Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: Ivacaftor
Intervention MeSH terms: Ivacaftor
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 33.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No
Keywords: Cystic Fibrosis

Summary:

      The purpose of this study is to provide information regarding the long-term safety and
      pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of
      age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will
      further explore the efficacy of long-term ivacaftor treatment in this population of patients
      with CF.
    

Eligibility criteria:

        Inclusion Criteria (Ivacaftor Arm):

          1. Completed the last study visit of the treatment period of the previous study
             (NCT01705145)

          2. Hematology, serum chemistry, and vital signs results on Day 1 with no clinically
             significant abnormalities that would interfere with the study assessments, as judged
             by the investigator

          3. As judged by the investigator, parent or legal guardian must be able to understand
             protocol requirements, restrictions, and instructions and the parent or legal guardian
             should be able to ensure that the subject assents to participation in the study to the
             degree the subject can assent, and that the subject will comply with and is likely to
             complete the study as planned

        Inclusion Criteria (Observational Arm):

        1. Subjects who completed their assigned study drug treatment in the previous study
        (NCT01705145) and elected not to enroll in the ivacaftor arm and subjects who prematurely
        discontinued treatment in the previous study and received at least 1 dose of study drug
        treatment in the previous study will be eligible for enrollment in the observational arm.

        Exclusion Criteria (Ivacaftor Arm):

          1. Subjects who prematurely discontinued from the previous study

          2. 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 subject

          3. Subjects with a history of study treatment intolerance as observed in their previous
             study that, in the opinion of the investigator, might pose an additional risk in
             administering study drug to the subject

          4. Subjects receiving commercially-available ivacaftor treatment

          5. Subject was unable to complete an adequate slit-lamp examination at the last
             ophthalmologic examination in the previous study

        Exclusion Criteria: (Observational Arm)

        1. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the
        observational arm.