Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ataluren
Intervention description: Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Masking description: This extension study was not blinded. The investigators and participants remained blinded to the Study 009 treatment assignments throughout participation in Study PTC124-GD-009e-CF.
Enrollment: 191.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No

Summary:

      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.
    

Eligibility criteria:

        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.