B/BE/20/BVW4 - Summary Notification Information Format (SNIF)Public information presented according to the provisions of Directive 2001/18/EC
Date receipt dossier
EU record number
B/BE/20/BVW4
EudraCT number
2019-002921-31
Company / Sponsor
Pfizer
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 AAV9, lacking all AAV viral genes and containing a miniaturized version of the human dystrophin gene
Method of transfer of nucleic acid of interest
Non-replicating recombinant adeno-associated virus serotype 9 (AAV9)
Administered biological material
Genetically modified virus
Route of administration
Intravenous
Locations in Belgium
UZ Gent, UZ Leuven, CHR Citadelle Liège
Nr of subjects
Approximately 99 patients overall and 3 patients in Belgium are foreseen.
Foreseen duration
Q3 2020 to end of treatment period ~Q4 2021
Type of procedure
Contained use and Deliberate release
Current status
Authorized
Pharmaceutical study code
C3391003