A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
NCT ID: NCT03537651
Last Updated: 2024-04-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
130 participants
INTERVENTIONAL
2018-04-25
2023-09-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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TEZ/IVA
Part A: Participants weighing less than (\<)40 kilograms (kg) at Day 1 received tezacaftor (TEZ) 50 milligrams (mg) once daily (qd)/ivacaftor (IVA) 75 mg every 12 hours (q12h) and the participants weighing greater than or equals to (\>=) 40 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Doses were adjusted upward for changes in body weight and/or age.
Part B: Participants weighing \<30 kg at Day 1 received TEZ 50 mg qd/IVA 75 mg q12h and the participants weighing \>=30 kg at Day 1 received TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 192 weeks. Doses were adjusted upward for changes in body weight and/or age.
TEZ/IVA
Fixed-dose combination tablet for oral administration.
IVA
Tablet for oral administration.
Interventions
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TEZ/IVA
Fixed-dose combination tablet for oral administration.
IVA
Tablet for oral administration.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Eligible CFTR Mutation
Exclusion Criteria
* History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator
* Ongoing participation in another study with investigational drug
6 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Providence Alaska Medical Center
Anchorage, Alaska, United States
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Nemours/ Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Johns Hopkins All Children's Hospital Outpatient Care Center
St. Petersburg, Florida, United States
Center for Advanced Pediatrics
Atlanta, Georgia, United States
St. Luke's CF Center of Idaho
Boise, Idaho, United States
Riley Hospital for Children Indiana University Health
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Hospital & Clinics of Minnesota
Minneapolis, Minnesota, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Dartmouth Hitchcock Medical Center
Manchester, New Hampshire, United States
UBMD Pediatrics/ CF Center of Western New York
Buffalo, New York, United States
Columbia University Medical Center
New York, New York, United States
SUNY Upstate Medical University
Syracuse, New York, United States
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, United States
Austin Children's Chest Associates
Austin, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Perth Children's Hospital
Nedlands, , Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton, , Australia
Lady Cilento Children's Hospital
South Brisbane, , Australia
The Children's Hospital at Westmead
Westmead, , Australia
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, , Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, , Belgium
British Columbia's Children's Hospital
Vancouver, British Columbia, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Quebec, Canada
Juliane Marie Center, Rigshopitalet
Copenhagen, , Denmark
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux, , France
Hopital Necker, Enfants Malades
Paris, , France
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne, , Germany
Universitätsklinikum Essen
Essen, , Germany
Clinic of J.W. Goethe University
Frankfurt, , Germany
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
Jena, , Germany
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
Tübingen, , Germany
Our Lady's Children's Hospital
Dublin, , Ireland
University Hospital Limerick
Limerick, , Ireland
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Łomianki, , Poland
Inselspital - Universitaetsspital Bern
Bern, , Switzerland
Kinderspital Zuerich
Zurich, , Switzerland
Leeds General Infirmary
Leeds, , United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Countries
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References
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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.
Sawicki GS, Chilvers M, McNamara J, Naehrlich L, Saunders C, Sermet-Gaudelus I, Wainwright CE, Ahluwalia N, Campbell D, Harris RS, Paz-Diaz H, Shih JL, Davies JC. A Phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children >/= 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant. J Cyst Fibros. 2022 Jul;21(4):675-683. doi: 10.1016/j.jcf.2022.02.003. Epub 2022 Feb 18.
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. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2017-002968-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX17-661-116
Identifier Type: -
Identifier Source: org_study_id
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