The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial of the genetically modified medicine CSL222 for the treatment of haemophilia B. The public consultation will take place from 28 April to 28 May 2025.
For each clinical trial application involving the use of a genetically modified organism, the government organises a 30-day public consultation, in accordance with the Royal Decree of 21 February 2005. As a citizen, you can give your opinion on this clinical trial application with a genetically modified medicine.
Patients with haemophilia B are deficient in the factor IX (FIX) protein, which is needed to form a blood clot to stop bleeding. This deficiency is caused by a fault in the gene responsible for the production of a functional protein.
The medicine CSL222 (Hemgenix®) is developed to stimulate the expression and the production of functional FIX in the liver of patients with haemophilia B. It is currently approved for use in adults.
In this clinical trial, the medicine CSL222 will be administered once via intravenous infusion to male adolescents (≥ 12 to < 18 years of age) with moderately severe to severe haemophilia B. Patients will continue to be followed up for five years.
The aim of this trial is to evaluate the safety, efficacity and tolerability of CSL222 in male adolescents with haemophilia B.
The clinical trial will take place at the University Hospital in Louvain.
How to give your opinion?
The public consultation runs from 28 April to 28 May 2025 inclusive. You can access the various data in the application file, as well as an online form to send your comments or ask your questions.
General information on GMO - public consultation.