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

NCT03992430 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 160

Last updated 2026-02-27

No results posted yet for this study

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

DRUG

Eteplirsen

Solution for intravenous (IV) infusion.

Sponsors & Collaborators

  • Sarepta Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
13 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-07-13
Primary Completion
2026-10-31
Completion
2026-10-31
FDA Drug
Yes

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

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03992430 on ClinicalTrials.gov