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NCT03992430 · ClinicalTrials.gov registry record · Phase 3
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
A Phase 3 study of Muscular Dystrophy, Duchenne, sponsored by Sarepta Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 160
- Enrollment target
- 20
- Study locations
NCT03992430: Active Phase 3 study of Muscular Dystrophy, Duchenne, sponsored by Sarepta Therapeutics.
NCT03992430 is a Phase 3 study of Muscular Dystrophy, Duchenne that is active but no longer recruiting, run by Sarepta Therapeutics. The registered enrollment target is 160 participants, above the 67-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (139% higher). The trial reports 20 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03992430, a Phase 3 study of Muscular Dystrophy, Duchenne, is active but no longer recruiting, sponsored by Sarepta Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 160 participants
- Enrollment target
- 20
- Study locations
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 |
What the registry record for NCT03992430 still lists
NCT03992430 is an interventional study that assigns participants to a tested intervention. The registered 160 participants enrollment target is mid-sized for trials with a published cap, above the 67-participant average among 4 other Muscular Dystrophy, Duchenne trials with a reported enrollment target (139% higher).
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.
NCT03992430 names 20 study sites across 5 states, led by Other, Alabama, Florida.
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.
What interventions are being tested in trial NCT03992430?
The interventions under investigation include: Eteplirsen (DRUG).
Who is sponsoring clinical trial NCT03992430?
This trial is sponsored by Sarepta Therapeutics, which has 33 total clinical trials registered on ClinicalTrials.gov.
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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