Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
NCT ID: NCT01946412
Last Updated: 2017-02-01
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
33 participants
INTERVENTIONAL
2013-12-31
2015-12-31
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
PARALLEL
TREATMENT
NONE
Study Groups
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Observational Arm
No interventions assigned to this group
Ivacaftor
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:
* 50 mg q12h for subjects 2 to \<6 years of age and \<14 kg,
* 75 mg q12h for subjects 2 to \<6 years of age and ≥ 14 kg, or
* 150 mg q12h for subjects ≥ 6 years of age.
Ivacaftor
Interventions
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Ivacaftor
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Hematology, serum chemistry, and vital signs results on Day 1 with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
3. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned
1\. Subjects who completed their assigned study drug treatment in the previous study (NCT01705145) and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.
Exclusion Criteria
2. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
3. Subjects with a history of study treatment intolerance as observed in their previous study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
4. Subjects receiving commercially-available ivacaftor treatment
5. Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study
1\. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
2 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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Adebayo Lawal, M.D.
Role: STUDY_DIRECTOR
Vertex Pharmaceuticals Incorporated
Locations
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Birmingham, Alabama, United States
Denver, Colorado, United States
Atlanta, Georgia, United States
Indianapolis, Indiana, United States
Boston, Massachusetts, United States
Grand Rapids, Michigan, United States
Minneapolis, Minnesota, United States
Kansas City, Missouri, United States
Omaha, Nebraska, United States
Salt Lake City, Utah, United States
Richmond, Virginia, United States
Seattle, Washington, United States
Vancouver, British Columbia, Canada
Edinburgh, , United Kingdom
Liverpool, , United Kingdom
London, , United Kingdom
Countries
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References
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Rosenfeld M, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Chilvers M, Higgins M, Tian S, Cooke J, Davies JC; KLIMB study group. An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB). J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30.
Other Identifiers
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VX11-770-109
Identifier Type: -
Identifier Source: org_study_id
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