Phase III trial investigating a AAV vector containing a variant of human factor IX gene administered to adult subjects with severe or moderately-severe hemophilia B

Study title: 
Phase III trial investigating a AAV vector containing a variant of human factor IX gene administered to adult subjects with severe or moderately-severe hemophilia B
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: 
12 Oct 2018
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

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