, selected dose | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 18 Years | Male | 15 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom |
| 436 | EUCTR2011-001266-17-CZ (EUCTR) | 20/12/2012 | 30/10/2012 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 INN or Proposed INN: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 437 | EUCTR2011-005040-10-BE (EUCTR) | 03/12/2012 | 04/09/2012 | A study to test if multiple injections of PRO045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophy | A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping MedDRA version: 18.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO045 Product Code: PRO045 INN or Proposed INN: PS220 | BioMarin Nederland B.V. | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | Belgium;Italy;United Kingdom | ||
| 438 | EUCTR2010-023744-33-GB (EUCTR) | 12/11/2012 | 20/09/2012 | Trial to find best steroid treatment for Duchenne muscular dystrophy | Duchenne muscular dystrophy: double-blind randomized trial to find optimum steroid regimen - FOR-DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Deflazacort Product Name: Deflazacort 6 mg Tablets INN or Proposed INN: Deflazacort Trade Name: Prednisone Product Name: Prednisone 5 mg tablets INN or Proposed INN: Prednisone | University of Rochester | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 300 | United States;Canada;Germany;Italy;United Kingdom | |||
| 439 | JPRN-UMIN000009307 | 2012/11/08 | 10/11/2012 | Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy | Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy - Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy | Duchenne muscular dystrophy | oral adm,inistration of aspirin | Kobe University Graduate School of Medicine | NULL | Complete: follow-up complete | 5years-old | Not applicable | Male | 6 | Phase 1,2 | Japan |
| 440 | EUCTR2011-001266-17-DK (EUCTR) | 11/10/2012 | 11/10/2012 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 15.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Phase 3 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 441 | EUCTR2011-004853-18-IT (EUCTR) | 18/09/2012 | 27/09/2012 | An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy | An Open-Label Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 15.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 15.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na Product Name: ataluren Product Code: PTC124 INN or Proposed INN: na | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;United Kingdom;Italy;Sweden | ||
| 442 | EUCTR2011-004853-18-GB (EUCTR) | 21/08/2012 | 29/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 443 | EUCTR2011-001266-17-BG (EUCTR) | 09/08/2012 | 29/05/2012 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 444 | EUCTR2011-001266-17-HU (EUCTR) | 02/08/2012 | 18/06/2012 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 INN or Proposed INN: NA | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Czech Republic;Hungary;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 445 | EUCTR2009-012037-30-ES (EUCTR) | 27/06/2012 | 29/03/2012 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 ? 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 280 | Phase 3 | France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden | ||
| 446 | EUCTR2011-004853-18-ES (EUCTR) | 13/06/2012 | 17/04/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Belgium;Spain;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 447 | EUCTR2010-020069-26-HU (EUCTR) | 25/05/2012 | 28/02/2012 | A phase III, randomized, double blind, placebo-controlledclinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - N/A | A phase III, randomized, double blind, placebo-controlledclinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - N/A | Duchenne muscular dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Japan;Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Norway;Netherlands;Germany | ||
| 448 | NCT01557400 (ClinicalTrials.gov) | May 20, 2012 | 15/3/2012 | Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Dystrophinopathy | Drug: Ataluren | PTC Therapeutics | NULL | Completed | N/A | N/A | Male | 94 | Phase 3 | Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 449 | EUCTR2011-004853-18-DE (EUCTR) | 02/05/2012 | 12/03/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 450 | EUCTR2011-004853-18-FR (EUCTR) | 02/05/2012 | 13/08/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 14.1;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 451 | EUCTR2011-004853-18-BE (EUCTR) | 27/04/2012 | 03/01/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 16.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 452 | EUCTR2011-004853-18-SE (EUCTR) | 26/04/2012 | 27/02/2012 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 17.1;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | ||
| 453 | EUCTR2009-012037-30-IT (EUCTR) | 27/03/2012 | 26/04/2012 | (DELOS) DuchEnne MD Long-term IdebenOne Study | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Idebenone Product Code: NA INN or Proposed INN: IDEBENONE | SANTHERA PHARMACEUTICALS (SWITZERLAND) LTD | NULL | Not Recruiting | Female: no Male: yes | 280 | Phase 3 | France;United States;Canada;Spain;Belgium;Austria;Netherlands;Germany;Italy;Sweden | ||
| 454 | EUCTR2011-001266-17-IT (EUCTR) | 20/03/2012 | 08/03/2012 | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 INN or Proposed INN: NA Other descriptive name: NA | GLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD. | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Russian Federation;Chile;Israel;United Kingdom;Italy;Czech Republic;Hungary;Canada;Argentina;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of | |||
| 455 | NCT03076814 (ClinicalTrials.gov) | March 2012 | 23/9/2014 | Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Tadalafil;Other: Placebo | Cedars-Sinai Medical Center | NULL | Withdrawn | 15 Years | 55 Years | Male | 0 | N/A | NULL | |
| 456 | NCT01580501 (ClinicalTrials.gov) | March 2012 | 17/4/2012 | PDE Inhibitors in DMD Study (Acute Dosing Study) | Functional Muscle Ischemia and PDE5 Inhibition in Duchenne Muscular Dystrophy: Acute Dosing Study | Duchenne Muscular Dystrophy | Drug: Tadalafil and Sildenafil | Cedars-Sinai Medical Center | NULL | Completed | 7 Years | 15 Years | Male | 12 | Phase 1 | United States |
| 457 | NCT02207283 (ClinicalTrials.gov) | March 2012 | 28/7/2014 | PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular Dystrophy | PDE5 Inhibition to Alleviate Functional Muscle Ischemia in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Tadalafil;Drug: Placebo | Cedars-Sinai Medical Center | NULL | Completed | 15 Years | 55 Years | Male | 12 | Phase 4 | NULL |
| 458 | NCT01618331 (ClinicalTrials.gov) | March 2012 | 7/6/2012 | Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled Study | The Effect of Protein Supplementation Doing Regular Exercise in Patients With Facioscapulohumeral Muscular Dystrophy - a Blinded RCT Study | Facioscapulohumeral Muscle Dystrophy | Behavioral: Regular exercise;Dietary Supplement: Protein-carbohydrate supplementation | Grete Andersen, MD | The Augustinus Foundation, Denmark.;Aase and Ejnar Danielsens Foundation;The Danish Rheumatism Association;AP Moeller Foundation | Completed | 18 Years | 65 Years | Both | 42 | N/A | Denmark |
| 459 | NCT01540409 (ClinicalTrials.gov) | February 27, 2012 | 23/2/2012 | Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy | Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy Who Participated in Study 4658-US-201 | Duchenne Muscular Dystrophy (DMD) | Drug: AVI-4658 (Eteplirsen) | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 13 Years | Male | 12 | Phase 2 | United States |
| 460 | NCT01648634 (ClinicalTrials.gov) | February 13, 2012 | 20/7/2012 | Nebivolol for the Prevention of Left Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Multi-center Study to Examine the Effect of Nebivolol, a Beta-Blockade Drug, for the Prevention of Ventricular Systolic Dysfunction in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy;Cardiomyopathy;Heart Failure | Drug: Nebivolol;Drug: Placebo | Assistance Publique - Hôpitaux de Paris | Association Française contre les Myopathies (AFM), Paris | Completed | 10 Years | 15 Years | Male | 51 | Phase 3 | France |
| 461 | NCT01540604 (ClinicalTrials.gov) | February 2012 | 23/2/2012 | CRD007 for the Treatment of Duchenne Muscular Dystrophy, Becker Muscular Dystrophy and Symptomatic Carriers | An Open-label, Un-controlled, Single-centre Trial Investigating the Efficacy and Safety of CRD007 in Children With Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or Children Being Symptomatic Carriers for DMD or BMD | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: CRD007 | Cardoz AB | NULL | Completed | 2 Years | 11 Years | Both | Phase 2 | Sweden | |
| 462 | NCT01521546 (ClinicalTrials.gov) | February 2012 | 26/1/2012 | Eplerenone for Subclinical Cardiomyopathy in Duchenne Muscular Dystrophy | Early Treatment With Aldosterone Antagonism Attenuates Cardiomyopathy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: eplerenone;Drug: placebo | Subha Raman | Ballou Skies | Completed | 7 Years | N/A | Male | 42 | N/A | United States |
| 463 | NCT02516085 (ClinicalTrials.gov) | January 2012 | 3/8/2015 | Improved Muscle Function in Duchenne Muscular Dystrophy Through L-Arginine and Metformin | Duchenne Muscular Dystrophy | Drug: Metformin;Drug: L-Arginine | University Hospital, Basel, Switzerland | NULL | Completed | 7 Years | 10 Years | Both | 5 | Phase 1 | NULL | |
| 464 | NCT01388764 (ClinicalTrials.gov) | January 2012 | 5/7/2011 | Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids | Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids | Dystrophinopathy;Duchenne Muscular Dystrophy;Becker's Muscular Dystrophy | Drug: L-arginine | Massachusetts General Hospital | NULL | Completed | 7 Years | 11 Years | Male | 7 | Phase 1 | United States |
| 465 | NCT01519349 (ClinicalTrials.gov) | January 2012 | 23/1/2012 | Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis | Phase I Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis. | Becker Muscular Dystrophy;Sporadic Inclusion Body Myositis | Biological: rAAV1.CMV.huFollistatin344 | Nationwide Children's Hospital | Parent Project Muscular Dystrophy;The Myositis Association (Grant Sponsor) | Completed | 18 Years | N/A | All | 15 | Phase 1 | United States |
| 466 | EUCTR2011-004667-76-SE (EUCTR) | 19/12/2011 | 21/10/2011 | An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD. | An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers | Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1;Classification code 10059117;Term: Becker's muscular dystrophy;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: pemirolast Product Code: CRD007 INN or Proposed INN: PEMIROLAST | Cardoz AB | NULL | Not Recruiting | Female: yes Male: yes | Sweden | ||||
| 467 | EUCTR2011-001266-17-ES (EUCTR) | 18/11/2011 | 03/10/2011 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 INN or Proposed INN: GSK2402968 | GlaxoSmithKline, S.A. | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 468 | EUCTR2011-001266-17-DE (EUCTR) | 16/11/2011 | 31/08/2011 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 469 | EUCTR2011-001266-17-NL (EUCTR) | 03/11/2011 | 28/09/2011 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Germany;Netherlands;Norway;Japan;Korea, Republic of | |||
| 470 | NCT01350154 (ClinicalTrials.gov) | November 2011 | 4/5/2011 | Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients | Does Modulation of the nNOS System in Patients With Muscular Dystrophy and Defect nNOS Signalling Affect Cardiac, Muscular or Cognitive Function? | Becker Muscular Dystrophy | Drug: Sildenafil;Drug: Placebo | Rigshospitalet, Denmark | Glostrup University Hospital, Copenhagen | Completed | 18 Years | 80 Years | Male | 17 | Phase 2 | Denmark |
| 471 | NCT01462292 (ClinicalTrials.gov) | October 26, 2011 | 3/10/2011 | A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) | An Exploratory Study to Assess Two Doses of GSK2402968 in the Treatment of Ambulant Boys With Duchenne Muscular Dystrophy | Muscular Dystrophies | Drug: GSK2402968 3mg/kg/week;Drug: GSK2402968 6 mg/kg/week;Drug: Placebo to match GSK2402968 3 mg/kg/week;Drug: Placebo to match GSK2402968 6 mg/kg/week | GlaxoSmithKline | NULL | Completed | 5 Years | N/A | Male | 51 | Phase 2 | United States |
| 472 | EUCTR2011-001266-17-GB (EUCTR) | 03/10/2011 | 31/08/2011 | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 473 | NCT01478022 (ClinicalTrials.gov) | October 2011 | 14/11/2011 | To Compare the Pharmacokinetics Profiles of ISO 20, IBU 200 and IBU Plus ISO Combinations 200 + 20 | Open, Randomized, 3 Period Cross-over Design, in Healthy Volunteers to Compare the Pharmacokinetics Profiles of 3 Treatments: ISO 20, IBU 200 and IBU Plus ISO Combinations (200 + 20) Administered Per os as Single Doses | Duchenne Muscular Dystrophy (DMD) | Drug: Isosorbide Dinitrate;Drug: Ibuprofen;Other: Ibuprofen and Isosorbide Dinitrate combination | Parent Project, Italy | NULL | Completed | 18 Years | 27 Years | All | 12 | Phase 1 | Italy |
| 474 | NCT01610440 (ClinicalTrials.gov) | October 2011 | 21/5/2012 | Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy | Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: human umbilical cord mesenchymal stem cells | Shenzhen Beike Bio-Technology Co., Ltd. | The Second Affiliated Hospital of Kunming Medical University | Recruiting | 5 Years | 12 Years | Both | 15 | Phase 1/Phase 2 | China |
| 475 | NCT01480245 (ClinicalTrials.gov) | September 2011 | 23/11/2011 | Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy | An Open-label Extension Study of the Long-term Safety, Tolerability and Efficacy of GSK2402968 in Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies | Drug: GSK2402968 | GlaxoSmithKline | NULL | Terminated | 5 Years | N/A | Male | 233 | Phase 3 | Argentina;Australia;Belgium;Brazil;Bulgaria;Canada;Chile;Czech Republic;Denmark;France;Germany;Hungary;Israel;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;United Kingdom |
| 476 | EUCTR2011-001266-17-BE (EUCTR) | 08/08/2011 | 30/06/2011 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 16.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 200 | Phase 3 | Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Poland;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | ||
| 477 | EUCTR2010-024659-10-DK (EUCTR) | 03/08/2011 | 21/06/2011 | Can certain muscle wasting diseases be improved with sildenafil? | Can stimulation of the nNOS system in muscle disease with nNOS insufficiency improve heart and skeletal muscle function and cognition? | Muscular dystrophy with nNOS insufficiency MedDRA version: 14.0;Level: LLT;Classification code 10028301;Term: Muscle disorder NOS;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Revatio Product Name: Sildenafil Product Code: EMEA/H/C/000638 | Rigshospitalet | NULL | Not Recruiting | Female: no Male: yes | Denmark | ||||
| 478 | NCT01422200 (ClinicalTrials.gov) | August 2011 | 22/8/2011 | Flu Vaccine Study in Neuromuscular Patients 2011 | Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular Diseases | Duchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular Dystrophy | Biological: 2011-2012 seasonal flu vaccine Subcutaneous;Biological: 2011-2012 seasonal flu vaccine Intramuscular | Children's Hospital Medical Center, Cincinnati | NULL | Completed | 3 Years | 35 Years | All | 22 | Phase 4 | United States |
| 479 | NCT01645098 (ClinicalTrials.gov) | August 2011 | 8/6/2012 | Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy | Sedation During Muscle Biopsy in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Ketamine;Drug: Dexmedetomidine | Nationwide Children's Hospital | NULL | Completed | N/A | N/A | Male | 53 | N/A | United States |
| 480 | NCT01396239 (ClinicalTrials.gov) | July 2011 | 8/7/2011 | Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability and Pharmacokinetics Study of AVI-4658(Eteplirsen),in the Treatment of Ambulant Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: AVI-4658 (Eteplirsen);Other: Placebo | Sarepta Therapeutics, Inc. | NULL | Completed | 7 Years | 13 Years | Male | 12 | Phase 2 | United States |
| 481 | EUCTR2010-020069-26-DK (EUCTR) | 15/06/2011 | 18/05/2011 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan | ||
| 482 | NCT01406873 (ClinicalTrials.gov) | June 2011 | 20/7/2011 | Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 | A Randomized, Placebo Controlled, Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type-1 (DM1) | Myotonic Dystrophy | Drug: Mexiletine;Drug: Placebo | University of Rochester | NULL | Completed | 18 Years | 80 Years | All | 42 | Phase 2 | United States |
| 483 | NCT01359670 (ClinicalTrials.gov) | May 2011 | 23/5/2011 | Tadalafil and Sildenafil for Duchenne Muscular Dystrophy | Functional Muscle Ischemia and PDE5A Inhibition in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Tadalafil;Drug: Sildenafil | Cedars-Sinai Medical Center | Parent Project Muscular Dystrophy | Completed | 7 Years | 15 Years | Male | 30 | Early Phase 1 | United States |
| 484 | EUCTR2010-020069-26-ES (EUCTR) | 11/04/2011 | 18/02/2011 | Ensayo clínico de fase III, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad de GSK2402968 en sujetos con distrofia muscular de Duchenne.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Ensayo clínico de fase III, aleatorizado, doble ciego, controlado con placebo para evaluar la eficacia y seguridad de GSK2402968 en sujetos con distrofia muscular de Duchenne.A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 13;Term: Distrofia muscular de Duchenne | Product Code: GSK2402968 | GlaxoSmithKline.S.A. | NULL | Not Recruiting | Female: no Male: yes | 190 | Phase 3 | France;Hungary;Czech Republic;Poland;Belgium;Spain;Denmark;Norway;Netherlands;Germany;Italy | ||
| 485 | EUCTR2010-020069-26-PL (EUCTR) | 30/03/2011 | 03/01/2011 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan | ||
| 486 | EUCTR2010-020069-26-BE (EUCTR) | 15/03/2011 | 25/11/2010 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Netherlands;Germany;Italy | ||
| 487 | EUCTR2010-020069-26-NL (EUCTR) | 02/03/2011 | 04/11/2010 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Germany;Netherlands;Italy | ||
| 488 | NCT01335295 (ClinicalTrials.gov) | March 2011 | 12/4/2011 | Safety Study of Flavocoxid in Duchenne Muscular Dystrophy | Open Pilot Trial to Test the Safety and Tolerability of Flavocoxid in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Flavocoxid | University of Messina | NULL | Completed | 4 Years | 16 Years | Male | 20 | Phase 1 | Italy |
| 489 | EUCTR2010-020069-26-CZ (EUCTR) | 24/02/2011 | 13/01/2011 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan | ||
| 490 | EUCTR2011-000176-33-IT (EUCTR) | 14/02/2011 | 23/02/2011 | Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND | Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND | Duchenne Muscolar Dystrophy MedDRA version: 9.1;Level: PT;Classification code 10013801 | INN or Proposed INN: Tacrolimus Product Name: hMABs Other descriptive name: HLA-identical allogeneic mesoangioblasts Product Name: hMABs Other descriptive name: HLA-identical allogeneic mesoangioblasts Product Name: hMABs Other descriptive name: HLA-identical allogeneic mesoangioblasts | FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | Italy | ||||
| 491 | NCT01183767 (ClinicalTrials.gov) | December 30, 2010 | 17/8/2010 | Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy | Sunphenon Epigallocatechin-Gallate (EGCg) in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Epigallocatechin-Gallate;Drug: Placebo | Charite University, Berlin, Germany | NULL | Completed | 5 Years | 10 Years | All | 33 | Phase 2/Phase 3 | Germany |
| 492 | NCT01254019 (ClinicalTrials.gov) | December 2, 2010 | 21/10/2010 | A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy | A Phase III, Randomized, Double Blind, Placebo-controlled Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies | Drug: GSK2402968 6mg/kg/week | GlaxoSmithKline | NULL | Completed | 5 Years | N/A | Male | 186 | Phase 3 | Argentina;Belgium;Brazil;Canada;Chile;Czechia;Denmark;France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan;Turkey;Czech Republic |
| 493 | NCT02245711 (ClinicalTrials.gov) | December 2010 | 12/9/2014 | Cell Therapy in Limb Girdle Muscular Dystrophy | Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | NULL | Withdrawn | 15 Years | 60 Years | All | 0 | Phase 1 | India |
| 494 | NCT01247207 (ClinicalTrials.gov) | November 30, 2010 | 22/11/2010 | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) | An Open-Label, Safety Study for Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | NULL | Enrolling by invitation | N/A | N/A | Male | 270 | Phase 3 | United States;Canada |
| 495 | EUCTR2010-018412-32-ES (EUCTR) | 18/11/2010 | 01/12/2010 | Estudio clínico de fase II, doble ciego, explorador, de grupos paralelos y controlado con placebo para evaluar dos pautas posológicas de GSK2402968 para la eficacia, seguridad, tolerabilidad y farmacocinética en sujetos ambulatorios con distrofia muscular de Duchenne | Estudio clínico de fase II, doble ciego, explorador, de grupos paralelos y controlado con placebo para evaluar dos pautas posológicas de GSK2402968 para la eficacia, seguridad, tolerabilidad y farmacocinética en sujetos ambulatorios con distrofia muscular de Duchenne | Distrofia muscular de Duchenne MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | France;Spain;Netherlands;Germany;United Kingdom | |||
| 496 | EUCTR2010-020069-26-IT (EUCTR) | 16/11/2010 | 21/12/2010 | A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - ND | A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - ND | Duchenne muscular dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801 | Product Name: GSK2402968 Product Code: GSK2402968 INN or Proposed INN: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Netherlands;Germany;Italy | ||
| 497 | NCT01207908 (ClinicalTrials.gov) | November 2010 | 22/9/2010 | Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy | IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: IGF-1;Other: Standard steroid treatment | Children's Hospital Medical Center, Cincinnati | Tercica- Subsidiary of Ipsen;Charley's Fund | Active, not recruiting | 5 Years | N/A | Male | 40 | Phase 1/Phase 2 | United States |
| 498 | NCT01239758 (ClinicalTrials.gov) | October 2010 | 1/11/2010 | Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: ACE-031 (Extension of cohort 1 from core study, A031-03);Biological: ACE-031 (Extension of cohort 2 from core study, A031-03);Biological: ACE-031 (Extension of cohort 3 from core study, A031-03) | Acceleron Pharma, Inc. | NULL | Terminated | 4 Years | N/A | Male | 11 | Phase 2 | Canada |
| 499 | EUCTR2010-020069-26-FR (EUCTR) | 16/09/2010 | 15/07/2010 | A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy | A randomized, double-blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in ambulant subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Norway;Netherlands;Germany;Italy | ||
| 500 | EUCTR2010-018412-32-NL (EUCTR) | 13/09/2010 | 24/05/2010 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy - | Duchenne Muscular Dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2 | United Kingdom;Germany;Netherlands;France;Spain | |||
| 501 | NCT01153932 (ClinicalTrials.gov) | September 2010 | 29/6/2010 | Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy | A Phase II, Double Blind, Exploratory, Parallel-group, Placebocontrolled Clinical Study to Assess Two Dosing Regimens of GSK2402968 for Efficacy, Safety, Tolerability and Pharmacokinetics in Ambulant Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies | Drug: GSK2402968;Drug: matched placebo | GlaxoSmithKline | NULL | Completed | 5 Years | N/A | Male | 53 | Phase 2 | Australia;Belgium;France;Germany;Israel;Netherlands;Spain;Turkey;United Kingdom |
| 502 | NCT01168908 (ClinicalTrials.gov) | September 2010 | 22/7/2010 | Revatio for Heart Disease in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy | Phase 2 Clinical Trial of Sildenafil for Cardiac Dysfunction in Duchenne Muscular Dystrophy and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Sildenafil | Hugo W. Moser Research Institute at Kennedy Krieger, Inc. | Johns Hopkins University | Terminated | 18 Years | 50 Years | Male | 20 | Phase 2 | United States |
| 503 | EUCTR2010-018412-32-DE (EUCTR) | 05/08/2010 | 31/03/2010 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2 | France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom | ||
| 504 | EUCTR2010-018412-32-FR (EUCTR) | 03/08/2010 | 03/08/2010 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;Belgium;Netherlands;Germany;United Kingdom | ||
| 505 | NCT01128855 (ClinicalTrials.gov) | July 12, 2010 | 20/5/2010 | A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects | A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies | Drug: 3 mg/kg GSK2402968;Drug: 6 mg/kg GSK2402968;Drug: 9 mg/kg GSK2402968;Drug: 12 mg/kg GSK2402968;Other: Placebo | GlaxoSmithKline | NULL | Completed | 9 Years | N/A | Male | 20 | Phase 1 | United States;France |
| 506 | EUCTR2009-016482-28-DE (EUCTR) | 25/06/2010 | 15/03/2010 | SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy | SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy - SUNIMUD | Duchenne Muscular Dystrophy;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Sunphenon EGCG INN or Proposed INN: Sunphenon | Charite Universitätsmedizin Berlin | NULL | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | Germany | ||
| 507 | EUCTR2009-017649-67-IE (EUCTR) | 22/06/2010 | 13/01/2010 | Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD | Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD | Bone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate use | Trade Name: Actonel Once a week 5mg film coated tablets Product Name: Risedronate sodium Product Code: Risedronate Trade Name: Calcichew D3 Forte Product Name: Calcichew D3 Forte Product Code: Calcichew D3 Forte | The Central Remedial Clinic and The Children's University Hospital | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 70 | Ireland | |||
| 508 | EUCTR2010-018412-32-GB (EUCTR) | 17/06/2010 | 16/04/2010 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2 | France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom | ||
| 509 | EUCTR2010-018412-32-BE (EUCTR) | 15/06/2010 | 07/04/2010 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 54 | Phase 2 | France;Spain;Belgium;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom | |||
| 510 | EUCTR2009-012037-30-NL (EUCTR) | 03/06/2010 | 17/12/2009 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Germany;Netherlands;Switzerland;Italy;Sweden | ||
| 511 | NCT01596803 (ClinicalTrials.gov) | May 1, 2010 | 5/4/2012 | Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD) | Effects of Antioxidants Supplementation on Muscular Function of Patients Affected by Facioscapulohumeral Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy | Procedure: Taking of blood;Dietary Supplement: needle biopsy of the vastus lateralis muscle;Dietary Supplement: Vit C Vit E Zn Se;Dietary Supplement: Placebo Vit E Placebo Vit C Zn Se | University Hospital, Montpellier | Hospital Clinical Research Project 2010;Association Amis FSH France;FSH Dutch Fondation The Netherland | Completed | 18 Years | 60 Years | All | 54 | N/A | France |
| 512 | EUCTR2009-013762-63-NL (EUCTR) | 16/04/2010 | 25/08/2009 | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188 | Prosensa Therapeutics B.V | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 513 | NCT01099761 (ClinicalTrials.gov) | April 2010 | 2/4/2010 | Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: ACE-031 0.5 mg/kg q4wk;Biological: ACE-031 1.0 mg/kg q2wk;Other: Placebo | Acceleron Pharma, Inc. | NULL | Terminated | 4 Years | N/A | Male | 24 | Phase 2 | Canada |
| 514 | EUCTR2009-012037-30-AT (EUCTR) | 10/03/2010 | 10/02/2010 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden | ||
| 515 | EUCTR2009-013762-63-IT (EUCTR) | 25/02/2010 | 27/04/2010 | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy - ND | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801 | Product Name: PRO044 Product Code: PRO044 | PROSENSA THERAPEUTICS BV | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 516 | EUCTR2009-012037-30-SE (EUCTR) | 23/02/2010 | 28/12/2009 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | France;United States;Belgium;Spain;Austria;Netherlands;Germany;Italy;Switzerland;Sweden | ||
| 517 | EUCTR2009-012037-30-FR (EUCTR) | 19/02/2010 | 17/12/2009 | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 240 | Phase 3 | Germany;Netherlands;Belgium;France;Spain;Italy;Austria;Sweden | ||
| 518 | EUCTR2009-013762-63-SE (EUCTR) | 19/02/2010 | 03/11/2009 | A study to assess the effect and safety of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne Disease | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous and intravenous doses of PRO044 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188 | Prosensa Therapeutics B.V | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 1/2 | Belgium;Netherlands;Italy;Sweden | ||
| 519 | NCT01126697 (ClinicalTrials.gov) | February 2010 | 18/5/2010 | Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies | PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy;Limb Girdle Muscular Dystrophy | Drug: Coenzyme Q10 and Lisinopril | Cooperative International Neuromuscular Research Group | United States Department of Defense | Completed | 8 Years | N/A | All | 63 | Phase 2/Phase 3 | United States;Canada;Japan |
| 520 | EUCTR2009-013169-24-GB (EUCTR) | 19/01/2010 | 17/03/2010 | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 12.0;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren 125 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 250 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: Ataluren 1000 mg Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics Inc | NULL | Not Recruiting | Female: no Male: yes | 30 | Phase 2 | United Kingdom | ||
| 521 | NCT01009294 (ClinicalTrials.gov) | January 13, 2010 | 5/11/2009 | Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/BMD) | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren;Drug: Chronic Corticosteroid Therapy | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 7 Years | N/A | Male | 6 | Phase 2 | United States;United Kingdom |
| 522 | NCT01070511 (ClinicalTrials.gov) | January 2010 | 17/2/2010 | Tadalafil in Becker Muscular Dystrophy | Functional Muscle Ischemia and PDE5A Inhibition in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Tadalafil;Drug: Placebo | Cedars-Sinai Medical Center | Muscular Dystrophy Association | Completed | 18 Years | 55 Years | Male | 48 | Phase 4 | United States |
| 523 | EUCTR2009-013762-63-BE (EUCTR) | 09/12/2009 | 03/11/2009 | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy | Product Name: PRO044 Product Code: PRO044 INN or Proposed INN: h44AON188 | Prosensa Therapeutics B.V | NULL | Not Recruiting | Female: no Male: yes | 24 | Phase 1;Phase 2 | Belgium;Netherlands;Italy;Sweden | ||
| 524 | NCT01037309 (ClinicalTrials.gov) | December 2009 | 21/12/2009 | Phase I/II Study of PRO044 in Duchenne Muscular Dystrophy (DMD) | A Phase I/IIa, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous and Intravenous Doses of PRO044 in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PRO044 SC;Drug: PRO044 IV | BioMarin Pharmaceutical | NULL | Completed | 5 Years | 16 Years | Male | 18 | Phase 1/Phase 2 | Belgium;Italy;Netherlands;Sweden |
| 525 | EUCTR2009-012037-30-DE (EUCTR) | 08/10/2009 | 07/08/2009 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Netherlands;Germany;Switzerland;Italy;Sweden | ||
| 526 | EUCTR2009-012037-30-BE (EUCTR) | 14/07/2009 | 27/05/2009 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Netherlands;Germany;Switzerland;Italy;Sweden | ||
| 527 | EUCTR2008-007648-32-ES (EUCTR) | 09/07/2009 | 04/05/2009 | Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora. | Un estudio de extensión en Fase 2b de PTC124 en sujetos con distrofia muscular de Duchenne y Becker mediada por mutación terminadora. | Distrofia muscular de Duchenne, Distrofia muscular de Becker MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Belgium;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 528 | NCT01027884 (ClinicalTrials.gov) | July 2009 | 8/12/2009 | Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD) | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10-18 Year Old Patients With Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne;Ambulatory Care | Drug: Placebo;Drug: Idebenone | Santhera Pharmaceuticals | NULL | Completed | 10 Years | 18 Years | Male | 65 | Phase 3 | United States;Austria;Belgium;France;Germany;Italy;Netherlands;Spain;Sweden;Switzerland |
| 529 | EUCTR2008-007648-32-GB (EUCTR) | 22/06/2009 | 29/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | Germany;United Kingdom;Belgium;France;Spain;Italy;Sweden | ||
| 530 | EUCTR2008-007648-32-DE (EUCTR) | 22/06/2009 | 08/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 531 | EUCTR2008-007648-32-SE (EUCTR) | 02/06/2009 | 17/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 532 | NCT02432885 (ClinicalTrials.gov) | June 2009 | 24/3/2015 | Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - ACE Inhibitor Therapy Trial | Myocardial Fibrosis Progression in Duchenne and Becker Muscular Dystrophy - Angiotensin-Converting-Enzyme (ACE) Inhibitor Therapy | Myocardial Fibrosis;Muscular Dystrophies | Drug: Enalapril | InCor Heart Institute | Federal University of Minas Gerais;University of Sao Paulo | Completed | 6 Years | N/A | Both | 76 | Phase 3 | NULL |
| 533 | EUCTR2008-007648-32-FR (EUCTR) | 28/05/2009 | 03/04/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense–Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 534 | EUCTR2008-007648-32-IT (EUCTR) | 27/05/2009 | 22/05/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy - ND | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801 MedDRA version: 9.1;Classification code 10059117 | Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 535 | EUCTR2007-005478-29-GB (EUCTR) | 27/04/2009 | 22/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 536 | EUCTR2008-007648-32-BE (EUCTR) | 14/04/2009 | 25/02/2009 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: ATALUREN Product Code: PTC124 INN or Proposed INN: ATALUREN Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 537 | EUCTR2007-005932-10-GB (EUCTR) | 30/03/2009 | 14/04/2009 | A double-blind randomised multi-centre, placebo-controlled trial of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with DMD without echo-detectable left ventricular dysfunction - DMD-Heart Protection | A double-blind randomised multi-centre, placebo-controlled trial of combined ACE-inhibitor and beta-blocker therapy in preventing the development of cardiomyopathy in genetically characterised males with DMD without echo-detectable left ventricular dysfunction - DMD-Heart Protection | Duchenne muscular dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Trade Name: Perindopril 2mg Tablets Product Name: Perindopril Trade Name: Cardicor (Merck brand) Product Name: Bisoprolol | Newcastle upon Tyne Hospitals NHS Foundation Trust | NULL | Not Recruiting | Female: no Male: yes | 140 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United Kingdom | ||
| 538 | NCT01982695 (ClinicalTrials.gov) | March 2009 | 29/10/2013 | Cardiomyopathy in DMD: Lisinopril vs. Losartan | Compare Efficacy of the Angiotensin Converting Enzyme Inhibitor (ACEi) Lisinopril With Angiotensin II Receptor Antagonist Losartan (ARB) for the Cardiomyopathy of Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD);Cardiomyopathy | Drug: Losartan;Drug: Lisinopril | Nationwide Children's Hospital | Boston Children’s Hospital;University of California, Davis;Unverisity of Kansas Medical Center;University of Minnesota - Clinical and Translational Science Institute;St. Louis Children's Hospital | Completed | N/A | N/A | Male | 23 | N/A | United States |
| 539 | NCT00847379 (ClinicalTrials.gov) | January 31, 2009 | 16/2/2009 | Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | 5 Years | N/A | Male | 173 | Phase 2 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 540 | NCT02241434 (ClinicalTrials.gov) | January 2009 | 12/9/2014 | Stem Cell Therapy in Duchenne Muscular Dystrophy | The Role of Autologous Bone Marrow Mononuclear Cell Therapy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | NULL | Withdrawn | 3 Years | 25 Years | All | 0 | Phase 1 | India |
| 541 | NCT02241928 (ClinicalTrials.gov) | January 2009 | 12/9/2014 | Stem Cell Therapy in Muscular Dystrophy | Role of Autologous Mononuclear Cell Therapy in Muscular Dystrophy | Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | NULL | Withdrawn | 6 Months | 60 Years | All | 0 | Phase 1 | India |
| 542 | NCT00844597 (ClinicalTrials.gov) | January 2009 | 24/12/2008 | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Clinical Study to Assess the Safety fo AVI-4658 in Subjects With Duchenne Muscular Dystrophy Due to a Frame-shift Mutation Amenable to Correction by Skipping Exon 51. | Duchenne Muscular Dystrophy | Drug: AVI-4658 for Injection | Sarepta Therapeutics | British Medical Research Council | Completed | 5 Years | 15 Years | Male | 19 | Phase 1/Phase 2 | United Kingdom |
| 543 | EUCTR2007-004695-39-GB (EUCTR) | 05/12/2008 | 28/03/2008 | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: AVI-4658 Product Code: AVI-4658 | AVI BioPharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | United Kingdom | ||||
| 544 | EUCTR2008-007236-18-IT (EUCTR) | 01/12/2008 | 11/12/2008 | Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - ND | Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - ND | Duchenne and Becher muscular dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Trade Name: DILATREND INN or Proposed INN: Carvedilol Trade Name: TRIATEC INN or Proposed INN: Ramipril | POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI | NULL | Not Recruiting | Female: no Male: yes | Italy | ||||
| 545 | NCT00819845 (ClinicalTrials.gov) | December 2008 | 8/1/2009 | Ramipril Versus Carvedilol in Duchenne and Becker Patients | Effects of Cardioprotective Therapy, Carvedilol vs Ramipril, in Patients Affected by Duchenne and Becker Muscular Dystrophy. Clinical Significance and Prognostic Value of Cardiac Magnetic Resonance Study. | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: carvedilol;Drug: ramipril | Catholic University, Italy | NULL | Recruiting | 2 Years | 45 Years | Male | 194 | Phase 4 | Italy |
| 546 | NCT02050776 (ClinicalTrials.gov) | December 2008 | 29/1/2014 | Stem Cell Therapy in Limb Girdle Muscular Dystrophy | The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study | Limb Girdle Muscular Dystrophy | Biological: Autologous bone marrow mononuclear cell transplantation | Neurogen Brain and Spine Institute | NULL | Withdrawn | 15 Years | 60 Years | All | 0 | Phase 1 | India |
| 547 | NCT00758225 (ClinicalTrials.gov) | September 2008 | 23/9/2008 | Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension) | A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001 | Duchenne Muscular Dystrophy | Drug: Idebenone | Santhera Pharmaceuticals | NULL | Completed | N/A | N/A | Male | 21 | Phase 2 | Belgium |
| 548 | EUCTR2007-007752-34-BE (EUCTR) | 14/08/2008 | 26/06/2008 | A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy | A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy | Duchennes Muscular Dystrophy MedDRA version: 12.0;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: Idebenone Product Code: SNT-MC17 INN or Proposed INN: Idebenone | Santhera Pharmaceuticals (Switzerland) Ltd | NULL | Not Recruiting | Female: no Male: yes | Phase 2 | Belgium | |||
| 549 | NCT00759876 (ClinicalTrials.gov) | August 13, 2008 | 23/9/2008 | Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD) | A Phase 2a Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | Genzyme, a Sanofi Company | Terminated | N/A | N/A | Male | 36 | Phase 2 | United States |
| 550 | EUCTR2007-005478-29-IT (EUCTR) | 03/06/2008 | 14/07/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy - ND | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy. MedDRA version: 9.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Code: PTC124 | PTC THERAPEUTICS, INC. | NULL | Not Recruiting | Female: no Male: yes | 165 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 551 | NCT01421992 (ClinicalTrials.gov) | June 2008 | 1/6/2011 | Methylphenidate in Myotonic Dystrophy Type 1 | Phase 2/3 Study of Efficacy and Tolerability of Methylphenidate in the Treatment of Excessive Daytime Sleepiness in Myotonic Dystrophy Type 1 | Dystrophia Myotonica 1 | Drug: Methylphenidate;Drug: Placebo | Laval University | NULL | Completed | 18 Years | 65 Years | Both | 28 | Phase 2/Phase 3 | Canada |
| 552 | EUCTR2007-005478-29-ES (EUCTR) | 20/05/2008 | 10/03/2008 | Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora | Estudio de fase 2b sobre la eficacia y la seguridad de PTC124 en pacientes con distrofia muscular de Duchenne y Becker mediada por mutación terminadora | Distrofia muscular de Duchenne, Distrofia muscular de Becker MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 165 | Phase 2 | France;Belgium;Spain;Germany;Italy;United Kingdom;Sweden | ||
| 553 | EUCTR2007-005478-29-SE (EUCTR) | 16/04/2008 | 22/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 554 | EUCTR2007-005478-29-DE (EUCTR) | 14/04/2008 | 28/01/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 555 | EUCTR2007-004819-54-SE (EUCTR) | 19/03/2008 | 31/01/2008 | A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration | Duchenne Muscular Dystrophy MedDRA version: 17.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | Belgium;Netherlands;Sweden | ||
| 556 | EUCTR2007-005478-29-FR (EUCTR) | 17/03/2008 | 24/01/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 165 | Phase 2 | France;Spain;Belgium;Germany;Italy;United Kingdom;Sweden | ||
| 557 | EUCTR2007-005478-29-BE (EUCTR) | 11/03/2008 | 27/02/2008 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy MedDRA version: 9.1;Classification code 10059117;Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 INN or Proposed INN: ataluren Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 | PTC Therapeutics, Inc. | NULL | Not Recruiting | Female: no Male: yes | 174 | Phase 2 | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | ||
| 558 | NCT01910649 (ClinicalTrials.gov) | March 2008 | 2/8/2012 | A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration | A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess the Effect, Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for Intravenous Dosing as an Alternative Route of Administration | Muscular Dystrophies | Drug: Drisapersen | BioMarin Pharmaceutical | NULL | Terminated | 5 Years | 16 Years | Male | 12 | Phase 2 | NULL |
| 559 | NCT00494195 (ClinicalTrials.gov) | March 2008 | 27/6/2007 | Gene Transfer Therapy for Treating Children and Adults With Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) | Phase I Gene Transfer of rAAV1.tMCK.Human-alpha-sarcoglycan for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) | Muscular Dystrophies | Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- First cohort;Genetic: Genetic: rAAV1.tMCK.human-alpha-sarcoglycan- Second cohort | Nationwide Children's Hospital | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS);Muscular Dystrophy Association | Completed | 5 Years | N/A | Both | 6 | Phase 1 | United States |
| 560 | NCT00592553 (ClinicalTrials.gov) | February 29, 2008 | 1/1/2008 | Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2B Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren;Drug: Placebo | PTC Therapeutics | NULL | Completed | 5 Years | N/A | Male | 174 | Phase 2 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 561 | EUCTR2007-005808-41-ES (EUCTR) | 25/02/2008 | 25/01/2008 | Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con CalcifediolMulticentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathy | Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con CalcifediolMulticentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathy | Portadores sintomaticos de una mutación en el gen disferlina.Symptomatic carriers of a mutation in the dysferlin gene MedDRA version: 9.1;Level: LLT;Classification code 10028356;Term: Muscular dystrophy | Trade Name: Hidroferol® Product Name: Calcifediol INN or Proposed INN: CALCIFEDIOL Other descriptive name: HIDROFEROL | Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Spain | ||||
| 562 | EUCTR2007-004819-54-NL (EUCTR) | 22/02/2008 | 07/01/2008 | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO051 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: PRO051 Product Code: h51AON23 | Prosensa Holding B.V. | NULL | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2 | Netherlands;Belgium;Sweden | ||
| 563 | NCT04337112 (ClinicalTrials.gov) | January 23, 2008 | 2/4/2020 | The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation | The Expanded Access Use of Viltolarsen for the Treatment of Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping | Muscular Dystrophy, Duchenne;DMD | Drug: viltolarsen | NS Pharma, Inc. | NULL | Approved for marketing | 3 Years | 12 Years | Male | NULL | ||
| 564 | EUCTR2007-004819-54-BE (EUCTR) | 22/01/2008 | 14/12/2007 | A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration | Duchenne Muscular Dystrophy MedDRA version: 17.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 Product Name: Drisapersen Product Code: PRO051 INN or Proposed INN: PRO051 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | Belgium;Netherlands;Sweden | ||
| 565 | NCT00577577 (ClinicalTrials.gov) | December 2007 | 18/12/2007 | Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1 | A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1 | Myotonic Dystrophy Type 1 | Drug: rhIGF-I/rhIGFBP-3;Drug: placebo | Insmed Incorporated | Muscular Dystrophy Association | Active, not recruiting | 21 Years | 65 Years | Both | 60 | Phase 2 | United States |
| 566 | NCT00606775 (ClinicalTrials.gov) | December 2007 | 22/1/2008 | The Preventive Efficacy of Carvedilol on Cardiac Dysfunction in Duchenne Muscular Dystrophy | Carvedilol for the Prevention of Minor Cardiac Damage and Cardiac Function in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy;Cardiomyopathies | Drug: Carvedilol | Suzuka Hospital | Nagoya University | Recruiting | 8 Years | 45 Years | Male | 60 | Phase 4 | Japan |
| 567 | NCT00159250 (ClinicalTrials.gov) | October 26, 2007 | 8/9/2005 | Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Duchenne Muscular Dystrophy | Drug: AVI-4658 (PMO) | Imperial College London | Department of Health, United Kingdom;Sarepta Therapeutics, Inc. | Completed | 10 Years | 17 Years | Male | 7 | Phase 1/Phase 2 | United Kingdom |
| 568 | EUCTR2006-003833-33-GB (EUCTR) | 13/06/2007 | 09/01/2009 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Duchenne Muscular Dystrophy MedDRA version: 9.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: AVI-4658 Product Code: AVI-4658 Other descriptive name: Phosphorodiamidate Morpholino Oligomer | Imperial College, London | NULL | Not Recruiting | Female: no Male: yes | 9 | Phase 1;Phase 2 | United Kingdom | ||
| 569 | EUCTR2006-006776-37-NL (EUCTR) | 14/05/2007 | 22/12/2006 | Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD | Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD | Healthy volunteers. Later the protocol will be applied to Facioscapular humeral dystrophy and/or other muscular dystrophies in general. MedDRA version: 8.1;Level: LLT;Classification code 10011328;Term: Creatine | Product Name: creatine-13C Product Code: Cr13C | Radboud University Nijmegen Medical Center | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Netherlands | ||||
| 570 | NCT00308113 (ClinicalTrials.gov) | April 2007 | 27/3/2006 | CoQ10 and Prednisone in Non-Ambulatory DMD | PITT0503: Clinical Trial of Coenzyme Q10 and Prednisone in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Prednisone;Dietary Supplement: Coenzyme Q10 | Cooperative International Neuromuscular Research Group | United States Department of Defense | Terminated | 10 Years | 18 Years | Male | 3 | Phase 3 | United States;Australia;Puerto Rico |
| 571 | NCT00451074 (ClinicalTrials.gov) | March 2007 | 21/3/2007 | Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons | A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations | Duchenne Muscular Dystrophy | Drug: Gentamicin infusions twice a week for six months | Nationwide Children's Hospital | National Institutes of Health (NIH);National Institute of Neurological Disorders and Stroke (NINDS) | Completed | 5 Years | 20 Years | Male | 12 | Phase 1 | United States |
| 572 | NCT01344798 (ClinicalTrials.gov) | November 2006 | 17/3/2011 | Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C | Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C | Limb Girdle Muscular Dystrophy Type 2C;Gamma-sarcoglycanopathy | Biological: AAV1-gamma-sarcoglycan vector injection | Genethon | NULL | Completed | 15 Years | N/A | Both | 9 | Phase 1 | France |
| 573 | NCT00428935 (ClinicalTrials.gov) | March 2006 | 26/1/2007 | Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy | Phase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: rAAV2.5-CMV-minidystrophin (d3990) | Nationwide Children's Hospital | Asklepios Biopharmaceutical, Inc. | Completed | 5 Years | 15 Years | Male | 6 | Phase 1 | United States |
| 574 | NCT00296621 (ClinicalTrials.gov) | February 2006 | 23/2/2006 | Effect of Oral Glutamine on Muscle Mass and Function in Duchenne Muscular Dystrophy | Efficacy Study of Oral Glutamine Supplementation in Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: L-Glutamine;Drug: placebo | Assistance Publique - Hôpitaux de Paris | NULL | Completed | N/A | N/A | Male | 30 | Phase 2 | France |
| 575 | NCT00264888 (ClinicalTrials.gov) | December 2005 | 9/12/2005 | Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy | A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: PTC124 | PTC Therapeutics | Muscular Dystrophy Association | Completed | 5 Years | N/A | Male | 38 | Phase 2 | United States |
| 576 | NCT00233519 (ClinicalTrials.gov) | November 2005 | 3/10/2005 | Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) | Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) | Myotonic Dystrophy | Drug: SomatoKine/IPLEX | University of Rochester | National Institute of Neurological Disorders and Stroke (NINDS);Imsmed Incorporated | Completed | 21 Years | 60 Years | All | 17 | Phase 1/Phase 2 | United States |
| 577 | NCT00654784 (ClinicalTrials.gov) | October 2005 | 3/4/2008 | Efficacy and Tolerability of Idebenone in Boys With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy | A Phase IIa Double Blind, Randomised, Placebo Controlled, Single Centre Study at the University of Leuven to Assess the Efficacy and Tolerability of Idebenone in 8 - 16 Year Old Males With Cardiac Dysfunction Associated With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy (DMD) | Drug: idebenone;Drug: placebo | Santhera Pharmaceuticals | NULL | Completed | 8 Years | 16 Years | Male | 21 | Phase 2 | Belgium |
| 578 | EUCTR2005-002520-33-BE (EUCTR) | 06/09/2005 | 04/10/2005 | A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy | A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Product Name: idebenone | Santhera Pharmaceuticals LLC | NULL | Not Recruiting | Female: no Male: yes | 21 | Phase 2 | Belgium | ||
| 579 | NCT00243789 (ClinicalTrials.gov) | September 2005 | 21/10/2005 | Study of Daily Pentoxifylline as a Rescue Treatment in Duchenne Muscular Dystrophy | A Double-Blinded Randomized Placebo Controlled Study of Daily Pentoxifylline as a Rescue Treatment in DMD | Muscular Dystrophy, Duchenne | Drug: Pentoxifylline | Cooperative International Neuromuscular Research Group | NULL | Completed | 7 Years | N/A | Male | 64 | Phase 1/Phase 2 | United States;Argentina;Australia;Canada;Israel;Italy |
| 580 | EUCTR2004-000622-67-GB (EUCTR) | 03/06/2005 | 10/02/2005 | A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy. | A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy. | Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy. | Product Name: MYO-029 INN or Proposed INN: Not Available Other descriptive name: Recombinant human anti-GDF-8 antibody | Wyeth Research Division of Wyeth Pharmaceuticals Inc. | NULL | Not Recruiting | Female: yes Male: yes | 136 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United Kingdom | ||
| 581 | EUCTR2005-000663-26-AT (EUCTR) | 25/04/2005 | 21/03/2005 | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Duchenne Muscular Dystrophy | Trade Name: Sandimmun Optoral Product Name: Sandimmun Optoral Product Code: 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00 Other descriptive name: Cyclosporin A Trade Name: Decortin Product Name: Decortin Product Code: not available Other descriptive name: Prednison | Universitätsklinik Freiburg | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | Austria | ||
| 582 | NCT00104078 (ClinicalTrials.gov) | February 2005 | 22/2/2005 | Study Evaluating MYO-029 in Adult Muscular Dystrophy | Becker Muscular Dystrophy;Facioscapulohumeral Muscular Dystrophy;Limb-Girdle Muscular Dystrophy | Drug: MYO-029 | Wyeth is now a wholly owned subsidiary of Pfizer | NULL | Completed | 18 Years | N/A | Both | 108 | Phase 1/Phase 2 | United States | |
| 583 | NCT00167609 (ClinicalTrials.gov) | November 2004 | 10/9/2005 | Efficacy and Safety of DHEA for Myotonic Dystrophy | Phase 3 Study of Oral Dehydroepiandrosterone (DHEA) in Adults With Myotonic Dystrophy | Myotonic Dystrophy | Drug: dehydroepiandrosterone 100 and 400 mg | University of Versailles | Association Française contre les Myopathies (AFM), Paris;AP-HP | Completed | 18 Years | 70 Years | Both | 75 | Phase 2/Phase 3 | France |
| 584 | NCT00110669 (ClinicalTrials.gov) | January 2004 | 12/5/2005 | High-dose Prednisone in Duchenne Muscular Dystrophy | A Randomized Study of Daily vs. High-dose Weekly Prednisone Therapy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: Prednisone | Cooperative International Neuromuscular Research Group | NULL | Completed | 4 Years | 10 Years | Male | 64 | Phase 3 | United States;India |
| 585 | NCT00102453 (ClinicalTrials.gov) | March 2002 | 29/1/2005 | Pentoxifylline in Duchenne Muscular Dystrophy | An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Pentoxifylline | Cooperative International Neuromuscular Research Group | NULL | Completed | 4 Years | 7 Years | Male | 17 | Phase 1/Phase 2 | United States |
| 586 | NCT00033813 (ClinicalTrials.gov) | January 2002 | 10/4/2002 | KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Oxatomide (tinset) | Cooperative International Neuromuscular Research Group | NULL | Completed | 5 Years | 10 Years | Male | 15 | Phase 2 | United States | |
| 587 | NCT00033189 (ClinicalTrials.gov) | September 2001 | 8/4/2002 | An Open-label Pilot Study of Coenzyme Q10 in Steroid-Treated Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Coenzyme Q10 | Cooperative International Neuromuscular Research Group | NULL | Completed | 5 Years | 11 Years | Male | 15 | Phase 2 | United States | |
| 588 | NCT00018109 (ClinicalTrials.gov) | June 2001 | 3/7/2001 | A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) | A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD) | Muscular Dystrophy, Duchenne | Drug: glutamine;Drug: creatine monohydrate | National Center for Research Resources (NCRR) | Children's Research Institute | Completed | 5 Years | 10 Years | Male | Phase 3 | United States | |
| 589 | NCT01882400 (ClinicalTrials.gov) | May 2001 | 17/6/2013 | Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy | Évaluation Multidimensionnelle de la réponse au Traitement de l'ostéoporose spontanée et Induite Par Les corticostéroïdes à l'Aide d'un Bisphosphonate à Administration Orale Chez Des Malades Porteurs d'Une Dystrophie Musculaire sévère. | Osteoporosis;Muscular Dystrophy;Cystic Fibrosis | Drug: Bisphosphonate treatment | Gilles Boire | Procter and Gamble | Completed | 5 Years | 18 Years | All | 11 | Phase 4 | Canada |
| 590 | NCT00016653 (ClinicalTrials.gov) | June 2000 | 21/5/2001 | Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy | A Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy | Muscular Dystrophy, Duchenne | Drug: Creatine Monohydrate;Drug: Glutamine | Cooperative International Neuromuscular Research Group | NULL | Completed | 5 Years | 9 Years | Male | 48 | Phase 2/Phase 3 | United States;Belgium;Israel;Puerto Rico |
| 591 | NCT00005574 (ClinicalTrials.gov) | February 2000 | 2/5/2000 | Gentamicin Treatment of Muscular Dystrophy | Gentamicin Treatment of Patients With Muscular Dystrophy Due to Nonsense Mutations in Dystrophin | Becker Muscular Dystrophy;Duchenne Muscular Dystrophy | Drug: Gentamicin | National Institute of Neurological Disorders and Stroke (NINDS) | NULL | Completed | N/A | N/A | Both | 4 | Phase 1 | United States |
| 592 | NCT00004685 (ClinicalTrials.gov) | January 1998 | 24/2/2000 | Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy | Muscular Dystrophy, Facioscapulohumeral | Drug: albuterol | Ohio State University | NULL | Completed | 18 Years | 80 Years | Both | 90 | N/A | NULL | |
| 593 | NCT00004646 (ClinicalTrials.gov) | April 1995 | 24/2/2000 | Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: prednisone | National Center for Research Resources (NCRR) | National Institute of Neurological Disorders and Stroke (NINDS);University of Rochester | Completed | 5 Years | 15 Years | Male | 20 | Phase 3 | NULL | |
| 594 | EUCTR2011-001266-17-Outside-EU/EEA (EUCTR) | 09/03/2012 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 32 | NULL | ||||
| 595 | EUCTR2019-000337-39-FR (EUCTR) | 05/04/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients who have completed study 4658-102 | An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients with Duchenne Muscular Dystrophy Who Have Completed Study 4658-102 | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc | NULL | NA | Female: no Male: yes | 15 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Belgium;United Kingdom | |||
| 596 | EUCTR2020-000698-26-BE (EUCTR) | 21/09/2020 | Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD) | Non-ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | NA | Female: no Male: yes | 96 | Phase 3 | United States;Czechia;Spain;Austria;Israel;Italy;Switzerland;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Netherlands | |||
| 597 | EUCTR2017-004554-42-DE (EUCTR) | 27/07/2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy: A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial Tamoxifen in Duchenne muscular dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | NA | Female: no Male: yes | 100 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | |||
| 598 | EUCTR2016-005001-39-Outside-EU/EEA (EUCTR) | 16/03/2017 | A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy. | Open-Label, Multiple-Dose, Efficacy, Safety, and Tolerability Study of Eteplirsen in Subjects with Duchenne Muscular Dystrophy who Participated in Study 4658-us-201 | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 599 | EUCTR2011-005040-10-FR (EUCTR) | 16/06/2015 | A study to test if multiple injections of PRO045 are safe and effective in people who suffer from Duchenne muscular dystrophy | A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple doses of PRO045 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO045 Product Code: PRO045 INN or Proposed INN: PS220 | Prosensa Therapeutics BV | NULL | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | France;Belgium;Italy;United Kingdom | |||
| 600 | EUCTR2018-000975-34-FR (EUCTR) | 28/06/2018 | A Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | NA | Female: no Male: yes | 40 | Phase 1 | United States;France;Canada;Belgium;Netherlands;Italy;United Kingdom | |||
| 601 | EUCTR2018-004009-22-DE (EUCTR) | 21/08/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 with Open-label Extension in Ambulatory Patients with Duchenne Muscular Dystrophy (DYSTANCE 51) | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden | |||
| 602 | EUCTR2018-004009-22-PL (EUCTR) | 18/06/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Netherlands;Japan;Sweden;Turkey;United States;Spain;Ireland | |||
| 603 | EUCTR2015-003681-87-IT (EUCTR) | 19/01/2021 | A study to assess the effect of BMN 044 in subjects with Duchennemuscular dystrophy (Extension study) | A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy - NA | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 044 Product Code: BMN 044 Other descriptive name: PS188 Product Name: BMN 044 Product Code: BMN 044 INN or Proposed INN: PS188 Other descriptive name: PS188 | BIOMARIN PHARMACEUTICAL INC. | NULL | NA | Female: no Male: yes | 50 | Phase 2 | Netherlands;Italy;Sweden;United States;Belgium | |||
| 604 | EUCTR2017-000397-10-FR (EUCTR) | 09/09/2019 | Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | NA | Female: no Male: yes | 185 | Phase 3 | United States;France;Canada;Belgium;Spain;Netherlands;Germany;Italy;United Kingdom | |||
| 605 | EUCTR2011-001266-17-FR (EUCTR) | 05/07/2011 | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety,tolerability and efficacy of GSK2402968 in subjects withDuchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 200 | Phase 3 | Taiwan;Spain;Turkey;Israel;Chile;Russian Federation;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 606 | EUCTR2019-000601-77-BE (EUCTR) | 01/08/2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-5051 Product Code: SRP-5051 INN or Proposed INN: SRP-5051 Other descriptive name: peptide-conjugated phosphorodiamidate morpholino oligomer for exon 51 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 60 | Phase 2 | United States;Canada;Spain;Belgium;Ireland;Germany;Netherlands;United Kingdom;Italy | |||
| 607 | EUCTR2010-020069-26-DE (EUCTR) | 22/06/2010 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan | |||
| 608 | EUCTR2016-000602-10-SE (EUCTR) | 22/09/2016 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Italy;Switzerland;United Kingdom;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | |||
| 609 | EUCTR2017-004625-32-DE (EUCTR) | 26/07/2018 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;Czechia;Spain;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Poland;Bulgaria;Germany;Sweden | |||
| 610 | EUCTR2017-004625-32-PL (EUCTR) | 07/11/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 260 | Phase 3 | United States;United Kingdom;Spain;Canada;Czech Republic;Sweden;Belgium;Ireland;Finland;Poland;Italy;Israel;Australia;France;Bulgaria;Germany | |||
| 611 | EUCTR2016-005000-26-Outside-EU/EEA (EUCTR) | 16/03/2017 | A research study of a new investigational medicinal product for the treatment of patients with Duchenne Muscular Dystrophy. | A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Efficacy, Safety, Tolerability, and Pharmacokinetics Study of AVI-4658 (Eteplirsen), a Phosphorodiamidate Morpholino Oligomer, Administered Over 28 Weeks in the Treatment of Ambulant Subjects with Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 12 | Phase 2 | United States | |||
| 612 | EUCTR2018-001762-42-FR (EUCTR) | 08/07/2019 | A research study to compare different doses of a new investigational medicinal product for treatment of certain patients with Duchenne muscular dystrophy | A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 INN or Proposed INN: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 152 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | Taiwan;Hong Kong;Spain;Ireland;Turkey;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Canada;Argentina;Belgium;Peru;Australia;Netherlands;Germany;New Zealand;Korea, Republic of | |||
| 613 | EUCTR2017-004554-42-BE (EUCTR) | 30/04/2019 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy:A multicenter, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open label extension of a multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Tamoxifen 20mg Hexal® Filmtabletten INN or Proposed INN: Tamoxifen Other descriptive name: TAMOXIFEN CITRATE | University of Basel Children's Hospital, Division of Neuropediatrics | NULL | NA | Female: no Male: yes | 100 | Phase 3 | France;Spain;Belgium;Turkey;Netherlands;Germany;United Kingdom;Switzerland | |||
| 614 | EUCTR2017-004279-30-DE (EUCTR) | 11/06/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | |||
| 615 | EUCTR2017-004279-30-SE (EUCTR) | 15/05/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | |||
| 616 | EUCTR2010-018412-32-Outside-EU/EEA (EUCTR) | 09/03/2012 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy - | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 54 | Phase 2 | Australia;Israel;Turkey | |||
| 617 | EUCTR2016-005023-92-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystropy Amenable to Exon 51 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 40 | Phase 2 | United States | |||
| 618 | EUCTR2019-002076-13-SE (EUCTR) | 17/10/2019 | This is a randomized, double-blind, Placebo-controlled sudy to evaluate the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3 Randomized, Double-blind, Placebo-controlled, Multi-center Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53 | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | Not Recruiting | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Spain;Turkey;Chile;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Brazil;Poland;Australia;Netherlands;Japan;Sweden;Korea, Republic of | |||
| 619 | EUCTR2010-024566-22-Outside-EU/EEA (EUCTR) | 09/03/2012 | A clinical study to investigate the pharmacokinetics safety, and tolerability of a single dose of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy (GSK Study Number DMD114118) | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 32 | United States | ||||
| 620 | EUCTR2012-004527-20-FR (EUCTR) | 25/09/2015 | Study of ataluren in patients with nonsense mutation Duchenne and Becker muscular dystrophy | A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients with Nonsense Mutation Dystrophinopathy - N/A | Nonsense mutation dystrophinopathy MedDRA version: 18.0;Level: PT;Classification code 10059117;Term: Becker's muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ataluren | PTC Therapeutics, Inc | NULL | Not Recruiting | Female: no Male: yes | 220 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Turkey;Chile;Israel;Italy;United Kingdom;Switzerland;France;Czech Republic;Canada;Poland;Brazil;Belgium;Australia;Germany;Korea, Republic of;Sweden | |||
| 621 | EUCTR2016-000602-10-IE (EUCTR) | 06/02/2018 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | Not Recruiting | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Germany;Netherlands;Sweden | |||
| 622 | EUCTR2020-000699-39-BE (EUCTR) | 28/01/2021 | Evaluation of Pamrevlumab for the Treatment of Ambulatory Patients Affected by Duchenne Muscular Dystrophy and just Treated with Corticosteroids | A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD) | Ambulatory Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Pamrevlumab Product Code: FG-3019 INN or Proposed INN: PAMREVLUMAB | FibroGen, Inc. | NULL | NA | Female: no Male: yes | 70 | Phase 3 | France;United States;Canada;Spain;Belgium;Austria;Australia;Netherlands;United Kingdom;Italy | |||
| 623 | EUCTR2021-000122-10-NO (EUCTR) | 24/02/2022 | A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD) | A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - RACER-53X | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Viltolarsen Product Code: NS-065/NCNP-01 INN or Proposed INN: VILTOLARSEN | NS Pharma, Inc. | NULL | NA | Female: no Male: yes | 74 | Phase 3 | United States;Taiwan;Hong Kong;Greece;Spain;Ukraine;Turkey;Russian Federation;Chile;United Kingdom;Italy;Mexico;Canada;Australia;Norway;Netherlands;China;Japan;New Zealand;Korea, Republic of | |||
| 624 | EUCTR2010-020047-12-FR (EUCTR) | 01/04/2011 | Effet d’un traitement préventif par Nebivolol chez les enfants atteints de dystrophie de Duchenne | Etude de l'effet d'un traitement préventif par Nebivolol sur l'apparition et la progression de la dysfonction cardiaque chez les enfants atteints de dystrophie de Duchenne - NEBIDYS | Enfants atteints de dystrophie de Duchenne MedDRA version: 13.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Temerit Product Name: Temerit INN or Proposed INN: Nébivolol | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | NULL | Not Recruiting | Female: no Male: yes | 70 | Phase 3 | France | |||
| 625 | EUCTR2017-002686-21-FR (EUCTR) | 29/01/2018 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 | Wave Life Sciences Ltd. | NULL | Not Recruiting | Female: no Male: yes | 32 | Phase 1 | United States;France;Czech Republic;Belgium;Spain;Ireland;Denmark;Netherlands;Italy;United Kingdom | |||
| 626 | EUCTR2016-000624-25-IT (EUCTR) | 19/01/2021 | An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy - na | Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 | ATYR PHARMA, INC. | NULL | Not Recruiting | Female: yes Male: yes | 24 | Phase 1;Phase 2 | Denmark;Italy | |||
| 627 | JPRN-JapicCTI-142538 | 12/05/2014 | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Intervention name : Tadalafil Dosage And administration of the intervention : During the double-blind period, tadalafil or matching placebo will be administered orally once daily at one of 2 target doses (0.3 mg/kg or 0.6 mg/kg).During the open-label extension period all patients will initially receive tadalafil. | Eli Lilly Japan K.K. | NULL | 7 | 14 | Male | Phase 3 | NULL | |||
| 628 | EUCTR2019-002921-31-DE (EUCTR) | 28/07/2020 | Phase 3 study to evaluate the safety and efficacy of PF-06939926 for the treatment of Duchenne muscular dystrophy. | A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY | DUCHENNE MUSCULAR DYSTROPHY (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PF-06939926 Product Code: PF-06939926 INN or Proposed INN: PF-06939926 Other descriptive name: Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene | Pfizer Inc. | NULL | NA | Female: no Male: yes | 99 | Phase 3 | United States;Taiwan;Spain;Israel;Russian Federation;United Kingdom;Switzerland;Italy;France;Canada;Belgium;Germany;China;Japan;Korea, Republic of | |||
| 629 | EUCTR2016-005024-28-Outside-EU/EEA (EUCTR) | 28/03/2017 | Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy | An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 24 | Phase 2 | United States | |||
| 630 | EUCTR2010-020069-26-Outside-EU/EEA (EUCTR) | 09/03/2012 | A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. | A phase III, randomized, double blind, placebo-controlledclinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy - N/A | Duchenne muscular dystrophy MedDRA version: 14.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 180 | Phase 3 | Argentina;Belgium;Brazil;Canada;Chile;Czech Republic;Denmark;France;Germany;Italy;Japan;Korea, Democratic People's Republic of;Netherlands;Norway;Poland;Russian Federation;Spain;Taiwan | |||
| 631 | EUCTR2011-005042-35-FR (EUCTR) | 18/09/2015 | A study to test if PRO053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of PRO053 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO053-induced DMD exon 53 skipping MedDRA version: 18.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO053 Product Code: PRO053 INN or Proposed INN: PS524 | Prosensa Therapeutics BV | NULL | NA | Female: no Male: yes | 42 | Phase 1;Phase 2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | |||
| 632 | EUCTR2018-004009-22-FR (EUCTR) | 26/03/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | NA | Female: no Male: yes | 150 | Phase 2;Phase 3 | United States;Spain;Ireland;Turkey;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden | |||
| 633 | EUCTR2010-020069-26-NO (EUCTR) | 27/04/2011 | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | A phase III, randomized, double blind, placebo-controlled clinical study toassess the efficacy and safety of GSK2402968 in subjects with Duchenne musculardystrophy. - N/A | Duchenne muscular dystrophy MedDRA version: 13.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | France;Hungary;Czech Republic;Spain;Poland;Belgium;Denmark;Netherlands;Germany;Norway;Italy | |||
| 634 | EUCTR2017-001223-49-PL (EUCTR) | 05/06/2018 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 INN or Proposed INN: ATALUREN | PTC Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 340 | Phase 3 | United States;Taiwan;Hong Kong;Thailand;Turkey;Russian Federation;India;Mexico;Canada;Jordan;Argentina;Poland;Brazil;Malaysia;Australia;Bulgaria;China;Japan;Korea, Republic of | |||
| 635 | EUCTR2017-000397-10-DE (EUCTR) | 22/01/2019 | Study in which all the patients take the same investigational drug with the aim of evaluate the long-term safety, tolerability, and efficacy of GIVINOSTAT in all the patients affected with Duchenne Muscolar Dystrophy who have been already treated in one of the GIVINOSTAT studies in the past | Open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD patients who have been previously treated in one of the GIVINOSTAT studies - Givinostat DMD long term study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 222 | Phase 3 | France;United States;Serbia;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | |||
| 636 | EUCTR2020-000980-21-Outside-EU/EEA (EUCTR) | 24/06/2021 | A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Subjects From =6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) | An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From =6 Months to <2 Years of Age with Nonsense Mutation Duchenne Muscular Dystrophy | Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD);Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: Translarna Product Name: Ataluren Product Code: PTC124 INN or Proposed INN: Ataluren | PTC Therapeutics, Inc. | NULL | NA | Female: no Male: yes | Phase 2 | United States | ||||
| 637 | EUCTR2016-000401-36-FR (EUCTR) | 16/05/2017 | Study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Randomised, double blind, placebo controlled, multicentre study to evaluate the efficacy and safety of givinostat in ambulant patients with Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 INN or Proposed INN: Givinostat (hydrochloride monohydrate) Other descriptive name: GIVINOSTAT | ITALFARMACO S.p.A. | NULL | Not Recruiting | Female: no Male: yes | 192 | Phase 3 | United States;France;Canada;Spain;Belgium;Israel;Netherlands;Germany;United Kingdom;Italy | |||
| 638 | EUCTR2017-004625-32-CZ (EUCTR) | 09/09/2019 | A long-term extension study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | Long-term, Open-label Extension Study for Patients with Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: CASIMERSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: GOLODIRSEN Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 260 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Finland;Spain;Ireland;Israel;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Sweden | |||
| 639 | EUCTR2016-005002-19-Outside-EU/EEA (EUCTR) | 28/03/2017 | Confirmatory Study of Eteplirsen in DMD Patients (PROMOVI) | An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy | Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EXONDYS 51™ Product Name: Eteplirsen Injection Product Code: AVI-4658 INN or Proposed INN: AVI-4658 Other descriptive name: ETEPLIRSEN | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 160 | Phase 3 | United States | |||
| 640 | EUCTR2009-009871-36-DE (EUCTR) | 06/08/2009 | Study within children with Duchenne Muscular Dystrophy | Effect and Safety of preventive Treatment with ACE-Inhibitor and Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy - DMD-Kardio | Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EnalHexal®, 5 mg INN or Proposed INN: ENALAPRIL Trade Name: EnaHexal®, 10mg INN or Proposed INN: ENALAPRIL Trade Name: MetoHEXAL® Succ ® 23,75 mg Retardtabletten INN or Proposed INN: METOPROLOL SUCCINATE Trade Name: MetoHEXAL® Succ ® 47,5 mg Retardtabletten INN or Proposed INN: METOPROLOL SUCCINATE Trade Name: MetoHEXAL® Succ ® 95 mg Retardtabletten INN or Proposed INN: METOPROLOL SUCCINATE | Friedrich- Alexander- Universität Erlangen Nürnberg | NULL | Not Recruiting | Female: no Male: yes | Germany | |||||
| 641 | EUCTR2017-004279-30-FR (EUCTR) | 14/05/2018 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone INN or Proposed INN: IDEBENONE Other descriptive name: IDEBENONE | Santhera Pharmaceuticals (Switzerland) Limited | NULL | NA | Female: no Male: yes | 266 | Phase 3 | United States;France;Spain;Belgium;Austria;Israel;Netherlands;Germany;United Kingdom;Switzerland;Italy;Sweden | |||
| 642 | EUCTR2018-004009-22-NL (EUCTR) | 13/05/2019 | Study of WVE-210201 in Patients (able to walk independently) with Duchenne Muscular Dystrophy | A Randomized, Double-blind, Placebo-controlled, Efficacy and Safety Study of WVE-210201 in Ambulatory Patients with Duchenne Muscular Dystrophy | Duchenne muscular dystrophy MedDRA version: 20.0;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 INN or Proposed INN: WVE-210201 Other descriptive name: suvodirsen | Wave Life Sciences UK Limited | NULL | NA | Female: no Male: yes | 150 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Spain;Ireland;Turkey;Italy;United Kingdom;France;Czech Republic;Canada;Poland;Belgium;Australia;Netherlands;Germany;Japan;Sweden | |||
| 643 | EUCTR2014-003346-27-IT (EUCTR) | 19/01/2021 | A Study to Evaluate the Safety, Tolerability, Immunogenicity and Biological Activity of ATYR1940 in Patients With Pediatric Onset FHSD | An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy - na | Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy MedDRA version: 20.0;Level: PT;Classification code 10064087;Term: Facioscapulohumeral muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 | ATYR PHARMA, INC. | NULL | Not Recruiting | Female: yes Male: yes | 16 | Phase 1;Phase 2 | France;United States;Italy | |||
| 644 | EUCTR2006-003075-12-IE (EUCTR) | 13/07/2006 | Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction. | Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction. | Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patients with skeletal myopathy such as DMD eventually progress to develop dilated cardiomyopathy as dystrophin is expressed in all forms of muscle including cardiac and smooth muscle. Often the cause of death in patients with DMD is cardiac failure and arrhythmia. | Trade Name: Eucardic Product Name: Eucardic Product Code: No code INN or Proposed INN: CARVEDILOL | Department Of Paediatric Cardiology | NULL | NA | Female: yes Male: yes | 30 | Phase 4 | Ireland | |||
| 645 | EUCTR2015-002069-52-FR (EUCTR) | 12/01/2017 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 45 or 53 Skipping MedDRA version: 19.1;Level: PT;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4045 Product Code: SRP-4045 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 45 skipping Product Name: SRP-4053 Product Code: SRP-4053 INN or Proposed INN: Not yet available Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping | Sarepta Therapeutics, Inc. | NULL | NA | Female: no Male: yes | 99 | Phase 3 | United States;France;Czech Republic;Canada;Spain;Belgium;Germany;United Kingdom | |||
| 646 | EUCTR2010-024566-22-FR (EUCTR) | 22/02/2011 | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 12.1;Level: LLT;Classification code 10013801;Term: Duchenne muscular dystrophy | Product Name: GSK2402968 Product Code: GSK2402968 | GlaxoSmithKline Research and Development LTD | NULL | NA | Female: no Male: yes | 32 | Phase 1 | France |