A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
A Phase 3, Open-label Study Evaluating the Long-term Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis
2 other identifiers
interventional
458
11 countries
97
Brief Summary
This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).
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 Aug 2019
Typical duration for phase_3
97 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
July 31, 2019
CompletedFirst Posted
Study publicly available on registry
August 2, 2019
CompletedStudy Start
First participant enrolled
August 9, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 14, 2022
CompletedStudy Completion
Last participant's last visit for all outcomes
December 14, 2022
CompletedResults Posted
Study results publicly available
July 6, 2023
CompletedJuly 6, 2023
June 1, 2023
3.4 years
July 31, 2019
June 14, 2023
June 14, 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 through Week 100
Study Arms (1)
ELX/TEZ/IVA
EXPERIMENTALPart A: Participants received ELX 200 milligram (mg) once daily (qd),TEZ 100 mg qd, and 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 received 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:
- Currently participating in study VX17-659-105 (NCT03447262)
You may not qualify if:
- History of drug intolerance in study VX17-659-105 that would pose an additional risk to the participant in the opinion of the investigator
- Current participation in an investigational drug trial (other than study VX17-659-105)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (97)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Stanford University
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Hartford Hospital
Hartford, Connecticut, 06102, United States
Yale New Haven Hospital
New Haven, Connecticut, 06510, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
Arnold Palmer Hospital Pulmonary and Sleep Medicine
Orlando, Florida, 32806, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, 33701, United States
St. Luke's CF Center of Idaho
Boise, Idaho, 83702, United States
Cystic Fibrosis Institute
Glenview, Illinois, 60025, United States
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, 60714, United States
Indiana University
Indianapolis, Indiana, 46202, United States
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
University of Kentucky
Lexington, Kentucky, 40536, United States
Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
UMass Memorial Medical Center
Worcester, Massachusetts, 01655, United States
Michigan Medicine
Ann Arbor, Michigan, 48109-5212, United States
University of Minnesota
Minneapolis, Minnesota, 55455, United States
University of Mississippi Medical Center
Jackson, Mississippi, 39216, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire, 03756, United States
Rutgers Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
Albany Medical College, Pulmonary and Critical Care Medicine
Albany, New York, 12208, United States
Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo
Buffalo, New York, 14222, United States
Northwell Health- Long Island Jewish Medical Center
New Hyde Park, New York, 11040, United States
Columbia University Medical Center
New York, New York, 10032, United States
SUNY Upstate Medical University
Syracuse, New York, 13210, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Santiago Reyes, M.D.
Oklahoma City, Oklahoma, 73112, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, 57105, United States
University of Tennessee Medical Center
Knoxville, Tennessee, 37920, United States
Children's Foundation Research Center / Le Bonheur Children's Hospital
Memphis, Tennessee, 38103, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, 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
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Providence Pediatric Pulmonary & Cystic Fibrosis Clinic
Spokane, Washington, 99204, United States
Royal Adelaide Hospital
Adelaide, Australia
The Prince Charles Hospital
Chermside, Australia
Alfred Hospital
Melbourne, VIC, Australia
Institute for Respiratory Health
Nedlands, Australia
Perth Children's Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton, Australia
Queensland Children's Hospital
South Brisbane, Australia
Stollery Children's Hospital
Edmonton, Canada
Queen Elizabeth II Health Sciences Center
Halifax, Canada
St. Michael's Hospital
Toronto, Canada
Juliane Marie Center, Rigshospitalet
Copenhagen, Denmark
Charite Paediatric Pulmonology Department
Berlin, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne, Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, Germany
Johann Wolfgang Goethe University
Frankfurt, Germany
Hannover Medical School
Hanover, Germany
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
Jena, Germany
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
Lübeck, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Pneumologisches Studienzentrum Muenchen-West
München, Germany
Cork University Hospital
Cork, Ireland
Beaumont Hospital
Dublin, Ireland
Children's Health Ireland at Crumlin
Dublin, Ireland
Children's Health Ireland at Temple Street
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
University Hospital Galway
Galway, Ireland
University Hospital Limerick (Adults)
Limerick, Ireland
Lady Davis Carmel Medical Center
Haifa, Israel
Pediatric Pulmonary Unit Rambam Medical Center
Haifa, Israel
Hadassah Medical Organization
Jerusalem, Israel
Schneider Children's Medical Center
Petah Tikva, Israel
Sheba Medical Center
Tel Litwinsky, Israel
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Łomianki, Poland
Hospital Universitari Vall d Hebron
Barcelona, Spain
Hospital Universitari Vall d´Hebron Servicio de Broncoscopia
Barcelona, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitario Infantil La Paz
Madrid, Spain
Parc Tauli Sabadell Hospital Universitari
Sabadell, Spain
Hospital Universitario Virgen del Rocio
Seville, Spain
Hospital Universitario y Politecnico La Fe
Valencia, Spain
Lindenhofspital - Quartier Bleu
Bern, Switzerland
Kinderspital Zuerich
Zurich, Switzerland
Papworth Hospital NHS Foundation Trust, Papworth Everard
Cambridge, United Kingdom
NHS Greater Glasgow and Clyde, Gartnavel General 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
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Nottingham University Hospitals NHS Trust, Queens Medical Center
Nottingham, United Kingdom
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
Penarth, 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
July 31, 2019
First Posted
August 2, 2019
Study Start
August 9, 2019
Primary Completion
December 14, 2022
Study Completion
December 14, 2022
Last Updated
July 6, 2023
Results First Posted
July 6, 2023
Record last verified: 2023-06
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