A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis Who Have an F508del-CFTR Mutation

• Status: Active, not recruiting
• Trial end date: 2023-03-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744), VX14-661-106 (Study 106, NCT02347657), VX14-661-107 (Study 107, NCT02516410), VX14-661-108 (Study 108, NCT02392234), VX14-661-109 (Study 109, NCT02412111), and VX14-661-111 (Study 111, NCT02508207). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

Part A:

• Subjects entering the Treatment Cohort must meet all of the following criteria:

• Elect to enroll in the Treatment Cohort

• Completed study drug Treatment Period in a parent study (NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111) or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207.

• Willing to remain on a stable CF regimen through the Safety Follow-up Visit.

• Subjects re-enrolling in the Part A Treatment Cohort must meet all of the following criteria:

• Previously received at least 4 weeks of study drug before discontinuing in Part A of Study NCT02565914 to participate in another qualified Vertex study.

• Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part A Study NCT02565914.

• Subjects entering the Part A Observational Cohort must meet the following criteria:

• <18 years of age (age on the date of informed consent/assent in the parent study)

• Completed study drug Treatment Period in a parent study or study drug treatment and the Safety Follow up Visit for subjects from NCT02508207, but do not elect to enroll in the NCT02565914 Treatment Cohort; or

• Received at least 4 weeks of study drug treatment and completed visits up to the last scheduled visit of the Treatment Period of a parent study (and the Safety Follow up Visit for subjects from NCT02508207), but do not meet eligibility criteria for enrollment into the Treatment Cohort

Part B:

Subjects who meet all of the following inclusion criteria will be eligible for Part B.

• Did not withdraw consent from the parent study or Part A of Study NCT02565914.

• Completed study drug treatment during the Treatment Period in Part A of - Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Study NCT02565914.

Subjects re enrolling in Part B must meet all of the following criteria:

• Previously received at least 4 weeks of study drug before discontinuing Study NCT02565914 to participate in another qualified Vertex study, which is defined as a Vertex study of investigational CFTR modulators that allows participation of subjects in Study NCT02565914.

• Completed the last required visit of another qualified Vertex study before or during the Returning Visit in Part B.

• Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit in Part B.

Part C:

• Subjects who meet all of the following inclusion criteria will be eligible for Part C.

• Did not withdraw consent from Part B of Study NCT02565914.

• Completed study drug treatment during Part B of NCT02565914.

• Willing to remain on a stable CF medication (and supplement) regimen through the 96 week visit of Part C.

Exclusion Criteria:

• History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk to the subject.

• Pregnant and nursing females.

• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.

• History of drug intolerance in the parent study that would pose an additional risk to the subject.

• Participation in an investigational drug trial (including studies investigating VX-661/ivacaftor or lumacaftor/ivacaftor) other than the parent studies of NCT02565914 or other eligible Vertex studies investigating VX-661 in combination with ivacaftor, or use of a commercially available CFTR modulator.

Other protocol defined Inclusion/Exclusion criteria may apply.