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 with Duchenne Muscular Dystrophy

Study title: 
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 with Duchenne Muscular Dystrophy
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: 
4 mrt 2026
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: 
Assessed

Information for the public

Information related to the decision procedure