Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

Stopped early · Phase 3

Conditions studied: Cystic Fibrosis

In brief

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Key facts

Study ID

NCT02456103

Run by

PTC Therapeutics

People needed

246

Starts

2015-08-31

Expected to finish

2017-06-02

Last updated by the study team

2020-04-27

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
• Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

You may not qualify if…

• Known hypersensitivity to any of the ingredients or excipients of the study drug.
• Ongoing participation in any other therapeutic clinical trial.

Where it is running

• Miller Children's Hospital Long Beach — Long Beach, California, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Children's Hospital and Research Center at Oakland — Oakland, California, United States
• Stanford University-Children's Hospital — Palo Alto, California, United States
• Children's Hospital Colorado — Aurora, Colorado, United States
• Nemours Children's Clinic — Jacksonville, Florida, United States
• University of Miami — Miami, Florida, United States
• Miami Children's Hospital — Miami, Florida, United States
• All Children's Hospital — St. Petersburg, Florida, United States
• Ann and Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• Indiana University — Indianapolis, Indiana, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• Morristown Medical Center — Morristown, New Jersey, United States
• Beth Israel Medical Center — New York, New York, United States
• New York University Langone Medical Center — New York, New York, United States
• Columbia University Medical Center — New York, New York, United States
• University of Cincinnati — Cincinnati, Ohio, United States
• Penn State Milton S. Hershey Medical Center — Hershey, Pennsylvania, United States
• Drexel University College of Medicine — Philadelphia, Pennsylvania, United States
• Texas Children's Hospital — Houston, Texas, United States
• University of Texas Health Science Center — Tyler, Texas, United States
• Childrens Hospital of Wisconsin — Milwaukee, Wisconsin, United States
• Hospital de Ninos Ricardo Gutierrez — Buenos Aires, Argentina
• University of Alabama at Birmingham — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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