drugset
drugset / Trial / NCT07542314

Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting

NCT07542314 ↗

Phase 4 Not yet recruiting 20 enrolled Sarepta Therapeutics, Inc.
Non-randomizedSingle-groupOpen-labelTreatment

Summary

The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.

Timeline

Start
2026-07-31
Primary completion
2027-03-31
Completion
2027-03-31

Drugs

EvaluationDrugModalityDoseRoute
Subject delandistrogene moxeparvovec Gene therapy (AAV / viral vector) — Intravenous
Background Sirolimus Small molecule — Oral

Indications

Duchenne muscular dystrophy