DMD research

New Roche study of Duchenne gene therapy Elevidys announced

Roche has announced early plans to initiate a new global Phase 3 clinical study of delandistrogene moxeparvovec (Elevidys), a gene therapy for Duchenne muscular dystrophy (DMD). The study is intended to generate additional placebo-controlled data to support potential future regulatory submissions in the UK and Europe.

Assess efficacy and safety

Roche says that the double-blind study will enrol approximately 100 ambulatory boys with DMD (likely between ages four and six) and will assess the efficacy and safety of the treatment over a 72‑week period. Participants initially assigned to placebo will be eligible to receive the gene therapy after the primary study period.

The study design builds on results from the Phase 3 EMBARK trial and incorporates feedback from regulators and the DMD community. Roche says that further details, including eligibility criteria, which countries the trials will take part in, trial sites and enrolment timelines, will be shared as planning progresses.

Read the full community letter from Roche.

Elevidys in the UK

Sarepta and Roche halted several clinical studies for Elevidys in April last year, following the tragic death of a young man with DMD, who passed away after being treated with Elevidys. These studies remain on hold and the companies are continuing to work with the relevant health authorities to determine next steps.  
 
This announcement is about a new study separate from those currently on hold that would involve a smaller cohort of ambulant patients, likely in a narrower age range.