Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Lumacaftor; Ivacaftor; Lumacaftor Placebo; Ivacaftor Placebo
Intervention description: Tablet; Tablet.; Matching placebo tablet.; Matching placebo tablet.
Intervention MeSH terms: Ivacaftor
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Factorial Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 312.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No

Summary:

      The purpose of this study is to evaluate of the safety, efficacy, pharmacokinetics (PK) and
      pharmacodynamic (PD) effects of lumacaftor (VX-809) alone and when coadministered with
      ivacaftor (VX-770) in participants with cystic fibrosis, homozygous or heterozygous for the
      F508del-CFTR mutation.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female participants with confirmed diagnosis of CF

          -  Must have the F508del-CFTR mutation on at least 1 allele.

          -  FEV1 greater than equal (>=) 40% of predicted normal for age, gender, and height
             (Knudson standards)(Cohort 1, 2, and 3); FEV1 40-90% of predicted normal for age,
             gender, and height (Hankinson standards (Cohort 4)

          -  Participant of child-bearing potential and who are sexually active must meet the
             contraception requirements

        Exclusion Criteria:

          -  History of any illness or condition that, in the opinion of the investigator might
             confound the results of the study or pose an additional risk in administering study
             drug to the participant (e.g., cirrhosis with portal hypertension).

          -  An acute illness including acute upper or lower respiratory infection, pulmonary
             exacerbation or changes in therapy (including antibiotics) for pulmonary disease
             within 14 days (Cohort 1, 2, and 3) or 28 days (Cohort 4) before receiving the first
             dose of study drug.

          -  History of solid organ or hematological transplantation.

          -  History of alcohol abuse or drug addiction in the past year, including cannabis,
             cocaine, and opiates.

          -  Ongoing participation in another therapeutic clinical study, or prior participation in
             an investigational drug study without appropriate washout

          -  Pregnant or nursing females; females of childbearing potential who are unwilling or
             unable to use an acceptable method of non-hormonal contraception

          -  Participants enrolled in Cohort 1 or Cohort 2 will not be eligible for Cohort 3

          -  Ongoing or prior participation in an investigational drug study within 30 days of the
             Screening Visit

          -  Heterozygous participants who participated in Cohort 2 and meet the eligibility
             criteria for Cohort 4 may participate in Cohort 4

          -  Evidence of lens opacity or cataract as determined by the ophthalmologic examination
             (Cohort 4 only)