113. Muscular dystrophy Clinical trials / Disease details


Clinical trials : 622 Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168

  
1 trial found
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1EUCTR2017-002213-60-GB
(EUCTR)
08/01/201911/12/2018A study to investigate the safety, tolerability, and efficacy of SGT-001 in male adolescents and children with Duchenne muscular dystrophyA 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.NULLAuthorised-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): noUnited States;United Kingdom