search
Back to results

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

Primary Purpose

Cystic Fibrosis

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
VX-121/TEZ/D-IVA
ELX/TEZ/IVA
IVA
Placebo (matched to VX-121/TEZ/D-IVA)
Placebo (matched to ELX/TEZ/IVA)
Placebo (matched to IVA)
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:

  • Participant has one of the following genotypes:

    • Homozygous for F508del;
    • Heterozygous for F508del and a gating (F/G) mutation;
    • Heterozygous for F508del and a residual function (F/RF) mutation;
    • At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation
  • Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy

Key Exclusion Criteria:

  • History of solid organ or hematological transplantation
  • Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Pregnant or breast-feeding females

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • University of Alabama at Birmingham
  • Banner University of Arizona Medical Center
  • University of Arkansas for Medical Sciences
  • Children's Hospital Los Angeles
  • Children's Hospital of Orange County
  • University of California Davis Medical Center
  • Children's Hospital of Colorado
  • National Jewish Health
  • Yale New Haven Hospital
  • Nemours Children's Specialty Care
  • University of Miami Miller School of Medicine
  • Central Florida Pulmonary Group, PA
  • Nemours Children's Specialty Care, Pensacola
  • Johns Hopkins All Children's Hospital Outpatient Care Center
  • The Emory Clinic at Chantilly
  • St. Luke's Cystic Fibrosis Center of Idaho
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • Northwestern Memorial Hospital
  • Indiana University
  • Riley Hospital for Children at Indiana University Health
  • University of Kentucky
  • Tulane Medical Center
  • Maine Medical Partners
  • Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
  • Boston Children's Hospital
  • UMass Memorial Medical Center
  • Michigan Medicine
  • Harper University Hospital
  • Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
  • University of Mississippi Medical Center
  • The Children's Mercy Hospital
  • Washington University School of Medicine
  • Billings Clinic
  • Morristown Medical Center
  • CF Therapeutics Development Center of Western New York
  • Northwell Health- Long Island Jewish Medical Center
  • Columbia University Medical Center
  • SUNY Upstate Medical University
  • Wake Forest Baptist Health
  • Akron 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
  • Santiago Reyes, M.D.
  • Oregon Health & Science University
  • Children's Hospital of Philadelphia
  • UPMC Children's Hospital of Pittsburgh
  • Prisma Health Richland Campus
  • Sanford Children's Speciality Clinic
  • University of Tennessee Medical Center
  • Vanderbilt University Medical Center
  • The University of Texas Southwestern Medical Center
  • Cook Children's Health Care System
  • University of Utah - Primary Children's Medical Center
  • Vermont Lung Center
  • University of Virginia Health System
  • Children's Hospital of the King's Daughters
  • University of Washington Medical Center
  • Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
  • West Virginia University
  • University Hospital and UW Health Clinics
  • Royal Prince Alfred Hospital
  • The Prince Charles Hospital
  • Alfred Hospital
  • Institute for Respiratory Health
  • Telethon Kids Institute
  • The Royal Children's Hospital
  • Sydney Children's Hospital
  • Mater Adult Hospital
  • Queensland Children's Hospital
  • Westmead Hospital
  • University of Graz
  • Medizinische Universität Innsbruck
  • Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde
  • Medizinische Universität Wien
  • Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen
  • Cliniques Universitaires de Bruxelles Hopital Erasme
  • Universitair Ziekenhuis Brussel - Campus Jette
  • Universitair Ziekenhuis Gent
  • Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
  • Cliniques Universitaires Saint-Luc
  • University of Calgary Medical Clinic of the Foothills Medical Centre
  • Stollery Children's Hospital
  • St. Joseph's Health Care London
  • Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
  • St. Michael's Hospital
  • British Columbia Children's Hospital
  • St. Paul's Hospital
  • Aarhus University Hospital
  • Juliane Marie Center, Rigshospitalet
  • Groupe Hospitaler Pellegrin, CHU De Bordeaux
  • CHU Lyon - Hopital Femme Mere-Enfant
  • Centre Hospitalier Intercommunal Creteil
  • Institut Cœur Poumon, CHU de Lille
  • Hopital Arnaud de Villeneuve
  • Centre Hospitalier Universitaire De Nantes - G. R. Laennec
  • Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
  • Hopital Necker, Enfants Malades
  • Hopital Cochin
  • Hopital Robert Debre
  • Centre Hospitalier Lyon Sud
  • Centre de Perharidy
  • CHU de Rouen - Hopital Charles Nicolle
  • Hôpital de Hautepierre, AX5
  • Hopital Foch (Suresnes), Hopital Foch, Adultes
  • Hopital Bretonneau
  • Centre hospitalier universitaire de Nancy, Hôpital de Braboi
  • Charite Paediatric Pulmonology Department
  • Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
  • Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
  • Pneumologisches Studienzentrum Muenchen-West
  • General Hospital of Thessaloniki "Papanikolaou"
  • General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki
  • National Koranyi Institute for TBC and Pulmonology
  • Pulmonology Institute Torokbalint
  • Cork University Hospital
  • Children's Health Ireland at Crumlin
  • Children's Health Ireland at Tallaght
  • Children's University Hospital, Dublin
  • St. Vincent's University Hospital
  • University Hospital Limerick (Adults)
  • University Hospital Limerick (Pediatrics)
  • Hadassah Medical Organization
  • Schneider Children's Medical Center of Israel
  • Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
  • Azienda Ospedaliero Universitaria Ospedale Riuniti
  • Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
  • IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
  • Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
  • Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
  • Azienda Ospedaliera di Verona - Ospedale Civile Maggiore
  • Academisch Medisch Centrum (Academic Medical Centre)
  • HagaZiekenhuis van den Haag
  • UMC St. Radboud
  • Erasmus Medical Center
  • University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
  • Greenlane Clinical Centre
  • Starship Children's Hospital
  • Canterbury Respiratory Research Group
  • Waikato Hospital
  • Haukeland Universitetssjukehus (CF)
  • Oslo University Hospital, Department of Paediatric Medicine
  • Pediatric Hospital Polanki named of Maciej Płażyński
  • Institute of Tuberculosis and Lung Diseases
  • Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
  • Sahlgrenska Universitetssjukhuset
  • Lund University Skanes Universitetssjukhus
  • Karolinska Universitetssjukhuset, Huddinge
  • Lindenhofspital - Quartier Bleu
  • Réseau Hospitalier Neuchâtelois
  • Kinderspital Zuerich
  • Birmingham Children's Hospital
  • Royal Papworth Hospital NHS Foundation Trust
  • Clinical Research Facility, Queen Elizabeth University Hospital
  • Leeds General Infirmary
  • St. James University Hospital
  • King's College Hospital
  • Royal Brompton Hospital
  • St. Bartholomew's Hospital
  • Royal Manchester Children's Hospital
  • Wythenshawe Hospital
  • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

VX-121/TEZ/D-IVA

ELX/TEZ/IVA

Arm Description

Participants will receive VX-121/TEZ/D-IVA in the morning.

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Outcomes

Primary Outcome Measures

Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24

Secondary Outcome Measures

Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24
Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-102)
Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-102)

Full Information

First Posted
September 29, 2021
Last Updated
June 16, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
search

1. Study Identification

Unique Protocol Identification Number
NCT05076149
Brief Title
A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation
Official Title
A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive CFTR Mutation and No F508del Mutation
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 27, 2021 (Actual)
Primary Completion Date
May 18, 2023 (Actual)
Study Completion Date
February 2024 (Anticipated)

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

5. Study Description

Brief Summary
The purpose of this study is to evaluate the efficacy and safety of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants who are homozygous for F508del, heterozygous for F508del and a gating (F/G) or residual function (F/RF) mutation, or have at least 1 other TCR CF transmembrane conductance regulator (CFTR) gene mutation and no F508del mutation.

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
600 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VX-121/TEZ/D-IVA
Arm Type
Experimental
Arm Description
Participants will receive VX-121/TEZ/D-IVA in the morning.
Arm Title
ELX/TEZ/IVA
Arm Type
Active Comparator
Arm Description
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention Type
Drug
Intervention Name(s)
VX-121/TEZ/D-IVA
Other Intervention Name(s)
VX-121/VX-661/CTP-656, VX-121/VX-661/VX-561, VX-121/tezacaftor/deutivacaftor
Intervention Description
Fixed-dose combination tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed-dose combination tablets for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo (matched to VX-121/TEZ/D-IVA)
Intervention Description
Placebo matched to VX-121/TEZ/D-IVA for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo (matched to ELX/TEZ/IVA)
Intervention Description
Placebo matched to ELX/TEZ/IVA for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo (matched to IVA)
Intervention Description
Placebo matched to IVA for oral administration.
Primary Outcome Measure Information:
Title
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 24
Time Frame
From Baseline Through Week 24
Secondary Outcome Measure Information:
Title
Absolute Change From Baseline in Sweat Chloride (SwCl) Through Week 24
Time Frame
From Baseline Through Week 24
Title
Proportion of Participants With SwCl <60 Millimole per Liter (mmol/L) Through Week 24 (Pooled With Data From Study VX20-121-102)
Time Frame
From Baseline Through Week 24
Title
Proportion of Participants With SwCl <30 mmol/L Through Week 24 (Pooled With Data From Study VX20-121-102)
Time Frame
From Baseline Through Week 24

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Participant has one of the following genotypes: Homozygous for F508del; Heterozygous for F508del and a gating (F/G) mutation; Heterozygous for F508del and a residual function (F/RF) mutation; At least 1 other TCR CFTR gene mutation identified as responsive to ELX/TEZ/IVA and no F508del mutation Forced expiratory volume in 1 second (FEV1) value >=40% and <=90% of predicted mean for age, sex, and height for participants currently receiving CFTR protein modulator therapy; FEV1 >=40% and <=80% for participants not currently receiving CFTR protein modulator therapy Key Exclusion Criteria: History of solid organ or hematological transplantation Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) Lung infection with organisms associated with a more rapid decline in pulmonary status Pregnant or breast-feeding females Other protocol defined Inclusion/Exclusion criteria may apply
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
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
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Children's Hospital of Orange County
City
Orange
State/Province
California
ZIP/Postal Code
92868
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
Children's Hospital of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
National Jewish Health
City
Denver
State/Province
Colorado
ZIP/Postal Code
80206
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
Nemours Children's Specialty Care
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Central Florida Pulmonary Group, PA
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Facility Name
Nemours Children's Specialty Care, Pensacola
City
Pensacola
State/Province
Florida
ZIP/Postal Code
32514
Country
United States
Facility Name
Johns Hopkins All Children's Hospital Outpatient Care Center
City
Saint Petersburg
State/Province
Florida
ZIP/Postal Code
33701
Country
United States
Facility Name
The Emory Clinic at Chantilly
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30324
Country
United States
Facility Name
St. Luke's Cystic Fibrosis Center of Idaho
City
Boise
State/Province
Idaho
ZIP/Postal Code
83702
Country
United States
Facility Name
Ann & Robert H. 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
Indiana University
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Riley Hospital for Children at Indiana University Health
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Kentucky
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
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 Clinical Rsearch Center
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
UMass Memorial Medical Center
City
Worcester
State/Province
Massachusetts
ZIP/Postal Code
01655
Country
United States
Facility Name
Michigan Medicine
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109-5212
Country
United States
Facility Name
Harper University Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49546
Country
United States
Facility Name
University of Mississippi Medical Center
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Billings Clinic
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
Morristown Medical Center
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07960
Country
United States
Facility Name
CF Therapeutics Development Center of Western New York
City
Buffalo
State/Province
New York
ZIP/Postal Code
14203
Country
United States
Facility Name
Northwell Health- Long Island Jewish Medical Center
City
New Hyde Park
State/Province
New York
ZIP/Postal Code
11040
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
SUNY Upstate Medical University
City
Syracuse
State/Province
New York
ZIP/Postal Code
13210
Country
United States
Facility Name
Wake Forest Baptist Health
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27104
Country
United States
Facility Name
Akron Children's Hospital
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
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
Santiago Reyes, M.D.
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73112
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Prisma Health Richland Campus
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29203
Country
United States
Facility Name
Sanford Children's Speciality Clinic
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57105
Country
United States
Facility Name
University of Tennessee Medical Center
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37920
Country
United States
Facility Name
Vanderbilt University Medical Center
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
The University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8558
Country
United States
Facility Name
Cook Children's Health Care System
City
Fort Worth
State/Province
Texas
ZIP/Postal Code
76104
Country
United States
Facility Name
University of Utah - Primary Children's Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
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 Health System
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
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
City
Spokane
State/Province
Washington
ZIP/Postal Code
99204
Country
United States
Facility Name
West Virginia University
City
Morgantown
State/Province
West Virginia
ZIP/Postal Code
26506
Country
United States
Facility Name
University Hospital and UW Health Clinics
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Royal Prince Alfred Hospital
City
Camperdown
Country
Australia
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Alfred Hospital
City
Melbourne, VIC
Country
Australia
Facility Name
Institute for Respiratory Health
City
Nedlands
Country
Australia
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville, VIC
Country
Australia
Facility Name
Sydney Children's Hospital
City
Randwick
Country
Australia
Facility Name
Mater Adult Hospital
City
South Brisbane
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Westmead Hospital
City
Westmead
Country
Australia
Facility Name
University of Graz
City
Graz
Country
Austria
Facility Name
Medizinische Universität Innsbruck
City
Innsbruck
Country
Austria
Facility Name
Uniklinikum Salzburg - Universitätsklinik für Pneumologie/Lungenheilkunde
City
Salzburg
Country
Austria
Facility Name
Medizinische Universität Wien
City
Vienna
Country
Austria
Facility Name
Abteilung fuer Pulmologie Klinikum Wels-Grieskirchen
City
Wels
Country
Austria
Facility Name
Cliniques Universitaires de Bruxelles Hopital Erasme
City
Brussel
Country
Belgium
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussel
Country
Belgium
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
Cliniques Universitaires Saint-Luc
City
Woluwe-Saint-Lambert
Country
Belgium
Facility Name
University of Calgary Medical Clinic of the Foothills Medical Centre
City
Calgary
Country
Canada
Facility Name
Stollery Children's Hospital
City
Edmonton
Country
Canada
Facility Name
St. Joseph's Health Care London
City
London
Country
Canada
Facility Name
Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu
City
Montreal
Country
Canada
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montréal
Country
Canada
Facility Name
Institut Universitaire de Cardiologie et Pneumologie de Quebec - Universite Laval
City
Québec
Country
Canada
Facility Name
St. Michael's Hospital
City
Toronto
Country
Canada
Facility Name
British Columbia Children's Hospital
City
Vancouver
Country
Canada
Facility Name
St. Paul's Hospital
City
Vancouver
Country
Canada
Facility Name
Aarhus University Hospital
City
Aarhus
Country
Denmark
Facility Name
Juliane Marie Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Groupe Hospitaler Pellegrin, CHU De Bordeaux
City
Bordeaux cedex
Country
France
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Facility Name
Centre Hospitalier Intercommunal Creteil
City
Créteil
Country
France
Facility Name
Institut Cœur Poumon, CHU de Lille
City
Lille
Country
France
Facility Name
Hopital Arnaud de Villeneuve
City
Montpellier Cedex 5
Country
France
Facility Name
Centre Hospitalier Universitaire De Nantes - G. R. Laennec
City
Nantes
Country
France
Facility Name
Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur
City
Nice
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Hopital Cochin
City
Paris
Country
France
Facility Name
Hopital Robert Debre
City
Paris
Country
France
Facility Name
Centre Hospitalier Lyon Sud
City
Pierre-Bénite
Country
France
Facility Name
Centre de Perharidy
City
Roscoff cedex
Country
France
Facility Name
CHU de Rouen - Hopital Charles Nicolle
City
Rouen Cedex, Seine Maritime
Country
France
Facility Name
Hôpital de Hautepierre, AX5
City
Strasbourg
Country
France
Facility Name
Hopital Foch (Suresnes), Hopital Foch, Adultes
City
Suresnes
Country
France
Facility Name
Hopital Bretonneau
City
Tours
Country
France
Facility Name
Centre hospitalier universitaire de Nancy, Hôpital de Braboi
City
Vandoeuvre Les Nancy
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
City
Erlangen
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Facility Name
Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin
City
Gießen
Country
Germany
Facility Name
Pneumologisches Studienzentrum Muenchen-West
City
Muenchen
Country
Germany
Facility Name
General Hospital of Thessaloniki "Papanikolaou"
City
Pilea Chortiatis
Country
Greece
Facility Name
General Hospital of Thessaloniki 'Hippokratio', 3rd Pediatrics Department of Aristotle University of Thessaloniki
City
Thessaloníki
Country
Greece
Facility Name
National Koranyi Institute for TBC and Pulmonology
City
Budapest
Country
Hungary
Facility Name
Pulmonology Institute Torokbalint
City
Torokbalint
Country
Hungary
Facility Name
Cork University Hospital
City
Cork
Country
Ireland
Facility Name
Children's Health Ireland at Crumlin
City
Dublin
Country
Ireland
Facility Name
Children's Health Ireland at Tallaght
City
Dublin
Country
Ireland
Facility Name
Children's University Hospital, Dublin
City
Dublin
Country
Ireland
Facility Name
St. Vincent's University Hospital
City
Dublin
Country
Ireland
Facility Name
University Hospital Limerick (Adults)
City
Limerick
Country
Ireland
Facility Name
University Hospital Limerick (Pediatrics)
City
Limerick
Country
Ireland
Facility Name
Hadassah Medical Organization
City
Jerusalem
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel
City
Petach Tikvah
Country
Israel
Facility Name
Sheba Medical Center - The Edmond and Lili Safra Children's Hospital
City
Tel HaShomer
Country
Israel
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
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
Country
Italy
Facility Name
Fondazione IRCCS Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena
City
Milan
Country
Italy
Facility Name
Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica
City
Orbassano
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
Academisch Medisch Centrum (Academic Medical Centre)
City
Amsterdam
Country
Netherlands
Facility Name
HagaZiekenhuis van den Haag
City
Den Haag
Country
Netherlands
Facility Name
UMC St. Radboud
City
Nijmegen
Country
Netherlands
Facility Name
Erasmus Medical Center
City
Rotterdam
Country
Netherlands
Facility Name
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
City
Utrecht
Country
Netherlands
Facility Name
Greenlane Clinical Centre
City
Auckland
Country
New Zealand
Facility Name
Starship Children's Hospital
City
Auckland
Country
New Zealand
Facility Name
Canterbury Respiratory Research Group
City
Christchurch Hospital
Country
New Zealand
Facility Name
Waikato Hospital
City
Hamilton
Country
New Zealand
Facility Name
Haukeland Universitetssjukehus (CF)
City
Bergen
Country
Norway
Facility Name
Oslo University Hospital, Department of Paediatric Medicine
City
Oslo
Country
Norway
Facility Name
Pediatric Hospital Polanki named of Maciej Płażyński
City
Gdańsk
Country
Poland
Facility Name
Institute of Tuberculosis and Lung Diseases
City
Rabka Zdrój
Country
Poland
Facility Name
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
City
Łomianki
Country
Poland
Facility Name
Sahlgrenska Universitetssjukhuset
City
Göteborg
Country
Sweden
Facility Name
Lund University Skanes Universitetssjukhus
City
Malmoe
Country
Sweden
Facility Name
Karolinska Universitetssjukhuset, Huddinge
City
Stockholm
Country
Sweden
Facility Name
Lindenhofspital - Quartier Bleu
City
Bern
Country
Switzerland
Facility Name
Réseau Hospitalier Neuchâtelois
City
Neuchâtel
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zürich
Country
Switzerland
Facility Name
Birmingham Children's Hospital
City
Birmingham
Country
United Kingdom
Facility Name
Royal Papworth Hospital NHS Foundation Trust
City
Cambridge
Country
United Kingdom
Facility Name
Clinical Research Facility, Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Facility Name
Leeds General Infirmary
City
Leeds
Country
United Kingdom
Facility Name
St. James University Hospital
City
Leeds
Country
United Kingdom
Facility Name
King's College Hospital
City
London
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
St. Bartholomew's Hospital
City
London
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
Country
United Kingdom
Facility Name
Wythenshawe Hospital
City
Manchester
Country
United Kingdom
Facility Name
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
City
Penarth
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Learn more about this trial

A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation

We'll reach out to this number within 24 hrs