A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Public ClinicalTrials.gov record NCT03992430. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Study identification
- NCT ID
- NCT03992430
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Industry
- Enrollment
- 160 participants
Conditions and interventions
Conditions
Interventions
- Eteplirsen Drug
Drug
Eligibility (public fields only)
- Age range
- 4 Years to 13 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 12, 2020
- Primary completion
- Oct 30, 2026
- Completion
- Oct 30, 2026
- Last update posted
- Feb 26, 2026
2020 – 2026
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | — |
| University of Florida | Gainesville | Florida | 32610-3010 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30318 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 56 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03992430, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 26, 2026 · Synced May 6, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03992430 live on ClinicalTrials.gov.