Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
NCT ID: NCT04537793
Last Updated: 2023-06-28
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
83 participants
INTERVENTIONAL
2020-11-19
2022-06-03
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 Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older
NCT05153317
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
NCT05882357
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
NCT04183790
Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT03691779
A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes
NCT04545515
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.
NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Part A: ELX/TEZ/IVA
Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.
ELX/TEZ/IVA
Fixed dose combination granules for oral administration.
IVA
Granules for oral administration.
Part B: ELX/TEZ/IVA
Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
ELX/TEZ/IVA
Fixed dose combination granules for oral administration.
IVA
Granules 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 granules for oral administration.
IVA
Granules 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
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation
2 Years
5 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.
Banner University of Arizona Medical Center
Tucson, Arizona, United States
Stanford University
Palo Alto, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 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
Washington University School of Medicine / St. Louis Children's Hospital
St Louis, Missouri, United States
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Oregon Health & Science University
Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States
Seattle Children's Hospital
Seattle, Washington, United States
Telethon Kids Institute
Nedlands, , Australia
The Royal Children's Hospital
Parkville, VIC, , Australia
Queensland Children's Hospital
South Brisbane, , Australia
The Hospital for Sick Children
Toronto, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
Charite Paediatric Pulmonology Department
Berlin, , Germany
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
Essen, , Germany
Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
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.
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.
Goralski JL, Hoppe JE, Mall MA, McColley SA, McKone E, Ramsey B, Rayment JH, Robinson P, Stehling F, Taylor-Cousar JL, Tullis E, Ahluwalia N, Chin A, Chu C, Lu M, Niu T, Weinstock T, Ratjen F, Rosenfeld M. Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67. doi: 10.1164/rccm.202301-0084OC.
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.
2020-002251-38
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX20-445-111
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.