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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
248

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2017

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 1, 2017

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 12, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 12, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 18, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 12, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 8, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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