113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 646 / Drugs : 471 - (DrugBank : 105) / Drug target genes : 59 - Drug target pathways : 170
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | EUCTR2019-003374-91-BE (EUCTR) | 30/06/2022 | 15/10/2021 | Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: SRP-9001 INN or Proposed INN: Not available Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 3 | France;United States;Taiwan;Hong Kong;Spain;Belgium;Germany;United Kingdom;Italy;Japan | ||
| 2 | EUCTR2019-003374-91-ES (EUCTR) | 21/02/2022 | 20/10/2021 | Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Code: SRP-9001 INN or Proposed INN: Not available Other descriptive name: ADENO-ASSOCIATED VIRUS SEROTYPE RH74 CONTAINING THE HUMAN MICRO-DYSTROPHIN GENE | Sarepta Therapeutics, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 120 | Phase 3 | France;United States;Taiwan;Hong Kong;Belgium;Spain;Germany;United Kingdom;Italy;Japan | ||
| 3 | NCT05096221 (ClinicalTrials.gov) | October 27, 2021 | 14/10/2021 | A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) | A Phase 3 Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Subjects With Duchenne Muscular Dystrophy (EMBARK) | Duchenne Muscular Dystrophy | Genetic: SRP-9001;Genetic: Placebo | Sarepta Therapeutics, Inc. | Hoffmann-La Roche | Active, not recruiting | 4 Years | 7 Years | Male | 126 | Phase 3 | United States;Belgium;Germany;Hong Kong;Italy;Japan;Spain;Taiwan;United Kingdom |
| 4 | NCT04626674 (ClinicalTrials.gov) | November 23, 2020 | 6/11/2020 | A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 (Delandistrogene Moxeparvovec) in Participants With Duchenne Muscular Dystrophy (DMD) | An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR) | Muscular Dystrophy, Duchenne | Genetic: SRP-9001 | Sarepta Therapeutics, Inc. | Hoffmann-La Roche | Enrolling by invitation | 3 Years | N/A | Male | 46 | Phase 1 | United States |