Study Stopped
At Sponsor's Discretion
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
2 other identifiers
interventional
484
11 countries
100
Brief Summary
This study will evaluate the long-term safety and tolerability of VX-659 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 Jul 2018
100 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
February 21, 2018
CompletedFirst Posted
Study publicly available on registry
February 27, 2018
CompletedStudy Start
First participant enrolled
July 13, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 9, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 9, 2020
CompletedResults Posted
Study results publicly available
January 25, 2022
CompletedJanuary 25, 2022
December 1, 2021
2.2 years
February 21, 2018
November 9, 2021
December 31, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
From Day 1 up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First in the Current Study 659-105 (up to Week 100)
Secondary Outcomes (16)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for Participants From the Parent Study 659-102
From Baseline at Week 72 (Study 659-105)
Absolute Change in ppFEV1 for Participants From the Parent Study 659-103
From Baseline at Week 72 (Study 659-105)
Absolute Change in Sweat Chloride (SwCl) for Participants From the Parent Study 659-102
From Baseline at Week 24 (Study 659-105)
Absolute Change in SwCl for Participants From the Parent Study 659-103
From Baseline at Week 24 (Study 659-105)
Number of Pulmonary Exacerbations (PEx) for Participants From the Parent Study 659-102
From Baseline up to Week 96 (Study 659-105)
- +11 more secondary outcomes
Study Arms (1)
VX-659/TEZ/IVA TC
EXPERIMENTALParticipants from parent studies VX17-659-102 (NCT03447249) or VX17-659-103 (NCT03460990) were administered VX-659 240 milligrams (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the TC treatment period for up to 96 weeks in the current study VX17-659-105.
Interventions
Fixed-dose combination tablets for oral administration qd in the morning.
Eligibility Criteria
You may qualify if:
- Completed study drug treatment in a parent study; 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 (100)
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 Medical Center
New Haven, Connecticut, 06511, United States
University of Miami Miller School of Medicine
Miami, Florida, 33136, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
Orlando Health, Inc.- Arnold Palmer Hospital for Children (APH)
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, 83712, United States
Cystic Fibrosis Center of Chicago
Glenview, Illinois, 60025, United States
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, 60714, United States
Indiana Clinical Research Center, IU Health University Hospital
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
Spectrum Health Medical Group Adult Cystic Fibrosis Care Center
Grand Rapids, Michigan, 49546, 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, Manchester
Manchester, New Hampshire, 03104, United States
Rutgers-Robert Wood Johnson Medical School
New Brunswick, New Jersey, 08901, United States
Albany Medical College
Albany, New York, 12208, United States
CF Therapeutics Development Center of Western New York
Buffalo, New York, 14203, 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
Cincinnati, Ohio, 45229, United States
Santiago Reyes, M.D.
Oklahoma City, Oklahoma, 73112, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Drexel University College of Medicine / Drexel Adult Cystic Fibrosis Center
Philadelphia, Pennsylvania, 19107, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Sanford Children's Specialty Clinic
Sioux Falls, South Dakota, 57105, United States
University of Tennessee Medical Center- Adult Cystic Fibrosis Clinic
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, Australia
Institute for Respiratory Health, Sir Charles Gairdner Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton, Australia
Telethon Kids Institute
Perth, Australia
Sydney Children's Hospital
Randwick, Australia
Lady Cilento 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
Clinic of J.W. Goethe University
Frankfurt, Germany
Medizinische Hochschule Hannover
Hanover, Germany
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
Jena, Germany
Universitatsklinikum Schleswig-Holstein, Klinik für Kinder- und Jugendmedizin
Lübeck, Germany
Klinikum Innenstadt, University of Munich
München, Germany
Pneumologische Praxis Pasing
München, Germany
Cork University Hospital
Cork, Ireland
Beaumont Hospital
Dublin, Ireland
Our Lady's Children's Hospital
Dublin, Ireland
St. Vincent's University Hospital
Dublin, Ireland
Temple Street Children's University Hospital
Dublin, Ireland
University Hospital Galway
Galway, Ireland
University Hospital Limerick
Limerick, Ireland
Lady Davis Carmel Medical Center
Haifa, Israel
Rambam Health Care Campus, Liver Unit
Haifa, Israel
Pediatrics Hadassah Medical Center
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
Clinical Research Facility, Queen Elizabeth University Hospital
Glasgow, United Kingdom
St. James University Hospital
Leeds, United Kingdom
Liverpool Head and Chest Hospital
Liverpool, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, United Kingdom
Wythenshaw e Hospital
Manchester, United Kingdom
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
Newcastle upon Tyne, United Kingdom
Wolfson Cystic Fibrosis Unit, City Campus
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
- Restriction Type
- OTHER
- 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
Study Record Dates
First Submitted
February 21, 2018
First Posted
February 27, 2018
Study Start
July 13, 2018
Primary Completion
September 9, 2020
Study Completion
September 9, 2020
Last Updated
January 25, 2022
Results First Posted
January 25, 2022
Record last verified: 2021-12