Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
NCT ID: NCT03601637
Last Updated: 2023-01-06
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
61 participants
INTERVENTIONAL
2018-09-07
2021-10-29
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Part A: LUM/IVA
Participants weighing 7 to less than (\<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to \<14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
IVA
FDC granules (LUM/IVA).
Part B: LUM/IVA
Participants weighing 7 to \<9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to \<14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing \>=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
IVA
FDC granules (LUM/IVA).
Interventions
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LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
IVA
FDC granules (LUM/IVA).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Homozygous for F508del (F/F)
Exclusion Criteria
* Solid organ or hematological transplantation
12 Months
23 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
Stanford University
Palo Alto, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Yale New Haven Medical Center
New Haven, Connecticut, United States
Nemours / Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
Johns Hopkins Hospital
Baltimore, Maryland, 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
University of North Carolina Hospitals
Chapel Hill, North Carolina, United States
Wake Forest University School of Medicine - Brenner Children's Hospital
Winston-Salem, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 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.
Rayment JH, Asfour F, Rosenfeld M, Higgins M, Liu L, Mascia M, Paz-Diaz H, Tian S, Zahigian R, McColley SA. A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
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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2017-004794-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX16-809-122
Identifier Type: -
Identifier Source: org_study_id
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