Study of Ataluren (PTC124) in Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ataluren
Intervention description: Ataluren will be administered per dose and schedule specified in the arm.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 61.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No
Keywords: Cystic fibrosis; Nonsense mutation; Premature stop codon; PTC124; Ataluren

Summary:

      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).
    

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.