A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: LUM/IVA
Intervention description: Participants received LUM/IVA every q12h.
Intervention MeSH terms: Ivacaftor
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 57.0
Sex: All
Minimum age: 2 Years
Healthy volunteers: No

Summary:

      A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With
      Cystic Fibrosis, Homozygous for F508del.
    

Eligibility criteria:

        Inclusion Criteria:

        Subjects entering the Treatment Cohort must meet the following criteria:

          -  Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study
             VX15-809-115 Part B (Study 115B, NCT02797132)

          -  Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit

        Subjects entering the Observational Cohort must meet 1 of the following criteria:

          -  Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B,
             but do not want to enroll in the Treatment Cohort.

          -  Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and
             the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at
             the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug
             interruption and either did not receive Vertex approval to enroll in the Treatment
             Cohort or do not want to enroll in the Treatment Cohort.

          -  Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of
             treatment and remained in the study from the time of treatment discontinuation through
             the Week 24 Visit and Safety Follow-up Visit, if required.

        Exclusion Criteria (Treatment Cohort Only):

          -  Prematurely discontinued LUM/IVA treatment in Study 115B.

          -  History of any comorbidity or laboratory abnormality that, in the opinion of the
             investigator, might confound the results of the study or pose an additional risk in
             administering LUM/IVA to the subject

          -  History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that
             would pose an additional risk to the subject in the opinion of investigator, and which
             should be discussed with the Vertex medical monitor.

          -  Subjects with a history of allergy or hypersensitivity to LUM/IVA.

          -  Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment
             interruption at the completion of Study 115B, for which no convincing alternative
             etiology is identified.

          -  QTc value at the completion of Study 115B that would pose an additional risk to the
             subject in the opinion of investigator, and which should be discussed with the Vertex
             medical monitor

          -  History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by
             the investigator.

          -  Participation in an investigational drug trial (including studies investigating LUM
             and/or IVA) other than Study 115B.