Give your opinion on a clinical trial with a genetically modified medicinal product for the treatment of hemophilia B.

The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial with the genetically modified medicinal product AMT-061 from the company uniQure biopharma B.V. The purpose of this trial is to demonstrate the efficacy of AMT-061 for the treatment of moderate to severe haemophilia B and to further describe its safety aspects. The public consultation runs from 17 October 2018 to 17 November 2018 inclusive.

Hemophilia B is an inherited bleeding disorder characterized by an increased bleeding tendency due to either a partial or complete deficiency of the essential blood coagulation Factor IX (FIX). The genetically modified medicinal product AMT-061 is developed to deliver the sequence encoding the human FIX at the liver to achieve a sufficient rise in constantly circulating FIX protein in adult patients suffering moderate to severe hemophilia B.

During the clinical trial, the subjects receive a single infusion of AMT-061 and are permitted to continue the routine Factor IX treatment in the first week after dosing. One week after IMP dosing, endogenous FIX activity will be assessed and FIX activity levels will be monitored throughout the study. After the post-treatment follow-up, subjects will enter a long-term follow-up for an additional four years to assess sustainability of efficacy and long-term safety.

The clinical trial will be conducted at the University Hospital in Leuven and at Cliniques universitaires Saint-Luc in Brussels.

As a citizen of Belgium you can comment on this clinical trial application. For each clinical trial application related to the use of genetically modified organisms, the authority organises a public consultation for thirty days (in accordance with the Royal Decree of 21 February 2005). You will have access to the data in the application file, as well as to an on-line form (HTML) (in Dutch and French) to send comments or ask questions about this file.

The public consultation will last until 17 November 2018 inclusive.

General information on GMOs and public consultation (HTML) (in Dutch and French)