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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)

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
VX-445/TEZ/IVA
IVA
Placebo
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

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

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

Sites / Locations

  • 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
  • UCSF Gateway Medical Center
  • National Jewish Health
  • University of Florida, Shands Hospital
  • Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital
  • Nemours Children's Specialty Care
  • Central Florida Pulmonary Group
  • Nemours Children's Hospital
  • Tampa General Hospital Cardiac and Lung Transplant Clinic
  • Children's Speciality Services at North Druid Hills
  • Augusta University
  • Ann & Robert Lurie Children's Hospital of Chicago
  • Northwestern Memorial Hospital
  • Saint Francis Medical Center/ Children's Hospital of Illinois/OSF
  • University of Kansas Medical Center
  • Tulane Medical Center
  • Maine Medical Partners
  • Massachusetts General Hospital Cystic Fibrosis Center
  • Harper University Hospital
  • Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
  • Billings Clinic
  • Nebraska Medical Center
  • Morristown Medical Center
  • University of New Mexico Clinical & Translational Science Center
  • Mount Sinai Beth Israel
  • New York Medical College
  • UNC Marsico Clinical Research Center
  • Akron Children's Hospital
  • UC Health Holmes
  • University Hospitals Cleveland Medical Center/ Rainbow Babies and Children's Hospital
  • Nationwide Children's Hospital
  • Dayton Children's Hospital
  • ProMedica Toledo Hospital/ Toledo Children's Hospital/ Pediatric Pulmonary & Cystic Fibrosis Center
  • University of Oklahoma Health Sciences Center
  • Children's Hospital of Philadelphia
  • Medical University of South Carolina
  • Austin Children's Chest Associates
  • The University of Texas Southwestern Medical Center
  • The University of Texas Health Science Center at Tyler
  • Vermont Lung Center
  • University of Virginia Primary Care Center
  • Children's Hospital of the King's Daughters
  • Children's Hospital of Richmond at VCU, Children's Pavilion
  • University of Washington Medical Center
  • West Virginia University
  • University of Wisconsin Hospitals and Clinics
  • CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital
  • 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

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

VX-445/TEZ/IVA TC

Arm Description

Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period.

Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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.

Outcomes

Primary Outcome Measures

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Secondary Outcome Measures

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Number of Pulmonary Exacerbations (PEx)
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).
Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time-to-first Pulmonary Exacerbation (PEx)
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
Absolute Change in Body Weight
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA

Full Information

First Posted
May 2, 2018
Last Updated
May 6, 2020
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT03525444
Brief Title
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)
Official Title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Study Type
Interventional

2. Study Status

Record Verification Date
May 2020
Overall Recruitment Status
Completed
Study Start Date
June 15, 2018 (Actual)
Primary Completion Date
April 24, 2019 (Actual)
Study Completion Date
April 24, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
405 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants who received placebo matched to VX-445/TEZ/IVA for 24 weeks in the TC treatment period.
Arm Title
VX-445/TEZ/IVA TC
Arm Type
Experimental
Arm Description
Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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.
Intervention Type
Drug
Intervention Name(s)
VX-445/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, VX-445/tezacaftor/ivacaftor
Intervention Description
Participants received VX-445/TEZ/IVA orally once daily in the morning.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Participants received IVA orally once daily in the evening
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Participants received placebo matched VX-445/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening.
Primary Outcome Measure Information:
Title
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame
From Baseline at Week 4
Secondary Outcome Measure Information:
Title
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame
From Baseline through Week 24
Title
Number of Pulmonary Exacerbations (PEx)
Description
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Time Frame
From Baseline through Week 24
Title
Absolute Change in Sweat Chloride (SwCl)
Description
Sweat samples were collected using an approved collection device.
Time Frame
From Baseline through Week 24
Title
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
Description
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time Frame
From Baseline through Week 24
Title
Absolute Change in Body Mass Index (BMI)
Description
BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2).
Time Frame
From Baseline at Week 24
Title
Absolute Change in Sweat Chloride
Description
Sweat samples were collected using an approved collection device.
Time Frame
From Baseline at Week 4
Title
Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score
Description
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time Frame
From Baseline at Week 4
Title
Time-to-first Pulmonary Exacerbation (PEx)
Description
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms.
Time Frame
From Baseline through Week 24
Title
Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline
Description
BMI was defined as weight in kg divided by height in m^2. Z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
Time Frame
From Baseline at Week 24
Title
Absolute Change in Body Weight
Time Frame
From Baseline at Week 24
Title
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)
Title
Observed Pre-dose Concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA
Time Frame
Pre-dose on Week 4, 8, 12, and 16

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Heterozygous for the F508del mutation (F/MF) Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height 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
Facility Information:
Facility Name
Banner University of Arizona Medical Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
Miller Children's Hospital/ Long Beach Memorial
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Valley Children's Hospital/ Children's Hospital of Central California
City
Madera
State/Province
California
ZIP/Postal Code
93636
Country
United States
Facility Name
Kaiser Permanente
City
Oakland
State/Province
California
ZIP/Postal Code
94611
Country
United States
Facility Name
University of California Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
UCSF Gateway Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
University of Florida, Shands Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital
City
Hollywood
State/Province
Florida
ZIP/Postal Code
33021
Country
United States
Facility Name
Nemours Children's Specialty Care
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
Central Florida Pulmonary Group
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Nemours Children's Hospital
City
Orlando
State/Province
Florida
ZIP/Postal Code
32827
Country
United States
Facility Name
Tampa General Hospital Cardiac and Lung Transplant Clinic
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Facility Name
Children's Speciality Services at North Druid Hills
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30324
Country
United States
Facility Name
Augusta University
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Ann & Robert Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Northwestern Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Saint Francis Medical Center/ Children's Hospital of Illinois/OSF
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61637
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Tulane Medical Center
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Maine Medical Partners
City
Portland
State/Province
Maine
ZIP/Postal Code
04102
Country
United States
Facility Name
Massachusetts General Hospital Cystic Fibrosis Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Harper University Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Facility Name
Billings Clinic
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Morristown Medical Center
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07960
Country
United States
Facility Name
University of New Mexico Clinical & Translational Science Center
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87131
Country
United States
Facility Name
Mount Sinai Beth Israel
City
New York
State/Province
New York
ZIP/Postal Code
10003
Country
United States
Facility Name
New York Medical College
City
Valhalla
State/Province
New York
ZIP/Postal Code
10595
Country
United States
Facility Name
UNC Marsico Clinical Research Center
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27517
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
Facility Name
UC Health Holmes
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45220
Country
United States
Facility Name
University Hospitals Cleveland Medical Center/ Rainbow Babies and Children's Hospital
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
Dayton Children's Hospital
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45404
Country
United States
Facility Name
ProMedica Toledo Hospital/ Toledo Children's Hospital/ Pediatric Pulmonary & Cystic Fibrosis Center
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43606
Country
United States
Facility Name
University of Oklahoma Health Sciences Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Austin Children's Chest Associates
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
The University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
The University of Texas Health Science Center at Tyler
City
Tyler
State/Province
Texas
ZIP/Postal Code
75708
Country
United States
Facility Name
Vermont Lung Center
City
Colchester
State/Province
Vermont
ZIP/Postal Code
05446
Country
United States
Facility Name
University of Virginia Primary Care Center
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Children's Hospital of the King's Daughters
City
Norfolk
State/Province
Virginia
ZIP/Postal Code
23507
Country
United States
Facility Name
Children's Hospital of Richmond at VCU, Children's Pavilion
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
West Virginia University
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
University of Wisconsin Hospitals and Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Women & Children's Hospital
City
North Adelaide
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
The Children's Hospital at Westmead
City
Westmead
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
University of Graz
City
Graz
Country
Austria
Facility Name
Medizinische Universitat Innsbruck
City
Innsbruck
Country
Austria
Facility Name
LKH - Universitätsklinikum der PMU Salzburg
City
Salzburg
Country
Austria
Facility Name
Medizinische Universitat Wien
City
Vienna
Country
Austria
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussels
Country
Belgium
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussels
Country
Belgium
Facility Name
UZ Antwerpen
City
Edegem
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gathuisberg
City
Leuven
Country
Belgium
Facility Name
British Columbia's Children's Hospital
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
St. Paul's Hospital
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montréal
State/Province
Quebec
Country
Canada
Facility Name
University of Calgary Medical Clinic of the Foothills Medical Centre
City
Calgary
Country
Canada
Facility Name
Centre Hospitalier De L'Universite Laval
City
Qubec
Country
Canada
Facility Name
Saint John Regional Hospital
City
Saint John
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
Vancouver Island Health Authority
City
Victoria
Country
Canada
Facility Name
Fakultni Nemocnice Brno
City
Brno
Country
Czechia
Facility Name
Fakultni nemocnice v Motole
City
Praha 5
Country
Czechia
Facility Name
Centre Hospitalier Lyon Sud
City
Benite Cedex
Country
France
Facility Name
Groupe Hospitaler Pellegrin, CHU De Bordeaux
City
Bordeaux Cedex
Country
France
Facility Name
CHU Marseille - Hopital Nord
City
Marseille
Country
France
Facility Name
CHU de Nice - Hopital Pasteur
City
Nice
Country
France
Facility Name
Hopital Cochin
City
Paris
Country
France
Facility Name
CHU de Rouen - Hopital Charles Nicolle
City
Rouen Cedex, Seine Maritime
Country
France
Facility Name
Hopital Foch (Suresnes), Hopital Foch, Adultes
City
Suresnes
Country
France
Facility Name
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
City
Erlangen
Country
Germany
Facility Name
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
City
Giessen
Country
Germany
Facility Name
Hannover Medical School
City
Hannover
Country
Germany
Facility Name
Heidelberg Cystic Fibrosis Center
City
Heidelberg
Country
Germany
Facility Name
Johannes Gutenberg-Universitaet
City
Mainz
Country
Germany
Facility Name
Dr. von Haunersches Kinderspital
City
München
Country
Germany
Facility Name
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
City
Tuebingen
Country
Germany
Facility Name
University Hospital Wuerzburg
City
Wuerzburg
Country
Germany
Facility Name
General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)
City
Maroúsi
Country
Greece
Facility Name
Azienda Ospedaliero Universitaria Ospedale Riuniti
City
Ancona
Country
Italy
Facility Name
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
City
Firenze
Country
Italy
Facility Name
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
City
Genova
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Policlinico G. Martino
City
Messina
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Facility Name
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
City
Potenza
Country
Italy
Facility Name
Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
City
Verona
Country
Italy
Facility Name
Academic Medical Center
City
Amsterdam
Country
Netherlands
Facility Name
HagaZiekenhuis van den Haag
City
Den Haag
Country
Netherlands
Facility Name
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
City
Heidelberglaan
Country
Netherlands
Facility Name
UMC St. Radboud
City
Nijmegen
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
Karolinska Univeritetssjukhuset, Huddinge
City
Stockholm
Country
Sweden
Facility Name
The Royal Belfast Hospital for Sick Children
City
Belfast
Country
United Kingdom
Facility Name
Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
City
Brimingham
Country
United Kingdom
Facility Name
Royal Hospital for Sick Children
City
Edinburgh
Country
United Kingdom
Facility Name
Western General Hospital
City
Edinburgh
Country
United Kingdom
Facility Name
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
City
Exeter
Country
United Kingdom
Facility Name
Leeds General Infirmary
City
Leeds
Country
United Kingdom
Facility Name
King's College Hospital
City
London
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
31697873
Citation
Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R; VX17-445-102 Study Group. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31.
Results Reference
derived

Learn more about this trial

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)

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