A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)

• Status: Active, not recruiting
• Trial end date: 2026-02-01
• Target enrollment: 0
• Participant gender: Male

Summary

This study will be comprised of 2 parts: Part 1 will be conducted to evaluate the safety and tolerability of two doses (high dose level 1 and high dose level 2) of eteplirsen in approximately 8 patients; Part 2 will be conducted for the selection of a high dose (high dose level 1 vs high dose level 2) (dose finding phase), and its comparison with the 30 mg/kg dose of eteplirsen (dose comparison phase), in approximately 144 DMD patients with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sarepta Therapeutics; Sarepta Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.

• Have achieved a mean 6-minute walk test (6MWT) distance of greater than equal to (>=) 300 and less than equal to (<=) 450 meters.

• Have intact right and left biceps muscles or an alternative upper arm muscle group.

• Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization.

• Have stable pulmonary function (forced vital capacity >= 50 percent (%) of predicted and no requirement for nocturnal ventilation).

Exclusion Criteria:

• Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.

• Current or previous treatment with gene therapy or any other experimental pharmacologic treatment for DMD; some exceptions apply.

• Previous treatment with Ezutromid in the last 1 week prior to first dose or Drisapersen in the last 36 weeks prior to first dose.

• Major surgery within 3 months prior to randomization.

• Presence of any other significant neuromuscular or genetic disease other than DMD.

• Presence of any known impairment of renal function and/or other clinically significant illness.

• Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than (<) 50% on the screening Echocardiogram or QTcF >= 450 millisecond based on the screening ECGs.

Other inclusion/exclusion criteria apply.