Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ivacaftor; Placebo
Intervention description: 150-mg tablet given orally q12h for up to 48 weeks; Tablet given orally q12h for up to 48 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: 52.0
Sex: All
Minimum age: 6 Years
Maximum age: 11 Years
Healthy volunteers: No
Keywords: Fibrosis; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Pathologic Processes

Summary:

      The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects
      with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis
      transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective
      potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators
      are pharmacological agents that increase the chloride ion transport properties of the channel
      in the presence of cyclic adenosine monophosphate (AMP)-dependent protein kinase A (PKA)
      activation.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Weighing at least 15 kg

          -  Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele

          -  Forced expiratory volume in 1 second (FEV1) of 40% to 105% (inclusive) of predicted
             normal for age, gender, and height (Knudson standards) at Screening

          -  Able to swallow tablets

          -  As judged by the investigator, parent or legal guardian and subject must have been
             able to understand protocol requirements, restrictions, and instructions, and the
             parent or legal guardian should have been able to ensure that the subject complied
             with, and was likely to complete, the study as planned

          -  Parent or legal guardian must have signed the informed consent form and corresponding
             assent must be obtained from the subject

          -  Willing to use at least 1 highly effective birth control method during the study

          -  No clinically significant abnormalities that would have interfered with the study
             assessments, as judged by the investigator

        Exclusion Criteria:

          -  History of any illness or condition that might confound the results of the study or
             pose an additional risk in administering study drug to the subject

          -  Acute respiratory infection, pulmonary exacerbation, or changes in therapy for
             pulmonary disease within 4 weeks of Day 1 of the study

          -  Abnormal liver function ≥ 3x the upper limit of normal

          -  Abnormal renal function at Screening

          -  History of solid organ or hematological transplantation

          -  Ongoing participation in another therapeutic clinical study or prior participation in
             an investigational drug study within 30 days prior to Screening

          -  Use of inhaled hypertonic saline treatment

          -  Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)