Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

Completed · Phase 3

Conditions studied: Duchenne Muscular Dystrophy

In brief

The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

Key facts

Study ID

NCT01247207

Run by

PTC Therapeutics

People needed

270

Starts

2010-11-30

Expected to finish

2026-02-10

Last updated by the study team

2026-03-09

Who can join

Age: any. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Phoenix Childrens Hospital — Phoenix, Arizona, United States
• Children's Hospital of Los Angeles — Los Angeles, California, United States
• UCLA — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• University of California-Davis — Sacramento, California, United States
• University of CA, San Francisco-Benioff Children's Hospital — San Francisco, California, United States
• Children's Hospital Colorado - Center for Cancer and Blood Disorders — Aurora, Colorado, United States
• Child Neurology Center of NW Florida NW — Gulf Breeze, Florida, United States
• Rare Disease Research, LLC — Atlanta, Georgia, United States
• Rush Univ Medical Center — Chicago, Illinois, United States
• University of Iowa Children's Hospital — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Children's Hospital of Boston/Harvard Medical School — Boston, Massachusetts, United States
• University of Michigan — Ann Arbor, Michigan, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University Medical School — St Louis, Missouri, United States
• Columbia University Pediatric Neuromuscular Center — New York, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• Cincinnati Childrens Hospital Medical Center — Cincinnati, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Shriners Hospital for Children-Portland — Portland, Oregon, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• University of Pittsburgh Medical Center — Pittsburgh, Pennsylvania, United States
• Childrens Medical Center Dallas — Dallas, Texas, United States
• Texas Children's Hospital — Houston, Texas, United States

Full record on ClinicalTrials.gov

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