Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
NCT ID: NCT04183790
Last Updated: 2025-05-18
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE3
64 participants
INTERVENTIONAL
2020-02-17
2024-02-24
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
NCT04537793
A Study Evaluating the Long-term Safety of VX-445 Combination Therapy
NCT04043806
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
NCT05153317
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
NCT06460506
Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT03691779
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
ELX/TEZ/IVA
Participants greater than or equal to (≥) 6 years and less than (\<) 12 years of age and weighing \<30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
IVA
Mono tablet for oral administration.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
ELX/TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration
IVA
Mono tablet for oral administration.
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
Exclusion Criteria
6 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital of Orange County
Orange, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
The Children's Mercy Hospital
Kansas City, Missouri, United States
Northwell Health- Long Island Jewish Medical Center
New Hyde Park, New York, United States
Clinical Research of Charlotte
Charlotte, North Carolina, United States
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Oregon Health & Science University
Portland, Oregon, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Queensland Children's Hospital
South Brisbane, , Australia
The Children's Hospital at Westmead
Westmead, , Australia
The Hospital for Sick Children
Toronto, , Canada
British Columbia's Children's Hospital
Vancouver, , Canada
Children's Health Ireland at Crumlin
Dublin, , Ireland
Children's Health Ireland at Temple Street
Dublin, , Ireland
Birmingham Children's Hospital - NHS Foundation Trust
Birmingham, , United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ramsey B, McKone EF, Tullis E, Mall MA, Taylor-Cousar JL, Waltz D, Ahluwalia N, Chu C, Scirica CV, Davies JC. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children >/=6 Years with Cystic Fibrosis and at Least One F508del Allele: A 192-Week, Phase 3, Open-Label Extension Study. Am J Respir Crit Care Med. 2025 Oct;211(10):1915-1925. doi: 10.1164/rccm.202502-0512OC.
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.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, Zelazoski L, Davies JC. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least One F508del Allele: A Phase 3, Open-Label Clinical Trial. Am J Respir Crit Care Med. 2023 Jul 1;208(1):68-78. doi: 10.1164/rccm.202301-0021OC.
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
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2019-001827-11
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX19-445-107
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.