Roche’s update on clinical hold in Duchenne muscular dystrophy
On the 18th March Sarepta announced the sad news regarding a young man with Duchenne muscular dystrophy who passed away following treatment with delandistrogene moxeparvovec, having suffered acute liver failure. There is still an ongoing assessment and analysis of information following this sad event.
The European Medicines Agency (EMA) has requested a temporary clinical hold on Study 104 (NCT06241950), Study 302 (ENVOL, NCT06128564), and Study 303 (ENVISION, NCT05881408) until the analysis into the cause of death is complete. Please find additional information about each of these studies below.
Roche is pausing enrollment and dosing of participants in these three studies at EU country study sites. For the Roche-sponsored ENVOL study enrollment and dosing is also being paused in the UK. Patient safety monitoring for already enrolled participants and ongoing collection of data will continue
Patient safety and well-being are Roche’s top priority and they will be collaborating closely with EMA to this effect.
- Study 104 (NCT06241950), a Sarepta-sponsored Phase I open-label, systemic gene delivery study to evaluate the safety, tolerability and expression of delandistrogene moxeparvovec in association with imlifidase in individuals aged 4 to 9 years with pre-existing antibodies to recombinant adeno-associated virus serotype, rAAVrh74.
- ENVOL (Study 302, NCT06128564), a Roche-sponsored Phase II study evaluating the safety of delandistrogene moxeparvovec and expression of delandistrogene moxeparvovec micro-dystrophin protein in young children, including babies and newborns
- ENVISION (Study 303, NCT05881408), a Sarepta-sponsored global Phase III study investigating the safety and efficacy of delandistrogene moxeparvovec in participants who are ambulatory (aged 8 to <18 years old) and non-ambulatory (no age limitation).
Reporting Side Effects
If you get any side effects during a clinical trial, talk to your doctor, pharmacist or nurse who are part of the clinical trial team. They will report the side effect(s) following the procedure detailed in the clinical trial process. Reporting side effects during clinical trials helps to provide more information on the safety of potential medicines.
If you would like to talk about the topic discussed in this article, please do reach out to us at info@actionduchenne.org.
