- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03537651
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation
October 4, 2023 updated by: Vertex Pharmaceuticals Incorporated
A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
This study will evaluate the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.
Study Overview
Status
Active, not recruiting
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
130
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Nedlands, Australia
- Perth Children's Hospital
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New Lambton, Australia
- John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
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South Brisbane, Australia
- Lady Cilento Children's Hospital
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Westmead, Australia
- The Children's Hospital at Westmead
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Brussels, Belgium
- Universitair Ziekenhuis Brussel - Campus Jette
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Leuven, Belgium
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
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British Columbia
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Vancouver, British Columbia, Canada
- British Columbia's Children's Hospital
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Ontario
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Toronto, Ontario, Canada
- The Hospital for Sick Children
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Quebec
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Montreal, Quebec, Canada
- McGill University Health Centre, Glen Site, Montreal Children's Hospital
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Copenhagen, Denmark
- Juliane Marie Center, Rigshopitalet
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Bordeaux cedex, France
- Groupe Hospitaler Pellegrin, CHU De Bordeaux
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Paris Cedex 15, France
- Hopital Necker, Enfants Malades
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Essen, Germany
- Universitatsklinikum Essen
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Frankfurt, Germany
- Clinic of J.W. Goethe University
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Giessen, Germany
- Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
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Hannover, Germany
- Medizinische Hochschule Hannover
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Jena, Germany
- Universitaetsklinikum Jena, Mukoviszidose-Zentrum
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Koeln, Germany
- Universitaetsklinkum Koeln, CF-Studienzentrum
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Tuebingen, Germany
- Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
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Dublin, Ireland
- Our Lady's Children's Hospital
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Limerick, Ireland
- University Hospital Limerick
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Lomianki, Poland
- Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
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Bern, Switzerland
- Inselspital - Universitaetsspital Bern
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Zürich, Switzerland
- Kinderspital Zuerich
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Leeds, United Kingdom
- Leeds General Infirmary
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London, United Kingdom
- Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
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Southampton, United Kingdom
- Southampton General Hospital
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Alabama
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Birmingham, Alabama, United States, 35233
- University of Alabama at Birmingham
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Alaska
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Anchorage, Alaska, United States, 99508
- Providence Alaska Medical Center
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Arkansas
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Little Rock, Arkansas, United States, 72202
- Arkansas Children's Hospital
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California
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Los Angeles, California, United States, 90027
- Children's Hospital Los Angeles
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Colorado
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Aurora, Colorado, United States, 80045
- Children's Hospital Colorado
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Delaware
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Wilmington, Delaware, United States, 19803
- Nemours/ Alfred I. duPont Hospital for Children
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Florida
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Saint Petersburg, Florida, United States, 33701
- Johns Hopkins All Children's Hospital Outpatient Care Center
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Georgia
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Atlanta, Georgia, United States, 30329
- Center for Advanced Pediatrics
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Idaho
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Boise, Idaho, United States, 83702
- St. Luke's CF Center of Idaho
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Indiana
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Indianapolis, Indiana, United States, 46202
- Riley Hospital for Children Indiana University Health
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Minnesota
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Minneapolis, Minnesota, United States, 55404
- Children's Hospital & Clinics of Minnesota
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Missouri
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Kansas City, Missouri, United States, 64108
- The Children's Mercy Hospital
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New Hampshire
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Manchester, New Hampshire, United States, 03756
- Dartmouth Hitchcock Medical Center
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New York
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Buffalo, New York, United States, 14203
- UBMD Pediatrics/ CF Center of Western New York
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New York, New York, United States, 10032
- Columbia University Medical Center
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Syracuse, New York, United States, 13202
- SUNY Upstate Medical University
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North Carolina
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Winston-Salem, North Carolina, United States, 27157
- Wake Forest Baptist Health
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Ohio
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Cleveland, Ohio, United States, 44106
- Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
- Children's Hospital of Pittsburgh of UPMC
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South Carolina
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Charleston, South Carolina, United States, 29425
- Medical University of South Carolina (MUSC)
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South Dakota
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Sioux Falls, South Dakota, United States, 57105
- Sanford Children's Speciality Clinic
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Texas
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Austin, Texas, United States, 78723
- Austin Children's Chest Associates
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Fort Worth, Texas, United States, 76104
- Cook Children's Medical Center
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Houston, Texas, United States, 77030
- Baylor College of Medicine
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Virginia
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Norfolk, Virginia, United States, 23507
- Children's Hospital of The King's Daughters
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Washington
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Seattle, Washington, United States, 98105
- Seattle Children's Hospital
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Wisconsin
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Milwaukee, Wisconsin, United States, 53226
- Children's Hospital of Wisconsin
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
6 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115.
- Eligible CFTR Mutation.
Exclusion Criteria:
- Pregnant and nursing females.
- 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.
Other protocol defined Inclusion/Exclusion criteria may apply.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: TEZ/IVA
TEZ 50 mg once daily (qd)/IVA 75 mg every 12 hours (q12h) or TEZ 100 mg qd/IVA 150 mg q12h based on body weight for participants aged 6 through 11 years at enrollment and TEZ 100 mg qd/IVA 150 mg q12h for participants aged >=12 years at enrollment.
Doses were adjusted upward for changes in body weight and/or age.
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Tablet for oral administration.
Other Names:
Fixed-dose combination tablet for oral administration.
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Part A: Day 1 up to Week 100
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Part A: Day 1 up to Week 100
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group)
Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116)
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LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS
Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116)
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LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Part A: Absolute Change in Sweat Chloride for 115/116 FAS (TEZ/IVA Group)
Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116)
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Sweat samples were collected using an approved collection device.
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Part A: Absolute Change in SwCl for 113B/116 FAS
Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116)
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Sweat samples were collected using an approved collection device.
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group)
Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116)
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF.
Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS
Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116)
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The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF.
Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group)
Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116)
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BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2).
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From Parent Study 115 Baseline at Week 96 (Study 116)
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Part A: Absolute Change in BMI for 113B/116 FAS
Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116)
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BMI was defined as weight in kg divided by m^2.
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From Parent Study 113B Baseline at Week 96 (Study 116)
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
General Publications
- 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.
- 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.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 25, 2018
Primary Completion (Actual)
October 28, 2020
Study Completion (Estimated)
July 1, 2024
Study Registration Dates
First Submitted
May 15, 2018
First Submitted That Met QC Criteria
May 24, 2018
First Posted (Actual)
May 25, 2018
Study Record Updates
Last Update Posted (Actual)
October 5, 2023
Last Update Submitted That Met QC Criteria
October 4, 2023
Last Verified
October 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- VX17-661-116
- 2017-002968-40 (EudraCT Number)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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