Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
NCT ID: NCT01705145
Last Updated: 2016-04-05
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
35 participants
INTERVENTIONAL
2013-01-31
2014-03-31
Brief Summary
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Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.
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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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Ivacaftor
Part A: Ivacaftor 50 milligram (mg) (for participants weighing less than \[\<\] 14 kilograms \[kg\]) or 75 mg (for participants weighing greater than or equal to \[\>=\] 14 kg) every 12 hours (q12h) from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study..
Part B: Ivacaftor 50 mg (for participants weighing \<14 kg) or 75 mg (for participants weighing \>=14 kg) q12h for 24 weeks during Part B of the study.
Ivacaftor
Interventions
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Ivacaftor
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Must have a CFTR gating mutation in at least 1 allele
* Aged 2 through 5 years at screening and Day 1
* Weight \>= 8 kg at screening and Day 1
* Hematology, serum chemistry, coagulation, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
Exclusion Criteria
* An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1
* Abnormal liver function, at screening
* History of solid organ or hematological transplantation
* Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
* Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening
2 Years
5 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Principal Investigators
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Margaret Rosenfeld, MD
Role: PRINCIPAL_INVESTIGATOR
Seattle Children's Hospital
Jane Davies, MD
Role: PRINCIPAL_INVESTIGATOR
Imperial College London
Locations
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Birmingham, Alabama, United States
Aurora, Colorado, United States
Atlanta, Georgia, United States
Indianapolis, Indiana, United States
Lexington, Kentucky, United States
Boston, Massachusetts, United States
Detroit, Michigan, United States
Grand Rapids, Michigan, United States
Minneapolis, Minnesota, United States
Kansas City, Missouri, United States
Omaha, Nebraska, United States
Pittsburgh, Pennsylvania, United States
Salt Lake City, Utah, United States
Charlottesville, Virginia, United States
Richmond, Virginia, United States
Seattle, Washington, United States
Vancouver, , Canada
Edinburgh, , United Kingdom
Liverpool, , United Kingdom
London, , United Kingdom
Countries
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References
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Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Robertson S, Green Y, Cooke J, Rosenfeld M; KIWI Study Group. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016 Feb;4(2):107-15. doi: 10.1016/S2213-2600(15)00545-7. Epub 2016 Jan 21.
Other Identifiers
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KIWI
Identifier Type: -
Identifier Source: secondary_id
VX11-770-108
Identifier Type: -
Identifier Source: org_study_id
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