A 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 Homozygous or Heterozygous for the F508del Mutation
2 other identifiers
interventional
507
12 countries
106
Brief Summary
The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation
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 Oct 2018
Typical duration for phase_3
106 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
May 2, 2018
CompletedFirst Posted
Study publicly available on registry
May 15, 2018
CompletedStudy Start
First participant enrolled
October 9, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 9, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
January 9, 2023
CompletedResults Posted
Study results publicly available
May 8, 2024
CompletedSeptember 24, 2025
September 1, 2025
4.3 years
May 2, 2018
January 8, 2024
September 22, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 196
Secondary Outcomes (16)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 102/105 Efficacy Set
From Baseline at Week 192
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/105 Efficacy Set
From Baseline at Week 192
Absolute Change in Sweat Chloride (SwCl) for 102/105 Efficacy Set
From Baseline at Week 192
Absolute Change in Sweat Chloride (SwCl) for 103/105 Efficacy Set
From Baseline at Week 192
Number of Pulmonary Exacerbations (PEx) for 102/105 Efficacy Set
From Baseline at Week 192
- +11 more secondary outcomes
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALTreatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks. Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks.
Interventions
Fixed-dose combination (FDC) tablet for oral administration.
Eligibility Criteria
You may qualify if:
- Completed study drug treatment in a parent study i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); or had study drug interruption(s) in a 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 drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
- Current participation in an investigational drug trial (other than a parent study)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (110)
Banner University of Arizona Medical Center
Tucson, Arizona, 85724, United States
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, 90806, United States
Children's Hospital Los Angeles
Los Angeles, California, 90027, United States
Valley Children's Hospital
Madera, California, 93636, United States
Kaiser Permanente
Oakland, California, 94611, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
UCSF Gateway Medical Center
San Francisco, California, 94143, United States
National Jewish Health
Denver, Colorado, 80206, United States
University of Florida, Shands Hospital
Gainesville, Florida, 32610, United States
Joe DiMaggio Cystic Fibrosis & Pulmonary Center
Hollywood, Florida, 33021, United States
Nemours Children's Specialty Care
Jacksonville, Florida, 32207, United States
Central Florida Pulmonary Group, PA
Orlando, Florida, 32803, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Tampa General Hospital Cardiac and Lung Transplant Clinic
Tampa, Florida, 33606, United States
Children's Specialty Services at North Druid Hills
Atlanta, Georgia, 30324, United States
Augusta University
Augusta, Georgia, 30912, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Northwestern Memorial Hospital
Chicago, Illinois, 60611, United States
Cystic Fibrosis Center, Children's Hospital of Illinois at OSF Saint Francis Medical Center
Peoria, Illinois, 61637, United States
The University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Tulane Medical Center
New Orleans, Louisiana, 70112, United States
Maine Medical Partners
Portland, Maine, 04102, United States
Massachusetts General Hospital Cystic Fibrosis Center Clinical Research Center
Boston, Massachusetts, 02114, United States
Harper University Hospital
Detroit, Michigan, 48201, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Billings Clinic Hospital
Billings, Montana, 59101, United States
Nebraska Medical Center
Omaha, Nebraska, 68198, United States
Morristown Medical Center
Morristown, New Jersey, 07960, United States
UNM Clinical and Translational Science Center
Albuquerque, New Mexico, 87131, United States
The Cystic Fibrosis Center, Mount Sinai Beth Israel
New York, New York, 10003, United States
New York Medical College
Valhalla, New York, 10595, United States
UNC Marsico Clinical Research Center
Chapel Hill, North Carolina, 27517, United States
Akron Children's Hospital
Akron, Ohio, 44308, 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
Dayton Children's Hospital
Dayton, Ohio, 45404, 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
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
Vermont Lung Center
Colchester, Vermont, 05446, United States
University of Virginia Primary Care Center
Charlottesville, Virginia, 22908, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
Children's Hospital of Richmond at VCU, Children's Pavilion
Richmond, Virginia, 23298, United States
University of Washington Medical Center
Seattle, Washington, 98195, United States
West Virginia University
Morgantown, West Virginia, 26506, United States
University of Wisconsin Hospitals and Clinics
Madison, Wisconsin, 53792, United States
CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin, 53226, United States
Women & Children's Hospital
North Adelaide, Australia
The Royal Children's Hospital
Parkville, Australia
Mater Misericordiae Ltd
South Brisbane, Australia
The Children's Hospital at Westmead
Westmead, Australia
Westmead Hospital
Westmead, Australia
University of Graz
Graz, Austria
Medizinische Universität Innsbruck
Innsbruck, Austria
LKH - Universitätsklinikum der PMU Salzburg
Salzburg, Austria
Medizinische Universitat Wien
Vienna, Austria
Cliniques Universitaires de Bruxelles Hopital Erasme
Brussels, Belgium
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
UZ Antwerpen
Edegem, Belgium
Universitair Ziekenhuis Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
University of Calgary Medical Clinic of the Foothills Medical Centre
Calgary, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Canada
Centre Hospitalier De L'Universite Laval
Québec, Canada
The Hospital for Sick Children
Toronto, Canada
British Columbia's Children's Hospital
Vancouver, Canada
St. Paul's Hospital
Vancouver, Canada
Fakultni Nemocnice Brno
Brno, Czechia
Fakultni nemocnice v Motole
Prague, Czechia
Centre Hospitalier Lyon Sud
Benite Cedex, France
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux, France
CHU Marseille - Hopital Nord
Marseille, France
CHU de Nice - Hopital Pasteur
Nice, France
Hopital Cochin
Paris, France
CHU de Rouen - Hopital Charles Nicolle
Rouen, France
Hopital Foch (Suresnes), Hopital Foch, Adultes
Suresnes, France
Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital
Erlangen, Germany
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen, Germany
Hannover Medical School
Hanover, Germany
Johannes Gutenberg-Universitaet
Mainz, Germany
Dr. von Haunersches Kinderspital
München, Germany
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
Tübingen, Germany
University Hospital Wuerzburg
Würzburg, Germany
General Hospital of Attika "Sismanoglio"(Adult CF center, NHS)
Marousi, Greece
Azienda Ospedaliero Universitaria Ospendali Riuniti
Ancona, Italy
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence, Italy
IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico
Genova, Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo
Potenza, 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
UMC St. Radboud
Nijmegen, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
HagaZiekenhuis van den Haag
The Hague, Netherlands
Karolinska Universitetssjukhuset, Huddinge
Stockholm, Sweden
Belfast City Hospital
Belfast, United Kingdom
Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital
Birmingham, United Kingdom
Western General Hospital
Edinburgh, United Kingdom
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
Exeter, United Kingdom
Leeds General Infirmary
Leeds, United Kingdom
King's College Hospital
London, United Kingdom
Royal Manchester Children's Hospital
Manchester, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Related Publications (3)
Daines CL, Polineni D, Tullis E, Costa S, Linnemann RW, Mall MA, McKone EF, Quon BS, Ringshausen FC, Selvadurai H, Taylor-Cousar JL, Withers NJ, Sawicki GS, Lee T, Ahluwalia N, Morlando Geiger J, Jennings M, Tan YV, Waltz D, Ramsey B, Griese M; VX17-445-105 Study Group. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Adults and Adolescents with Cystic Fibrosis and at Least One F508del Allele: A Phase 3 Open-Label Extension Study. Am J Respir Crit Care Med. 2025 Oct;211(10):1901-1914. doi: 10.1164/rccm.202411-2231OC.
PMID: 40209082DERIVEDHeneghan 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
May 2, 2018
First Posted
May 15, 2018
Study Start
October 9, 2018
Primary Completion
January 9, 2023
Study Completion
January 9, 2023
Last Updated
September 24, 2025
Results First Posted
May 8, 2024
Record last verified: 2025-09