113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 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 | 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 |
| 3 | 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 |
| 4 | 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 |
| 5 | 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 |
| 6 | 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 |
| 7 | 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 |
| 8 | 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 |
| 9 | 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 |
| 10 | 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 |
| 11 | 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 |
| 12 | 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 |
| 13 | 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 |
| 14 | 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 |
| 15 | 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 |
| 16 | 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 |
| 17 | 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 |
| 18 | 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 |
| 19 | 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 |
| 20 | 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 |
| 21 | 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 |
| 22 | 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 |
| 23 | 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 |
| 24 | 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 |
| 25 | 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 |
| 26 | 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 |
| 27 | 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 |
| 28 | 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 |
| 29 | 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 |
| 30 | 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 |
| 31 | 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 |
| 32 | 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 | |
| 33 | 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 |
| 34 | 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 |
| 35 | 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 |
| 36 | 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 |
| 37 | 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 |
| 38 | 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 |
| 39 | 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 |
| 40 | 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 |
| 41 | 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 | ||
| 42 | 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 |
| 43 | 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 |
| 44 | 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 |
| 45 | 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 |
| 46 | 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 | ||
| 47 | 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 |
| 48 | 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 |
| 49 | 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 |
| 50 | 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 |
| 51 | 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 |
| 52 | 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 |
| 53 | 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 |
| 54 | 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 |
| 55 | 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 |
| 56 | 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 |
| 57 | 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 |
| 58 | 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 | ||
| 59 | 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 |
| 60 | 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 |
| 61 | 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 |
| 62 | 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 |
| 63 | 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 |
| 64 | 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 |
| 65 | 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 |
| 66 | 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 |
| 67 | 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 |
| 68 | 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 |
| 69 | 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 |
| 70 | 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 |
| 71 | 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 |
| 72 | 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 |
| 73 | 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 |
| 74 | 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 |
| 75 | 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 |
| 76 | 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 |
| 77 | 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 |
| 78 | 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 |
| 79 | 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 |
| 80 | 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 |
| 81 | 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 | |
| 82 | 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 |
| 83 | 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 |
| 84 | 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 |
| 85 | 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 |
| 86 | 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 |
| 87 | 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 |
| 88 | 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 |
| 89 | 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 |
| 90 | 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 |
| 91 | 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 |
| 92 | 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 |
| 93 | 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 |
| 94 | 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 |
| 95 | 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 |
| 96 | 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 |
| 97 | 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 |
| 98 | 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 |
| 99 | 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 |
| 100 | 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 |
| 101 | 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 | ||
| 102 | 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 | |
| 103 | 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 |
| 104 | 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 |
| 105 | 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 |
| 106 | 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 |
| 107 | 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 |
| 108 | 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 |
| 109 | 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 |
| 110 | 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 |
| 111 | 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 |
| 112 | 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 |
| 113 | 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 |
| 114 | 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 |
| 115 | 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 |
| 116 | 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 |
| 117 | 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 |
| 118 | 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 |
| 119 | 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 |
| 120 | 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 |
| 121 | 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 |
| 122 | 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 |
| 123 | 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 |
| 124 | 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 |
| 125 | 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 |
| 126 | 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 |
| 127 | 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 |
| 128 | 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 |
| 129 | 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 |
| 130 | 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 |
| 131 | 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 |
| 132 | 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 |
| 133 | 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 |
| 134 | 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 |
| 135 | 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 |
| 136 | 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 |
| 137 | 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 |
| 138 | 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 |
| 139 | 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 |
| 140 | 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 |
| 141 | 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 | ||
| 142 | 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 |
| 143 | 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 |
| 144 | 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 |
| 145 | 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 | |
| 146 | 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 |
| 147 | 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 |
| 148 | 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 |
| 149 | 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 |
| 150 | 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 | |
| 151 | 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 |
| 152 | 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 | |
| 153 | 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 |
| 154 | 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 |
| 155 | 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 |
| 156 | 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 |
| 157 | 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 |
| 158 | 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 |
| 159 | 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 |
| 160 | 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 |
| 161 | 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 |
| 162 | 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 |
| 163 | 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 |
| 164 | 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 |
| 165 | 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 |
| 166 | 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 |
| 167 | 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 |
| 168 | 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 |
| 169 | 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 |
| 170 | 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 |
| 171 | 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 |
| 172 | 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 |
| 173 | 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 |
| 174 | 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 |
| 175 | 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 |
| 176 | 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 |
| 177 | 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 |
| 178 | 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 |
| 179 | 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 |
| 180 | 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 |
| 181 | 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 |
| 182 | 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 |
| 183 | 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 |
| 184 | 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 | ||
| 185 | 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 |
| 186 | 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 |
| 187 | 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 |
| 188 | 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 |
| 189 | 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 |
| 190 | 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 |
| 191 | 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 |
| 192 | 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 |
| 193 | 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 |
| 194 | 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 |
| 195 | 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 | |
| 196 | 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 |
| 197 | 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 |
| 198 | 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 |
| 199 | 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 | |
| 200 | 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 | |
| 201 | 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 | |
| 202 | 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 |
| 203 | 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 |
| 204 | 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 |
| 205 | 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 | |
| 206 | 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 |