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)

Study 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)
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: 
23 feb 2024
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: 
Under evaluation

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