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

Completed · Phase 3 · Has a placebo group

Conditions studied: Cystic Fibrosis

In brief

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).

Key facts

Study ID

NCT01614470

Run by

Vertex Pharmaceuticals Incorporated

People needed

39

Starts

2012-07-01

Expected to finish

2013-10-01

Last updated by the study team

2014-10-29

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Study site — Tampa, Florida, United States
• Study site — Atlanta, Georgia, United States
• Study site — Chicago, Illinois, United States
• Study site — Boston, Massachusetts, United States
• Study site — Ann Arbor, Michigan, United States
• Study site — Minneapolis, Minnesota, United States
• Study site — St Louis, Missouri, United States
• Study site — Houston, Texas, United States
• Study site — Leuven, Belgium
• Study site — Lyon, France
• Study site — Montpellier, France
• Study site — Paris, France

Full record on ClinicalTrials.gov

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