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NCT03992430

PHASE3

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

Sponsor: Sarepta Therapeutics, Inc.

View on ClinicalTrials.gov

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.

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

Key Details

Gender

MALE

Age Range

4 Years - 13 Years

Study Type

INTERVENTIONAL

Enrollment

160

Start Date

2020-07-13

Completion Date

2026-10-31

Last Updated

2026-02-27

Healthy Volunteers

Conditions

Muscular Dystrophy, Duchenne

Interventions

DRUG

Eteplirsen

Solution for intravenous (IV) infusion.

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.

Locations (59)

University of Alabama at Birmingham

Birmingham, Alabama, United States

University of Florida

Gainesville, Florida, United States

Rare Disease Research, LLC

Atlanta, Georgia, United States

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, Greece

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, Mexico

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, Poland

National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"

Bucharest, Romania

Clinic for Neurology and Psychiatry for Children and Youth

Belgrade, Serbia

University Children's Hospital

Belgrade, Serbia

Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"

Belgrade, Serbia

University Medical Centre Ljubljana

Ljubljana, Slovenia

Kyungpook National University Chilgok Hospital

Daegu, South Korea

Seoul National University Hospital

Seoul, South Korea

Samsung Medical Center

Seoul, South Korea

Pusan National University Yangsan Hospital

Yangsan, South Korea

Hospital Sant Joan de Deu

Barcelona, Spain

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, Spain

Universitätsspital Basel

Basel, Switzerland

Kaohsiung Medical University

Kaohsiung City, Taiwan

National Taiwan University Hospital

Taipei, Taiwan

Akdeniz Universitesi Tip Fakultesi

Antalya, Turkey (Türkiye)

Mersin University Medical Faculty

Mersin, Turkey (Türkiye)

Leeds Teaching Hospitals NHS Trust

Leeds, West Yorkshire, United Kingdom

Birmingham Heartlands Hospital

Birmingham, United Kingdom

UCL Institute of Child Health Great Ormond Street

London, United Kingdom

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (59)