B/BE/25/BVW8 - Informationsblatt für die Öffentlichkeit (vom Anmelder erstellt)Public information according to the provisions of Directive 2001/18/EC
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
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
Authorized