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 | EUCTR2017-002213-60-GB (EUCTR) | 08/01/2019 | 11/12/2018 | A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophy | A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and children with Duchenne muscular dystrophy | Duchenne muscular dystrophy MedDRA version: 20.1;Level: PT;Classification code 10052655;Term: Duchenne muscular dystrophy gene carrier;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10013801;Term: Duchenne muscular dystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Product Name: SGT-001 Product Code: rAAV9-CK8-h-µD5 INN or Proposed INN: SGT-001 Other descriptive name: rAAV9-CK8-h-µD5 | Solid Biosciences Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 32 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;United Kingdom | ||
| 2 | NCT03368742 (ClinicalTrials.gov) | December 6, 2017 | 5/12/2017 | Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | A Randomized, Controlled, Open-label, Single-ascending Dose, Phase I/II Study to Investigate the Safety and Tolerability, and Efficacy of Intravenous SGT-001 in Male Adolescents and Children With Duchenne Muscular Dystrophy | Duchenne Muscular Dystrophy | Genetic: SGT-001 | Solid Biosciences, LLC | NULL | Active, not recruiting | 4 Years | 17 Years | Male | 16 | Phase 1/Phase 2 | United States |