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

  • End date
    Nov 30, 2024
  • participants needed
  • sponsor
    Sarepta Therapeutics, Inc.
Updated on 21 October 2022
walk test
muscular dystrophy
dmd gene
deletion mutation
forced vital capacity


This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to 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 be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.

Condition Muscular Dystrophy, Duchenne
Treatment Eteplirsen
Clinical Study IdentifierNCT03992430
SponsorSarepta Therapeutics, Inc.
Last Modified on21 October 2022


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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
Have intact right and left biceps muscles or an alternative upper arm muscle group
Able to walk independently without assistive devices
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; and Eteplirsen (previous or current use)
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)
Other inclusion/exclusion criteria apply
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