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drugset / Trial / NCT06128564

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)

NCT06128564 ↗

Phase 2 Active not recruiting 13 enrolled Hoffmann-La Roche Sarepta Therapeutics, Inc. · collab
NaSingle-groupOpen-labelTreatment

Summary

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.

Timeline

Start
2023-11-29
Primary completion
2026-03-04
Completion
2030-02-18

Drugs

EvaluationDrugModalityDoseRoute
Subject delandistrogene moxeparvovec Gene therapy (AAV / viral vector) — Intravenous

Indications

Duchenne muscular dystrophy