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Clinical Trials in the USA / NCT03992430
Active, not recruiting Phase 3

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

NCT03992430 · tracked via the Priya Life Science USA tracker
Sponsor
Sarepta Therapeutics, Inc.
Phase
Phase 3
Started
2020-07-13
Last updated
2026-02-27

Condition(s) studied

Muscular Dystrophy, Duchenne

Investigational drug(s) / intervention(s)

Eteplirsen

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

Sex
MALE
Min age
4 Years
Max age
13 Years
Healthy volunteers
No
Inclusion Criteria: * Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping. * Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit. * Able to walk independently without assistive devices. * Have intact right and left biceps muscles or an alternative upper arm muscle group. * Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study. * For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening. Exclusion Criteria: * Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization. * Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; Eteplirsen (previous or current use); and Tamoxifen in the last 4 weeks prior to first dose. * Major surgery within 3 months prior to randomization. * Presence of any other significant neuromuscular or genetic disease other than DMD. * Presence of any known impairment of renal function and/or other clinically significant illness. * Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than \<50% on the screening echocardiogram or Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs). Other inclusion/exclusion criteria apply.

Primary outcome measure(s)

Trial sites (59)

FacilityCityRegionStatus
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

Other trials for the same condition

Official registry record

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