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New drug aims to help boys with duchenne walk longer

NCT ID NCT03179631

Disease control Sponsor: PTC Therapeutics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 a drug called ataluren in 360 boys with Duchenne muscular dystrophy caused by a specific genetic mistake (nonsense mutation). The main goal was to see if ataluren could help them walk farther over 72 weeks compared to a placebo. All participants were also taking steroids. The trial is now complete.

What this could mean

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

Active substance
ataluren
What this could lead to
If it works, ataluren could slow the loss of walking ability in boys with a specific genetic form of Duchenne muscular dystrophy.
What could go wrong
This is a completed Phase 3 trial, but results may not show a meaningful benefit over placebo. The drug only works for a small subset of patients with a nonsense mutation.

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

360 people

The number who actually took part.

Started

Jul 2017

Finished

Jul 2023

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

5 years and older

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: * Male sex * Age ≥5 years * Phenotypic evidence of Duchenne Muscular Dystrophy * Nonsense point mutation in the dystrophin gene * Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment * 6MWD ≥150 meters * Ability to perform timed function tests within 30 seconds * Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions. Exclusion Criteria: * Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment. * Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy. * Prior or ongoing therapy with ataluren. * Known hypersensitivity to any of the ingredients or excipients of the study drug * Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial. * History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period. * Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy. * Uncontrolled clinical symptoms and signs of congestive heart failure * Elevated serum creatinine or cystatin C at screening.

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Conditions

The condition(s) this trial relates to.

atrophic muscular disease Duchenne muscular dystrophy hereditary disease muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder nervous system disorder neuromuscular disease X-linked disease

As listed by the trial registrant

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

Genetic diseases, inborn Genetic diseases, X-linked Muscular diseases Muscular disorders, atrophic Muscular dystrophies Muscular dystrophy, Duchenne Musculoskeletal disease Nervous system diseases Neuromuscular diseases

Contacts and locations

Locations

  • "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre

    Saint Petersburg, 194100, Russia

  • All India Institute of Medical Sciences

    New Delhi, 110029, India

  • Apollo Children's Hospital Chennai

    Chennai, Tamil Nadu, 600006, India

  • Apollo Gleneagles Hospital

    Kolkata, West Bengal, 700054, India

  • Children's Hospital of Fudan University

    Shanghai, 200032, China

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Michigan

    Detroit, Michigan, 48201, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Childrens Hospital London Health Sciences Centre

    London, Ontario, N6A5W9, Canada

  • Childrens Hospital of Eastern Ontario

    Ottawa, Ontario, K1H8L1, Canada

  • Christian Medical College Hospital Vellore

    Vellore, Tamil Nadu, 632004, India

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University College of Physicians & Surgeons

    New York, New York, 10032, United States

  • Cook Childrens Medical Center

    Fort Worth, Texas, 76104, United States

  • General Hospital of Chinese Armed Police Forces

    Beijing, 100039, China

  • Hospital Angeles Chihuahua

    Chihuahua City, 31217, Mexico

  • Hospital Tunku Azizah Kuala Lumpur

    Kuala Lumpur, 50586, Malaysia

  • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP

    São Paulo, 05403-000, Brazil

  • Hospital de Clínicas da Universidade Federal de Minas Gerais

    Belo Horizonte, 30130-100, Brazil

  • Indiana University Health - Riley Child Neurology

    Indianapolis, Indiana, 46202, United States

  • Instituto Nacional de Pediatría

    Mexico City, 04530, Mexico

  • Instituto Nacional de Rehabilitacion

    Tlalpan, 14389, Mexico

  • Istanbul University- Instanbul Medical Faculty

    Istanbul, 34093, Turkey (Türkiye)

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 80756, Taiwan

  • Loma Linda University Children's Hospital

    San Bernardino, California, 92408, United States

  • National Institute of Mental Health and Neurosciences

    Bengaluru, Karnataka, 560029, India

  • National Taiwan University Hospital

    Taipei, 10002, Taiwan

  • Nizam's Institute of Medical Sciences (NIMS)

    Hyderabad, Telangana, 500082, India

  • Northwest Florida Clinical Research Group, LLC

    Gulf Breeze, Florida, 32561, United States

  • P.D. Hinduja Hospital

    Māhīm, Maharashtra, 400016, India

  • PTC Clinical Site

    Multiple Locations, Japan

  • Panchshil Hospital

    Ahmedabad, Gujarat, 380005, India

  • Perth Children's Hospital

    Nedlands, Western Australia, 6009, Australia

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Postgraduate Institute of Medical Education and Research

    Chandigarh, 160012, India

  • Pusan National University Yangsan Hospital

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Queen Mary Hospital

    Hong Kong, SAR, Hong Kong

  • Queensland Children's Hospital

    South Brisbane, Q4101, Australia

  • Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev

    Moscow, 125412, Russia

  • Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie

    Warsaw, 02-097, Poland

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Seoul National University Hospital

    Seoul, 3080, South Korea

  • Shenzhen Children's Hospital

    Shenzhen, 518038, China

  • Shriners Hospital for Children

    Portland, Oregon, 97239, United States

  • Siriraj Hospital

    Bangkok, 10700, Thailand

  • Stanford University Medical Center

    Palo Alto, California, 94305, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • The Childrens Hospital at Westmead

    Westmead, New South Wales, 2145, Australia

  • The First Affiliated Hospital of Fujian Medical University

    Fuzhou, 350005, China

  • The Royal Childrens Hospital

    Parkville, Victoria, 3052, Australia

  • UMHAT Sofiamed

    Sofia, 1793, Bulgaria

  • Universidade Federal do Rio de Janeiro

    Rio de Janeiro, 21.941-912, Brazil

  • University Malaya Medical Centre (UMMC)

    Pantai, 59100, Malaysia

  • University Of Utah

    Salt Lake City, Utah, 84112, United States

  • University of California (UC) Davis Medical Center

    Sacramento, California, 95817, United States

  • University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland

    Oakland, California, 94143, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Michigan - CS Mott Children's Hospital

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of Pittsburgh School of Medicine

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Puerto Rico - School of Medicine

    San Juan, 00936-5067, Puerto Rico

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78229-3900, United States

  • Xiangya Hospital Central South University

    Hunan, 410008, China

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