NCT03992430

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

A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 160 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years – 13 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
DRUG	Eteplirsen	Solution for intravenous (IV) infusion.

Timeline

• Start date: 2020-07-13
• Primary completion: 2026-10-31
• Completion: 2026-10-31
• First posted: 2019-06-20
• Last updated: 2026-02-27

Locations

59 sites across 26 countries: United States, Colombia, Czechia, Denmark, France, Germany, Greece, Hungary, India, Ireland, Italy, Jordan, Mexico, Netherlands, New Zealand, Norway, Poland, Romania, Serbia, Slovenia, South Korea, Spain, Switzerland, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study