Give your opinion on a genetically modified drug for the treatment of Duchenne muscular dystrophy

The FPS Public Health and FAMHP would like to invite you to take part in the public consultation on a clinical trial testing the genetically modified drug PF-06939926 for the treatment of Duchenne muscular dystrophy. The public consultation will run from 23 September 2020 to 23 October 2020.

For each clinical trial application related to the use of genetically modified organisms, the authority organises a public consultation lasting 30 days (in accordance with the Royal Decree of 21 February 2005). This gives you the opportunity, as a citizen, to share your opinion on the clinical trial application for the genetically modified drug PF-06939926 for the treatment of Duchenne muscular dystrophy.

This clinical trial will be conducted on male patients (aged ≥ 4 to <8 years old) with a genetic diagnosis of Duchenne muscular dystrophy and who are being treated with a stable daily glucocorticoid regimen. Patients will be randomly placed in the treatment group or in the placebo group. Patients in the treatment group will receive a single infusion of PF-06939926. Patients in the placebo group will receive a single dose of the treatment after one year of enrolment in the trial. All participants will be monitored for five years after the administration of PF-06939926.

The main objective of this trial is to demonstrate the superior efficacy of treatment with PF-06939926 compared to placebo.

The clinical trial will take place at the UZ Ghent, UZ Leuven and the CHR de la Citadelle, Liège.

How can I share my opinion?

The public consultation will run until 23 October 2020 inclusive. You can now access the various data in the application file, as well as an online form to send us your comments or ask your questions about this file.

General information on public consultations on GMOs.

Last updated on
25/09/2020