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New drugs aim to keep boys with duchenne walking longer

NCT ID NCT02500381

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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested two drugs, casimersen and golodirsen, in 228 boys with Duchenne muscular dystrophy who have specific genetic mutations. The goal was to see if these drugs could slow muscle decline, measured by how fast they could climb four steps. Participants received either the drug or a placebo for 96 weeks, with some continuing in an open-label extension.

What this could mean

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

Active substance
Casimersen (SRP-4045) and Golodirsen (SRP-4053)
What this could lead to
If successful, these drugs could slow muscle decline and help boys with Duchenne muscular dystrophy walk longer and maintain strength.
What could go wrong
This is a Phase 3 trial, but Duchenne is a severe, progressive disease. The drugs only work for specific genetic mutations, and long-term benefits are not guaranteed.

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

228 people

The number who actually took part.

Started

Sep 2016

Finished

Oct 2025

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

6 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: * Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping * Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight). * Intact right and left biceps brachii muscles or 2 alternative upper arm muscle groups * Mean 6MWT ≥300 meters and ≤450 meters * Stable pulmonary function: forced vital capacity (FVC) ≥50% predicted Exclusion Criteria: * Treatment with gene therapy at any time * Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24 weeks prior to Week 1 * Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1 * Major surgery within 3 months prior to Week 1 * Presence of other clinically significant illness * Other inclusion/exclusion criteria may 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.

Duchenne muscular dystrophy

Contacts and locations

Locations

  • Alberta Childrens Hospital

    Calgary, Alberta, T3B 6A8, Canada

  • Alder Hey Childrens Hospital

    Liverpool, L12 2AP, United Kingdom

  • Ann and Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Az Ospedaliera Universitaria Policlinico G Martino

    Messina, 98125, Italy

  • Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna

    Ferrara, 44124, Italy

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • CHR de la Citadelle

    Liège, 4000, Belgium

  • Center for Integrative Rare Disease Research (CIRDR)

    Atlanta, Georgia, 30318, United States

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353, Germany

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital at Westmead

    Westmead, 2145, Australia

  • Children's Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1, Canada

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Children's Hospital of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Children's Medical Center Dallas

    Dallas, Texas, 75235, United States

  • Children's and Women's Health Centre of British Columbia

    Vancouver, British Columbia, V6H 3V4, Canada

  • Cincinnati Children's Hospital Medical Center (CCHMC)

    Cincinnati, Ohio, 45229, United States

  • Clinic for Neurology and Psychiatry for Children and Youth

    Belgrade, 11000, Serbia

  • DOM Centro de Reumatologia

    Buenos Aires, 1111, Argentina

  • David Geffen School of Medicine, UCLA

    Los Angeles, California, 90095, United States

  • Deenanth Mangeshkar Hospital

    Pune, Maharashtra, 411004, India

  • Drottning Silvias Barn Och Ungdomssjukhus

    Gothenburg, SE-41685, Sweden

  • Fakultni nemocnice v Motole

    Prague, 15008, Czechia

  • Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation

    Moscow, 125412, Russia

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Great Ormond Street Hospital (GOSH)

    London, WC1N 1EH, United Kingdom

  • Hospital Sant Joan de Deu

    Barcelona, 08950, Spain

  • Hospital Universitari i Politecnic La Fe de Valencia

    Valencia, Spain

  • Hospital de La Santa Creu i Sant Pau

    Barcelona, 08041, Spain

  • Hôpital Armand Trousseau

    Paris, 75012, France

  • Hôpital Des Enfants

    Toulouse, Haute-Garonne, 31059, France

  • IASO Children's Hospital

    Marousi, 15123, Greece

  • Instituto de Investigaciones Clínicas para la Salud A.C

    Durango, 34000, Mexico

  • Ippokratio General Hospital of Thessaloniki

    Thessaloniki, 54642, Greece

  • Istituto Giannina Gaslini

    Genoa, 16147, Italy

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • LMU Klinikum der Universität

    München, Bavaria, 80337, Germany

  • Las Vegas Clinic

    Las Vegas, Nevada, 89145, United States

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS1 3EX, United Kingdom

  • London Health Sciences Centre

    London, Ontario, N6A 5W9, Canada

  • NW Florida Clinical Research Group, LLC

    Gulf Breeze, Florida, 32561, United States

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Neurociencias Estudios Clínicos S.C

    Culiacán, 80020, Mexico

  • Neuromuscular Research Center

    Phoenix, Arizona, 85028, United States

  • Policlinico Universitario A Gemelli

    Rome, 00168, Italy

  • Pusan National University Yangsan Hospital

    Yangsan, 50612, South Korea

  • Queensland Children's Hospital

    South Brisbane, 4101, Australia

  • Rady Children's Hospital San Diego/ UCSD

    San Diego, California, 92123, United States

  • Reference Centre for Neuromuscular Diseases

    Nantes, 44093, France

  • Rigshospitalet Copenhagen University Hospital

    København Ø, 2100, Denmark

  • Royal Children's Hospital Melbourne

    Parkville, Victoria, 3052, Australia

  • Royal Hospital for Children (Glasgow)

    Glasgow, G51 4TF, United Kingdom

  • Royal Instituite of Child Neurosciences

    Ahmedabad, Gujarat, 380054, India

  • Royal Victoria Infirmary

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • Samodzielny Publiczny Centralny Szpital Kliniczny

    Warsaw, Masovian Voivodeship, 02-097, Poland

  • Schneider Children's Medical Center of Israel

    Petah Tikvah, 49102, Israel

  • Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete

    Budapest, 1083, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Shriners Hospital for Children

    Portland, Oregon, 97239, United States

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Stanford University School of Medicine/Medical Center

    Stanford, California, 94305, United States

  • State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg

    Yekaterinburg, Russia

  • The Children's University Hospital

    Dublin, D1, Ireland

  • Universitair Ziekenhuis Gent

    Ghent, 9000, Belgium

  • Universitair Ziekenhuis Leuven

    Leuven, 3000, Belgium

  • University Hospital Brno

    Brno, 61300, Czechia

  • University Hospital Freiburg

    Freiburg im Breisgau, 79106, Germany

  • University Multiprofile Hospital for Active Treatment Aleksandrovska EAD

    Sofia, Sofia-Grad, 1431, Bulgaria

  • University of Florida

    Gainesville, Florida, 32610, United States

  • University of Iowa Children's Hospital

    Iowa City, Iowa, 52242, United States

  • University of Kansas, Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Rochester Clinical Research Center

    Rochester, New York, 14642, United States

  • University of Utah

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Essen

    Essen, 45122, Germany

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-952, Poland

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Other studies related to the condition(s) this trial covers.