B/BE/18/BVW7 - Summary Notification Information Format (SNIF) Public information presented according to the provisions of Directive 2001/18/EC
Long title
Phase III, open-label, single-dose, multi-centre multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately-severe hemophilia B
Date receipt dossier
EU record number
B/BE/18/BVW7
EudraCT number
2017-004305-40
Company / Sponsor
uniQure biopharma B.V.
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 wild type modified to express the Padua derivative of human coagulation factor IX cDNA
Method of transfer of nucleic acid of interest
Replication competent adeno-associated viral vector
Administered biological material
Recombinant virus
Route of administration
Intravenous
Locations in Belgium
UZ Leuven; Cliniques Universitaires Saint-Luc (Brussels)
Nr of subjects
56
Foreseen duration
Q1-2019 until Q4-2021
Type of procedure
Contained use and Deliberate release
Current status
Authorized