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Experimental drug ataluren tested for safety in duchenne MD patients

NCT ID NCT01247207

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested the safety of a drug called ataluren (also known as Translarna) in 270 people with Duchenne muscular dystrophy caused by a specific type of genetic mistake (nonsense mutation). Participants had already taken ataluren in earlier studies. The goal was to monitor side effects, lab results, and physical changes over time. The drug is meant to help the body produce a working version of the dystrophin protein, which is missing in Duchenne.

What this could mean

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

Active substance
ataluren (PTC124, Translarna)
What this could lead to
If successful, this could confirm that ataluren is safe enough to use long-term for managing Duchenne muscular dystrophy in people with a specific genetic mutation.
What could go wrong
This is an open-label safety study, not designed to prove the drug works. It only includes people who already took ataluren, so results may not apply to new patients. Side effects are still possible.

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

About 270 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2010

Finished

Feb 2026

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed. * History of exposure to ataluren in a prior PTC study or treatment plan and effected nmDBMD siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). * Fertile men, who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug. * Willingness and ability to comply with scheduled visits, drug administration and return plan, study procedures, laboratory tests, and study restrictions Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered. Exclusion Criteria: * Exposure to another investigational drug within 1 month prior to start of study treatment. * Eligibility for another ataluren clinical trial that is actively enrolling study participants. * Positive for Hepatitis B core antibody or Hepatitis C antibody at screening for ataluren naïve participants (siblings) or participants who have a temporary treatment gap of 1 year before entering study * Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate). * Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy. * Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant or make it unlikely that follow-up would be completed.

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Conditions

The condition(s) this trial relates to.

Duchenne muscular dystrophy neuromuscular disease caused by qualitative or quantitative defects of dystrophin

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 Children's Hospital

    Calgary, Alberta, Canada

  • British Columbia Children's Hospital

    Vancouver, British Columbia, Canada

  • CHU de Québec - Université Laval

    Québec, Canada

  • Child Neurology Center of NW Florida NW

    Gulf Breeze, Florida, 32561, United States

  • Children's Hospital Colorado - Center for Cancer and Blood Disorders

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Boston/Harvard Medical School

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of East Ontario

    Ottawa, Ontario, Canada

  • Children's Hospital of Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Childrens Medical Center Dallas

    Dallas, Texas, 75207, United States

  • Cincinnati Childrens Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Columbia University Pediatric Neuromuscular Center

    New York, New York, 10032, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • London Health Sciences Centre

    London, Ontario, Canada

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Phoenix Childrens Hospital

    Phoenix, Arizona, 85016, United States

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Rush Univ Medical Center

    Chicago, Illinois, 60612, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • Shriners Hospital for Children-Portland

    Portland, Oregon, 97239, United States

  • Stanford University

    Palo Alto, California, 94304, United States

  • Texas Children's Hospital

    Houston, Texas, 77030, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • University of CA, San Francisco-Benioff Children's Hospital

    San Francisco, California, 94158, United States

  • University of California-Davis

    Sacramento, California, 95817, 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 Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 16148, United States

  • University of Texas Health Science Center at San Antonio

    San Antonio, Texas, 78229, United States

  • University of Utah

    Salt Lake City, Utah, 84112, United States

  • Washington University Medical School

    St Louis, Missouri, 63110, United States

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