Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
2 other identifiers
interventional
251
12 countries
84
Brief Summary
This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Dec 2019
Typical duration for phase_3
84 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 14, 2019
CompletedFirst Posted
Study publicly available on registry
August 15, 2019
CompletedStudy Start
First participant enrolled
December 5, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 16, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
December 16, 2022
CompletedResults Posted
Study results publicly available
January 16, 2024
CompletedJanuary 16, 2024
December 1, 2023
3 years
August 14, 2019
December 15, 2023
December 15, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Baseline up to Week 100
Secondary Outcomes (6)
Part A: Absolute Change From Parent Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)
From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI)
From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in BMI Z-score
From Baseline at Week 96
Part A: Absolute Change From Parent Study Baseline in Body Weight
From Baseline at Week 96
- +1 more secondary outcomes
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALPart A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks.
Interventions
Fixed-dose combination (FDC) tablet for oral administration.
Eligibility Criteria
You may qualify if:
- Completed study drug treatment in parent study (VX18-445-104); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
You may not qualify if:
- History of study drug intolerance in parent study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (84)
Banner University of Arizona Medical Center
Tucson, Arizona, 85724, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806, United States
Stanford University
Palo Alto, California, 94304, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of California San Francisco, Lung Transplant Program
San Francisco, California, 94143, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803, United States
Indiana University
Indianapolis, Indiana, 46202, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center
Boston, Massachusetts, 02114, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Michigan Medicine
Ann Arbor, Michigan, 48109-5212, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Mount Sinai Beth Israel
New York, New York, 10003, United States
Columbia University Medical Center
New York, New York, 10032, United States
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514, United States
UC Health Holmes
Cincinnati, Ohio, 45220, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center
Toledo, Ohio, 43606, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Dell Children's Medical Group
Austin, Texas, 78723, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, Australia
Telethon Kids Institute
Nedlands, Australia
The Royal Children's Hospital
Parkville, Australia
Mater Adult Hospital
South Brisbane, Australia
Queensland Children's Hospital
South Brisbane, Australia
Westmead Hospital
Westmead, Australia
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
McGill University Health Center
Montreal, Canada
St. Paul's Hospital
Vancouver, Canada
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Centre Hospitalier Lyon Sud
Benite Cedex, France
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux, France
CHRU de Lille - Hopital Albert Calmette
Lille, France
CHU Marseille - Hopital Nord
Marseille, France
CHU de Montpellier - Hopital Arnaud de Villeneuve
Montpellier, France
Hopital Cochin
Paris, France
Hopital Necker, Enfants Malades
Paris, France
Hopital Pontchaillou CHU de Rennes
Rennes, France
Charite Paediatric Pulmonology Department
Berlin, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen, Germany
Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie
Halle, Germany
Pneumologisches Studienzentrum Muenchen-West
München, Germany
University Hospital Wuerzburg
Würzburg, Germany
Beaumont Hospital
Dublin, Ireland
Children's Health Ireland at Crumlin
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
University Hospital Limerick (Adults)
Limerick, Ireland
Azienda Ospedaliero Universitaria Ospedale Riuniti
Ancona, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Ospedale Pediatrico Bambino Gesu
Rome, Italy
Azienda Ospedaliera di Verona-Ospedale Civile Maggiore
Verona, Italy
Academic Medical Center
Amsterdam, Netherlands
University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis
Heidelberglaan, Netherlands
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Virgen de la Arrixaca
Murcia, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, United Kingdom
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Exeter, United Kingdom
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Liverpool Heart and Chest Hospital
Liverpool, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, United Kingdom
Wythenshawe Hospital
Manchester, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Related Publications (2)
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.
PMID: 37983082DERIVEDSouthern 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.
PMID: 33331662DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
- Expanded Access
- Yes
Study Record Dates
First Submitted
August 14, 2019
First Posted
August 15, 2019
Study Start
December 5, 2019
Primary Completion
December 16, 2022
Study Completion
December 16, 2022
Last Updated
January 16, 2024
Results First Posted
January 16, 2024
Record last verified: 2023-12
Data Sharing
- IPD Sharing
- Will not share
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.