Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients with the disease. Ataluren is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. Funding source for this study is the FDA OOPD.

Key facts

Study ID

NCT01140451

Run by

PTC Therapeutics

People needed

191

Starts

2010-08-12

Expected to finish

2013-12-02

Last updated by the study team

2020-10-19

Who can join

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

You may qualify if…

• Completion of blinded study drug treatment in the previous Phase 3 study (PTC124-GD-009-CF).
• Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).
• In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 4-week follow up period.
• Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

You may not qualify if…

• Known hypersensitivity to any of the ingredients or excipients of the study drug (list provided at study sites).
• Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3 study.
• Ongoing participation in any other therapeutic clinical trial.
• Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.

Where it is running

• University of Alabama-Birmingham — Birmingham, Alabama, United States
• Miller Children's Hospital Long Beach — Long Beach, California, United States
• Lucile Packard Children's Hospital — Palo Alto, California, United States
• The Children's Hospital — Aurora, Colorado, United States
• University of Miami — Miami, Florida, United States
• Emory University Cystic Fibrosis Center — Atlanta, Georgia, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• Johns Hopkins Children's Center — Baltimore, Maryland, United States
• Children's Hospital Boston — Boston, Massachusetts, United States
• Washington University — St Louis, Missouri, United States
• St. Vincent's Hospital — New York, New York, United States
• New York Medical College — Valhalla, New York, United States
• University of North Carolina — Chapel Hill, North Carolina, United States
• Rainbow Babies & Children's Hospital — Cleveland, Ohio, United States
• Childrens Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
• Hôpital Erasme — Brussels, Belgium
• Hôpital Universitaire des Enfants Reine Fabiola — Brussels, Belgium
• University Hospital Brussels — Brussels, Belgium
• University Hospital Leuven — Leuven, Belgium
• University of Toronto — Toronto, Canada
• Hôpital Cochin — Paris, France
• Hôpital Necker - Enfants Malades — Paris, France
• Hôpital des Enfants — Toulouse, France
• Klinikum der Universität Köln — Cologne, Germany
• Hadassah University Hospital - Mount Scopus — Jerusalem, Israel

Full record on ClinicalTrials.gov

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