A Phase 3 Study of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

NCT ID: NCT03447249

Last Updated: 2020-03-13

Study Results

Results available

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

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

385 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-03-07

Study Completion Date

2019-02-05

Brief Summary

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This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).

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

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Participants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.

VX-659/TEZ/IVA TC

Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.

Group Type EXPERIMENTAL

VX-659/TEZ/IVA

Intervention Type DRUG

Participants received VX-659/TEZ/IVA orally once daily in the morning.

IVA

Intervention Type DRUG

Participants received IVA orally once daily in the evening.

Interventions

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VX-659/TEZ/IVA

Participants received VX-659/TEZ/IVA orally once daily in the morning.

Intervention Type DRUG

IVA

Participants received IVA orally once daily in the evening.

Intervention Type DRUG

Placebo

Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.

Intervention Type DRUG

Other Intervention Names

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VX-659/VX-661/VX-770 VX-659/tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

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Inclusion Criteria

* Heterozygous for F508del and an MF mutation (as defined in the protocol)
* Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Exclusion Criteria

* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation
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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University of Alabama at Birmingham

Birmingham, Alabama, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status

Hartford Health

Hartford, Connecticut, United States

Site Status

Yale New Haven Medical Center

New Haven, Connecticut, United States

Site Status

University of Miami/ Miller School of Medicine

Miami, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Arnold Palmer Hospital

Orlando, Florida, United States

Site Status

Johns Hopkins All Children's Hospital Outpatient Care Center

St. Petersburg, Florida, United States

Site Status

St. Luke's CF Center of Idaho

Boise, Idaho, United States

Site Status

Cystic Fibrosis Center of Chicago

Glenview, Illinois, United States

Site Status

Advocate Children's Hospital - Park Ridge/ North Suburban Pulmonary and Critical Care Consultants

Niles, Illinois, United States

Site Status

Indiana Clinical Research Center, IU Health University Hospital

Indianapolis, Indiana, United States

Site Status

The University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

Kentucky Clinic

Lexington, Kentucky, United States

Site Status

Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, United States

Site Status

The Johns Hopkins Hospital

Baltimore, Maryland, United States

Site Status

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status

University of Massachusetts Memorial Medical Center

Worcester, Massachusetts, United States

Site Status

University of Michigan Health System

Ann Arbor, Michigan, United States

Site Status

Helen DeVos Children's Hospital CF Center

Grand Rapids, Michigan, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

University of Mississippi Medical Center

Jackson, Mississippi, United States

Site Status

Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Washington University School of Medicine/ St. Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Dartmouth Hitchcock Medical Center, Lebanon

Lebanon, New Hampshire, United States

Site Status

Rutgers-Robert Wood Johnson Medical School

New Brunswick, New Jersey, United States

Site Status

Albany Medical College

Albany, New York, United States

Site Status

Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo

Buffalo, New York, United States

Site Status

Northwell Health, Long Island Jewish Medical Center

New Hyde Park, New York, United States

Site Status

Columbia University Medical Center

New York, New York, United States

Site Status

SUNY Upstate Medical University

Syracuse, New York, United States

Site Status

Clinical Research of Charlotte

Charlotte, North Carolina, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Respiratory Diseases of Children and Adolescents

Oklahoma City, Oklahoma, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, United States

Site Status

Sanford Research/ USD

Sioux Falls, South Dakota, United States

Site Status

University of Tennessee Medical Center - Adult Cystic Fibrosis Clinic

Knoxville, Tennessee, United States

Site Status

Children's Foundation Research Center/ Le Bonheur Children's Hospital

Memphis, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

Cook Children's Medical Center

Fort Worth, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Utah / Primary Children's Medical Center

Salt Lake City, Utah, United States

Site Status

Seattle Children's Hospital

Seattle, Washington, United States

Site Status

Providence Pediatric Pulmonary & Allergy/Immunology Clinic

Spokane, Washington, United States

Site Status

The Alfred Hospital

Melbourne, Victoria, Australia

Site Status

Royal Adelaide Hospital

Adelaide, , Australia

Site Status

Prince Charles Hospital

Chermside, , Australia

Site Status

Royal Brisbane & Women's Hospital

Herston, , Australia

Site Status

Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital

Nedlands, , Australia

Site Status

John Hunter Hospital & Hunter Medical Research Institute

New Lambton Heights, , Australia

Site Status

Sydney Children's Hospital, Randwick

Randwick, , Australia

Site Status

Princess Margaret Hospital for Children

Subiaco, , Australia

Site Status

Stollery Children's Hospital

Edmonton, Alberta, Canada

Site Status

Queen Elizabeth II Health Sciences Center

Halifax, Nova Scotia, Canada

Site Status

St. Michael's Hospital

Toronto, , Canada

Site Status

Juliane Marie Center, Rigshospitalet

Copenhagen, , Denmark

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

University Hospital Cologne

Cologne, , Germany

Site Status

Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen

Essen, , Germany

Site Status

Clinic of J.W Goethe University

Frankfurt, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder - und Jugendmedizin

Jena, , Germany

Site Status

Universitatsklinikum Schleswig-Holstein, Klinik fur Kinder- und Jugendmedizin

Lübeck, , Germany

Site Status

Klinikum Innenstadt, University of Munich

München, , Germany

Site Status

Pneumologische Praxis Pasing

München, , Germany

Site Status

Beaumont Hospital

Dublin, , Ireland

Site Status

Children's University Hospital Temple Street

Dublin, , Ireland

Site Status

Cork University Hospital

Dublin, , Ireland

Site Status

Our Lady's Children's Hospital

Dublin, , Ireland

Site Status

St. Vincent's University Hospital

Dublin, , Ireland

Site Status

National University of Ireland

Galway, , Ireland

Site Status

University Hospital Limerick

Limerick, , Ireland

Site Status

Lady Davis Carmel Medical Center

Haifa, , Israel

Site Status

Pediatric Pulmonary Unit Rambam Medical Center

Haifa, , Israel

Site Status

Hadassah Medical Organization

Jerusalem, , Israel

Site Status

Schneider Children's Medical Center

Petah Tikva, , Israel

Site Status

Sheba Medical Center

Tel Litwinsky, , Israel

Site Status

Instytut Matki i Dziecka

Warsaw, , Poland

Site Status

Hospital Universitari Vall d Hebron

Barcelona, , Spain

Site Status

Hospital Universitari Vall d'Hebron Servicio de Broncoscopia

Barcelona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario Infantil La Paz

Madrid, , Spain

Site Status

Coporacio Sanitaria Parc Tauli

Sabadell, , Spain

Site Status

Hospital Universitario Virgen del Rocio

Seville, , Spain

Site Status

Hospital Universitario y Politecnico La Fe

Valencia, , Spain

Site Status

Lindenhofspital - Quartier Bleu

Bern, , Switzerland

Site Status

Kinderspital Zuerich

Zurich, , Switzerland

Site Status

Universitaetsspital Zuerich

Zurich, , Switzerland

Site Status

Papworth Hospital NHS Foundation Trust, Papworth Everard

Cambridge, , United Kingdom

Site Status

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status

The Leeds Teaching Hospitals NHS Trust, St. James University Hospital

Leeds, , United Kingdom

Site Status

Liverpool Heart and Chest Hospital

Liverpool, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, , United Kingdom

Site Status

Wythenshawe Hospital

Manchester, , United Kingdom

Site Status

The Newcastle Upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status

Nottingham University Hospitals NHS Trust, Queens Medical Center

Nottingham, , United Kingdom

Site Status

University Hospital Llandough

Penarth, , United Kingdom

Site Status

Countries

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United States Australia Canada Denmark Germany Ireland Israel Poland Spain Switzerland United Kingdom

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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2017-004132-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX17-659-102

Identifier Type: -

Identifier Source: org_study_id

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