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

January 7, 2019 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

156

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Chermside, Australia
      • Clayton, Australia
      • Melbourne, Australia
      • South Brisbane, Australia
      • Westmead, Australia
  • Austria
      • Innsbruck, Austria
  • Belgium
      • Bruxelles, Belgium
      • Gent, Belgium
      • Leuven, Belgium
  • Canada
      • Calgary, Canada
      • Toronto, Canada
      • Vancouver, Canada
  • France
      • Paris Cedex 14, France
  • Germany
      • Dresden, Germany
      • Erlangen, Germany
      • Essen, Germany
      • Frankfurt, Germany
      • Giessen, Germany
      • Heidelberg, Germany
      • Jena, Germany
      • Munchen, Germany
  • Ireland
      • Cork, Ireland
      • Dublin, Ireland
  • Italy
      • Bari, Italy
      • Milano, Italy
      • Roma, Italy
  • United Kingdom
      • Belfast, United Kingdom
      • Birmingham, United Kingdom
      • Cardiff, United Kingdom
      • Glasgow, United Kingdom
      • Leeds, United Kingdom
      • London, United Kingdom
      • Manchester, United Kingdom
      • Newcastle, United Kingdom
      • Southampton, United Kingdom
  • United States
    • Alabama
      • Birmingham, Alabama, United States
    • California
      • Oakland, California, United States
      • San Diego, California, United States
    • Colorado
      • Aurora, Colorado, United States
    • District of Columbia
      • Washington, District of Columbia, United States
    • Florida
      • Orlando, Florida, United States
    • Georgia
      • Augusta, Georgia, United States
    • Indiana
      • Indianapolis, Indiana, United States
    • Kentucky
      • Lexington, Kentucky, United States
    • Massachusetts
      • Boston, Massachusetts, United States
    • Michigan
      • Ann Arbor, Michigan, United States
      • Detroit, Michigan, United States
      • Grand Rapids, Michigan, United States
    • Missouri
      • Saint Louis, Missouri, United States
    • Nebraska
      • Omaha, Nebraska, United States
    • New Hampshire
      • Lebanon, New Hampshire, United States
      • Manchester, New Hampshire, United States
    • New Jersey
      • Morristown, New Jersey, United States
      • New Brunswick, New Jersey, United States
    • New York
      • Albany, New York, United States
      • New York, New York, United States
    • Ohio
      • Cincinnati, Ohio, United States
      • Toledo, Ohio, United States
    • Oklahoma
      • Oklahoma City, Oklahoma, United States
    • Oregon
      • Portland, Oregon, United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
      • Pittsburgh, Pennsylvania, United States
    • South Carolina
      • Charleston, South Carolina, United States
    • Tennessee
      • Knoxville, Tennessee, United States
      • Nashville, Tennessee, United States
    • Texas
      • Houston, Texas, United States
    • Utah
      • Salt Lake City, Utah, United States
    • West Virginia
      • Morgantown, West Virginia, United States

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Ivacaftor (Run-in Period)
Ivacaftor 150 milligram (mg) tablet orally every 12 hours for 4 weeks.
Drug: Ivacaftor
Other Names:
  • VX-770
Experimental: 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.
Drug: Ivacaftor
Other Names:
  • VX-770
Drug: Tezacaftor/Ivacaftor
Other Names:
  • VX-661/VX-770
Active Comparator: Ivacaftor monotherapy (Active comparator period)
Ivacaftor 150 mg tablet orally every 12 hours as monotherapy for 8 weeks.
Drug: Ivacaftor
Other Names:
  • VX-770

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
Time Frame: Baseline, Through Week 8
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Through Week 8

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8
Time Frame: Baseline, Through Week 8
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Through Week 8
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score From Baseline Through Week 8
Time Frame: Baseline, Through Week 8
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Baseline, Through Week 8
Absolute Change From Baseline in Sweat Chloride Through Week 8
Time Frame: Baseline, Through Week 8
Sweat samples were collected using an approved collection device.
Baseline, Through Week 8
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline up to Week 16
Baseline up to Week 16
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1-VX-661), Ivacaftor (IVA) and IVA Metabolite (M1-IVA)
Time Frame: Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period
Predose on Week -2 for Run-in period; Pre-dose on Week 2 for Active comparator period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 1, 2015

Primary Completion (Actual)

September 1, 2017

Study Completion (Actual)

September 1, 2017

Study Registration Dates

First Submitted

April 3, 2015

First Submitted That Met QC Criteria

April 3, 2015

First Posted (Estimate)

April 8, 2015

Study Record Updates

Last Update Posted (Actual)

January 8, 2019

Last Update Submitted That Met QC Criteria

January 7, 2019

Last Verified

January 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX14-661-109

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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