B/BE/26/BVW3 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy: A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long-term safety follow up part, in ambulant boys
Date receipt dossier
EU record number
B/BE/26/BVW3
EudraCT number
2023-505187-11-00
Company / Sponsor
GENETHON
Phase
I/II/III
Treated organism
Humans
Indication category
Neuromuscular disorders
Disease
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 dystrophin (hMD1) gene
Method of transfer of nucleic acid of interest
Non-replicating recombinant vector derived from adeno-associated virus serotype 8
Administered biological material
Genetically modified virus
Route of administration
Intravenous
Locations in Belgium
Universitair Ziekenhuis Leuven ; Huderf Brussels
Nr of subjects
Up to 73 patients overall and 10 patients in Belgium
Foreseen duration
May 2026 – November 2031
Type of procedure
Contained use and Deliberate release
Current status
Authorized