A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

14 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-05-31

Study Completion Date

2017-08-31

Brief Summary

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To evaluate the efficacy of ivacaftor treatment, as measured by lung clearance index (LCI), in subjects with cystic fibrosis (CF) who have a specified CF transmembrane conductance regulator (CFTR) gating mutation

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Detailed Description

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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

DOUBLE

Participants Investigators

Study Groups

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Part 1-Sequence 1

ivacaftor in Treatment Period 1 →washout→placebo in Treatment Period 2

Group Type EXPERIMENTAL

ivacaftor

Intervention Type DRUG

Placebo

Intervention Type DRUG

Part 1 - Sequence 2

placebo in Treatment Period 1→washout→ivacaftor in Treatment Period 2

Group Type EXPERIMENTAL

ivacaftor

Intervention Type DRUG

Placebo

Intervention Type DRUG

Part 2: ivacaftor

open label period

Group Type EXPERIMENTAL

ivacaftor

Intervention Type DRUG

Interventions

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ivacaftor

Intervention Type DRUG

Placebo

Intervention Type DRUG

Other Intervention Names

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VX-770

Eligibility Criteria

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Inclusion Criteria

* Male or female with confirmed diagnosis of CF.
* Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
* Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator.

Exclusion Criteria

* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
* Abnormal liver function, at Screening, defined as ≥3 × 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), and total bilirubin
* History of solid organ or hematological transplantation
* Any clinically significant "non-CF-related" illness within 2 weeks before Day 1
* Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
* Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening

Minimum Eligible Age

3 Years

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No