Phase III Trial of AMT-061 in adults with haemophilia B
Research type
Research Study
Full title
Phase III, open-label, single-dose, multi-center 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
IRAS ID
247085
Contact name
Syed Rashid Saeed Kamzi
Contact email
Sponsor organisation
uniQure biopharma B.V.
Eudract number
2017-004305-40
Duration of Study in the UK
7 years, 0 months, 0 days
Research summary
AMT-061 is a gene therapy being developed and studied for the treatment of patients with Haemophilia B. Haemophilia B is a congenital disease characterised by increased bleeding tendencies due to either a partial or complete deficiency of the essential blood coagulation Factor IX (FIX). The deficiency is the result of mutations of the respective clotting factor genes. Haemophilia B is an X-linked, recessive condition, since it occurs almost exclusively in males, with females typically asymptomatic carriers.
This is a Global Phase III clinical trial, approximately 56 patients are expected to be enrolled at 40 sites across Europe, North America and South Africa. This study is a part of the AMT-061 program and will run in parallel to the CT-AMT-061-01 study currently active in the USA. This study consists of a screening period, lead-in phase, dosing, post-dose follow-up and long-term follow up. The study design (6 months-lead in phase) allows for the collection and analysis of interim data from the Phase IIb study before the study therapy is administered to any patient.
The primary objective of this study is to demonstrate the effect of AMT-061 on endogenous Factor IX activity 6 months after a single dose of AMT-061. The secondary objectives of this study are to demonstrate the non-inferiority of AMT-061 as compared to standard of care continuous routine Factor IX prophylaxis in terms of prevention of bleeding and to demonstrate additional efficacy and safety aspects of systemic administration of AMT-061.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
18/SC/0342
Date of REC Opinion
6 Sep 2018
REC opinion
Further Information Favourable Opinion