Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)

• 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 the R117H-CFTR mutation.

• 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

• Must have at least 1 allele of the R117H CFTR mutation

• Percent predicted forced expiratory volume in 1 second (FEV1) 40 percent (%) to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years) 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:

• CFTR gene mutation leading to CFTR channel with gating defect (that is, any 1 of the following mutations: G551D, G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D)

• 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

• Abnormal liver function, at screening, defined as greater than or equal to (>=) 3 time upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), total bilirubin

• Colonization with organisms associated with a more rapid decline in pulmonary status (for example, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening

• 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

• Any "non-CF-related" illness within 2 weeks before Day 1 (first dose of study drug). "Illness" was defined as an acute (serious or non-serious) condition (for example, gastroenteritis)

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