Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

• Status: Completed
• Trial end date: 2013-10-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D cystic fibrosis transmembrane regulator (CFTR) gating mutation (any one of the following CFTR mutations: G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D).

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

Details

• Lead Sponsor: Vertex Pharmaceuticals Incorporated
• Collaborators: Cystic Fibrosis Foundation; Cystic Fibrosis Foundation Therapeutics
• Treatments: Ivacaftor

Criteria

Inclusion Criteria:

• Male or female with confirmed diagnosis of CF

• At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D

• Percent predicted forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent (%) predicted normal for age, sex, and height

• 6 years of age or older

• Minimum weight of 15 kilogram (kg) at screening

• Females of childbearing potential must not be pregnant

• Willing to comply with contraception requirements

Exclusion Criteria:

• G551D-CFTR mutation on at least 1 allele

• History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject

• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug

• History of solid organ or hematological transplantation

• History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug

• Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening

• Use of inhaled hypertonic saline treatment

• Use of any inhibitors or inducers of cytochrome (CYP) P450 3A

• Evidence of cataract or lens opacity at screening