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Higher dose of muscular dystrophy drug shows promise in new trial

NCT ID NCT03992430

Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 3 trial tests whether higher doses of eteplirsen (Exondys 51) can safely improve muscle function in boys with Duchenne muscular dystrophy whose genetic mutation allows exon 51 skipping. About 160 ambulatory boys will receive one of three doses (30, 100, or 200 mg/kg) to see if higher doses lead to better walking and movement outcomes over 144 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
eteplirsen (Exondys 51)
What this could lead to
If successful, higher doses of eteplirsen could slow muscle decline and improve mobility in boys with Duchenne muscular dystrophy.
What could go wrong
This is an early-stage dose-finding study, so benefits are unproven. Higher doses may increase side effects without added efficacy.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

160 people

The number who actually took part.

Started

Jul 2020

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 to 13 years

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Muscular dystrophy, Duchenne

Contacts and locations

Locations

  • Akdeniz Universitesi Tip Fakultesi

    Antalya, 07059, Turkey (Türkiye)

  • All India Institute of Medical Sciences

    New Delhi, 110029, India

  • Aster RV Hospital

    Bengaluru, 560078, India

  • Birmingham Heartlands Hospital

    Birmingham, B9 5SS, United Kingdom

  • Brno Klinika detske neurologie

    Brno, 613 00, Czechia

  • CHRU de Strasbourg

    Strasbourg, 67098, France

  • Charité Universitätsmedizin Berlin CVK

    Berlin, 13353, Germany

  • Children's Department and Department for Children's Habilitation at Stavanger University Hospital

    Stavanger, 4011, Norway

  • Children's Health Ireland (CHI) at Temple Street

    Dublin, D01 XD99, Ireland

  • Christian Medical College

    Vellore, 632004, India

  • Clinic for Neurology and Psychiatry for Children and Youth

    Belgrade, 11000, Serbia

  • Deenanath Mangeshkar Hospital & Research Centre

    Pune, 411004, India

  • Fakultni nemocnice v Motole

    Prague, 150 06, Czechia

  • Fondazione Policlinico Universitario A. Gemelli- IRCCS

    Rome, 00168, Italy

  • Hopital Armand Trousseau

    Paris, 75571, France

  • Hopital Femme Mere Enfant

    Bron, 69677, France

  • Hospital Pablo Tobón Uribe

    Medellín, 50034, Colombia

  • Hospital Sant Joan de Deu

    Barcelona, 8950, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, 46026, Spain

  • Hospital Universitario San Ignacio

    Bogotá, 110231, Colombia

  • IASO Children's Hospital

    Marousi, Attica, 151 23, Greece

  • IRCCS Instituto Gianna Gaslini

    Genova, 16147, Italy

  • Instituto Neurologico de Colombia (INDEC)

    Medellín, 50012, Colombia

  • Instituto de Investigaciones Clinicas para la Salud A.C

    Durango, 34000, Mexico

  • Irbid Specialty Hospital

    Irbid, 22110, Jordan

  • Istiklal Hosptial (IST)

    Amman, 11196, Jordan

  • Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)

    Madurai, 626022, India

  • Kaohsiung Medical University

    Kaohsiung City, 80756, Taiwan

  • Klinika Neurologii Rozwojowej

    Gdansk, Pomeranian Voivodeship, 80-211, Poland

  • Kyungpook National University Chilgok Hospital

    Daegu, 41404, South Korea

  • Leeds Teaching Hospitals NHS Trust

    Leeds, West Yorkshire, LS1 3EX, United Kingdom

  • Leids Universitair Medisch Centrum

    Leiden, 2333 ZC, Netherlands

  • Mersin University Medical Faculty

    Mersin, 33110, Turkey (Türkiye)

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

    Belgrade, 190133, Serbia

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

    Bucharest, 41408, Romania

  • National Taiwan University Hospital

    Taipei, 10071, Taiwan

  • Neurociencias Estudios Clínicos S.C.

    Culiacán, Sinaloa, 80020, Mexico

  • New Zealand Clinical Research - Auckland

    Auckland, 1010, New Zealand

  • Nizam's Institute of Medical Sciences

    Hyderabad, 500082, India

  • Oslo Universitetssykehus HF Rikshospitalet

    Oslo, 0450, Norway

  • Pharmaceutical Research Center/Jordan University of Science and Technology

    Irbid, 22110, Jordan

  • Pusan National University Yangsan Hospital

    Yangsan, 50612, South Korea

  • Radboud University Nijmegen Medical Centre

    Nijmegen, 6525GA, Netherlands

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30318, United States

  • Rigshospitalet Copenhagen University Hospital

    Copenhagen, 2100, Denmark

  • Royal Institute of Child Neurosciences

    Ahmedabad, 380054, India

  • Samsung Medical Center

    Seoul, 6351, South Korea

  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

    Budapest, 1082, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Sir Ganga Ram Hospital

    New Delhi, 110060, India

  • The Specialty Hospital (TSH)/Advanced Clinical Center

    Amman, 11194, Jordan

  • UCL Institute of Child Health Great Ormond Street

    London, WC1N 1EH, United Kingdom

  • University Children's Hospital

    Belgrade, 11000, Serbia

  • University Medical Centre Ljubljana

    Ljubljana, 1000, Slovenia

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Florida

    Gainesville, Florida, 32610-3010, United States

  • Universitätsklinikum Essen

    Essen, 45147, Germany

  • Universitätsklinikum Freiburg

    Freiburg im Breisgau, 79106, Germany

  • Universitätsspital Basel

    Basel, 4031, Switzerland

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