Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ivacaftor; Placebo
Intervention description: Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.; Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.
Intervention MeSH terms: Ivacaftor
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 70.0
Sex: All
Minimum age: 6 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects
      with cystic fibrosis (CF) who have the R117H-CFTR mutation.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female with confirmed diagnosis of CF

          -  Must have at least 1 allele of the R117H CFTR mutation

          -  Percent predicted forced expiratory volume in 1 second (FEV1) 40 percent (%) to 90%
             (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years)
             predicted normal for age, sex, and height

          -  6 years of age or older

          -  Minimum weight of 15 kilogram (kg) at screening

          -  Females of childbearing potential must not be pregnant

          -  Willing to comply with contraception requirements

        Exclusion Criteria:

          -  CFTR gene mutation leading to CFTR channel with gating defect (that is, any 1 of the
             following mutations: G551D, G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P,
             or G1349D)

          -  History of any illness or condition that might confound the results of the study or
             pose an additional risk in administering ivacaftor to the subject

          -  An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
             therapy (including antibiotics) for pulmonary disease within 4 weeks before the first
             dose of study drug

          -  Abnormal liver function, at screening, defined as greater than or equal to (>=) 3 time
             upper limit of normal (ULN), of any 3 or more of the following: serum aspartate
             transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase
             (GGT), serum alkaline phosphatase (ALP), total bilirubin

          -  Colonization with organisms associated with a more rapid decline in pulmonary status
             (for example, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium
             abscessus) at screening

          -  History of solid organ or hematological transplantation

          -  History of alcohol, medication or illicit drug abuse within 1 year before the first
             dose of study drug

          -  Ongoing participation in another therapeutic clinical study or prior participation in
             an investigational drug study within 30 days before screening

          -  Any "non-CF-related" illness within 2 weeks before Day 1 (first dose of study drug).
             "Illness" was defined as an acute (serious or non-serious) condition (for example,
             gastroenteritis)

          -  Use of any inhibitors or inducers of cytochrome (CYP) P450 3A