A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Phase

Condition

Lung Disease

Cystic Fibrosis

Scar Tissue

Treatment

IVA

Placebo

VX-445/TEZ/IVA

Clinical Study ID

NCT03525444

VX17-445-102

2018-000183-28

Ages > 12
All Genders

Study Summary

This study will evaluate the efficacy of VX-445 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).

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

Heterozygous for the F508del mutation (F/MF)
Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean forage, sex, and height

Exclusion

Key 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 Other protocol defined Inclusion/Exclusion criteria may apply

Study Design

IVA

June 15, 2018

April 24, 2019

Connect with a study center

Women & Children's Hospital
North Adelaide,
Australia
Site Not Available
The Royal Children's Hospital
Parkville,
Australia
Site Not Available
Mater Adult Hospital
South Brisbane,
Australia
Site Not Available
The Children's Hospital at Westmead
Westmead,
Australia
Site Not Available
Westmead Hospital
Westmead,
Australia
Site Not Available
University of Graz
Graz,
Austria
Site Not Available
Medizinische Universitat Innsbruck
Innsbruck,
Austria
Site Not Available
LKH - Universitätsklinikum der PMU Salzburg
Salzburg,
Austria
Site Not Available
Medizinische Universitat Wien
Vienna,
Austria
Site Not Available
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels,
Belgium
Site Not Available
Universitair Ziekenhuis Brussel - Campus Jette
Brussels,
Belgium
Site Not Available
UZ Antwerpen
Edegem,
Belgium
Site Not Available
Universitair Ziekenhuis Gent
Gent,
Belgium
Site Not Available
Universitaire Ziekenhuizen Leuven - Campus Gathuisberg
Leuven,
Belgium
Site Not Available
British Columbia's Children's Hospital
Vancouver, British Columbia
Canada
Site Not Available
St. Paul's Hospital
Vancouver, British Columbia
Canada
Site Not Available
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montréal, Quebec
Canada
Site Not Available
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary,
Canada
Site Not Available
Centre Hospitalier De L'Universite Laval
Qubec,
Canada
Site Not Available
Saint John Regional Hospital
Saint John,
Canada
Site Not Available
The Hospital for Sick Children
Toronto,
Canada
Site Not Available
Vancouver Island Health Authority
Victoria,
Canada
Site Not Available
Fakultni Nemocnice Brno
Brno,
Czechia
Site Not Available
Fakultni nemocnice v Motole
Praha 5,
Czechia
Site Not Available
Centre Hospitalier Lyon Sud
Benite Cedex,
France
Site Not Available
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux Cedex,
France
Site Not Available
CHU Marseille - Hopital Nord
Marseille,
France
Site Not Available
CHU de Nice - Hopital Pasteur
Nice,
France
Site Not Available
Hopital Cochin
Paris,
France
Site Not Available
CHU de Rouen - Hopital Charles Nicolle
Rouen Cedex, Seine Maritime,
France
Site Not Available
Hopital Foch (Suresnes), Hopital Foch, Adultes
Suresnes,
France
Site Not Available
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen,
Germany
Site Not Available
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen,
Germany
Site Not Available
Hannover Medical School
Hannover,
Germany
Site Not Available
Heidelberg Cystic Fibrosis Center
Heidelberg,
Germany
Site Not Available
Johannes Gutenberg-Universitaet
Mainz,
Germany
Site Not Available
Dr. von Haunersches Kinderspital
München,
Germany
Site Not Available
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
Tuebingen,
Germany
Site Not Available
University Hospital Wuerzburg
Wuerzburg,
Germany
Site Not Available
General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)
Maroúsi,
Greece
Site Not Available
Azienda Ospedaliero Universitaria Ospedale Riuniti
Ancona,
Italy
Site Not Available
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Firenze,
Italy
Site Not Available
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova,
Italy
Site Not Available
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina,
Italy
Site Not Available
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milano,
Italy
Site Not Available
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
Potenza,
Italy
Site Not Available
Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
Verona,
Italy
Site Not Available
Academic Medical Center
Amsterdam,
Netherlands
Site Not Available
HagaZiekenhuis van den Haag
Den Haag,
Netherlands
Site Not Available
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Heidelberglaan,
Netherlands
Site Not Available
UMC St. Radboud
Nijmegen,
Netherlands
Site Not Available
Erasmus Medical Center
Rotterdam,
Netherlands
Site Not Available
Karolinska Univeritetssjukhuset, Huddinge
Stockholm,
Sweden
Site Not Available
The Royal Belfast Hospital for Sick Children
Belfast,
United Kingdom
Site Not Available
Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
Brimingham,
United Kingdom
Site Not Available
Royal Hospital for Sick Children
Edinburgh,
United Kingdom
Site Not Available
Western General Hospital
Edinburgh,
United Kingdom
Site Not Available
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Exeter,
United Kingdom
Site Not Available
Leeds General Infirmary
Leeds,
United Kingdom
Site Not Available
King's College Hospital
London,
United Kingdom
Site Not Available
Royal Manchester Children's Hospital
Manchester,
United Kingdom
Site Not Available
Southampton General Hospital
Southampton,
United Kingdom
Site Not Available
Banner University of Arizona Medical Center
Tucson, Arizona 85724
United States
Site Not Available
University of Arkansas for Medical Sciences
Little Rock, Arkansas 72205
United States
Site Not Available
Miller Children's Hospital/ Long Beach Memorial
Long Beach, California 90806
United States
Site Not Available
Children's Hospital Los Angeles
Los Angeles, California 90027
United States
Site Not Available
Valley Children's Hospital/ Children's Hospital of Central California
Madera, California 93636
United States
Site Not Available
Kaiser Permanente
Oakland, California 94611
United States
Site Not Available
University of California Davis Medical Center
Sacramento, California 95817
United States
Site Not Available
UCSF Gateway Medical Center
San Francisco, California 94143
United States
Site Not Available
National Jewish Health
Denver, Colorado 80206
United States
Site Not Available
University of Florida, Shands Hospital
Gainesville, Florida 32610
United States
Site Not Available
Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital
Hollywood, Florida 33021
United States
Site Not Available
Nemours Children's Specialty Care
Jacksonville, Florida 32207
United States
Site Not Available
Central Florida Pulmonary Group
Orlando, Florida 32803
United States
Site Not Available
Nemours Children's Hospital
Orlando, Florida 32827
United States
Site Not Available
Tampa General Hospital Cardiac and Lung Transplant Clinic
Tampa, Florida 33606
United States
Site Not Available
Children's Speciality Services at North Druid Hills
Atlanta, Georgia 30324
United States
Site Not Available
Augusta University
Augusta, Georgia 30912
United States
Site Not Available
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Site Not Available
Northwestern Memorial Hospital
Chicago, Illinois 60611
United States
Site Not Available
Saint Francis Medical Center/ Children's Hospital of Illinois/OSF
Peoria, Illinois 61637
United States
Site Not Available
University of Kansas Medical Center
Kansas City, Kansas 66160
United States
Site Not Available
Tulane Medical Center
New Orleans, Louisiana 70112
United States
Site Not Available
Maine Medical Partners
Portland, Maine 04102
United States
Site Not Available
Massachusetts General Hospital Cystic Fibrosis Center
Boston, Massachusetts 02114
United States
Site Not Available
Harper University Hospital
Detroit, Michigan 48201
United States
Site Not Available
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota 55404
United States
Site Not Available
Billings Clinic
Billings, Montana 59101
United States
Site Not Available
Nebraska Medical Center
Omaha, Nebraska 68198
United States
Site Not Available
Morristown Medical Center
Morristown, New Jersey 07960
United States
Site Not Available
University of New Mexico Clinical & Translational Science Center
Albuquerque, New Mexico 87131
United States
Site Not Available
Mount Sinai Beth Israel
New York, New York 10003
United States
Site Not Available
New York Medical College
Valhalla, New York 10595
United States
Site Not Available
UNC Marsico Clinical Research Center
Chapel Hill, North Carolina 27517
United States
Site Not Available
Akron Children's Hospital
Akron, Ohio 44308
United States
Site Not Available
UC Health Holmes
Cincinnati, Ohio 45220
United States
Site Not Available
University Hospitals Cleveland Medical Center/ Rainbow Babies and Children's Hospital
Cleveland, Ohio 44106
United States
Site Not Available
Nationwide Children's Hospital
Columbus, Ohio 43205
United States
Site Not Available
Dayton Children's Hospital
Dayton, Ohio 45404
United States
Site Not Available
ProMedica Toledo Hospital/ Toledo Children's Hospital/ Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio 43606
United States
Site Not Available
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma 73104
United States
Site Not Available
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Site Not Available
Medical University of South Carolina
Charleston, South Carolina 29425
United States
Site Not Available
Austin Children's Chest Associates
Austin, Texas 78723
United States
Site Not Available
The University of Texas Southwestern Medical Center
Dallas, Texas 75390
United States
Site Not Available
The University of Texas Health Science Center at Tyler
Tyler, Texas 75708
United States
Site Not Available
Vermont Lung Center
Colchester, Vermont 05446
United States
Site Not Available
University of Virginia Primary Care Center
Charlottesville, Virginia 22908
United States
Site Not Available
Children's Hospital of the King's Daughters
Norfolk, Virginia 23507
United States
Site Not Available
Children's Hospital of Richmond at VCU, Children's Pavilion
Richmond, Virginia 23298
United States
Site Not Available
University of Washington Medical Center
Seattle, Washington 98195
United States
Site Not Available
West Virginia University
Morgantown, West Virginia 26506
United States
Site Not Available
University of Wisconsin Hospitals and Clinics
Madison, Wisconsin 53792
United States
Site Not Available
CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin 53226
United States
Site Not Available

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A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Phase

Condition

Treatment

Clinical Study ID

Study Summary

Eligibility Criteria

Inclusion

Exclusion

Study Design

Connect with a study center

Women & Children's Hospital

The Royal Children's Hospital

Mater Adult Hospital

The Children's Hospital at Westmead

Westmead Hospital

University of Graz

Medizinische Universitat Innsbruck

LKH - Universitätsklinikum der PMU Salzburg

Medizinische Universitat Wien

Cliniques Universitaires de Bruxelles Hopital Erasme

Universitair Ziekenhuis Brussel - Campus Jette

UZ Antwerpen

Universitair Ziekenhuis Gent

Universitaire Ziekenhuizen Leuven - Campus Gathuisberg

British Columbia's Children's Hospital

St. Paul's Hospital

McGill University Health Centre, Glen Site, Montreal Children's Hospital

University of Calgary Medical Clinic of the Foothills Medical Centre

Centre Hospitalier De L'Universite Laval

Saint John Regional Hospital

The Hospital for Sick Children

Vancouver Island Health Authority

Fakultni Nemocnice Brno

Fakultni nemocnice v Motole

Centre Hospitalier Lyon Sud

Groupe Hospitaler Pellegrin, CHU De Bordeaux

CHU Marseille - Hopital Nord

CHU de Nice - Hopital Pasteur

Hopital Cochin

CHU de Rouen - Hopital Charles Nicolle

Hopital Foch (Suresnes), Hopital Foch, Adultes

Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital

Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin

Hannover Medical School

Heidelberg Cystic Fibrosis Center

Johannes Gutenberg-Universitaet

Dr. von Haunersches Kinderspital

Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin

University Hospital Wuerzburg

General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)

Azienda Ospedaliero Universitaria Ospedale Riuniti

Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer

IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico

Azienda Ospedaliera Universitaria Policlinico G. Martino

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo

Azienda Ospedaliera di Verona-Ospedale Civile Maggiore

Academic Medical Center

HagaZiekenhuis van den Haag

University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis

UMC St. Radboud

Erasmus Medical Center

Karolinska Univeritetssjukhuset, Huddinge

The Royal Belfast Hospital for Sick Children

Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital

Royal Hospital for Sick Children

Western General Hospital

Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital

Leeds General Infirmary

King's College Hospital

Royal Manchester Children's Hospital

Southampton General Hospital

Banner University of Arizona Medical Center

University of Arkansas for Medical Sciences

Miller Children's Hospital/ Long Beach Memorial

Children's Hospital Los Angeles

Valley Children's Hospital/ Children's Hospital of Central California

Kaiser Permanente

University of California Davis Medical Center