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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

39 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-07-31

Study Completion Date

2013-10-31

Brief Summary

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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).

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Detailed Description

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Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical benefit in subjects with CF. Results from Phase 3 studies (VX08-770-102 \[Study 102\] \[NCT00909532\] and VX08-770-103 \[Study 103\] \[NCT00909727\]) showed that ivacaftor is effective in the treatment of subjects with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR channel function (measured by reduction in sweat chloride concentration) and corresponding substantial, durable improvements in lung function, pulmonary exacerbations, respiratory symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and reasons for premature discontinuation and results of safety assessments.

Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States for the treatment of CF in subjects 6 years of age and older who have a G551D mutation in the CFTR gene.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Part 1: Ivacaftor First, Then Placebo

Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 mg tablet, oral use, administered twice a day (q12h)

Placebo

Intervention Type DRUG

oral use, administered twice a day (q12h)

Part 1: Placebo First, Then Ivacaftor

Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 mg tablet, oral use, administered twice a day (q12h)

Placebo

Intervention Type DRUG

oral use, administered twice a day (q12h)

Part 2: Ivacaftor

Ivacaftor 150 mg tablet orally twice daily for 16 weeks.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 mg tablet, oral use, administered twice a day (q12h)

Interventions

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Ivacaftor

150 mg tablet, oral use, administered twice a day (q12h)

Intervention Type DRUG

Placebo

oral use, administered twice a day (q12h)

Intervention Type DRUG

Other Intervention Names

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Kalydeco VX-770

Eligibility Criteria

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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

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No