A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor

NCT ID: NCT02412111

Last Updated: 2019-01-08

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

156 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-06-30

Study Completion Date

2017-09-30

Brief Summary

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This is a Phase 3, randomized, double-blind, ivacaftor-controlled, parallel-group, multicenter study of tezacaftor in combination with ivacaftor in subjects aged 12 years and older with CF who are heterozygous for the F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive.

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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Ivacaftor (Run-in Period)

Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

VX-661 + Ivacaftor (Active comparator period)

VX-661 100 mg and ivacaftor 150 mg fixed-dose combination tablet orally once daily in the morning and ivacaftor 150 mg tablet orally once daily in the evening for 8 weeks.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

Tezacaftor/Ivacaftor

Intervention Type DRUG

Ivacaftor monotherapy (Active comparator period)

Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.

Group Type ACTIVE_COMPARATOR

Ivacaftor

Intervention Type DRUG

Interventions

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Ivacaftor

Intervention Type DRUG

Tezacaftor/Ivacaftor

Intervention Type DRUG

Other Intervention Names

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VX-770 VX-661/VX-770

Eligibility Criteria

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Inclusion Criteria

* Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
* FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height during screening
* 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.
* Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
* Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Birmingham, Alabama, United States

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Oakland, California, United States

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San Diego, California, United States

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Aurora, Colorado, United States

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Washington D.C., District of Columbia, United States

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Orlando, Florida, United States

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Augusta, Georgia, United States

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Indianapolis, Indiana, United States

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Lexington, Kentucky, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Grand Rapids, Michigan, United States

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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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Lebanon, New Hampshire, United States

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Manchester, New Hampshire, United States

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Morristown, New Jersey, United States

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New Brunswick, New Jersey, United States

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Albany, New York, United States

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New York, New York, United States

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Cincinnati, Ohio, United States

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Toledo, Ohio, United States

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Oklahoma City, Oklahoma, United States

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Portland, Oregon, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Charleston, South Carolina, United States

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Knoxville, Tennessee, United States

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Nashville, Tennessee, United States

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Houston, Texas, United States

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Salt Lake City, Utah, United States

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Morgantown, West Virginia, United States

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Chermside, , Australia

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Clayton, , Australia

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Melbourne, , Australia

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South Brisbane, , Australia

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Westmead, , Australia

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Innsbruck, , Austria

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Brussels, , Belgium

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Ghent, , Belgium

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Leuven, , Belgium

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Calgary, , Canada

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Toronto, , Canada

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Vancouver, , Canada

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Paris, , France

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Dresden, , Germany

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Erlangen, , Germany

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Essen, , Germany

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Frankfurt, , Germany

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Giessen, , Germany

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Heidelberg, , Germany

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Jena, , Germany

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München, , Germany

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Cork, , Ireland

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Dublin, , Ireland

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Bari, , Italy

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Milan, , Italy

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Roma, , Italy

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Belfast, , United Kingdom

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Birmingham, , United Kingdom

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Cardiff, , United Kingdom

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Glasgow, , United Kingdom

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Leeds, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Newcastle, , United Kingdom

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Southampton, , United Kingdom

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Countries

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United States Australia Austria Belgium Canada France Germany Ireland Italy United Kingdom

References

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McKone EF, DiMango EA, Sutharsan S, Barto TL, Campbell D, Ahluwalia N, Higgins M, Owen CA, Tullis E. A phase 3, randomized, double-blind, parallel-group study to evaluate tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for F508del-CFTR and a gating mutation. J Cyst Fibros. 2021 Mar;20(2):234-242. doi: 10.1016/j.jcf.2020.11.003. Epub 2020 Dec 16.

Reference Type DERIVED
PMID: 33339768 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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VX14-661-109

Identifier Type: -

Identifier Source: org_study_id

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