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

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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

NCT03992430•Sarepta Therapeutics, Inc.

View on ClinicalTrials.gov

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.

Eligibility

Age

4 - 13 years

Sex

MALE

Healthy Volunteers

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.
•Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
•Able to walk independently without assistive devices.
•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 and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.
•For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening.

Exclusion Criteria

•Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
•Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; Eteplirsen (previous or current use); and Tamoxifen in the last 4 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 Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs).

Locations (59)

University of Alabama at Birmingham

Birmingham, Alabama, United States

University of Florida

Gainesville, Florida, United States

Rare Disease Research, LLC

Atlanta, Georgia, United States

Hospital Universitario San Ignacio

Bogotá, Colombia

Instituto Neurologico de Colombia (INDEC)

Medellín, Colombia

Hospital Pablo Tobón Uribe

Medellín, Colombia

Brno Klinika detske neurologie

Brno, Czechia

Fakultni nemocnice v Motole

Prague, Czechia

Rigshospitalet Copenhagen University Hospital

Copenhagen, Denmark

Hopital Femme Mere Enfant

Bron, France

Key Dates

Start Date

July 13, 2020

Primary Completion Date

October 31, 2026

Completion Date

October 31, 2026

Last Updated

February 27, 2026

Enrollment

160

ACTUAL participants

Conditions

Muscular Dystrophy, Duchenne

Interventions

Eteplirsen

DRUG

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372

Muscular Dystrophy, Duchenne

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COMPLETED

Regular Physical Exercise in Duchenne Muscular Dystrophy

NCT03963453

Muscular Dystrophy, Duchenne

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COMPLETEDPHASE1/PHASE2

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

NCT03769116

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 27, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Regular Physical Exercise in Duchenne Muscular Dystrophy

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

An Open-Label Extension Study for Patients With Duchenne Muscular Dystrophy Who Participated in Studies of SRP-5051 (Vesleteplirsen)

Trunk Oriented Exercises Versus Whole-body Vibration for Duchenne Muscular Dystrophy

Validating Cardiac MRI Biomarkers and Genotype-Phenotype Correlations for DMD