B/BE/24/BVW4 - Informationsblatt für die Öffentlichkeit (vom Anmelder erstellt)Public information according to the provisions of Directive 2001/18/EC
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
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
Authorized