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

NCT ID: NCT02392234

Last Updated: 2018-06-12

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

248 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-03-31

Study Completion Date

2017-02-28

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

VX-661/Ivacaftor combination

Group Type EXPERIMENTAL

VX-661/Ivacaftor

Intervention Type DRUG

Fixed dose combination tablet, oral use

Ivacaftor

Intervention Type DRUG

Tablet, oral use

Placebo matched to Ivacaftor

Intervention Type DRUG

Tablet, oral use

Ivacaftor monotherapy

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

Tablet, oral use

Placebo matched to VX-661/ ivacaftor

Intervention Type DRUG

Fixed dose combination tablet, oral use

Placebo

Group Type PLACEBO_COMPARATOR

Placebo matched to VX-661/ ivacaftor

Intervention Type DRUG

Fixed dose combination tablet, oral use

Placebo matched to Ivacaftor

Intervention Type DRUG

Tablet, oral use

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

VX-661/Ivacaftor

Fixed dose combination tablet, oral use

Intervention Type DRUG

Ivacaftor

Tablet, oral use

Intervention Type DRUG

Placebo matched to VX-661/ ivacaftor

Fixed dose combination tablet, oral use

Intervention Type DRUG

Placebo matched to Ivacaftor

Tablet, oral use

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-661+VX-770 IVA, VX-770

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Birmingham, Alabama, United States

Site Status

Phoenix, Arizona, United States

Site Status

Tucson, Arizona, United States

Site Status

Long Beach, California, United States

Site Status

Oakland, California, United States

Site Status

Palo Alto, California, United States

Site Status

Sacramento, California, United States

Site Status

Aurora, Colorado, United States

Site Status

Denver, Colorado, United States

Site Status

Gainesville, Florida, United States

Site Status

Miami, Florida, United States

Site Status

Orlando, Florida, United States

Site Status

Pensacola, Florida, United States

Site Status

Tampa, Florida, United States

Site Status

Atlanta, Georgia, United States

Site Status

Chicago, Illinois, United States

Site Status

Park Ridge, Illinois, United States

Site Status

Iowa City, Iowa, United States

Site Status

Baltimore, Maryland, United States

Site Status

Boston, Massachusetts, United States

Site Status

Ann Arbor, Michigan, United States

Site Status

Grand Rapids, Michigan, United States

Site Status

Minneapolis, Minnesota, United States

Site Status

St Louis, Missouri, United States

Site Status

Lebanon, New Hampshire, United States

Site Status

New York, New York, United States

Site Status

Syracuse, New York, United States

Site Status

Valhalla, New York, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Akron, Ohio, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Cleveland, Ohio, United States

Site Status

Toledo, Ohio, United States

Site Status

Portland, Oregon, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Charleston, South Carolina, United States

Site Status

Sioux Falls, South Dakota, United States

Site Status

Memphis, Tennessee, United States

Site Status

Austin, Texas, United States

Site Status

Dallas, Texas, United States

Site Status

Tyler, Texas, United States

Site Status

Salt Lake City, Utah, United States

Site Status

Norfolk, Virginia, United States

Site Status

Seattle, Washington, United States

Site Status

Spokane, Washington, United States

Site Status

Milwaukee, Wisconsin, United States

Site Status

Adelaide, , Australia

Site Status

Melbourne, , Australia

Site Status

South Brisbane, , Australia

Site Status

Westmead, , Australia

Site Status

Ghent, , Belgium

Site Status

Montreal, , Canada

Site Status

Québec, , Canada

Site Status

Toronto, , Canada

Site Status

Vancouver, , Canada

Site Status

Marseille, Bouches-du-Rhone, France

Site Status

Montpellier, Herault, France

Site Status

Rennes, Ille Et Vilaine, France

Site Status

Lille, Nord, France

Site Status

Paris, Paris, France

Site Status

Bron, Rhone, France

Site Status

Bordeaux, , France

Site Status

Munich, Bavaria, Germany

Site Status

München, Bavaria, Germany

Site Status

Hanover, Lower Saxony, Germany

Site Status

Essen, North Rhine-Westphalia, Germany

Site Status

Jena, Thuringia, Germany

Site Status

Berlin, , Germany

Site Status

Haifa, , Israel

Site Status

Jerusalem, , Israel

Site Status

Petah Tikva, , Israel

Site Status

Ramat Gan, , Israel

Site Status

Ancona, , Italy

Site Status

Milan, , Italy

Site Status

Orbassano, , Italy

Site Status

Potenza, , Italy

Site Status

Roma, , Italy

Site Status

Verona, , Italy

Site Status

Amsterdam, , Netherlands

Site Status

Rotterdam, , Netherlands

Site Status

The Hague, , Netherlands

Site Status

Utrecht, , Netherlands

Site Status

Bern, , Switzerland

Site Status

Sankt Gallen, , Switzerland

Site Status

Zurich, , Switzerland

Site Status

Exeter, Devon, United Kingdom

Site Status

London, Greater London, United Kingdom

Site Status

Manchester, Greater Manchester, United Kingdom

Site Status

Southampton, Hampshire, United Kingdom

Site Status

Liverpool, Lancashire, United Kingdom

Site Status

Glasgow, Strathclyde, United Kingdom

Site Status

Leeds, West Yorkshire, United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Australia Belgium Canada France Germany Israel Italy Netherlands Switzerland United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

Reference Type DERIVED
PMID: 37983082 (View on PubMed)

Acaster S, Mukuria C, Rowen D, Brazier JE, Wainwright CE, Quon BS, Duckers J, Quittner AL, Lou Y, Sosnay PR, McGarry LJ. Development of the Cystic Fibrosis Questionnaire-Revised-8 Dimensions: Estimating Utilities From the Cystic Fibrosis Questionnaire-Revised. Value Health. 2023 Apr;26(4):567-578. doi: 10.1016/j.jval.2022.12.002. Epub 2022 Dec 9.

Reference Type DERIVED
PMID: 36509366 (View on PubMed)

Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

Reference Type DERIVED
PMID: 33331662 (View on PubMed)

Rowe SM, Daines C, Ringshausen FC, Kerem E, Wilson J, Tullis E, Nair N, Simard C, Han L, Ingenito EP, McKee C, Lekstrom-Himes J, Davies JC. Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis. N Engl J Med. 2017 Nov 23;377(21):2024-2035. doi: 10.1056/NEJMoa1709847. Epub 2017 Nov 3.

Reference Type DERIVED
PMID: 29099333 (View on PubMed)

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2014-004788-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX14-661-108

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.