A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-02-29

Study Completion Date

2017-06-30

Brief Summary

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To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

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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

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Placebo

Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.

Group Type PLACEBO_COMPARATOR

Tezacaftor/Ivacaftor matching placebo

Intervention Type DRUG

Ivacaftor matching placebo

Intervention Type DRUG

TEZ/IVA

Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.

Group Type EXPERIMENTAL

Tezacaftor/Ivacaftor

Intervention Type DRUG

Tezacaftor/Ivacaftor FDC

Ivacaftor

Intervention Type DRUG

Interventions

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Tezacaftor/Ivacaftor

Tezacaftor/Ivacaftor FDC

Intervention Type DRUG

Ivacaftor

Intervention Type DRUG

Tezacaftor/Ivacaftor matching placebo

Intervention Type DRUG

Ivacaftor matching placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Male and female participants, homozygous for the F508del CFTR mutation
* Confirmed diagnosis of CF by sweat chloride testing
* Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
* Stable CF disease as judged by the investigator.

Exclusion Criteria

* History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
* History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
* History of solid organ or hematological transplantation
* Pregnant or nursing females
* Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
* In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No