Study of Ataluren (PTC124) in Cystic Fibrosis

Launched by PTC THERAPEUTICS · Apr 7, 2014

Keywords

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Gender

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Eligibility criteria

• 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.