Study of Ataluren (PTC124) in Cystic Fibrosis

An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)

The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).

Key Inclusion Criteria:

• Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
• Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
• Body weight greater than or equal to (≥) 16 kilograms (kg).
• Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
• Confirmed laboratory values within the central laboratory ranges at screening.
• In male and female participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period.
• Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, laboratory tests, and study restrictions.

Key Exclusion Criteria:

• Chronic use of systemic tobramycin within 4 weeks prior to screening.
• Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
• Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
• Known hypersensitivity to any of the ingredients or excipients of the study drug.
• Exposure to another investigational drug within 4 weeks prior to screening.
• Treatment with intravenous antibiotics within 3 weeks prior to screening.
• History of solid organ or hematological transplantation.
• Ongoing immunosuppressive therapy (other than corticosteroids).
• Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
• Known portal hypertension.
• Pregnancy or breast-feeding.