A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4

Study title: 
A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4
Long title: 
A phase 3 multinational, open-label, systemic gene delivery study to evaluate the safety and efficacy of SRP-9003 in subjects with limb girdle muscular dystrophy 2E/R4
Date receipt dossier: 
9 Feb 2024
EU record number: 
B/BE/24/BVW4
EudraCT number: 
2022-503112-17-00
Company / Sponsor: 
Sarepta Therapeutics
Phase: 
III
Treated organism: 
Humans
Indication category: 
Neuromuscular disorders
Disease: 
Treatment of Limb Girdle Muscular Dystrophy
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV, serotype rh74, lacking all AAV viral genes and carrying the full-length sarcoglycan-beta (SGCB) gene
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus serotype rh74
Administered biological material: 
Genetically modified virus
Route of administration: 
Intravenous
Locations in Belgium: 
UZ Leuven, UZ Gent
Nr of subjects: 
Up to 15 patients overall and 4 patients in Belgium
Foreseen duration: 
from May 2024 till End January 2031
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation