B/BE/24/BVW5 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
Phase 3, open-label, single-arm study to evaluate efficacy and safety of FIX gene transfer with PF-06838435 (rAAV-Spark100-hFIX-R338L) in adult male participants with moderately severe to severe hemophilia B (FIX:C ≤2%) (BeneGene-2)
Date receipt dossier
EU record number
B/BE/24/BVW5
EudraCT number
2022-502844-11-00
Company / Sponsor
Pfizer
Phase
III
Treated organism
Humans
Indication category
Genetic disorders
Disease
Hemophilia B
Therapeutic approach
Gene therapy
Genetic modification
Recombinant adeno associated viral vector containing the codon-optimised human factor IX (FIX) minigene (R338L variant)
Method of transfer of nucleic acid of interest
Non-pathogenic recombinant competent adeno-associated viral vector
Administered biological material
Genetically modified virus
Route of administration
Intravenous
Locations in Belgium
Cliniques Universitaires Saint-Luc (Brussels)
Nr of subjects
Up to 50 patients overall and 2 patients in Belgium
Foreseen duration
From March 2024 till March 2030
Type of procedure
Contained use and Deliberate release
Current status
Authorized