113. 筋ジストロフィー ［臨床試験数：289，薬物数：306（DrugBank：83），標的遺伝子数：57，標的パスウェイ数：156］
Searched query = "Muscular dystrophy", "Dystrophinopathies", "Myotilinopathy", "Laminopathy", "Caveolinopathy", "LGMD1C", "Desminopathy", "Sarcoglycanopathy", "α dystroglycanopathy", "FCMD", "Walker Warburg syndrome", "Muscle eye brain disease", "Myotonic dystrophy", "Integrin α7 deficient CMD", "Rigid spine syndrome"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03340675 | January 2020 | 4 November 2019 | 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 | Not recruiting | 7 Years | N/A | Male | 48 | Phase 2 | |
| 2 | NCT04129294 | November 6, 2019 | 11 November 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. | Recruiting | 4 Years | 17 Years | Male | 6 | Phase 1/Phase 2 | Japan |
| 3 | NCT04060199 | October 23, 2019 | 11 November 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;Japan |
| 4 | NCT03907072 | September 4, 2019 | 11 November 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. | Recruiting | 5 Years | 12 Years | Male | 150 | Phase 2/Phase 3 | United States;Belgium;Canada;Czechia;France;Italy;Sweden | |
| 5 | NCT04004000 | August 23, 2019 | 9 September 2019 | Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 | 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) | Facioscapulohumeral Muscular Dystrophy 1 | Drug: Losmapimod | Fulcrum Therapeutics | Recruiting | 18 Years | 65 Years | All | 16 | Phase 2 | Netherlands | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT04003974 | August 9, 2019 | 11 November 2019 | Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) | A Phase 2, Randomized, Double-Blind, Placebo-Controlled, 24-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy (FSHD) | Drug: Losmapimod;Drug: Placebo oral tablet | Fulcrum Therapeutics | Recruiting | 18 Years | 65 Years | All | 66 | Phase 2 | United States;Canada;France;Germany;Spain | |
| 7 | NCT03895528 | August 2019 | 29 July 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 | Not recruiting | 12 Months | N/A | All | Phase 3 | |||
| 8 | NCT03992430 | August 2019 | 15 July 2019 | A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients | 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 | Not recruiting | 7 Years | 13 Years | Male | 152 | Phase 3 | ||
| 9 | NCT04004065 | June 26, 2019 | 4 November 2019 | A Phase 2 Study for Dose Determination of SRP-5051, Then Dose Expansion in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, Then Dose Expansion, in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment | Duchenne Muscular Dystrophy | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | Recruiting | 7 Years | 21 Years | Male | 24 | Phase 2 | United States;Canada | |
| 10 | NCT03985878 | June 19, 2019 | 24 June 2019 | A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy | 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 | Not recruiting | 2 Years | 5 Years | Male | 15 | Phase 2 | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03959189 | June 17, 2019 | 4 November 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. | Recruiting | 18 Years | 60 Years | All | 12 | Phase 1 | United States | |
| 12 | NCT03783923 | May 15, 2019 | 30 September 2019 | A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | A Multicenter Randomized Placebo-Controlled Phase 3 Study on the Safety and Efficacy of Deflazacort (Emflaza®) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) | Limb-Girdle Muscular Dystrophy | Drug: Deflazacort;Drug: Placebo | PTC Therapeutics | Recruiting | 18 Years | N/A | All | 100 | Phase 3 | United States;Canada;Denmark;France;Germany;Norway;Russian Federation;Sweden | |
| 13 | NCT03943290 | May 10, 2019 | 14 October 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. | Recruiting | 18 Years | N/A | All | 150 | Phase 2 | United States;Canada;Spain | |
| 14 | NCT03433807 | May 9, 2019 | 26 August 2019 | 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 | Not recruiting | 8 Years | N/A | All | Phase 3 | United States | ||
| 15 | NCT03936894 | May 1, 2019 | 28 October 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 Research Institute | Foundation to Eradicate Duchenne | Recruiting | 2 Years | 5 Years | Male | 6 | Phase 1/Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03917719 | March 14, 2019 | 17 June 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 | Recruiting | 4 Years | 10 Years | Male | 100 | Phase 3 | United States | |
| 17 | EUCTR2015-001192-48-FR | 04/03/2019 | 30 April 2019 | Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes. | Traitement du ptosis des dystrophies musculaires oculo-pharyngées par autogreffes de myoblastes. - TTT-PT-DOP | Patients atteints d’une DMOP et présentant un ptosis MedDRA version: 18.0 Level: LLT Classification code 10010498 Term: Congenital hereditary muscular dystrophy System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Myoblastes autologues Pharmaceutical Form: Suspension for injection | CHU CAEN | Authorised | Female: yes Male: yes | 10 | Phase 2 | France | |||
| 18 | NCT03789734 | March 2019 | 14 January 2019 | Safety Study of BLS-M22 in Healthy Volunteers | A Dose Block-randomized, Double-blind, Placebo-controlled and Dose-escalation Phase I Clinical Trial to Evaluate Safety of BLS-M22 Following Single/Multiple Oral Administration in Healthy Adult Volunteers | Muscular Dystrophy, Duchenne | Biological: BLS-M22;Other: Placebo | BioLeaders Corporation | Not recruiting | 19 Years | 55 Years | All | 37 | Phase 1 | Korea, Republic of | |
| 19 | EUCTR2018-000464-29-DE | 31/01/2019 | 30 April 2019 | A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy | A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD | Duchenne muscular dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 Pharmaceutical Form: Capsule, soft INN or Proposed INN: EDASALONEXENT Current Sponsor code: CAT-1004 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Capsule, soft Route of administration of the placebo: Oral use Product Name: Edasalonexent Product Code: CAT-1004 Pharmaceutical Form: Capsule, soft INN or Proposed INN: EDASALONEXENT Current Sponsor code: CAT-1004 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Pharmaceutical form of the placebo: Capsule, soft Route of administration of the placebo: Oral use | Catabasis Pharmaceuticals, Inc. | Authorised | Female: no Male: yes | 126 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | |||
| 20 | EUCTR2017-004554-42-ES | 22/01/2019 | 28 February 2019 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMD A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial | Duchenne muscular dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: TAMOX Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Tamoxifen CAS Number: 54965-24-1 Other descriptive name: TAMOXIFEN CITRATE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | University of Basel Children's Hospital, Division of Neuropediatrics | Authorised | Female: no Male: yes | 100 | Phase 3 | France;Belgium;Spain;Turkey;Netherlands;Germany;United Kingdom;Switzerland | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT03779646 | January 16, 2019 | 28 January 2019 | 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 |
| 22 | NCT03769116 | December 22, 2018 | 11 November 2019 | A Randomized, Double-blind, Placebo-controlled Study of SRP-9001 for Duchenne Muscular Dystrophy (DMD) | A 48-Week, Randomized, Double-Blind, Placebo-Controlled, Systemic, Gene-Delivery Clinical Trial for Duchenne Muscular Dystrophy Using SRP-9001 With a 96-Week Extension | Muscular Dystrophy, Duchenne | Drug: SRP-9001;Drug: Placebo | Sarepta Therapeutics, Inc. | Recruiting | 4 Years | 7 Years | Male | 40 | Phase 2 | United States | |
| 23 | NCT03648827 | December 21, 2018 | 14 October 2019 | 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 | Not recruiting | 2 Years | 7 Years | Male | 20 | Phase 2 | United States | |
| 24 | NCT03675126 | December 19, 2018 | 26 August 2019 | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 | An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 | Muscular Dystrophy, Duchenne | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | Recruiting | 7 Years | N/A | Male | 54 | Phase 1/Phase 2 | United States;Canada | |
| 25 | JPRN-jRCTs031180038 | 18/12/2018 | 22 July 2019 | Tranilast-MD | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD | muscular dystrophy muscular dystrophy ,heart failure ,tranilast;muscular dystrophies | Tranilast 300 mg / day is administered three times per minute. Treatment for 28 weeks (in principle, outpatient administration). As of the 28th week, reconfirmation of consent regarding continuation of administration is confirmed, and if confirmation is obtained, further treatment for 116 weeks is carried out. | Tsuyoshi Matsumura | Recruiting | >=13 age old | Both | 20 | N/A | None | ||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | JPRN-UMIN000031965 | 2018/12/14 | 2 April 2019 | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy | A multicenter, open-label, single-arm study of a TRPV2 inhibitor against cardiomyopathy of muscular dystrophy - Tranilast-MD | Heart failure patients with muscular dystrophy who showed brain natriuretic peptide (BNP) of 100 pg/mL or more in spite of myocardial protection treatment | Tranilast 300 mg/day is administered three times per day as the study treatment. The start date of study treatment is defined as the medication start date, and 28 weeks of study treatment will be performed (in principle, by outpatient administration). We reconfirm consent for continuation of administration at 28 weeks, and if consent is obtained, further treatment for 116 weeks will be carried out. As long as there are no particular problems, the outpatient visit during the observation period is in 4 weeks (21-35 days), 12 weeks (77-91 days), 20 weeks (134-147 days), 24 weeks (161-175 days), and 28 weeks (190-203 days) after starting medication. A prescription until the next outpatient visit will be issued at the consultation. The outpatient visit after consent reconfirmation is done at 12-weeks intervals after 36 weeks, and the prescription until the next outpatient visit will be issued until the next 144 weeks after starting medication at the consultation. Evaluation of various examination findings including clinical findings, cardiac functions, respiratory functions, motor function, QOL Questionnaire (MDQoL-60, SF-12), and adverse events at the designated timing. In order to confirm TRPV2 inhibitory effect by tranilast and to assess its effectiveness as a biomarker, central laboratory tests for the TRPV2 expression analysis will be performed. | National Hospital Organization Toneyama National Hospital | Recruiting | 13years-old | Not applicable | Male and Female | 20 | Not selected | Japan | |
| 27 | NCT03777319 | December 5, 2018 | 11 February 2019 | 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 | Recruiting | 4 Years | 7 Years | Male | 24 | Phase 1 | United States |
| 28 | EUCTR2016-004623-23-GB | 19/11/2018 | 30 April 2019 | A Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy | A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents with Congenital Myotonic Dystrophy - Efficacy & Safety of Tideglusib in Congenital Myotonic Dystrophy | Treatment of child and adolescent congenital myotonic dystrophy. MedDRA version: 20.0 Level: PT Classification code 10068871 Term: Myotonic dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10068871 Term: Myotonic dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: Tideglusib CAS Number: 865854-05-3 Current Sponsor code: AMO-02 Other descriptive name: 4-benzyl-2-naphthalen-1-yl-1,2,4-thiadiazolidine-3,5-dione Concentration unit: mg milligram(s) Concentration type: up to Concentration number: 1000- Pharmaceutical form of the placebo: Powder for oral suspension Route of administration of the placebo: Oral use | AMO Pharma Ltd | Authorised | Female: yes Male: yes | 56 | Phase 2;Phase 3 | United States;Canada;United Kingdom | |||
| 29 | EUCTR2017-001629-41-NL | 15/11/2018 | 28 February 2019 | Study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy | A randomised, double-blind, placebo-controlled study to evaluate the micro-macroscopic effects on muscles, the safety and tolerability, and the efficacy of givinostat in patients with Becker Muscular Dystrophy - NA | Distrofia Muscolare di Becker (DMB) MedDRA version: 20.0 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (hydrochloride monohydrate) Product Code: ITF2357 Pharmaceutical Form: Oral suspension INN or Proposed INN: Givinostat (hydrochloride monohydrate) CAS Number: 732302-99-7 Current Sponsor code: ITF2357 Other descriptive name: GIVINOSTAT Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use | ITALFARMACO S.P.A. | Authorised | Female: no Male: yes | 48 | Phase 2 | Netherlands | |||
| 30 | NCT03642145 | October 31, 2018 | 15 July 2019 | 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 | Not recruiting | 2 Years | 4 Years | Male | 0 | Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT03652259 | October 26, 2018 | 4 November 2019 | Gene Delivery Clinical Trial of SRP-9003 for Patients With LGMD2E (Beta-sarcoglycan Deficiency) | A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (ß-Sarcoglycan Deficiency). | Limb-Girdle Muscular Dystrophy, Type 2E | Drug: SRP-9003 | Sarepta Therapeutics, Inc. | Recruiting | 4 Years | 15 Years | All | 6 | Phase 1/Phase 2 | United States | |
| 32 | NCT03703882 | October 2, 2018 | 22 October 2019 | 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 | Not recruiting | 4 Years | 7 Years | Male | 131 | Phase 3 | United States;Australia;Canada;Germany;Ireland;Israel;Sweden;United Kingdom | |
| 33 | EUCTR2017-002704-27-SE | 15/08/2018 | 27 August 2018 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1.33- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 4- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Prednisone Pharmaceutical Form: Tablet INN or Proposed INN: PREDNISONE CAS Number: 53-03-2 Other descriptive name: PREDNISONE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | ReveraGen BioPharma, Inc. | Authorised | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | |||
| 34 | NCT03558958 | August 8, 2018 | 9 September 2019 | 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 | Not recruiting | 12 Years | 25 Years | Male | 10 | Phase 2 | United States |
| 35 | NCT03532542 | August 2, 2018 | 11 November 2019 | An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy | Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen | Duchenne Muscular Dystrophy | Drug: Casimersen;Drug: Golodirsen | Sarepta Therapeutics, Inc. | Recruiting | 7 Years | 23 Years | Male | 260 | Phase 3 | United States;Belgium;Italy;Spain;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | NCT03603288 | July 4, 2018 | 28 October 2019 | 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 | Recruiting | 11 Years | N/A | Male | 266 | Phase 3 | United States;Austria;Belgium;France;Germany;Italy;Spain;Switzerland;United Kingdom | |
| 37 | NCT03603171 | July 1, 2018 | 26 August 2019 | Clinical Outcome Measures in Myotonic Dystrophy Type 2 | Observational Trial in Myotonic Dystrophy Type 2 to Define Specific Clinical Outcome Measures | Myotonic Dystrophy Type 2 | Diagnostic Test: DM1-ActivC;Diagnostic Test: R-PAct;Diagnostic Test: Beck depression inventory;Diagnostic Test: McGill pain questionnaire;Diagnostic Test: Brief Pain Inventory Short-Form;Diagnostic Test: Fatigue and Daytime Sleepiness Scale;Diagnostic Test: Myotonia Behaviour scale;Diagnostic Test: Hand opening time;Diagnostic Test: Pressure pain threshold;Diagnostic Test: Manual muscle testing;Diagnostic Test: Quantitative muscle testing;Diagnostic Test: Scale for Assessment and Rating of Ataxia;Diagnostic Test: Berg balance scale;Diagnostic Test: Quick motor function test;Diagnostic Test: GSGC;Diagnostic Test: 30 seconds sit to stand test;Diagnostic Test: Functional Index-2;Diagnostic Test: Six minute walking test;Diagnostic Test: Myotonia (from Individualised Neuromuscular Quality of Life Questionnaire) | Prof. Dr. Benedikt Schoser | Recruiting | 18 Years | 90 Years | All | 60 | Phase 2 | Germany | |
| 38 | NCT03439670 | June 19, 2018 | 22 October 2019 | 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 | Recruiting | 4 Years | 7 Years | Male | 120 | Phase 2 | United States;Australia;Belgium;Canada;Czechia;Israel;Netherlands;Spain;Sweden;United Kingdom;Germany;Italy |
| 39 | EUCTR2017-004139-35-BE | 18/06/2018 | 20 August 2018 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Duchenne Muscular Distrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosyntropin acetate Product Code: MNK-1411 Pharmaceutical Form: Suspension for injection INN or Proposed INN: TETRACOSACTIDE HEXAACETATE CAS Number: 22633-88-1 Other descriptive name: Cosyntropin acetate Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Suspension for injection Route of administration of the placebo: Subcutaneous use | Mallinckrodt ARD Inc. | Authorised | Female: no Male: yes | 132 | Phase 2 | United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands | |||
| 40 | EUCTR2017-004139-35-ES | 12/06/2018 | 18 June 2018 | Clinical Trial in Male Subjects with Muscular Disease | A Multicenter, Randomized, Parallel Group, Double Blind, Multiple Dose, Placebo Controlled Study to Assess the Efficacy and Safety of MNK-1411 in Male Subjects 4 to 8 Years of Age With Duchenne Muscular Dystrophy | Duchenne Muscular Distrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Cosyntropin acetate Product Code: MNK-1411 Pharmaceutical Form: Suspension for injection INN or Proposed INN: TETRACOSACTIDE HEXAACETATE CAS Number: 22633-88-1 Other descriptive name: Cosyntropin acetate Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 1- Pharmaceutical form of the placebo: Suspension for injection Route of administration of the placebo: Subcutaneous use | Mallinckrodt ARD Inc. | Authorised | Female: no Male: yes | 132 | Phase 2 | United States;Serbia;Spain;Turkey;Chile;Israel;United Kingdom;Italy;Canada;Argentina;Belgium;Germany;Netherlands | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | NCT03354039 | June 12, 2018 | 17 June 2019 | 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 | Recruiting | 78 Months | 16 Years | Male | 99 | Phase 3 | Germany;Netherlands;Spain;Switzerland;United Kingdom | |
| 42 | NCT03424460 | June 11, 2018 | 22 October 2019 | Venous Thromboembolism in Myotonic Dystrophy Type 1 | Venous Thromboembolism in Myotonic Dystrophy Type 1 | Myotonic Dystrophy 1 | Genetic: RNA extraction;Biological: Monocytes and megacaryocytes culture and RNA extraction;Biological: Haemostasis tests | Assistance Publique - Hôpitaux de Paris | AFM-Téléthon (Funding);Recherche Clinique Paris Descartes Necker Cochin Sainte Anne | Recruiting | 18 Years | N/A | All | 69 | N/A | France |
| 43 | EUCTR2017-002686-21-BE | 28/05/2018 | 30 April 2019 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 Pharmaceutical Form: Solution for infusion INN or Proposed INN: WVE-210201 Current Sponsor code: WVE-210201 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 60- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Intravenous use | Wave Life Sciences Ltd. | Not Recruiting | Female: no Male: yes | 32 | Phase 1 | France;United States;Canada;Belgium;Netherlands;Italy;United Kingdom | |||
| 44 | NCT03406780 | April 4, 2018 | 23 April 2019 | A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy | A Phase 2, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy | Muscular Dystrophies;Muscular Dystrophy, Duchenne;Muscular Disorders, Atrophic;Muscular Diseases;Neuromuscular Diseases;Nervous System Diseases;Genetic Diseases, X-Linked;Genetic Diseases, Inborn | Biological: CAP-1002;Drug: Placebo | Capricor Inc. | Not recruiting | 10 Years | N/A | Male | 18 | Phase 2 | United States | |
| 45 | NCT03375255 | February 5, 2018 | 9 September 2019 | A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD) | A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment | Muscular Dystrophy, Duchenne | Drug: SRP-5051 | Sarepta Therapeutics, Inc. | Not recruiting | 12 Years | N/A | Male | 15 | Phase 1 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT03508947 | January 24, 2018 | 23 April 2019 | 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. | Not recruiting | 5 Years | 18 Years | Male | 36 | Phase 1 | United States;Belgium;Canada;France;Italy;Netherlands;United Kingdom | |
| 47 | EUCTR2017-001223-49-BG | 16/01/2018 | 20 August 2018 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use | PTC Therapeutics, Inc. | Authorised | Female: no Male: yes | 250 | Phase 3 | United States;Taiwan;Hong Kong;Turkey;Russian Federation;India;Mexico;Canada;Argentina;Poland;Malaysia;Brazil;Australia;Bulgaria;China;Japan;Korea, Republic of | |||
| 48 | EUCTR2017-003568-10-GB | 05/01/2018 | 21 May 2018 | An Extension Study to Assess the Long-term Safety and Efficacy of 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) - VBP15-LTE | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: Vamorolone CAS Number: 13209-41-1 Other descriptive name: EV substance code: SUB188638 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: Vamorolone CAS Number: 13209-41-1 Other descriptive name: EV substance code: SUB188638 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: Vamorolone CAS Number: 13209-41-1 Other descriptive name: EV substance code: SUB188638 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: Vamorolone CAS Number: 13209-41-1 Other descriptive name: EV substance code: SUB188638 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | ReveraGen BioPharma Inc. | Authorised | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | |||
| 49 | EUCTR2018-000464-29-IE | 10 December 2018 | A study to compare edasalonexent with placebo in pediatric patients with Duchenne Muscular dystrophy. | A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - Phase 3 Study of Edasalonexent in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 Pharmaceutical Form: Capsule, soft INN or Proposed INN: Edasalonexent Current Sponsor code: CAT-1004 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 100-250 Pharmaceutical form of the placebo: Capsule, soft Route of administration of the placebo: Oral use | Catabasis Pharmaceuticals, Inc. | Not Available | Female: no Male: yes | 126 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | ||||
| 50 | EUCTR2018-000464-29-SE | 7 January 2019 | A study to compare edasalonexent with placebo in patients with Duchenne Muscular dystrophy. | A RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, GLOBAL PHASE 3 STUDY OF EDASALONEXENT IN PEDIATRIC PATIENTS WITH DUCHENNE MUSCULAR DYSTROPHY - POLARIS DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Edasalonexent Product Code: CAT-1004 Pharmaceutical Form: Capsule, soft INN or Proposed INN: Edasalonexent Current Sponsor code: CAT-1004 Concentration unit: mg milligram(s) Concentration type: range Concentration number: 100-250 Pharmaceutical form of the placebo: Capsule, soft Route of administration of the placebo: Oral use | Catabasis Pharmaceuticals, Inc. | Not Available | Female: no Male: yes | 126 | Phase 3 | United States;Canada;Ireland;Australia;Israel;Germany;United Kingdom;Sweden | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | EUCTR2018-000975-34-FR | 20 August 2018 | A Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | A Multicenter, Open-Label Extension Study of WVE-210201 in Patients previously enrolled in WVE-DMDX51-001 | Duchenne muscular dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 Pharmaceutical Form: Solution for infusion INN or Proposed INN: WVE-210201 Current Sponsor code: WVE-210201 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 60- | Wave Life Sciences Ltd. | Not Available | Female: no Male: yes | 40 | Phase 1 | United States;France;Canada;Belgium;Netherlands;Italy;United Kingdom | ||||
| 52 | NCT03123913 | December 18, 2017 | 18 March 2019 | 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 | Recruiting | 18 Years | 65 Years | Male | 20 | Phase 1 | United States | |
| 53 | NCT03238235 | December 12, 2017 | 16 September 2019 | 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 | Recruiting | 18 Years | 65 Years | Male | 48 | Phase 2 | Italy;Netherlands | |
| 54 | NCT03375164 | December 11, 2017 | 24 June 2019 | Systemic Gene Delivery Clinical Trial for Duchenne Muscular Dystrophy | Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin | Duchenne Muscular Dystrophy | Drug: rAAVrh74.MHCK7.micro-dystrophin | Sarepta Therapeutics | Not recruiting | 3 Months | 7 Years | Male | 4 | Phase 1/Phase 2 | United States | |
| 55 | EUCTR2016-001654-18-DE | 13/11/2017 | 28 February 2019 | Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy | A randomized, double blind, placebo-controlled, study to assess the efficacy, safety, and tolerability of RO7239361 in ambulatory boys with Duchenne Muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: RO7239361 Injections, 7.5 mg/Syringe (10.7 mg/mL) Product Code: RO7239361 Pharmaceutical Form: Injection INN or Proposed INN: Anti-myostatin Adnectin Current Sponsor code: RO7239361 Other descriptive name: BMS-986089-01, anti-myostatin Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10.7- Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe Route of administration of the placebo: Subcutaneous use Product Name: RO7239361 Injections, 15 mg/Syringe (21.4 mg/mL) Product Code: RO7239361 Pharmaceutical Form: Injection INN or Proposed INN: Anti-myostatin Adnectin Current Sponsor code: RO7239361 Other descriptive name: BMS-986089-01, anti-myostatin Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 21.4- Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe Route of administration of the placebo: Subcutaneous use Product Name: RO7239361 Injections, 35 mg/Syringe (50 mg/mL) Product Code: RO7239361 Pharmaceutical Form: Injection INN or Proposed INN: Anti-myostatin Adnectin Current Sponsor code: RO7239361 Other descriptive name: BMS-986089-01, anti-myostatin Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Solution for injection in pre-filled syringe | F. Hoffmann-La Roche Ltd | Authorised | Female: no Male: yes | 159 | Phase 3 | United States;Spain;United Kingdom;Italy;France;Canada;Argentina;Belgium;Australia;Germany;Netherlands;Japan;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | NCT03333590 | November 6, 2017 | 20 May 2019 | Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy | Phase I/IIa Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 | Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.GALGT2 | Kevin Flanigan | Not recruiting | 4 Years | N/A | Male | 6 | Phase 1/Phase 2 | United States | |
| 57 | NCT03218995 | August 16, 2017 | 26 August 2019 | 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. | Recruiting | 6 Months | 48 Months | Male | 12 | Phase 2 | Belgium;France;Italy;United Kingdom;Germany | |
| 58 | NCT03863119 | July 15, 2017 | 18 March 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. | Not recruiting | N/A | N/A | Male | N/A | United States | ||
| 59 | NCT03039686 | July 6, 2017 | 4 November 2019 | 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 | Not recruiting | 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 | |
| 60 | NCT03167255 | July 6, 2017 | 1 April 2019 | 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) | Not recruiting | 4 Years | 10 Years | Male | 16 | Phase 2 | United States;Canada |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | NCT03179631 | July 6, 2017 | 11 November 2019 | 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 | Recruiting | 5 Years | N/A | Male | 250 | Phase 3 | United States;Australia;Brazil;Bulgaria;Canada;China;Hong Kong;India;Japan;Jordan;Korea, Republic of;Malaysia;Mexico;Poland;Puerto Rico;Russian Federation;Sri Lanka;Taiwan;Thailand;Turkey;Argentina;Chile | |
| 62 | NCT02851797 | June 1, 2017 | 7 October 2019 | 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 | Recruiting | 6 Years | 17 Years | Male | 213 | Phase 3 | United States;Belgium;Canada;France;Germany;Italy;Netherlands;Spain;United Kingdom |
| 63 | EUCTR2016-000951-29-BE | 29/05/2017 | 28 May 2018 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: eteplirsen CAS Number: 1173755-55-9 Current Sponsor code: AVI-4658 Other descriptive name: ETEPLIRSEN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Sarepta Therapeutics, Inc | Authorised | Female: no Male: yes | 12 | Phase 2 | France;Belgium;Germany;Italy | |||
| 64 | EUCTR2016-004462-26-GB | 16/02/2017 | 28 February 2019 | A Study to Assess Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | VBP15-002 A Phase IIa Open-Label, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 19.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.25- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.75- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 2.0- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 6.0- | ReveraGen BioPharma, Inc. | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom | |||
| 65 | EUCTR2016-004263-38-SE | 13/02/2017 | 10 October 2018 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne Muscular Dystrophy (DMD) MedDRA version: 19.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Current Sponsor code: 17a,21-dihydroxy-16a-methyl-pregna-1,4,9(11)-triene-3,20-dione Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.25- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.75- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 2.0- Product Name: Vamorolone Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 6.0- | ReveraGen BioPharma Inc. | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | EUCTR2016-004263-38-GB | 10/02/2017 | 28 February 2019 | An Extension Study to Assess the Long-term Safety and Efficacy of Vamorolone in Boys With Duchenne Muscular Dystrophy (DMD) | A Phase II Open-label, Multicenter Extension Study to Assess the Longterm Safety and Efficacy of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamarolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.25- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0.75- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 2.0- Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: vamorolone CAS Number: 13209-41-1 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 6.0- | ReveraGen BioPharma, Inc. | Not Recruiting | Female: no Male: yes | 48 | Phase 2 | United States;Canada;Australia;Israel;United Kingdom;Sweden | |||
| 67 | NCT03038399 | February 2, 2017 | 14 October 2019 | 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 | Not recruiting | 4 Years | 7 Years | Male | 46 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 68 | EUCTR2017-000397-10-IT | 5 February 2018 | Studio nel quale tutti i pazienti prendono lo stesso farmaco sperimentale con lo scopo di valutare a lungo termine la sicurezza, la tollerabilità e l'efficacia del GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in uno studio con GIVINOSTAT. | Studio in aperto a lungo termine volto a valutare la sicurezza, la tollerabilità e l'efficacia di GIVINOSTAT in pazienti affetti da distrofia muscolare di Duchenne che sono stati precedentemente trattati in studio con GIVINOSTAT. - Studio con Givinostat in DMD a lungo termine | Distrofia Muscolare di Duchenne (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat (idrocloruro monoidrato) Product Code: ITF2357 Pharmaceutical Form: Oral suspension INN or Proposed INN: Givinostat (idrocloruro monoidrato) CAS Number: 732302-99-7 Current Sponsor code: ITF2357 Other descriptive name: GIVINOSTAT Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | ITALFARMACO S.p.A. | Not Available | Female: no Male: yes | 100 | Phase 3 | France;United States;Canada;Belgium;Spain;Netherlands;Germany;United Kingdom;Italy | ||||
| 69 | EUCTR2017-001223-49-PL | 15 October 2018 | A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension | Nonsense Mutation Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Translarna 125 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use Trade Name: Translarna 250 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use Trade Name: Translarna 1000 mg granules for oral suspension Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Granules in sachet INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- Pharmaceutical form of the placebo: Granules in sachet Route of administration of the placebo: Oral use | PTC Therapeutics, Inc. | Not Available | Female: no Male: yes | 250 | Phase 3 | United States;Taiwan;Hong Kong;Turkey;Russian Federation;India;Mexico;Canada;Argentina;Malaysia;Poland;Brazil;Australia;Bulgaria;China;Japan;Korea, Republic of | ||||
| 70 | EUCTR2017-002686-21-FR | 25 June 2018 | A study to assess the safety and tolerability of different doses of WVE-210201 in patients with Duchenne Muscular Dystrophy | A Multicenter, Double-blind, Placebo-controlled, Phase 1 Study of WVE-210201 Administered Intravenously to Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: WVE-210201 Product Code: WVE-210201 Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: WVE-210201 Current Sponsor code: WVE-210201 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Intravenous use | Wave Life Sciences Ltd. | Not Available | Female: no Male: yes | 32 | Phase 1 | United States;France;Czech Republic;Belgium;Spain;Ireland;Denmark;Netherlands;Italy;United Kingdom | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | EUCTR2017-002704-27-BE | 30 April 2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD IIb | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1.33- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 4- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Prednisone Pharmaceutical Form: Tablet INN or Proposed INN: PREDNISONE CAS Number: 53-03-2 Other descriptive name: PREDNISONE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | ReveraGen BioPharma, Inc. | Not Available | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Germany;Netherlands;Sweden | ||||
| 72 | EUCTR2017-002704-27-NL | 28 February 2019 | A Study to Assess the efficacy and the safety of Vamorolone in Boys with Duchenne Muscular Dystrophy (DMD) | A Phase IIb Randomized, Double-blind, Parallel Group, Placebo- and Active-controlled Study with Double-Blind Extension to Assess the Efficacy and Safety of Vamorolone in Ambulant Boys with Duchenne Muscular Dystrophy (DMD) - VISION DMD | Duchenne muscular dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 1.33- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Vamorolone Product Code: VBP15 Pharmaceutical Form: Oral suspension INN or Proposed INN: VAMOROLONE CAS Number: 13209-41-1 Current Sponsor code: VBP15 Concentration unit: % (W/W) percent weight/weight Concentration type: equal Concentration number: 4- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use Product Name: Prednisone Pharmaceutical Form: Tablet INN or Proposed INN: PREDNISONE CAS Number: 53-03-2 Other descriptive name: PREDNISONE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | ReveraGen BioPharma, Inc. | Not Available | Female: no Male: yes | 120 | Phase 2 | United States;Greece;Spain;Israel;United Kingdom;Italy;Czech Republic;Canada;Belgium;Australia;Netherlands;Germany;Sweden | ||||
| 73 | EUCTR2017-004279-30-SE | 30 April 2019 | A clinical study to assess the long-term safety and efficacy of idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study. | A Phase III Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of Idebenone in Patients with Duchenne Muscular Dystrophy (DMD) who completed the SIDEROS study - SIDEROS-E | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IDEBENONE CAS Number: 58186-27-9 Other descriptive name: IDEBENONE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- | Santhera Pharmaceuticals (Switzerland) Limited | Not Available | Female: no Male: yes | 266 | Phase 3 | France;United States;Belgium;Spain;Austria;Israel;Netherlands;Germany;Italy;United Kingdom;Switzerland;Sweden | ||||
| 74 | EUCTR2017-004554-42-DE | 19 November 2018 | The study examines boys suffering from Duchenne muscular dystrophy. We are carrying out this study to examine the effect and tolerance of Tamoxifen in this disease. | Tamoxifen in Duchenne muscular dystrophy - TAMDMD A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial | Duchenne muscular dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: TAMOXIFEN Pharmaceutical Form: Film-coated tablet INN or Proposed INN: Tamoxifen CAS Number: 54965-24-1 Other descriptive name: TAMOXIFEN CITRATE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | University of Basel Children's Hospital, Division of Neuropediatrics | Not Available | Female: no Male: yes | 100 | Phase 3 | Turkey;Netherlands;Germany;Switzerland | ||||
| 75 | NCT02740972 | December 2016 | 26 August 2019 | 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 | Not recruiting | 4 Years | 9 Years | Male | 16 | Phase 2 | United States;Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 76 | EUCTR2016-000602-10-DE | 17/11/2016 | 30 April 2019 | A clinical study to assess how effective and safe is idebenone treatment in patients with Duchenne Muscular Dystrophy (DMD) who are currently receiving Glucocorticoid steroids | A Phase III Double-blind, Randomized, Placebo-Controlled Study assessing the Efficacy, Safety and Tolerability of Idebenone in Patients with Duchenne Muscular Dystrophy Receiving Glucocorticoid Steroids - SIDEROS | Duchenne Muscular Dystrophy (DMD) MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Raxone Product Name: Idebenone Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IDEBENONE CAS Number: 58186-27-9 Other descriptive name: IDEBENONE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | Santhera Pharmaceuticals (Switzerland) Limited | Authorised | Female: no Male: yes | 266 | Phase 3 | United States;Spain;Ireland;Austria;Israel;Switzerland;United Kingdom;Italy;France;Hungary;Belgium;Bulgaria;Netherlands;Germany;Sweden | |||
| 77 | NCT02835079 | November 2016 | 20 August 2018 | Treatment Effect of Tamoxifen on Patients With DMD | Treatment Effect of Tamoxifen on Patients With DMD | Duchenne Muscular Dystrophy | Drug: Tamoxifen | Hadassah Medical Organization | Not recruiting | 5 Years | 16 Years | Male | 19 | Phase 1 | ||
| 78 | NCT02927080 | November 2016 | 11 November 2019 | 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. | Not recruiting | 18 Years | N/A | All | 95 | Phase 2 | United States;Canada;Spain | |
| 79 | NCT02964377 | November 2016 | 28 January 2019 | 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. | Not recruiting | 8 Years | 17 Years | Male | 15 | Phase 1/Phase 2 | United States |
| 80 | NCT03236662 | November 2016 | 18 June 2018 | (-)- 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. | Not recruiting | 18 Years | 70 Years | Male | 10 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 81 | NCT02907619 | October 13, 2016 | 20 May 2019 | An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy | A MULTICENTER, OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG TERM SAFETY OF PF-06252616 IN BOYS WITH DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Biological: PF-06252616 | Pfizer | Not recruiting | 6 Years | 18 Years | Male | 59 | Phase 2 | United States;Canada;Italy;Japan;United Kingdom | |
| 82 | NCT02858908 | October 2016 | 11 June 2018 | 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 | Not recruiting | 12 Years | 45 Years | All | Phase 2 | United Kingdom | ||
| 83 | NCT02877784 | October 2016 | 4 November 2019 | Screening in Oculopharyngeal Muscular Dystrophy | Determination of Accurate Screening Tools for Dysphagia in Oculopharyngeal Muscular Dystrophy | Oculopharyngeal Muscular Dystrophy | Other: The Center for Neurologic Studies Bulbar Function Scale;Device: Iowa Oral Performance Instrument;Drug: Capsaicin;Procedure: Videofluoroscopic swallowing study;Other: Swallowing Related Quality of Life Questionnaire;Other: Functional Oral Intake Scale;Other: Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised;Other: Eating Assessment Tool-10;Other: Communicative Effectiveness Survey | University of Florida | Recruiting | 18 Years | 80 Years | All | 30 | Early Phase 1 | United States | |
| 84 | NCT02500381 | September 28, 2016 | 22 July 2019 | Study of SRP-4045 and SRP-4053 in DMD Patients | 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. | Recruiting | 7 Years | 13 Years | Male | 222 | Phase 3 | United States;Australia;Belgium;Bulgaria;Canada;Czechia;France;Germany;Hungary;Israel;Italy;Poland;Spain;Sweden;United Kingdom | |
| 85 | NCT02760277 | July 28, 2016 | 26 August 2019 | 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) | Not recruiting | 4 Years | 7 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 86 | NCT02836418 | July 12, 2016 | 16 December 2017 | 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. | Not recruiting | 16 Years | 25 Years | All | 8 | Phase 1/Phase 2 | United States;Denmark;Italy | |
| 87 | EUCTR2014-002008-25-FR | 06/07/2016 | 30 April 2019 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 19.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4053 Product Code: SRP-4053 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Not Available CAS Number: 1422959-91-8 Current Sponsor code: SRP-4053 Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Solution for infusion Route of administration of the placebo: Intravenous use | Sarepta Therapeutics, Inc. | Not Recruiting | Female: no Male: yes | 48 | Phase 1;Phase 2 | France;Italy;United Kingdom | |||
| 88 | NCT02878694 | July 2016 | 5 September 2016 | Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft | Treatment of Ptosis to Muscular Dystrophy Oculopharyngeal by Myoblast Autologous Graft | Muscular Dystrophy, Oculopharyngeal;Ptosis | Biological: Myoblast autologous graft | University Hospital, Caen | University Hospital, Rouen;Rennes University Hospital;Centre Hospitalier Universitaire, Amiens;University Hospital, Lille;University Hospital, Brest;Groupe Hospitalier Pitie-Salpetriere | Not recruiting | 18 Years | 75 Years | Both | 10 | Phase 2/Phase 3 | France |
| 89 | EUCTR2016-000624-25-DK | 22/06/2016 | 22 May 2017 | An Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Fascioscapulohumeral Muscular Dystrophy | Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 19.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Not available yet Current Sponsor code: ATYR1940 Other descriptive name: ATYR1940 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- | aTyr Pharma, Inc. | Not Recruiting | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Denmark;Italy | |||
| 90 | NCT02819557 | June 16, 2016 | 4 February 2019 | Study of Ataluren in =2 to <5 Year-Old Males 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 | Not recruiting | 2 Years | 5 Years | Male | 14 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 91 | NCT02760264 | June 2016 | 18 January 2018 | 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 6.0 mg/kg/day;Drug: Vamorolone 2.0 mg/kg/day;Drug: Vamorolone 0.75 mg/kg/day;Drug: Vamorolone 0.25 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 | Not recruiting | 4 Years | 6 Years | Male | 48 | Phase 2 | United States;Australia;Canada;Israel;Sweden;United Kingdom |
| 92 | NCT02808585 | June 2016 | 8 April 2019 | 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. | Not recruiting | 18 Years | N/A | All | 29 | Phase 2 | United States | |
| 93 | NCT02858362 | June 2016 | 20 May 2019 | PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD | A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With SMT C1100 in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Drug: ezutromid | Summit Therapeutics | Not recruiting | 5 Years | 10 Years | Male | 40 | Phase 2 | United States;United Kingdom | |
| 94 | EUCTR2014-005296-81-BE | 02/05/2016 | 8 August 2016 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the efficacy, safety and tolerability. | A 24 week Randomized Double-Blind, Placebo-Controlled Study followed by 72 week open-label extension to assess the efficacy, safety and tolerability of drisapersen sodium in subjects with Duchenne Muscular Dystrophy - Drisapersen confirmatory study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen Pharmaceutical Form: Solution for injection INN or Proposed INN: Drisapersen CAS Number: 1251830-50-8 Current Sponsor code: BMN-051 Other descriptive name: DRISAPERSEN SODIUM Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | BioMarin Pharmaceutical Inc. | Not Recruiting | Female: no Male: yes | 104 | Phase 3 | United States;Spain;Turkey;Russian Federation;Israel;United Kingdom;Italy;France;Czech Republic;Canada;Poland;Belgium;Australia;Germany;Japan;Sweden | |||
| 95 | NCT02752048 | May 2016 | 11 February 2019 | 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. | Not recruiting | 5 Years | N/A | Male | 36 | Phase 2 | Japan | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 96 | EUCTR2016-000067-16-GB | 20/04/2016 | 10 September 2018 | A clinical trial to investigate how safe and effective tideglusib is, as treatment for adolescents and adults with myotonic dystrophy diagnosed before they were 12 years old. | A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400 mg Or 1000 mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy | Treatment of adolescent and adult congenital and juvenile onset myotonic dystrophy MedDRA version: 19.1 Level: PT Classification code 10068871 Term: Myotonic dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Tideglusib Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: Tideglusib Other descriptive name: 4-Benzyl-2-naphthalen-1-yl-1,2,4- thiadiazolidine-3,5-dione Concentration unit: mg milligram(s) Concentration type: up to Concentration number: 1000- Pharmaceutical form of the placebo: Powder for oral suspension Route of administration of the placebo: Oral use | AMO Pharma Ltd. | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United Kingdom | |||
| 97 | NCT02704325 | April 2016 | 12 February 2018 | Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 | Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 | Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.GALGT2;Other: PLACEBO (Saline) | Kevin Flanigan | Not recruiting | 9 Years | N/A | Male | 0 | Phase 1/Phase 2 | United States | |
| 98 | NCT02958202 | April 2016 | 5 February 2018 | 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 | Not recruiting | 5 Years | N/A | Male | 7 | Phase 2 | Belgium;Italy;Netherlands;Sweden | |
| 99 | NCT02603562 | March 30, 2016 | 16 December 2017 | Evaluate Safety and Biological Activity of ATYR1940 in Patients With Early Onset Facioscapulohumeral Muscular Dystrophy | An Open-Label, Intrapatient Dose-Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Early Onset and Other Pediatric Onset Facioscapulohumeral Muscular Dystrophy | Facioscapulohumeral Muscular Dystrophy (FSHD) | Biological: ATYR1940;Biological: Placebo | aTyr Pharma, Inc. | Not recruiting | 12 Years | 25 Years | All | 8 | Phase 1/Phase 2 | United States;France;Italy | |
| 100 | EUCTR2015-004333-27-GB | 19/01/2016 | 28 February 2019 | A clinical trial to test how the study medication (SMT C1100) works and how safe it is when given to children with Duchenne Muscular Dystrophy | A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD | Duchenne Muscular Dystrophy MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 Pharmaceutical Form: Oral suspension INN or Proposed INN: Pending CAS Number: 945531-77-1 Current Sponsor code: SMT C1100 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 200- | Summit (Oxford) Limited | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | United States;United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 101 | JPRN-UMIN000020580 | 2016/01/15 | 2 April 2019 | Pilot study of tranilast for cardiomyopathy of muscular dystrophy | muscular dystrophy | Tranilast 300mg/day for three months (oral intake) | National Hospital Organization Toneyama National Hospital | National Cerebral and Cardiovascular Center | Not Recruiting | 20years-old | Not applicable | Male and Female | 2 | Not applicable | Japan | |
| 102 | EUCTR2015-003681-87-BE | 12/01/2016 | 8 August 2016 | A study to assess the effect of BMN 044 in subjects with Duchenne muscular dystrophy (Extension study) | A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 044 Product Code: BMN 044 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: PS188 (company code) CAS Number: 1802402-63-6 Current Sponsor code: PS188 Other descriptive name: PS188 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Product Name: BMN 044 Product Code: BMN 044 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: PS188 (company code) CAS Number: 1802402-63-6 Current Sponsor code: PS188 Other descriptive name: PS188 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | BioMarin Pharmaceutical Inc. | Not Recruiting | Female: no Male: yes | 50 | Phase 2 | United States;Belgium;Netherlands;Italy;Sweden | |||
| 103 | NCT02485938 | January 2016 | 20 August 2018 | 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. | Not recruiting | 12 Years | N/A | Male | 25 | Phase 1/Phase 2 | United States | |
| 104 | EUCTR2016-000951-29-FR | 24 July 2017 | A research study of a new investigational medicinal product for the treatment of young Duchenne Muscular Dystrophy patients | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Exondys 51 Product Name: Eteplirsen Product Code: AVI-4658 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: eteplirsen CAS Number: 1173755-55-9 Current Sponsor code: AVI-4658 Other descriptive name: ETEPLIRSEN Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Sarepta Therapeutics, Inc | Not Available | Female: no Male: yes | 12 | Phase 1 | France | ||||
| 105 | EUCTR2015-001910-88-DK | 17/12/2015 | 2 October 2017 | A Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies | An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies | Limb Girdle Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy MedDRA version: 19.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Not available yet Current Sponsor code: ATYR1940 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- Pharmaceutical form of the placebo: Concentrate for solution for infusion Route of administration of the placebo: Intravenous use | aTyr Pharma, Inc. | Not Recruiting | Female: yes Male: yes | 16 | Phase 1;Phase 2 | France;United States;Denmark;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 106 | EUCTR2015-001955-54-NL | 08/12/2015 | 8 August 2016 | A clinical study to provide drisapersen (study medication) to patients with Duchenne disease (muscular disease) and to assess the safety, tolerability and effect by giving the medication long term. | An open-label extension study of the long-term safety, tolerability and efficacy of drisapersen in subjects with Duchenne Muscular Dystrophy. - Drisapersen extension study | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: Drisapersen Pharmaceutical Form: Solution for injection | BioMarin Nederland BV | Not Recruiting | Female: no Male: yes | 220 | Phase 3 | Portugal;United States;Belarus;Taiwan;Spain;Ukraine;Russian Federation;Chile;Israel;Switzerland;Italy;France;Australia;Denmark;Netherlands;Korea, Republic of;Uruguay;Turkey;Austria;United Kingdom;Czech Republic;Hungary;Argentina;Poland;Belgium;Brazil;Bulgaria;Norway;Germany;Japan;Sweden | |||
| 107 | NCT02515669 | December 2, 2015 | 26 August 2019 | 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 | Not recruiting | 5 Years | 10 Years | Male | 43 | Phase 1/Phase 2 | United States;Canada | |
| 108 | NCT02653833 | December 2015 | 16 December 2017 | 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 | Recruiting | 18 Years | 45 Years | Male | 19 | Early Phase 1 | United States | |
| 109 | NCT02636686 | November 27, 2015 | 29 January 2018 | 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 | Not recruiting | 5 Years | 80 Years | Male | N/A | 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 | ||
| 110 | EUCTR2015-002530-50-ES | 04/11/2015 | 19 February 2018 | Rimeporide in patients with Duchenne Muscular Dystrophy | A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy. - Rim4DMD | Duchenne Muscular Dystrophy (DMD) MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Rimeporide Product Code: EMD 87580 Pharmaceutical Form: Capsule, hard INN or Proposed INN: N(4,5Bis methanesulfonyl2methylbenzoyl) guanidine hydrochloride monohydrate Current Sponsor code: EMD 87580 Other descriptive name: Rimeporide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 25- | EspeRare | Not Recruiting | Female: no Male: yes | 20 | Phase 1 | France;Spain;Italy;United Kingdom;Switzerland | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 111 | NCT02571205 | November 2015 | 1 October 2018 | 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 | Not recruiting | 12 Years | 17 Years | Male | 15 | Phase 1/Phase 2 | United Kingdom | |
| 112 | NCT02606136 | November 2015 | 4 February 2019 | 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 | Not recruiting | 12 Years | N/A | Male | 22 | Phase 2 | United States | |
| 113 | NCT02530905 | October 14, 2015 | 3 December 2018 | 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 | Not recruiting | 7 Years | 21 Years | Male | 12 | Phase 1 | United States | |
| 114 | NCT02592941 | October 2015 | 12 March 2018 | 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 | Not recruiting | 5 Years | N/A | All | N/A | United States | |
| 115 | NCT02667483 | October 2015 | 15 April 2019 | 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. | Not recruiting | 5 Years | 10 Years | Male | 7 | Phase 1/Phase 2 | Japan |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 116 | NCT02531217 | September 2015 | 2 July 2018 | Safety, Tolerability, PK, and Activity of ATYR1940 in Patients With Muscular Dystrophy - Study Extension | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients With Fascioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy | Biological: ATYR1940 | aTyr Pharma, Inc. | Not recruiting | 18 Years | 65 Years | All | 9 | Phase 1/Phase 2 | United States;Italy;Netherlands | |
| 117 | NCT03067831 | September 2015 | 18 June 2018 | Bone Marrow-Derived Autologous Stem Cells for the Treatment of Duchenne Muscular Dystrophy | Safety and Efficacy of Purified Autologous Bone Marrow-Derived Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy. | Duchenne Muscular Dystrophy | Biological: Stem Cells | Stem Cells Arabia | Recruiting | 4 Years | 25 Years | All | 20 | Phase 1/Phase 2 | Jordan | |
| 118 | EUCTR2015-001912-36-NL | 04/08/2015 | 22 May 2017 | A Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Exposure of ATYR1940 in Adult Patients with Genetic Myopathy | An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) | Facioscapulohumeral muscular dystrophy MedDRA version: 19.0 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Not available yet Current Sponsor code: ATYR1940 Other descriptive name: ATYR1940 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- | aTyr Pharma, Inc. | Not Recruiting | Female: yes Male: yes | 32 | Phase 1;Phase 2 | United States;Netherlands;Italy | |||
| 119 | EUCTR2015-001967-38-GB | 03/08/2015 | 11 April 2016 | A research project for boys with Duchenne muscular dystrophy aged 5-9 years. It aims to find out how safe and well tolerated different doses of the experimental medicine are, when given by mouth after breakfast and evening meal. | A Phase I, 2-Part, Open-label, Multiple Oral Dose Study of the Safety, Tolerability and Pharmacokinetics of up to 2 Formulations of SMT C1100 in Healthy Adult Male Subjects and a Selected Formulation of SMT C1100 in Paediatric Subjects with Duchenne Muscular Dystrophy (DMD) - SMTC11004 - Phase 1 study in healthy volunteers and DMD patients | Duchenne muscular dystrophy (DMD) MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SMT C1100 (F5; nanosuspension) Product Code: SMT C1100 (F5) Pharmaceutical Form: Oral suspension Current Sponsor code: SMT-C1100 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 100- Product Name: SMT C1100 (F6; Powder for Oral Suspension) Product Code: SMT C1100 (F6) Pharmaceutical Form: Powder for oral suspension Current Sponsor code: SMT-C1100 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 250- | Summit (Oxford) Limited | Authorised | Female: no Male: yes | Phase 1 | United Kingdom | ||||
| 120 | NCT02420379 | July 1, 2015 | 11 March 2019 | 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 | Not recruiting | 4 Years | 6 Years | Male | 33 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 121 | NCT02434627 | June 2015 | 24 October 2016 | Sodium Nitrate for Muscular Dystrophy | Sodium Nitrate for Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Sodium Nitrate | Cedars-Sinai Medical Center | Recruiting | 15 Years | 45 Years | Male | 12 | Phase 1 | United States | |
| 122 | NCT02484560 | June 2015 | 3 August 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 | Not recruiting | 8 Years | 14 Years | Male | 10 | Phase 1 | Turkey |
| 123 | NCT02525302 | May 2015 | 25 March 2019 | 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 | Not recruiting | 6 Years | 20 Years | Male | 10 | Phase 2 | United States | |
| 124 | NCT02369731 | April 2015 | 16 December 2017 | Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystrophy | Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care | Muscular Dystrophy, Duchenne | Drug: Translarna | PTC Therapeutics | TREAT-NMD;Cooperative International Neuromuscular Research Group (CINRG) | Recruiting | N/A | N/A | All | 200 | N/A | Austria;France;Germany;Israel;Sweden |
| 125 | NCT02354352 | March 20, 2015 | 25 February 2019 | 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 | Not recruiting | 7 Years | N/A | Male | 52 | Phase 3 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 126 | NCT02376816 | March 2015 | 16 December 2017 | Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy | Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.Micro-dystrophin | Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.micro-Dystrophin | Jerry R. Mendell | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Not recruiting | 7 Years | N/A | Male | 2 | Phase 1 | United States |
| 127 | NCT01976091 | February 2015 | 26 August 2019 | Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA | Phase I/IIa Gene Transfer Clinical Trial for LGMD2D (Alpha-sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA | Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) | Drug: scAAVrh74.tMCK.hSGCA | Jerry R. Mendell | Not recruiting | 7 Years | N/A | All | 6 | Phase 1/Phase 2 | United States | |
| 128 | NCT02383511 | February 2015 | 7 September 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 | Not recruiting | 5 Years | 13 Years | Male | 12 | Phase 1 | United Kingdom | |
| 129 | NCT02310906 | January 13, 2015 | 24 June 2019 | 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 | 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 | Not recruiting | 6 Years | 15 Years | Male | 39 | Phase 1/Phase 2 | United States;France;Italy;United Kingdom |
| 130 | NCT02328482 | January 2015 | 29 July 2019 | 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. | Not recruiting | 18 Years | 80 Years | All | 9 | Phase 3 | Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 131 | NCT02354781 | January 2015 | 16 December 2017 | Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy | Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: rAAV1.CMV.huFollistin344 | Jerry R. Mendell | Duchenne Alliance;Milo Therapeutics | Not recruiting | 7 Years | N/A | Male | 3 | Phase 1/Phase 2 | United States |
| 132 | JPRN-UMIN000016092 | 2014/12/31 | 2 April 2019 | Analysis of the effects of animal fat free diet and aspirin for prostaglandins in patients with myotonic dystrophy | myotonic dystrophy | 1st stage (1w): animal fat free diet 2nd stage (1w): animal fat free diet and aspirin (330mg/day) | National Hospital Organization Toneyama National Hospital | Graduate school of engineering Osaka University | Not Recruiting | Not applicable | Not applicable | Male and Female | 10 | Not applicable | Japan | |
| 133 | EUCTR2014-002072-92-GB | 30/12/2014 | 19 September 2016 | A Phase 2 Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Duchenne Muscular Dystrophy | A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study to Evaluate The Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 In Ambulatory Boys With Duchenne Muscular Dystrophy | Duchenne's Muscular Dystrophy MedDRA version: 19.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: PF-06252616 Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: PF-06252616 Current Sponsor code: PF-06252616 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Solution for injection/infusion Route of administration of the placebo: Intravenous use Product Code: PF-06252616 Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: PF-06252616 Current Sponsor code: PF-06252616 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 260- Pharmaceutical form of the placebo: Solution for injection/infusion Route of administration of the placebo: Intravenous use | Pfizer Inc. 235 East 42nd Street, New York, NY 10017 | Authorised | Female: no Male: yes | 105 | Phase 2 | Canada;Poland;Australia;Bulgaria;Japan;United Kingdom;Italy;United States | |||
| 134 | EUCTR2014-003100-78-GB | 01/12/2014 | 30 March 2015 | A clinical trial to test if the drug SMT C1100 is safe and well absorbed compared to placebo when given to children with Duchenne Muscular Dystrophy (DMD), who follow a balanced diet. | SMT C11003 - A Phase 1b placebo-controlled, multi-centre, randomized, double-blind 3-period dose escalation study to evaluate the pharmacokinetics (PK) and safety of SMT C1100 in paediatric patients with Duchenne Muscular Dystrophy (DMD) who follow a balanced diet. | Duchenne Muscular Dystrophy MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Code: SMT C1100 Pharmaceutical Form: Oral suspension INN or Proposed INN: Not available CAS Number: 945531-77-1 Current Sponsor code: SMT C1100 Other descriptive name: SMT C1100 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use | Summit (Oxford) Limited | Authorised | Female: no Male: yes | Phase 1b | United Kingdom | ||||
| 135 | NCT01805024 | December 2014 | 11 June 2018 | 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 | Not recruiting | 5 Years | 16 Years | All | 20 | Phase 1 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 136 | NCT02251600 | December 2014 | 16 December 2017 | 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 | Not recruiting | 4 Years | 16 Years | Male | 24 | Phase 1 | United States | |
| 137 | NCT02295748 | December 2014 | 16 December 2017 | 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 | Not recruiting | 4 Years | N/A | Male | 24 | Phase 1 | United States | |
| 138 | NCT02312011 | December 2014 | 2 July 2018 | 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. | Not recruiting | 20 Years | 55 Years | All | 48 | Phase 1/Phase 2 | United States | |
| 139 | NCT02329769 | December 2014 | 16 December 2017 | 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 | Not recruiting | 9 Years | 20 Years | Male | 15 | Phase 2 | Belgium;Italy;Netherlands;Sweden | |
| 140 | EUCTR2013-003605-26-SE | 26/11/2014 | 5 September 2016 | A study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy (Extension study to PRO044-CLIN-01) | A phase II, open label, extension study to assess the effect of PRO044 in patients with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO044 Product Code: PRO044 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: PS188 (company code) CAS Number: 1802402-63-6 Current Sponsor code: PS188 Other descriptive name: PS188 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | BioMarin Nederland B.V. | Not Recruiting | Female: no Male: yes | 18 | Phase 2 | Belgium;Netherlands;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 141 | NCT02310763 | November 24, 2014 | 14 January 2019 | A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy | A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY | Duchenne Muscular Dystrophy | Biological: PF-06252616;Drug: Placebo | Pfizer | Not recruiting | 6 Years | 15 Years | Male | 121 | Phase 2 | United States;Australia;Bulgaria;Canada;Italy;Japan;Poland;United Kingdom | |
| 142 | NCT02255552 | November 17, 2014 | 15 July 2019 | 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 | Not recruiting | 7 Years | 16 Years | Male | 109 | Phase 3 | United States | |
| 143 | NCT02036463 | November 2014 | 2 March 2015 | 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 | Not recruiting | 3 Years | 6 Years | Male | 0 | Phase 2 | United States |
| 144 | NCT02286947 | November 2014 | 20 May 2019 | 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 | Not recruiting | 7 Years | 21 Years | Male | 24 | Phase 2 | United States | |
| 145 | EUCTR2014-002210-23-DK | 14/10/2014 | 30 April 2019 | Can local botulinum toxin improve swallowing difficulties in persons with msucle disease? | Can local intramuscular botulinum toxin improve dysphagia in patients with myopathic dysphagia and constriction of the cricoid muscle? | Oculopharyngesl muscle dystrophy, inclusion body myositis MedDRA version: 18.1 Level: LLT Classification code 10019897 Term: Hereditary progressive muscular dystrophy System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Xeomin Product Name: Xeomin Pharmaceutical Form: Concentrate and solvent for solution for injection CAS Number: 93384-43-1 Other descriptive name: BOTULINUM TOXIN TYPE A Concentration unit: U/ml unit(s)/millilitre Concentration type: equal Concentration number: 100- | Rigshospitalet | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Denmark | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 146 | EUCTR2013-004427-37-IT | 01/10/2014 | 9 March 2015 | Efficacy and tolerability of ibuprofen and isosorbide dinitrate (20 mg, 40 mg, 60 mg, 80 mg) | Multicentre, randomised, double-blind, dose titration design in patients with Duchenne muscular dystrophy to evaluate the efficacy and the tolerability of the combinations of Ibuprofen (200 mg bid) and Isosorbide Dinitrate (20 mg, 40 mg, 60 mg, 80 mg) - ESPP001 | Restrictive respiratory syndrome with frequent pulmonary infections and cardiomyopathy. wasting of skeletal muscle, severe local inflammation and, at least initially, muscle regeneration. MedDRA version: 17.0 Level: PT Classification code 10064571 Term: Gene mutation System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Trade Name: Isosorbide Dinitrate 20 mg Product Name: Isosorbide Dinitrate Pharmaceutical Form: Tablet INN or Proposed INN: ISOSORBIDE DINITRATE CAS Number: 87-33-2 Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: IBUPROFEN 200MG Pharmaceutical Form: Tablet INN or Proposed INN: IBUPROFEN Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: Isosorbide Dinitrate 40 mg Product Name: Isosorbide Dinitrate Pharmaceutical Form: Tablet INN or Proposed INN: ISOSORBIDE DINITRATE CAS Number: 87-33-2 Trade Name: Isosorbide Dinitrate 60 mg Product Name: Isosorbide Dinitrate Pharmaceutical Form: Tablet INN or Proposed INN: ISOSORBIDE DINITRATE CAS Number: 87-33-2 Trade Name: Isosorbide Dinitrate 80 mg Product Name: Isosorbide Dinitrate Pharmaceutical Form: Tablet INN or Proposed INN: ISOSORBIDE DINITRATE CAS Number: 87-33-2 | PARENT PROJECT ONLUS | Authorised | Female: no Male: yes | 188 | Italy | ||||
| 147 | EUCTR2014-001753-17-NL | 19/09/2014 | 16 February 2015 | A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Biological Activity of ATYR1940 in Adults with Genetic Myopathy | A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies | Facioscapulohumeral muscular dystrophy MedDRA version: 17.1 Level: PT Classification code 10064087 Term: Facioscapulohumeral muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ATYR1940 Product Code: ATYR1940 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: N/A Current Sponsor code: ATYR1940 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 25- Pharmaceutical form of the placebo: Concentrate for solution for infusion Route of administration of the placebo: Intravenous use | aTyr Pharma, Inc. | Authorised | Female: yes Male: yes | 44 | France;United States;Netherlands;Italy | ||||
| 148 | EUCTR2014-002008-25-GB | 10/09/2014 | 8 January 2018 | A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patients | A 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping - N/A | Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SRP-4053 Product Code: SRP-4053 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Not Available CAS Number: 1422959-91-8 Current Sponsor code: SRP-4053 Other descriptive name: Phosphorodiamidate morpholino oligomer for exon 53 skipping Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Solution for infusion Route of administration of the placebo: Intravenous use | Sarepta Therapeutics, Inc. | Authorised | Female: no Male: yes | 48 | Phase 1;Phase 2 | France;United States;Italy;United Kingdom | |||
| 149 | NCT01834066 | September 2014 | 19 February 2015 | Study Safety and Efficacy of Bone Marrow Derived Autologous Cells for the Treatment of Muscular Dystrophy. | Safety and Efficacy of Bone Marrow Autologous Cells in Muscular Dystrophy. It is Self Funded (Patients' Own Funding) Clinical Trial | Muscular Dystrophy;Duchenne Muscular Dystrophy, | Biological: Stem Cell | Chaitanya Hospital, Pune | Recruiting | 6 Years | 25 Years | Both | 25 | Phase 1/Phase 2 | India | |
| 150 | NCT02235844 | September 2014 | 30 September 2019 | Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) | Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD) | Duchenne's Muscular Dystrophy | Biological: Umbilical Cord Mesenchymal Stem Cells | Allergy and Asthma Consultants, Wichita, Kansas | Aidan Foundation;Neil H. Riordan PhD | Not recruiting | 28 Years | 31 Years | Male | 1 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 151 | NCT02246478 | September 2014 | 6 October 2015 | 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. | Not recruiting | 5 Years | 15 Years | Male | 21 | Phase 1 | Japan | |
| 152 | NCT02251457 | August 2014 | 18 March 2019 | 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 | Not recruiting | 18 Years | 100 Years | All | 35 | Phase 1 | United States |
| 153 | JPRN-UMIN000014836 | 2014/05/01 | 2 April 2019 | A pilot study to evaluate efficacy and safety of resveratrol for muscular dystrophy. | Duchenne muscular dystrophy, Becker muscular dystrophy, Fukuyama congenital muscular dystrophy | 500mg resveratrol is administrated daily for 8 weeks. Subsequently, resveratrol is increased to 1500mg by 500mg every 8 weeks. | Sapporo Medical University | Not Recruiting | 12years-old | Not applicable | Male and Female | 10 | Not selected | Japan | ||
| 154 | NCT02196467 | May 2014 | 26 August 2019 | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients | Transplantation of Myoblasts to Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy | Biological: Myoblast transplantation;Procedure: Saline injection | CHU de Quebec-Universite Laval | Recruiting | 16 Years | N/A | Male | 10 | Phase 1/Phase 2 | Canada | |
| 155 | NCT02167217 | April 17, 2014 | 11 June 2018 | 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 | Not recruiting | 1 Month | 30 Months | Male | 24 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 156 | NCT02090959 | March 31, 2014 | 17 June 2019 | 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 | Not recruiting | 7 Years | 18 Years | Male | 219 | Phase 3 | Switzerland;Turkey;United Kingdom;United States;Czech Republic;Bulgaria;Canada;Chile;Czechia;France;Germany;Israel;Italy;Korea, Republic of;Poland;Spain;Sweden;Australia;Belgium;Brazil | |
| 157 | NCT02015481 | February 2014 | 16 December 2017 | 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. | Not recruiting | 18 Years | 80 Years | All | 25 | Phase 2 | United States;Canada;Israel | |
| 158 | NCT01890798 | January 2014 | 19 February 2015 | 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 | Not recruiting | 5 Years | N/A | Male | 0 | Phase 3 | ||
| 159 | NCT02056808 | November 2013 | 19 February 2015 | 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. | Not recruiting | 5 Years | 11 Years | Male | 12 | Phase 1 | United Kingdom | |
| 160 | NCT02285673 | November 2013 | 19 February 2015 | Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy | Efficacy of Umbilical Cord Mesenchymal Stem Cells in Duchenne Muscular Dystrophy: Phase 1/2 Study | Duchenne Muscular Dystrophy | Biological: Umbilical Cord Mesenchymal Stem Cell | Acibadem University | Recruiting | 7 Years | 20 Years | Male | 10 | Phase 1/Phase 2 | Turkey | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 161 | NCT01970735 | October 2013 | 19 February 2015 | Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2 | Clinical, Genetic and Epigenetic Characterization of Patients With FSHD Type 1 and FSHD Type 2 | Muscular Dystrophy, Facioscapulohumeral | Biological: Blood test | Centre Hospitalier Universitaire de Nice | Recruiting | 18 Years | 75 Years | Both | 100 | N/A | France | |
| 162 | NCT01978366 | October 2013 | 26 August 2019 | 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 | Akashi Therapeutics | Not recruiting | 6 Years | 20 Years | Male | 17 | Phase 2 | United States | |
| 163 | NCT01995032 | October 2013 | 11 June 2018 | 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 | Not recruiting | 78 Months | 10 Years | All | 47 | Phase 3 | Switzerland | |
| 164 | NCT02847975 | October 2013 | 20 August 2018 | Sodium Nitrate to Improve Blood Flow | Sodium Nitrate to Improve Blood Flow | Becker Muscular Dystrophy | Dietary Supplement: Sodium nitrate | Cedars-Sinai Medical Center | Not recruiting | 15 Years | 55 Years | Male | 11 | Phase 1 | United States | |
| 165 | EUCTR2013-001194-25-GB | 25/09/2013 | 3 April 2017 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 18.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 5 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 10 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 20 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | Eli Lilly and Company | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;United Kingdom;Italy;France;Canada;Argentina;Belgium;Denmark;Germany;Netherlands;Korea, Republic of | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 166 | EUCTR2013-001732-21-FR | 06/09/2013 | 19 February 2018 | A phase II study of metformin in myotonic dystrophy type 1 patients | A randomized, double blind, placebo-controlled phase II study of metformin in myotonic dystrophy type 1 patients - Myomet | Myotonic dystrophy type 1 (DM1) also known as Steinert disease;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: metformin 500mg Pharmaceutical Form: Tablet INN or Proposed INN: METFORMIN CAS Number: 657-24-9 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Centre d'Etude des Cellules Souches (CECS) | Not Recruiting | Female: yes Male: yes | 30 | Phase 2 | France | |||
| 167 | NCT01865084 | September 2013 | 16 December 2017 | 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 | Not recruiting | 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 | |
| 168 | NCT02147639 | September 2013 | 19 February 2015 | Effects of Sodium Nitrate on Blood Flow in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Dietary Supplement: Sodium Nitrate;Dietary Supplement: Sodium Nitrate - double dose;Dietary Supplement: Placebo;Procedure: Increased exercise intensity | Cedars-Sinai Medical Center | Recruiting | 15 Years | 45 Years | Male | 20 | Phase 2/Phase 3 | United States | ||
| 169 | EUCTR2011-005042-35-GB | 23/08/2013 | 14 March 2016 | A study to test if BMN 053 is safe and effective in people who suffer from Duchenne muscular dystrophy | A Phase I/II, open-label, dose escalating with 48 week treatment study to assess the safety and tolerability, pharmacokinetics, pharmacodynamics and efficacy of BMN 053 (previously known as PRO053) in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by BMN 053-induced DMD exon 53 skipping MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: BMN 053 Product Code: BMN 053 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: PS524 Current Sponsor code: PS524 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | BioMarin Pharmaceutical Inc. | Authorised | Female: no Male: yes | 45 | Phase 1/2 | United States;Turkey;Chile;Israel;Italy;United Kingdom;France;Hungary;Argentina;Poland;Belgium;Brazil;Australia;Bulgaria;Netherlands;Germany;Japan | |||
| 170 | EUCTR2013-002115-99-GB | 23/08/2013 | 3 April 2017 | A Clinical Trial to investigate if the drug SMT C1100 is safe and well tolerated in children with DMD when given orally one to three times a day. | SMT C1100 - A Phase 1, Open-label, Single and Multiple Oral Dose, Safety, Tolerability and Pharmacokinetic Study in Paediatric Patients with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: SMT C1100 Pharmaceutical Form: Oral suspension INN or Proposed INN: 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole CAS Number: 945531-77-1 Current Sponsor code: SMT C1100 Concentration unit: mg/g milligram(s)/gram Concentration type: equal Concentration number: 200- | Summit Corporation plc | Not Recruiting | Female: no Male: yes | Phase 1 | United Kingdom | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 171 | EUCTR2013-001194-25-IT | 09/08/2013 | 14 March 2016 | A clinical trial looking at the use and safety of tadalafil for the treatment of Duchenne Muscular Dystrophy in children. | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Cialis 2.5 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 5 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 10 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use Trade Name: Cialis 20 mg film-coated tablets Pharmaceutical Form: Film-coated tablet INN or Proposed INN: TADALAFIL CAS Number: 171596-29-5 Current Sponsor code: LY450190 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Film-coated tablet Route of administration of the placebo: Oral use | Eli Lilly and Company | Not Recruiting | Female: no Male: yes | 306 | Phase 3 | United States;Taiwan;Spain;Russian Federation;Italy;United Kingdom;France;Canada;Argentina;Belgium;Denmark;Netherlands;Germany;Korea, Republic of | |||
| 172 | NCT01918384 | August 2013 | 14 September 2015 | 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 | Not recruiting | 4 Years | N/A | Male | 21 | Phase 2 | Japan |
| 173 | NCT01921374 | August 2013 | 15 February 2016 | Mother-caregivers of Children With Duchenne Muscular Dystrophy | Profile of Mother-caregivers of Children With Duchenne Muscular Dystrophy | Other Diseases or Conditions | Biological: sleep parameters;Biological: Hormonal profile;Biological: Inflammatory profile;Biological: Cardiovascular profile;Biological: Metabolic profile | Monica Levy Andersen | Not recruiting | 25 Years | 65 Years | Female | 60 | N/A | Brazil | |
| 174 | NCT01957059 | June 2013 | 16 December 2017 | 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 | Not recruiting | 5 Years | 18 Years | Male | 9 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom | |
| 175 | NCT02018731 | June 2013 | 23 May 2016 | 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 | Not recruiting | 18 Years | N/A | Both | 20 | Phase 2 | Switzerland | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 176 | NCT01803412 | May 1, 2013 | 25 June 2018 | 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 | Not recruiting | 5 Years | N/A | Male | 53 | Phase 3 | United States;Canada | |
| 177 | NCT01761292 | May 2013 | 5 March 2018 | A 2-Part Study to Assess the Safety and Tolerability, pk, Effects on Histology and Some Clinical Parameters of Givinostat in Ambulant 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 | Not recruiting | 7 Years | 11 Years | Male | 20 | Phase 1/Phase 2 | Italy | |
| 178 | NCT01847573 | May 2013 | 26 August 2019 | 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 | Akashi Therapeutics | Not recruiting | 6 Years | 20 Years | Male | 17 | Phase 1/Phase 2 | United States | |
| 179 | NCT01856868 | May 2013 | 28 January 2019 | 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. | Not recruiting | 18 Years | 60 Years | Male | 7 | Phase 1/Phase 2 | United States |
| 180 | NCT02814110 | March 1, 2013 | 16 December 2017 | 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 of Muscle Strength in Patients With Muscular Dystrophy | Drug: Granulocyte colony-stimulating factor (Filgrastim) | Medical University of Bialystok | Recruiting | 5 Years | 15 Years | All | 27 | Phase 1 | Poland | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 181 | NCT01826422 | March 2013 | 8 February 2016 | Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients | Effect of Eicosapentaenoic Fatty Acid(EPA)and Docosahexaenoic Fatty Acids(DHA) Supplementation in the Inflammation State and Metabolic Disorders in Patients With Duchenne Muscular Dystrophy or Becker Muscular Dystrophy | Muscular Dystrophy, Duchenne | Dietary Supplement: EPA and DHA;Dietary Supplement: Placebo Comparator: Sunflower oil | Coordinación de Investigación en Salud, Mexico | Instituto Nacional de Rehabilitacion | Recruiting | 6 Years | 18 Years | Male | 70 | Phase 2 | Mexico |
| 182 | NCT01826487 | March 2013 | 15 April 2019 | Phase 3 Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy | A Phase 3 Efficacy and Safety 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;Drug: Placebo | PTC Therapeutics | Not recruiting | 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 | |
| 183 | EUCTR2012-002566-12-IT | 04/02/2013 | 19 February 2018 | A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy | A Two-Part Study to Assess the Safety and Tolerability, Pharmacokinetics, and Effects on Histology and Different Clinical Parameters of Givinostat in Ambulant Children with Duchenne Muscular Dystrophy - DSC | Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: SOC Classification code 10028395 Term: Musculoskeletal and connective tissue disorders System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Givinostat Product Code: ITF2357 Pharmaceutical Form: Oral suspension INN or Proposed INN: Givinostat CAS Number: 732302-99-7 Current Sponsor code: ITF2357 Other descriptive name: givinostat Concentration unit: mg milligram(s) Concentration type: up to Concentration number: 100- Product Name: Givinostat Product Code: ITF2357 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Givinostat CAS Number: 732302-99-7 Current Sponsor code: ITF2357 Other descriptive name: Givinostat Concentration unit: mg milligram(s) Concentration type: up to Concentration number: 100- | ITALFARMACO | Not Recruiting | Female: no Male: yes | 20 | Phase 2 | Italy | |||
| 184 | NCT01603407 | January 2013 | 29 April 2019 | Finding the Optimum Regimen for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy: Double-blind Randomized Trial to Find Optimum Steroid Regimen | Duchenne Muscular Dystrophy | Drug: Prednisone;Drug: Deflazacort | University of Rochester | Newcastle University;University Medical Center Freiburg;National Institute of Neurological Disorders and Stroke (NINDS) | Not recruiting | 4 Years | 7 Years | Male | 196 | Phase 3 | United States;Canada;Germany;Italy;United Kingdom |
| 185 | NCT01826474 | January 2013 | 16 December 2017 | 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 | Not recruiting | 5 Years | 18 Years | Male | 15 | Phase 1/Phase 2 | Belgium;France;Italy;Netherlands;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 186 | EUCTR2011-005040-10-BE | 03/12/2012 | 8 August 2016 | A study to test if multiple injections of PRO045 under the skin are safe and effective in people who suffer from Duchenne muscular dystrophy | A phase IIb, open-label study to assess the efficacy, safety, pharmacodynamics and pharmacokinetics of multiple subcutaneous doses of PRO045 in subjects with Duchenne muscular dystrophy | Duchenne muscular dystrophy resulting from a mutation correctable by PRO045-induced DMD exon 45 skipping MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: PRO045 Product Code: PRO045 Pharmaceutical Form: Solution for injection INN or Proposed INN: PS220 Current Sponsor code: PS220 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | BioMarin Nederland B.V. | Not Recruiting | Female: no Male: yes | 45 | Phase 1;Phase 2 | Belgium;Italy;United Kingdom | |||
| 187 | JPRN-UMIN000009307 | 2012/11/08 | 2 April 2019 | Inhibition of prostagrandin synthesis for the treatment of Duchenne musclar dystrophy | Duchenne muscular dystrophy | oral adm,inistration of aspirin | Kobe University Graduate School of Medicine | Not Recruiting | 5years-old | Not applicable | Male | 6 | Phase 1,2 | Japan | ||
| 188 | EUCTR2011-004853-18-GB | 21/08/2012 | 28 February 2019 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 18.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 18.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | PTC Therapeutics, Inc. | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | France;Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | |||
| 189 | EUCTR2011-004853-18-BE | 27/04/2012 | 12 March 2018 | Study of ataluren in patients with Nonsense Mutation Duchenne and Becker muscular dystrophy | An Open-Label Study for Previously Treated Ataluren (PTC124®) Patients with Nonsense Mutation Dystrophinopathy | Nonsense mutation dystrophinopathy MedDRA version: 16.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 16.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: ataluren Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ataluren CAS Number: 775304-57-9 Current Sponsor code: PTC124 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | PTC Therapeutics, Inc | Not Recruiting | Female: no Male: yes | 96 | Phase 3 | Canada;Spain;Belgium;Australia;Israel;Germany;Italy;United Kingdom;Sweden | |||
| 190 | NCT01580501 | March 2012 | 19 February 2015 | 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 | Not recruiting | 7 Years | 15 Years | Male | 12 | Phase 1 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 191 | NCT01618331 | March 2012 | 19 February 2015 | Protein Supplementation and Exercise in Patients With FSH Muscular Dystrophy- a Randomized Placebo Controlled Study | The Effect of Protein Supplementation Doing Regular Exercise in Patients With Facioscapulohumeral Muscular Dystrophy - a Blinded RCT Study | Facioscapulohumeral Muscle Dystrophy | Behavioral: Regular exercise;Dietary Supplement: Protein-carbohydrate supplementation | Grete Andersen, MD | The Augustinus Foundation, Denmark.;Aase and Ejnar Danielsens Foundation;The Danish Rheumatism Association;AP Moeller Foundation | Not recruiting | 18 Years | 65 Years | Both | 42 | N/A | Denmark |
| 192 | NCT02207283 | March 2012 | 8 August 2016 | 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 | Not recruiting | 15 Years | 55 Years | Male | 12 | Phase 4 | ||
| 193 | NCT03076814 | March 2012 | 30 October 2017 | Functional Muscle Ischemia With Tadalafil Treatment in Becker Muscular Dystrophy | Becker Muscular Dystrophy | Drug: Tadalafil;Other: Placebo | Cedars-Sinai Medical Center | Not recruiting | 15 Years | 55 Years | Male | 0 | N/A | |||
| 194 | NCT01540409 | February 27, 2012 | 15 July 2019 | 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 | Not recruiting | 7 Years | 13 Years | Male | 12 | Phase 2 | United States | |
| 195 | NCT01648634 | February 13, 2012 | 11 June 2018 | 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 | Not recruiting | 10 Years | 15 Years | Male | 51 | Phase 3 | France |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 196 | NCT01521546 | February 2012 | 19 October 2017 | 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 | Not recruiting | 7 Years | N/A | Male | 42 | N/A | United States |
| 197 | NCT01540604 | February 2012 | 19 February 2015 | 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 | Not recruiting | 2 Years | 11 Years | Both | Phase 2 | Sweden | ||
| 198 | NCT01388764 | January 2012 | 19 February 2015 | 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 | Not recruiting | 7 Years | 11 Years | Male | 7 | Phase 1 | United States | |
| 199 | NCT01519349 | January 2012 | 16 December 2017 | Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis | Phase I Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis. | Becker Muscular Dystrophy;Sporadic Inclusion Body Myositis | Biological: rAAV1.CMV.huFollistatin344 | Nationwide Children's Hospital | Parent Project Muscular Dystrophy;The Myositis Association (Grant Sponsor) | Not recruiting | 18 Years | N/A | All | 15 | Phase 1 | United States |
| 200 | NCT02516085 | January 2012 | 17 August 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 | Not recruiting | 7 Years | 10 Years | Both | 5 | Phase 1 | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 201 | EUCTR2011-004667-76-SE | 19/12/2011 | 13 May 2013 | An open single center study investigating the effects and side-effects of CRD007 in children with Duchenne Muscular Dystrophy (DMD -an inherited disorder which results in muscle degeneration) or Becker Muscular Dystrophy (BMD- an inherited disorder characterized by slowly progressive muscle weakness of the legs and pelvis) or children being symptomatic carriers for DMD or BMD. | An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers | Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy (BMD) and symptomatic carriers for DMD or BMD MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 10059117 Term: Becker's muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 14.1 Level: PT Classification code 10052655 Term: Duchenne muscular dystrophy gene carrier System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: pemirolast Product Code: CRD007 Pharmaceutical Form: Tablet INN or Proposed INN: PEMIROLAST CAS Number: 69372-19-6 Current Sponsor code: CRD007 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- | Cardoz AB | Not Recruiting | Female: yes Male: yes | Sweden | |||||
| 202 | NCT01350154 | November 2011 | 19 February 2015 | 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 | Not recruiting | 18 Years | 80 Years | Male | 17 | Phase 2 | Denmark |
| 203 | NCT01462292 | October 26, 2011 | 16 December 2017 | 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 | Not recruiting | 5 Years | N/A | Male | 51 | Phase 2 | United States | |
| 204 | EUCTR2011-001266-17-GB | 03/10/2011 | 28 February 2019 | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 Pharmaceutical Form: Injection Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | GlaxoSmithKline Research and Development LTD | Not Recruiting | Female: no Male: yes | 200 | Phase 3 | Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 205 | NCT01478022 | October 2011 | 10 September 2018 | 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 | Not recruiting | 18 Years | 27 Years | All | 12 | Phase 1 | Italy | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 206 | NCT01610440 | October 2011 | 19 February 2015 | Safety and Efficacy of Umbilical Cord Mesenchymal Stem Cell Therapy for Patients With Duchenne Muscular Dystrophy | Phase I/II Study of Stem Cell Therapy in Patients With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: human umbilical cord mesenchymal stem cells | Shenzhen Beike Bio-Technology Co., Ltd. | The Second Affiliated Hospital of Kunming Medical University | Recruiting | 5 Years | 12 Years | Both | 15 | Phase 1/Phase 2 | China |
| 207 | NCT01480245 | September 2011 | 16 December 2017 | 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 | Not recruiting | 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 | |
| 208 | EUCTR2011-001266-17-BE | 08/08/2011 | 21 August 2017 | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy | An open-label extension study of the long-term safety, tolerability and efficacy of GSK2402968 in subjects with Duchenne Muscular Dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 16.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: GSK2402968 Product Code: GSK2402968 Pharmaceutical Form: Injection Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | GlaxoSmithKline Research and Development LTD | Not Recruiting | Female: no Male: yes | 200 | Phase 3 | Taiwan;Spain;Turkey;Israel;Russian Federation;Chile;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Argentina;Belgium;Poland;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Norway;Japan;Korea, Republic of | |||
| 209 | NCT01422200 | August 2011 | 19 February 2015 | Flu Vaccine Study in Neuromuscular Patients 2011 | Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular Diseases | Duchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular Dystrophy | Biological: 2011-2012 seasonal flu vaccine | Children's Hospital Medical Center, Cincinnati | Recruiting | 3 Years | 35 Years | Both | 30 | N/A | United States | |
| 210 | NCT01645098 | August 2011 | 19 October 2017 | 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 | Not recruiting | N/A | N/A | Male | 53 | N/A | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 211 | NCT01396239 | July 2011 | 20 May 2019 | 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 | Not recruiting | 7 Years | 13 Years | Male | 12 | Phase 2 | United States | |
| 212 | NCT01406873 | June 2011 | 11 June 2018 | 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 | Not recruiting | 18 Years | 80 Years | All | 42 | Phase 2 | United States | |
| 213 | NCT01359670 | May 2011 | 11 June 2018 | 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 | Not recruiting | 7 Years | 15 Years | Male | 30 | N/A | United States |
| 214 | NCT01335295 | March 2011 | 19 February 2015 | 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 | Not recruiting | 4 Years | 16 Years | Male | 20 | Phase 1 | Italy | |
| 215 | EUCTR2011-000176-33-IT | 14/02/2011 | 19 March 2012 | Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND | Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND | Duchenne Muscolar Dystrophy MedDRA version: 9.1 Level: PT Classification code 10013801 | Pharmaceutical Form: Capsule, hard INN or Proposed INN: Tacrolimus Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 1- Product Name: hMABs Pharmaceutical Form: Suspension for injection Other descriptive name: HLA-identical allogeneic mesoangioblasts Concentration unit: % percent Concentration type: equal Concentration number: 1- Product Name: hMABs Pharmaceutical Form: Suspension for injection Other descriptive name: HLA-identical allogeneic mesoangioblasts Concentration unit: % percent Concentration type: equal Concentration number: 1- Product Name: hMABs Pharmaceutical Form: Suspension for injection Other descriptive name: HLA-identical allogeneic mesoangioblasts Concentration unit: % percent Concentration type: equal Concentration number: 1- | FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | Authorised | Female: no Male: yes | Italy | |||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 216 | NCT01183767 | December 30, 2010 | 10 December 2018 | 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 | Not recruiting | 5 Years | 10 Years | All | 33 | Phase 2/Phase 3 | Germany | |
| 217 | NCT02245711 | December 2010 | 5 November 2018 | Cell Therapy in Limb Girdle Muscular Dystrophy | Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy | Limb Girdle Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | Not recruiting | 15 Years | 60 Years | All | 0 | Phase 1 | India | |
| 218 | NCT01247207 | November 30, 2010 | 14 October 2019 | Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD) | An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients With Nonsense Mutation Dystrophinopathy | Duchenne Muscular Dystrophy | Drug: Ataluren | PTC Therapeutics | Recruiting | N/A | N/A | Male | 160 | Phase 3 | United States;Canada | |
| 219 | NCT01207908 | November 2010 | 19 February 2015 | 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 | Not recruiting | 5 Years | N/A | Male | 40 | Phase 1/Phase 2 | United States |
| 220 | NCT01239758 | October 2010 | 19 February 2015 | Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy | An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: ACE-031 (Extension of cohort 1 from core study, A031-03);Biological: ACE-031 (Extension of cohort 2 from core study, A031-03);Biological: ACE-031 (Extension of cohort 3 from core study, A031-03) | Acceleron Pharma, Inc. | Not recruiting | 4 Years | N/A | Male | 11 | Phase 2 | Canada | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 221 | NCT01153932 | September 2010 | 19 February 2015 | 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 | Not recruiting | 5 Years | N/A | Male | 53 | Phase 2 | Australia;Belgium;France;Germany;Israel;Netherlands;Spain;Turkey;United Kingdom | |
| 222 | NCT01168908 | September 2010 | 11 June 2018 | 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 | Not recruiting | 18 Years | 50 Years | Male | 20 | Phase 2 | United States |
| 223 | EUCTR2010-018412-32-DE | 05/08/2010 | 3 February 2014 | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | A phase II, double blind, exploratory, parallel-group, placebo-controlled clinical study to assess two dosing regimens of GSK2402968 for efficacy, safety, tolerability and pharmacokinetics in ambulant subjects with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy MedDRA version: 14.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Code: GSK2402968 Pharmaceutical Form: Injection Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | GlaxoSmithKline Research and Development LTD | Not Recruiting | Female: no Male: yes | 54 | Phase 2 | France;Spain;Turkey;Australia;Israel;Netherlands;Germany;United Kingdom | |||
| 224 | NCT01128855 | July 12, 2010 | 16 December 2017 | 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 | Not recruiting | 9 Years | N/A | Male | 20 | Phase 1 | United States;France | |
| 225 | EUCTR2009-016482-28-DE | 25/06/2010 | 10 October 2018 | SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy | SUNIMUD - Sunphenon EGCg (Epigallocatechin-Gallate) in Duchenne Muscular Dystrophy - SUNIMUD | Duchenne Muscular Dystrophy;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Sunphenon EGCG Pharmaceutical Form: Capsule, hard INN or Proposed INN: Sunphenon Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Pharmaceutical form of the placebo: Capsule, hard Route of administration of the placebo: Oral use | Charite Universitätsmedizin Berlin | Not Recruiting | Female: no Male: yes | 40 | Phase 2 | Germany | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 226 | EUCTR2009-017649-67-IE | 22/06/2010 | 19 March 2012 | Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD | Bone Health in Duchenne Muscular Dystrophy- a case controlled study of Risedronate use - Risedronate in DMD | Bone Health in Duchenne Muscular Dystrophy- a randomised controlled study of Risedronate use | Trade Name: Actonel Once a week 5mg film coated tablets Product Name: Risedronate sodium Product Code: Risedronate Pharmaceutical Form: Film-coated tablet Trade Name: Calcichew D3 Forte Product Name: Calcichew D3 Forte Product Code: Calcichew D3 Forte Pharmaceutical Form: Chewable tablet | The Central Remedial Clinic and The Children's University Hospital | Authorised | Female: no Male: yes | 70 | Ireland | ||||
| 227 | NCT01596803 | June 2010 | 19 February 2015 | Effects Antioxidants Supplementation on Muscular Function Patients Facioscapulohumeral Dystrophy (FSHD) | Effects of Antioxidants Supplementation on Muscular Function of Patients Affected by Facioscapulohumeral Dystrophy (FSHD) | Facioscapulohumeral Muscular Dystrophy | Procedure: Taking of blood;Dietary Supplement: needle biopsy of the vastus lateralis muscle;Dietary Supplement: Vit C Vit E Zn Se;Dietary Supplement: Placebo Vit E Placebo Vit C Zn Se | University Hospital, Montpellier | Hospital Clinical Research Project 2010;Association Amis FSH France;FSH Dutch Fondation The Netherland | Recruiting | 18 Years | 60 Years | Both | 54 | N/A | France |
| 228 | EUCTR2009-013762-63-NL | 16/04/2010 | 5 August 2013 | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | A phase I/IIa, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of PRO044 in patients with Duchenne muscular dystrophy | Duchenne Muscular Dystrophy | Product Name: PRO044 Product Code: PRO044 Pharmaceutical Form: Solution for injection INN or Proposed INN: h44AON188 Current Sponsor code: PRO044 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 92- | Prosensa Therapeutics B.V | Not Recruiting | Female: no Male: yes | 18 | Phase 1/2a | Belgium;Netherlands;Italy;Sweden | |||
| 229 | NCT01099761 | April 2010 | 16 December 2017 | Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: ACE-031 0.5 mg/kg q4wk;Biological: ACE-031 1.0 mg/kg q2wk;Other: Placebo | Acceleron Pharma, Inc. | Not recruiting | 4 Years | N/A | Male | 24 | Phase 2 | Canada | |
| 230 | NCT01126697 | February 2010 | 25 June 2018 | 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 | Not recruiting | 8 Years | N/A | All | 63 | Phase 2/Phase 3 | United States;Japan;Canada |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 231 | EUCTR2009-013169-24-GB | 19/01/2010 | 19 March 2012 | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients with Nonsense–Mutation-Mediated Duchenne/Becker Muscular Dystrophy - Study of Ataluren in Nonambulatory Patients with DMD/BMD | Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy MedDRA version: 12.0 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy | Product Name: Ataluren 125 mg Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Product Name: Ataluren 250 mg Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: Ataluren 1000 mg Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | PTC Therapeutics Inc | Not Recruiting | Female: no Male: yes | 30 | Phase 2a | United Kingdom | |||
| 232 | NCT01070511 | January 2010 | 19 February 2015 | 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 | Not recruiting | 18 Years | 55 Years | Male | 48 | Phase 4 | United States |
| 233 | EUCTR2010-020047-12-FR | 2 October 2017 | Effet d’un traitement préventif par Nebivolol chez les enfants atteints de dystrophie de Duchenne | Etude de l'effet d'un traitement préventif par Nebivolol sur l'apparition et la progression de la dysfonction cardiaque chez les enfants atteints de dystrophie de Duchenne - NEBIDYS | Enfants atteints de dystrophie de Duchenne MedDRA version: 13.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Temerit Product Name: Temerit Pharmaceutical Form: Tablet INN or Proposed INN: Nébivolol CAS Number: 118457-15-1 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP) | Not Available | Female: no Male: yes | Phase 3 | France | |||||
| 234 | EUCTR2010-020069-26-DE | 21 October 2013 | A phase III, randomized, double blind, placebo-controlled 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. - N/A | Duchenne muscular dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Code: GSK2402968 Pharmaceutical Form: Injection Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Injection Route of administration of the placebo: Subcutaneous use | GlaxoSmithKline Research and Development LTD | Not Recruiting | Female: no Male: yes | 180 | Phase 3 | Taiwan;Spain;Korea, Democratic People's Republic of;Russian Federation;Chile;Italy;France;Hungary;Czech Republic;Canada;Argentina;Poland;Belgium;Brazil;Denmark;Netherlands;Germany;Norway;Japan | ||||
| 235 | EUCTR2010-024566-22-FR | 2 October 2017 | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 12.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy | Product Name: GSK2402968 Product Code: GSK2402968 Pharmaceutical Form: Injection* Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | GlaxoSmithKline Research and Development LTD | Not Available | Female: no Male: yes | 32 | Phase 1 | France | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 236 | EUCTR2010-024566-22-Outside-EU/EEA | 20 March 2012 | A clinical study to investigate the pharmacokinetics safety, and tolerability of a single dose of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy (GSK Study Number DMD114118) | A double-blind, escalating dose, randomized, placebo-controlled study to assess the pharmacokinetics, safety and tolerability of single subcutaneous injections of GSK2402968 in non-ambulant subjects with Duchenne muscular dystrophy - N/A | Duchenne Muscular Dystrophy MedDRA version: 14.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders | Product Name: GSK2402968 Product Code: GSK2402968 Pharmaceutical Form: Injection Current Sponsor code: GSK2402968 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Subcutaneous use | GlaxoSmithKline Research and Development LTD | Not Available | Female: no Male: yes | 32 | United States | |||||
| 237 | NCT01009294 | November 30, 2009 | 15 April 2019 | Study of Ataluren (PTC124®) in Nonambulatory Patients 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 (PTC124) | PTC Therapeutics | Genzyme, a Sanofi Company | Not recruiting | 7 Years | N/A | Male | 6 | Phase 2 | United States;United Kingdom |
| 238 | EUCTR2009-012037-30-BE | 14/07/2009 | 12 May 2014 | Hereditary neuromuscular disease | A Phase III Double-Blind, Randomised, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in 10 – 18 Year Old Patients with Duchenne Muscular Dystrophy - DELOS | Duchenne Muscular Dystrophy MedDRA version: 17.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Idebenone Product Code: SNT-MC17 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IDEBENONE CAS Number: 58186279 Current Sponsor code: SNT-MC17 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Santhera Pharmaceuticals (Switzerland) Limited | Authorised | Female: no Male: yes | 266 | Phase 3 | France;United States;Spain;Belgium;Austria;Netherlands;Germany;Italy;Switzerland;Sweden | |||
| 239 | NCT02432885 | June 2009 | 11 May 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 | Not recruiting | 6 Years | N/A | Both | 76 | Phase 3 | |
| 240 | EUCTR2008-007648-32-BE | 14/04/2009 | 19 March 2012 | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Extension Study of PTC124 in Subjects with Nonsense Mutation Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy MedDRA version: 9.1 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy | Product Name: ATALUREN Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Product Name: ATALUREN Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: ATALUREN Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension INN or Proposed INN: ATALUREN CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 1000- | PTC Therapeutics, Inc. | Not Recruiting | Female: no Male: yes | 174 | Phase 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 241 | NCT01982695 | March 2009 | 16 December 2017 | 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 | Not recruiting | N/A | N/A | Male | 23 | N/A | United States |
| 242 | NCT00844597 | January 2009 | 19 October 2017 | 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 | Not recruiting | 5 Years | 15 Years | Male | 19 | Phase 1/Phase 2 | United Kingdom |
| 243 | NCT00847379 | January 2009 | 16 December 2017 | Phase 2b Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2b Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: Ataluren (PTC124) | PTC Therapeutics | Genzyme, a Sanofi Company | Not recruiting | N/A | N/A | Male | 173 | Phase 2/Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom |
| 244 | NCT02241434 | January 2009 | 5 November 2018 | Stem Cell Therapy in Duchenne Muscular Dystrophy | The Role of Autologous Bone Marrow Mononuclear Cell Therapy in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | Not recruiting | 3 Years | 25 Years | All | 0 | Phase 1 | India | |
| 245 | NCT02241928 | January 2009 | 5 November 2018 | Stem Cell Therapy in Muscular Dystrophy | Role of Autologous Mononuclear Cell Therapy in Muscular Dystrophy | Muscular Dystrophy | Biological: Stem Cell | Neurogen Brain and Spine Institute | Not recruiting | 6 Months | 60 Years | All | 0 | Phase 1 | India | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 246 | EUCTR2009-009871-36-DE | 25 January 2016 | Study within children with Duchenne Muscular Dystrophy | Effect and Safety of preventive Treatment with ACE-Inhibitor and Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy - DMD-Kardio | Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: EnalHexal®, 5 mg Pharmaceutical Form: Tablet INN or Proposed INN: ENALAPRIL CAS Number: 75847733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: EnaHexal®, 10mg Pharmaceutical Form: Tablet INN or Proposed INN: ENALAPRIL CAS Number: 75847733 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: MetoHEXAL® Succ ® 23,75 mg Retardtabletten Pharmaceutical Form: Tablet INN or Proposed INN: METOPROLOL SUCCINATE CAS Number: 98418474 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 23.75- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: MetoHEXAL® Succ ® 47,5 mg Retardtabletten Pharmaceutical Form: Tablet INN or Proposed INN: METOPROLOL SUCCINATE CAS Number: 98418474 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 47.5- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use Trade Name: MetoHEXAL® Succ ® 95 mg Retardtabletten Pharmaceutical Form: Tablet INN or Proposed INN: METOPROLOL SUCCINATE CAS Number: 98418474 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 95- Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Friedrich- Alexander- Universität Erlangen Nürnberg | Not Recruiting | Female: no Male: yes | Germany | ||||||
| 247 | EUCTR2007-004695-39-GB | 05/12/2008 | 26 June 2012 | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients | Duchenne Muscular Dystrophy MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy | Product Name: AVI-4658 Product Code: AVI-4658 Pharmaceutical Form: Concentrate for solution for injection CAS Number: 1173755-55-9 Current Sponsor code: AVI-4658 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | AVI BioPharma, Inc. | Not Recruiting | Female: no Male: yes | United Kingdom | |||||
| 248 | EUCTR2008-007236-18-IT | 01/12/2008 | 5 August 2013 | Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - ND | Effects of cardioprotective therapy, carvedilol vs ramipril, in patients affected by Duchenne and Becker muscular dystrophy. Clinical significance and prognostic value of Cardiac Magnetic Resonance study. - ND | Duchenne and Becher muscular dystrophy MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy | Trade Name: DILATREND Pharmaceutical Form: Tablet INN or Proposed INN: Carvedilol Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 6.25- Trade Name: TRIATEC Pharmaceutical Form: Tablet INN or Proposed INN: Ramipril Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2.5- | POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI | Not Recruiting | Female: no Male: yes | Italy | |||||
| 249 | NCT00819845 | December 2008 | 8 February 2016 | 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 | Recruiting | 2 Years | 45 Years | Male | 194 | Phase 4 | Italy | |
| 250 | NCT02050776 | December 2008 | 5 November 2018 | Stem Cell Therapy in Limb Girdle Muscular Dystrophy | The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study | Limb Girdle Muscular Dystrophy | Biological: Autologous bone marrow mononuclear cell transplantation | Neurogen Brain and Spine Institute | Not recruiting | 15 Years | 60 Years | All | 0 | Phase 1 | India | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 251 | NCT00758225 | September 2008 | 19 February 2015 | 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 | Not recruiting | N/A | N/A | Male | 21 | Phase 2 | Belgium | |
| 252 | EUCTR2007-007752-34-BE | 14/08/2008 | 19 March 2012 | A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy | A Phase II open-label extension study to obtain long-term safety, tolerability and efficacy data of Idebenone in the treatment of Duchenne Muscular Dystrophy | Duchennes Muscular Dystrophy MedDRA version: 12.0 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy | Product Name: Idebenone Product Code: SNT-MC17 Pharmaceutical Form: Tablet INN or Proposed INN: Idebenone Current Sponsor code: SNT-MC17 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 150- | Santhera Pharmaceuticals (Switzerland) Ltd | Not Recruiting | Female: no Male: yes | Phase 2 | Belgium | ||||
| 253 | NCT00759876 | July 31, 2008 | 15 April 2019 | 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 | Not recruiting | N/A | N/A | Male | 36 | Phase 2 | United States |
| 254 | NCT01421992 | June 2008 | 19 February 2015 | 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 | Not recruiting | 18 Years | 65 Years | Both | 28 | Phase 2/Phase 3 | Canada | |
| 255 | EUCTR2007-005478-29-FR | 17/03/2008 | 19 March 2012 | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects with Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy, Becker Muscular Dystrophy MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy MedDRA version: 9.1 Level: LLT Classification code 10059117 Term: Becker's muscular dystrophy | Product Name: PTC124 Product Code: PTC124 Pharmaceutical Form: Powder for oral suspension CAS Number: 775304-57-9 Current Sponsor code: PTC124 Other descriptive name: PTC0161480, PTC124 Compound 1a, RPS2505, PTC-C124, 291844 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125, 250, 1000- Pharmaceutical form of the placebo: Powder for oral suspension Route of administration of the placebo: Oral use | PTC Therapeutics, Inc. | Authorised | Female: no Male: yes | 165 | Phase 2b | United Kingdom;Germany;Belgium;France;Spain;Italy;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 256 | EUCTR2007-005808-41-ES | 25/02/2008 | 19 March 2012 | Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathy | Estudio multicéntrico para comprobar la respuesta clínico-analítica de pacientes portadores sintomáticos de Disferlinopatía al tratamiento con Calcifediol Multicentric study to assess the clinical and analytical response to calcifediol in symptomatic carriers of dysferlin myopathy | Portadores sintomaticos de una mutación en el gen disferlina. Symptomatic carriers of a mutation in the dysferlin gene MedDRA version: 9.1 Level: LLT Classification code 10028356 Term: Muscular dystrophy | Trade Name: Hidroferol® Product Name: Calcifediol Pharmaceutical Form: Oral liquid INN or Proposed INN: CALCIFEDIOL CAS Number: 19356-17-3 Other descriptive name: HIDROFEROL Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 0,266- | Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau | Authorised | Female: yes Male: yes | Spain | |||||
| 257 | NCT00592553 | February 2008 | 16 December 2017 | Phase 2b Study of PTC124 in Duchenne/Becker Muscular Dystrophy (DMD/BMD) | A Phase 2b Efficacy and Safety Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne Muscular Dystrophy and Becker Muscular Dystrophy | Duchenne Muscular Dystrophy;Becker Muscular Dystrophy | Drug: PTC124 High Dose;Drug: PTC124 Low Dose;Drug: Placebo | PTC Therapeutics | Not recruiting | 5 Years | N/A | Male | 174 | Phase 2 | United States;Australia;Belgium;Canada;France;Germany;Israel;Italy;Spain;Sweden;United Kingdom | |
| 258 | EUCTR2007-004819-54-BE | 22/01/2008 | 8 August 2016 | A study to assess the effect and safety of multiple subcutaneous doses of drisapersen in patients with Duchenne Disease | A phase I/II, open label, escalating dose, pilot study to assess the effect, safety, tolerability and pharmacokinetics of multiple subcutaneous doses of drisapersen in patients with Duchenne muscular dystrophy and to assess the potential for intravenous dosing as an alternative route of administration | Duchenne Muscular Dystrophy MedDRA version: 17.1 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: Drisapersen Product Code: PRO051 Pharmaceutical Form: Solution for injection INN or Proposed INN: PRO051 Current Sponsor code: PRO051 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- Product Name: Drisapersen Product Code: PRO051 Pharmaceutical Form: Solution for injection INN or Proposed INN: PRO051 Current Sponsor code: PRO051 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | Prosensa Therapeutics BV | Not Recruiting | Female: no Male: yes | 12 | Phase 1;Phase 2 | Belgium;Netherlands;Sweden | |||
| 259 | NCT00577577 | December 2007 | 19 February 2015 | 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 | Not recruiting | 21 Years | 65 Years | Both | 60 | Phase 2 | United States |
| 260 | NCT00606775 | December 2007 | 19 February 2015 | 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 |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 261 | NCT00159250 | October 26, 2007 | 28 October 2019 | 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) | Sarepta Therapeutics, Inc. | Department of Health, United Kingdom | Not recruiting | 10 Years | 17 Years | Male | 7 | Phase 1/Phase 2 | United Kingdom |
| 262 | EUCTR2006-003833-33-GB | 13/06/2007 | 31 October 2016 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658 | Duchenne Muscular Dystrophy MedDRA version: 9.1 Level: LLT Classification code 10013801 Term: Duchenne muscular dystrophy | Product Name: AVI-4658 Product Code: AVI-4658 Pharmaceutical Form: Solution for injection Current Sponsor code: AVI-4658 Other descriptive name: Phosphorodiamidate Morpholino Oligomer Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 5- Pharmaceutical form of the placebo: Solution for injection Route of administration of the placebo: Intramuscular use | Imperial College, London | Not Recruiting | Female: no Male: yes | 9 | Phase 1;Phase 2 | United Kingdom | |||
| 263 | EUCTR2006-006776-37-NL | 14/05/2007 | 19 March 2012 | Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD | Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD | Healthy volunteers. Later the protocol will be applied to Facioscapular humeral dystrophy and/or other muscular dystrophies in general. MedDRA version: 8.1 Level: LLT Classification code 10011328 Term: Creatine | Product Name: creatine-13C Product Code: Cr13C Pharmaceutical Form: Powder for oral solution Pharmaceutical form of the placebo: Powder for oral solution Route of administration of the placebo: Oral use | Radboud University Nijmegen Medical Center | Authorised | Female: yes Male: yes | Netherlands | |||||
| 264 | NCT00308113 | April 2007 | 19 October 2017 | 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 | Not recruiting | 10 Years | 18 Years | Male | 3 | Phase 3 | United States;Australia;Puerto Rico |
| 265 | NCT00451074 | March 2007 | 19 February 2015 | 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) | Not recruiting | 5 Years | 20 Years | Male | 12 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 266 | NCT01344798 | November 2006 | 19 February 2015 | Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C | Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C | Limb Girdle Muscular Dystrophy Type 2C;Gamma-sarcoglycanopathy | Biological: AAV1-gamma-sarcoglycan vector injection | Genethon | Not recruiting | 15 Years | N/A | Both | 9 | Phase 1 | France | |
| 267 | NCT00428935 | March 2006 | 19 February 2015 | Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy | Phase 1 Clinical Trial of rAAV2.5-CMV-mini-Dystrophin Gene Vector in Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Biological: rAAV2.5-CMV-minidystrophin (d3990) | Nationwide Children's Hospital | Asklepios Biopharmaceutical, Inc. | Not recruiting | 5 Years | 15 Years | Male | 6 | Phase 1 | United States |
| 268 | NCT00296621 | February 2006 | 19 February 2015 | 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 | Not recruiting | N/A | N/A | Male | 30 | Phase 2 | France | |
| 269 | EUCTR2006-003075-12-IE | 2 October 2017 | Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction. | Effect of carvedilol on left ventricular systolic and diastolic function and the neurohormonal axis in patients with Duchenne muscular dystrophy and left ventricular dysfunction. | Duchenne muscular dystrophy is an X-linked inherited disorder which primarily affects skeletal muscle through a mutation in dystrophin which maps to the long arm of the X chromosome (Xp21). Patients with skeletal myopathy such as DMD eventually progress to develop dilated cardiomyopathy as dystrophin is expressed in all forms of muscle including cardiac and smooth muscle. Often the cause of death in patients with DMD is cardiac failure and arrhythmia. | Trade Name: Eucardic Product Name: Eucardic Product Code: No code Pharmaceutical Form: Tablet INN or Proposed INN: CARVEDILOL CAS Number: 72956093 Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 3.125mg-6.25mg | Department Of Paediatric Cardiology | Not Available | Female: yes Male: yes | 30 | Phase 4 | Ireland | ||||
| 270 | NCT00264888 | December 2005 | 19 February 2015 | 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 | Not recruiting | 5 Years | N/A | Male | 38 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 271 | NCT00233519 | November 2005 | 19 October 2017 | 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 | Not recruiting | 21 Years | 60 Years | All | 17 | Phase 1/Phase 2 | United States |
| 272 | NCT00654784 | October 2005 | 19 October 2017 | 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 | Not recruiting | 8 Years | 16 Years | Male | 21 | Phase 2 | Belgium | |
| 273 | EUCTR2005-002520-33-BE | 06/09/2005 | 19 March 2012 | A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy | A Phase IIa double blind, randomised, placebo controlled, single centre study at the university of Leuven to assess the efficacy and tolerability of idebenone in 10 - 16 year old males with cardiac dysfunction associated with Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Product Name: idebenone Pharmaceutical Form: Tablet Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Santhera Pharmaceuticals LLC | Not Recruiting | Female: no Male: yes | 21 | Phase 2a | Belgium | |||
| 274 | NCT00243789 | September 2005 | 19 February 2015 | 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 | Not recruiting | 7 Years | N/A | Male | 64 | Phase 1/Phase 2 | United States;Argentina;Australia;Canada;Israel;Italy | |
| 275 | EUCTR2004-000622-67-GB | 03/06/2005 | 1 May 2012 | A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy. | A Double-Blind, Placebo-Controlled, Randomized, Multiple Ascending Dose, Safety Study of MYO-029 Administered to Adult Patients with Becker, Facioscapulohumeral and Limb-Girdle Muscular Dystrophy. | Becker Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophy, Limb-Girdle Muscular Dystrophy. | Product Name: MYO-029 Pharmaceutical Form: Powder for injection* INN or Proposed INN: Not Available Current Sponsor code: MYO-029 Other descriptive name: Recombinant human anti-GDF-8 antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 70- Pharmaceutical form of the placebo: Powder for injection* Route of administration of the placebo: Intravenous use | Wyeth Research Division of Wyeth Pharmaceuticals Inc. | Authorised | Female: yes Male: yes | 136 | United Kingdom | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 276 | EUCTR2005-000663-26-AT | 25/04/2005 | 3 April 2017 | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Immunsuppressive Therapy in Duchenne Muscular Dystrophy - DMD-CsA | Duchenne Muscular Dystrophy | Trade Name: Sandimmun Optoral Product Name: Sandimmun Optoral Product Code: 29180.00.00, 34681.00.00, 34681.01.00, 34681.02.00 Pharmaceutical Form: Capsule* Other descriptive name: Cyclosporin A Concentration unit: mg/kg milligram(s)/kilogram Concentration type: range Concentration number: 3,5-4- Pharmaceutical form of the placebo: Capsule* Route of administration of the placebo: Oral use Trade Name: Decortin Product Name: Decortin Product Code: not available Pharmaceutical Form: Tablet Other descriptive name: Prednison Concentration unit: mg/kg milligram(s)/kilogram Concentration type: equal Concentration number: 0,75 -10 d on/ 10 d off | Universitätsklinik Freiburg | Not Recruiting | Female: no Male: yes | 150 | Phase 2;Phase 3 | Austria | |||
| 277 | NCT00104078 | February 2005 | 19 February 2015 | 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 | Not recruiting | 18 Years | N/A | Both | 108 | Phase 1/Phase 2 | United States | ||
| 278 | NCT00167609 | November 2004 | 19 February 2015 | 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 | Not recruiting | 18 Years | 70 Years | Both | 75 | Phase 2/Phase 3 | France |
| 279 | NCT00110669 | January 2004 | 19 February 2015 | 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 | Not recruiting | 4 Years | 10 Years | Male | 64 | Phase 3 | United States;India | |
| 280 | NCT00102453 | March 2002 | 19 February 2015 | 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 | Not recruiting | 4 Years | 7 Years | Male | 17 | Phase 1/Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 281 | NCT00033813 | January 2002 | 19 February 2015 | 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 | Not recruiting | 5 Years | 10 Years | Male | 15 | Phase 2 | United States | ||
| 282 | NCT00033189 | September 2001 | 19 February 2015 | 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 | Not recruiting | 5 Years | 11 Years | Male | 15 | Phase 2 | United States | ||
| 283 | NCT00018109 | June 2001 | 19 February 2015 | 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 | Not recruiting | 5 Years | 10 Years | Male | Phase 3 | United States | |
| 284 | NCT01882400 | May 2001 | 18 January 2018 | 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 | Not recruiting | 5 Years | 18 Years | All | 11 | Phase 4 | Canada |
| 285 | NCT00016653 | June 2000 | 19 February 2015 | 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 | Not recruiting | 5 Years | 9 Years | Male | 48 | Phase 2/Phase 3 | United States;Belgium;Israel;Puerto Rico | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 286 | NCT00005574 | February 2000 | 19 February 2015 | 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) | Not recruiting | N/A | N/A | Both | 4 | Phase 1 | United States | |
| 287 | NCT00004685 | January 1998 | 7 April 2015 | Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy | Muscular Dystrophy, Facioscapulohumeral | Drug: albuterol | Ohio State University | Not recruiting | 18 Years | 80 Years | Both | 90 | N/A | |||
| 288 | NCT00004646 | April 1995 | 19 February 2015 | 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 | Not recruiting | 5 Years | 15 Years | Male | 20 | Phase 3 | ||
| 289 | JPRN-JapicCTI-142538 | 23 April 2019 | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Intervention name : Tadalafil Dosage And administration of the intervention : During the double-blind period, tadalafil or matching placebo will be administered orally once daily at one of 2 target doses (0.3 mg/kg or 0.6 mg/kg).During the open-label extension period all patients will initially receive tadalafil. | Eli Lilly Japan K.K. | 7 | 14 | Male | Phase 3 |