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This is an extension study evaluating the long-term use of the drug Ataluren (PTC124) in Cystic Fibrosis patients who completed a prior Phase 3 study. It included patients who had completed the previous study. It was a Phase 3 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * 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. Exclusion Criteria: * 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.