Clinical Trial

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

Study acronym: MIS51ON
Active, Not Recruiting Phase 3
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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.
Protocol Amendment History 27 amendments
This ClinicalTrials.gov record has been amended 27 times since 2019-06-18; most recent amendment 2026-02-25.
Status change: Recruiting → Active, Not Recruiting 2023-11-28
Status change: Active, Not Recruiting → Recruiting 2022-01-19
Status change: Recruiting → Active, Not Recruiting 2021-01-15
Status change: Not Yet Recruiting → Recruiting 2019-12-05
Trial Details
NCT Number NCT03992430
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Muscular Dystrophy, Duchenne
Enrollment 160 participants
Start Date 2020-07-13
Primary Completion 2026-10-31 (estimated)
Study Completion 2026-10-31 (estimated)
Updated on ClinicalTrials.gov 2026-02-27