A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
NCT ID: NCT03992430
Last Updated: 2024-12-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
160 participants
INTERVENTIONAL
2020-07-13
2026-10-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Part 1: Eteplirsen
Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks.
Eteplirsen
Solution for intravenous (IV) infusion.
Part 2: Eteplirsen 30 mg/kg
Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.
Eteplirsen
Solution for intravenous (IV) infusion.
Part 2: Eteplirsen 100 mg/kg
Randomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks.
Eteplirsen
Solution for intravenous (IV) infusion.
Part 2: Eteplirsen 200 mg/kg
Randomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks.
Eteplirsen
Solution for intravenous (IV) infusion.
Interventions
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Eteplirsen
Solution for intravenous (IV) infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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).
4 Years
13 Years
MALE
No
Sponsors
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Sarepta Therapeutics, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Sarepta Therapeutics, Inc.
Locations
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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
Countries
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Other Identifiers
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2018-001762-42
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2024-511492-15-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
4658-402
Identifier Type: -
Identifier Source: org_study_id