B/BE/25/BVW3 - 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-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
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
Authorized