A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
Condition(s) studied
Investigational drug(s) / intervention(s)
Eteplirsen: Solution for intravenous (IV) infusion.
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.
Eligibility
Primary outcome measure(s)
- Part 1: Incidence of Adverse Events (AEs) — Up to Week 148
- Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis) — Baseline, Week 144
- Part 2: Change from Baseline at Week 72 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis) — Baseline, Week 72 or Week 96
Trial sites (59)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | |
| University of Florida | Gainesville | Florida | |
| Rare Disease Research, LLC | Atlanta | Georgia | |
| Hospital Universitario San Ignacio | Bogotá | Colombia | |
| Instituto Neurologico de Colombia (INDEC) | Medellín | Colombia | |
| Hospital Pablo Tobón Uribe | Medellín | Colombia | |
| Brno Klinika detske neurologie | Brno | Czechia | |
| Fakultni nemocnice v Motole | Prague | Czechia | |
| Rigshospitalet Copenhagen University Hospital | Copenhagen | Denmark | |
| Hopital Femme Mere Enfant | Bron | France | |
| Hopital Armand Trousseau | Paris | France | |
| CHRU de Strasbourg | Strasbourg | France | |
| Charité Universitätsmedizin Berlin CVK | Berlin | Germany | |
| Universitätsklinikum Essen | Essen | Germany | |
| Universitätsklinikum Freiburg | Freiburg im Breisgau | Germany | |
| IASO Children's Hospital | Marousi | Attica | |
| Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete | Budapest | Hungary | |
| Royal Institute of Child Neurosciences | Ahmedabad | India | |
| Aster RV Hospital | Bengaluru | India | |
| Nizam's Institute of Medical Sciences | Hyderabad | India | |
| Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.) | Madurai | India | |
| All India Institute of Medical Sciences | New Delhi | India | |
| Sir Ganga Ram Hospital | New Delhi | India | |
| Deenanath Mangeshkar Hospital & Research Centre | Pune | India | |
| Christian Medical College | Vellore | India | |
| Children's Health Ireland (CHI) at Temple Street | Dublin | Ireland | |
| IRCCS Instituto Gianna Gaslini | Genova | Italy | |
| Fondazione Policlinico Universitario A. Gemelli- IRCCS | Rome | Italy | |
| The Specialty Hospital (TSH)/Advanced Clinical Center | Amman | Jordan | |
| Istiklal Hosptial (IST) | Amman | Jordan | |
| Irbid Specialty Hospital | Irbid | Jordan | |
| Pharmaceutical Research Center/Jordan University of Science and Technology | Irbid | Jordan | |
| Neurociencias Estudios Clínicos S.C. | Culiacán | Sinaloa | |
| Instituto de Investigaciones Clinicas para la Salud A.C | Durango | Mexico | |
| Leids Universitair Medisch Centrum | Leiden | Netherlands | |
| Radboud University Nijmegen Medical Centre | Nijmegen | Netherlands | |
| New Zealand Clinical Research - Auckland | Auckland | New Zealand | |
| Oslo Universitetssykehus HF Rikshospitalet | Oslo | Norway | |
| Children's Department and Department for Children's Habilitation at Stavanger University Hospital | Stavanger | Norway | |
| Klinika Neurologii Rozwojowej | Gdansk | Pomeranian Voivodeship |
+ 19 more sites — see the full list on the official registry below.
More Sarepta Therapeutics, Inc. trials in the USA
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03992430 on ClinicalTrials.gov ↗ ← All trials in the USA