A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation

May 8, 2018 updated by: Vertex Pharmaceuticals Incorporated

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy and Safety of Ivacaftor and 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 Allele With a CFTR Mutation Predicted to Have Residual Function

The purpose of this study is to evaluate the efficacy and safety of VX-661 in combination with ivacaftor (IVA, VX-770) and IVA monotherapy in participants with Cystic Fibrosis (CF) who are heterozygous for F508del-CFTR allele and a second allele with a CFTR mutation predicted to have residual function.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

248

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
      • Adelaide, Australia
      • Melbourne, Australia
      • South Brisbane, Australia
      • Westmead, Australia
  • Belgium
      • Gent, Belgium
  • Canada
      • Montreal, Canada
      • Quebec City, Canada
      • Toronto, Canada
      • Vancouver, Canada
  • France
      • Bordeaux Cedex, France
    • Bouches-du-Rhone
      • Marseille cedex 20, Bouches-du-Rhone, France
    • Herault
      • Montpellier cedex 5, Herault, France
    • Ille Et Vilaine
      • Rennes cedex 09, Ille Et Vilaine, France
    • Nord
      • Lille cedex, Nord, France
    • Paris
      • Paris cedex 14, Paris, France
      • Paris cedex 15, Paris, France
    • Rhone
      • Bron, Rhone, France
  • Germany
      • Berlin, Germany
    • Bayern
      • Muenchen, Bayern, Germany
      • Munchen, Bayern, Germany
    • Niedersachsen
      • Hannover, Niedersachsen, Germany
    • Nordrhein Westfalen
      • Essen, Nordrhein Westfalen, Germany
    • Thueringen
      • Jena, Thueringen, Germany
  • Israel
      • Haifa, Israel
      • Jerusalem, Israel
      • Petach-Tikva, Israel
      • Ramat-Gan, Israel
  • Italy
      • Ancona, Italy
      • Milano, Italy
      • Orbassano, Italy
      • Potenza, Italy
      • Roma, Italy
      • Verona, Italy
  • Netherlands
      • Amsterdam, Netherlands
      • Den Haag, Netherlands
      • Rotterdam, Netherlands
      • Utrecht, Netherlands
  • Switzerland
      • Bern, Switzerland
      • St Gallen, Switzerland
      • Zuerich, Switzerland
  • United Kingdom
    • Devon
      • Exeter, Devon, United Kingdom
    • Greater London
      • London, Greater London, United Kingdom
    • Greater Manchester
      • Manchester, Greater Manchester, United Kingdom
    • Hampshire
      • Southampton, Hampshire, United Kingdom
    • Lancashire
      • Liverpool, Lancashire, United Kingdom
    • Strathclyde
      • Glasgow, Strathclyde, United Kingdom
    • West Yorkshire
      • Leeds, West Yorkshire, United Kingdom
  • United States
    • Alabama
      • Birmingham, Alabama, United States
    • Arizona
      • Phoenix, Arizona, United States
      • Tucson, Arizona, United States
    • California
      • Long Beach, California, United States
      • Oakland, California, United States
      • Palo Alto, California, United States
      • Sacramento, California, United States
    • Colorado
      • Aurora, Colorado, United States
      • Denver, Colorado, United States
    • Florida
      • Gainesville, Florida, United States
      • Miami, Florida, United States
      • Orlando, Florida, United States
      • Pensacola, Florida, United States
      • Tampa, Florida, United States
    • Georgia
      • Atlanta, Georgia, United States
    • Illinois
      • Chicago, Illinois, United States
      • Park Ridge, Illinois, United States
    • Iowa
      • Iowa City, Iowa, United States
    • Maryland
      • Baltimore, Maryland, United States
    • Massachusetts
      • Boston, Massachusetts, United States
    • Michigan
      • Ann Arbor, Michigan, United States
      • Grand Rapids, Michigan, United States
    • Minnesota
      • Minneapolis, Minnesota, United States
    • Missouri
      • Saint Louis, Missouri, United States
    • New Hampshire
      • Lebanon, New Hampshire, United States
    • New York
      • New York, New York, United States
      • Syracuse, New York, United States
      • Valhalla, New York, United States
    • North Carolina
      • Chapel Hill, North Carolina, United States
    • Ohio
      • Akron, Ohio, United States
      • Cincinnati, Ohio, United States
      • Cleveland, Ohio, United States
      • Toledo, Ohio, United States
    • Oregon
      • Portland, Oregon, United States
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States
      • Pittsburgh, Pennsylvania, United States
    • South Carolina
      • Charleston, South Carolina, United States
    • South Dakota
      • Sioux Falls, South Dakota, United States
    • Tennessee
      • Memphis, Tennessee, United States
    • Texas
      • Austin, Texas, United States
      • Dallas, Texas, United States
      • Tyler, Texas, United States
    • Utah
      • Salt Lake City, Utah, United States
    • Virginia
      • Norfolk, Virginia, United States
    • Washington
      • Seattle, Washington, United States
      • Spokane, Washington, United States
    • Wisconsin
      • Milwaukee, Wisconsin, 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

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

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Heterozygous for F508del-CFTR and a second allele with a CFTR mutation predicted to have residual function
  • Forced Expiratory Volume in 1 Second (FEV1) greater than or equal to (≥) 40 percent (%) and less than or equal to (≤) 90% of predicted normal for age, sex, and height during screening
  • Sweat chloride value ≥60 millimole per liter (mmol/L) during screening OR as documented in the participant's medical record
  • 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 participant
  • An acute upper or lower respiratory infection, pulmonary exacerbation
  • History of solid organ or hematological transplantation
  • Ongoing or prior participation in an investigational drug study (including studies investigating VX-661, lumacaftor [VX-809], and/or ivacaftor) within 30 days of screening
  • Pregnant and nursing females
  • Sexually active participants 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: Crossover Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo matched to Ivacaftor
Tablet, oral use
Drug: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use
Experimental: VX-661/Ivacaftor combination
Drug: VX-661/Ivacaftor
Fixed dose combination tablet, oral use
Other Names:
  • VX-661+VX-770
Drug: Ivacaftor
Tablet, oral use
Other Names:
  • IVA, VX-770
Drug: Placebo matched to Ivacaftor
Tablet, oral use
Experimental: Ivacaftor monotherapy
Drug: Ivacaftor
Tablet, oral use
Other Names:
  • IVA, VX-770
Drug: Placebo matched to VX-661/ ivacaftor
Fixed dose combination tablet, oral use

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Average of Week 4 and Week 8
Time Frame: Baseline, Week 4 and Week 8 of each treatment period
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Week 4 and Week 8 of each treatment period

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change From Study Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Average of Week 4 and Week 8
Time Frame: Baseline, Week 4 and Week 8 of each treatment period
The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
Baseline, Week 4 and Week 8 of each treatment period
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 28
Day 1 up to Week 28
Relative Change From Study Baseline in ppFEV1 at Average of Week 4 and Week 8
Time Frame: Baseline, Week 4 and Week 8 of each treatment period
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Baseline, Week 4 and Week 8 of each treatment period
Absolute Change From Study Baseline in Sweat Chloride at Average of Week 4 and Week 8
Time Frame: Baseline, Week 4 and Week 8 of each treatment period
Baseline, Week 4 and Week 8 of each treatment period
Trough Plasma Concentrations (Ctrough) of VX-661, VX-661 Metabolites (M1 VX-661), IVA and IVA Metabolite (M1 IVA) After Administration of VX-661/IVA Combination Therapy
Time Frame: Pre-morning dose on Week 8 of each treatment period
Pre-morning dose on Week 8 of each treatment period
Ctrough of IVA and IVA Metabolite (M1 IVA) After Administration of IVA Monotherapy
Time Frame: Pre-morning dose on Week 8 of each treatment period
Pre-morning dose on Week 8 of each treatment 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

March 1, 2015

Primary Completion (Actual)

February 1, 2017

Study Completion (Actual)

February 1, 2017

Study Registration Dates

First Submitted

March 12, 2015

First Submitted That Met QC Criteria

March 12, 2015

First Posted (Estimate)

March 18, 2015

Study Record Updates

Last Update Posted (Actual)

June 12, 2018

Last Update Submitted That Met QC Criteria

May 8, 2018

Last Verified

May 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX14-661-108
  • 2014-004788-18 (EudraCT Number)

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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