Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation
NCT ID: NCT04235140
Last Updated: 2024-09-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
52 participants
INTERVENTIONAL
2020-02-24
2023-08-22
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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LUM/IVA
Participants weighing 7 to less than (\<) 9 kilograms (kg) received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h in the treatment period of 96 weeks. Participants weighing greater than or equal to (\>=)14 kg received LUM 150 mg/IVA 188 mg FDC q12h in the treatment period of 96 weeks.
LUM/IVA
LUM/IVA granules for oral administration
Interventions
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LUM/IVA
LUM/IVA granules for oral administration
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B
* Participants Not From Study 122
* Subjects will be 1 to less than 2 years of age
* Homozygous for the F508del mutation (F/F)
Exclusion Criteria
* Solid organ or hematological transplantation
12 Months
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
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 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
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, United States
University of Rochester Medical Center
Rochester, New York, United States
NC TraCS Institute - CTRC University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Medical Center of Dallas
Dallas, Texas, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, United States
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, , Canada
The Hospital for Sick Children
Toronto, , Canada
British Columbia's Children's Hospital
Vancouver, , Canada
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.
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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VX19-809-124
Identifier Type: -
Identifier Source: org_study_id
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