ClinicaliQ Trial Snapshot
- A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) — Active Not Recruiting • Phase II • Gastroenterology • NCT06128564.
- Gene therapy trial testing safety and effectiveness of a new treatment for very young children with Duchenne muscular dystrophy over five years.
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What This Trial Is Studying
This open-label, single-arm study will evaluate the safety and expression of delandistrogene moxeparvovec in participants with DMD. Participants will be in the study for approximately 264 weeks. Conditions: Duchenne Muscular Dystrophy Interventions: delandistrogene moxeparvovec Lead Sponsor: Hoffmann-La Roche Planned Enrollment: 13 participants