A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy

Study title: 
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
Long title: 
A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males With Duchenne Muscular Dystrophy
Date receipt dossier: 
23 jan 2026
EU record number: 
B/BE/25/BVW8
EudraCT number: 
2025-522949-22-00
Company / Sponsor: 
Solid Biosciences Inc.
Phase: 
III
Treated organism: 
Humans
Indication category: 
Neuromuscular disorders
Disease: 
Treatment of Duchenne Muscular Dystrophy
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV, lacking all AAV viral genes and carrying the human micro-dystrophin (h-µD5) gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus serotype 9
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravenous
Locations in Belgium: 
UZ Leuven, Huderf Brussels
Nr of subjects: 
Up to 80 patients overall and 6 patients in Belgium
Foreseen duration: 
From February 2026 till end December 2033
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

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