HomeTrialNCT03992430
🔬NCT03992430Phase 3ACTIVE NOT RECRUITING

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Study Summary

Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram \[mg/kg\] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

Interventions / Drugs
View on ClinicalTrials.gov ↗
Study Details
Enrollment160 participants
Primary Completion
Lead SponsorSarepta Therapeutics, Inc.
Data Retrieved2026-04-11 02:55:56.327675+00:00
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