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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 · View on ClinicalTrials.gov ↗
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 Studied
Interventions
- DRUG Eteplirsen
Study Locations (20)
Other
- Hospital Universitario San Ignacio — Bogotá
- Instituto Neurologico de Colombia (INDEC) — Medellín
- Hospital Pablo Tobón Uribe — Medellín
- Brno Klinika detske neurologie — Brno
- Fakultni nemocnice v Motole — Prague
- Rigshospitalet Copenhagen University Hospital — Copenhagen
- Hopital Femme Mere Enfant — Bron
- Hopital Armand Trousseau — Paris
- CHRU de Strasbourg — Strasbourg
- Charité Universitätsmedizin Berlin CVK — Berlin
- Universitätsklinikum Essen — Essen
- Universitätsklinikum Freiburg — Freiburg im Breisgau
- Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete — Budapest
- Royal Institute of Child Neurosciences — Ahmedabad
- Aster RV Hospital — Bengaluru
- Nizam's Institute of Medical Sciences — Hyderabad
Alabama
- University of Alabama at Birmingham — Birmingham
Florida
- University of Florida — Gainesville
Georgia
- Rare Disease Research, LLC — Atlanta
Attica
- IASO Children's Hospital — Marousi
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 160 participants |
| Start Date | 2020-07-13 |
| Est. Completion | 2026-10-31 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03992430
The ClinicalTrials.gov registry entry for NCT03992430 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 160 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sarepta Therapeutics, which has 10 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Muscular Dystrophy, Duchenne appearing as the primary indexed condition, and to 1 intervention — of which Eteplirsen is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03992430 reports 20 study locations spanning 5 distinct geographic areas — top geographies include Other, Alabama, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03992430 about?
NCT03992430 is a clinical study titled "A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)". 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 20...
What is the current status of trial NCT03992430?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 160 participants. The study started on 2020-07-13. Estimated completion is 2026-10-31.
What conditions does trial NCT03992430 study?
This clinical trial studies the following conditions: Muscular Dystrophy, Duchenne. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03992430?
The interventions under investigation include: Eteplirsen (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03992430?
This trial is sponsored by Sarepta Therapeutics, which has 10 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03992430 being conducted?
This trial has 20 study locations across Alabama, Florida, Georgia, Attica. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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