Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B

Study title: 
Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B
Long title: 
Phase 3, Open-label, Single-dose, Multicenter Study Investigating Efficacy, Safety, and Tolerability of CSL222 (Etranacogene Dezaparvovec) Administered to Adolescent Male Subjects (≥ 12 to < 18 Years of Age) with Severe or Moderately Severe Hemophilia B
Date receipt dossier: 
22 Apr 2025
EU record number: 
B/BE/25/BVW3
EudraCT number: 
2023-505805-18
Company / Sponsor: 
CSL Behring LLC
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 PADUA factor IX (FIX) gene
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: 
University Hospital Leuven (UZ Leuven)
Nr of subjects: 
Up to 18 patients overall
Foreseen duration: 
From 01-Oct-2025 till 23-Oct-2033
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

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