Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ivacaftor; Placebo
Intervention description: Tablet; Tablet
Intervention MeSH terms: Ivacaftor
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 140.0
Sex: All
Minimum age: 12 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 safety and efficacy of ivacaftor in
      participants with cystic fibrosis (CF) who were aged 12 years or older and were homozygous
      for the F508del-CF transmembrane conductance regulator (CFTR) mutation. Ivacaftor is a potent
      and selective CFTR 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:

          -  Confirmed diagnosis of cystic fibrosis (CF) and homozygous for F508del-CFTR mutation

          -  Forced expiratory volume in 1 second (FEV1) of at least 40% of predicted normal for
             age, gender, and height

          -  Willing to use at least 2 highly effective birth control methods during the study

          -  No clinically significant abnormalities that would have interfered with the study
             assessments, as judged by the investigator

          -  Able to understand and comply with protocol requirements, restrictions, and
             instructions and likely to complete the study as planned, 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

          -  History of alcohol, medication or illicit drug abuse within one year prior to Day 1

          -  Abnormal liver function >=3 x the upper limit of normal

          -  Abnormal renal function at Screening

          -  History of solid organ or hematological transplantation

          -  Pregnant or breast-feeding (for women)

          -  Ongoing participation in another therapeutic clinical study or prior participation in
             an investigational drug study within 30 days prior to screening

          -  Previous participation in a VX-809 study

          -  Used inhaled hypertonic saline treatment

          -  Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP3A4)