A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Date receipt dossier

18 September, 2020

EU record number

B/BE/20/BVW4

EudraCT number

2019-002921-31

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

Information for the public

B/BE/20/BVW4 - Summary Notification Information Format (SNIF)

Public information presented according to the provisions of Directive 2001/18/EC

B/BE/20/BVW4 - Fiche d'information destinée au public (préparée par le notifiant)

B/BE/20/BVW4 - Informatie fiche bestemd voor het publiek (voorbereid door de kennisgever)

Information related to the decision procedure

B/BE/20/BVW4 - Advice Biosafety Advisory Council

5 February 2021 - The Biosafety Advisory Council issues a positive advice (with conditions) for this deliberate release

B/BE/20/BVW4 - Decision of the Federal Ministers (FINAL DECISION)

5 March 2021: The Federal Ministers give a final decision (positive) for this trial