a study to evaluate the safety and efficacy of long term treatment with vx-661 in combination with ivacaftor in participants with cystic fibrosis who have an f508del-cftr mutation

Conditions: Cystic Fibrosis

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), vx14-661-111 (study 111, nct02508207), vx15-661-112 (nct02730208), and vx16-661-114 (nct03150719). the study is designed to evaluate the safety and efficacy of long-term treatment of vx-661 in combination with ivacaftor.


Eligibility criteria:
inclusion criteria: part a: * participants 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 participants from nct02508207. * willing to remain on a stable cf regimen through the safety follow-up visit. * participants 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. * participants 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 participants from nct02508207), but do not meet eligibility criteria for enrollment into the treatment cohort part b: participants 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. participants 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 participants 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: * participants 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.