Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
NCT ID: NCT03691779
Last Updated: 2021-10-22
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
71 participants
INTERVENTIONAL
2018-10-02
2020-08-07
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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 in Part A received ELX 100 milligrams (mg) once daily (qd)/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) in the treatment period for 15 days.
ELX/TEZ/IVA
Fixed-dose combination tablet orally once daily in the morning.
IVA
IVA tablet orally once daily in the evening.
Part B: ELX/TEZ/IVA
Participants in Part B weighing less than (\<) 30 kilograms (kg) at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing greater than equals to (\>=) 30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.
ELX/TEZ/IVA
Fixed-dose combination tablet orally once daily in the morning.
IVA
IVA tablet orally once daily in the evening.
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 tablet orally once daily in the morning.
IVA
IVA tablet orally once daily in the evening.
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
* Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.
Exclusion Criteria
* Lung infection with organisms associated with a more rapid decline in pulmonary status.
* Solid organ or hematological transplantation.
6 Years
11 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
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.
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.
Zemanick ET, Taylor-Cousar JL, Davies J, Gibson RL, Mall MA, McKone EF, McNally P, Ramsey BW, Rayment JH, Rowe SM, Tullis E, Ahluwalia N, Chu C, Ho T, Moskowitz SM, Noel S, Tian S, Waltz D, Weinstock TG, Xuan F, Wainwright CE, McColley SA. A Phase 3 Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 through 11 Years of Age with Cystic Fibrosis and at Least One F508del Allele. Am J Respir Crit Care Med. 2021 Jun 15;203(12):1522-1532. doi: 10.1164/rccm.202102-0509OC.
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.
2018-001695-38
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX18-445-106
Identifier Type: -
Identifier Source: org_study_id