A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
NCT ID: NCT03277196
Last Updated: 2024-10-23
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
86 participants
INTERVENTIONAL
2017-08-16
2023-10-02
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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Ivacaftor Arm
Participants less than (\<) 24 months of age and weighing 5 to less than (\<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to \<14 kg received 50 mg IVA q12h, and those weighing 14 to \<25 kg received 75 mg IVA q12h.
Participants more than or equal (\>=) 24 months of age and weighing \<14 kg received 50 mg IVA q12h, and those weighing more than or equal to (\>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks.
Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight.
IVA
Granules for oral administration.
Observational Arm
No interventions assigned to this group
Interventions
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IVA
Granules for oral administration.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
* As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).
Ivacaftor Arm: Participants Not From Study 124 Part B:
* Confirmed diagnosis of CF, or 2 CF-causing mutations.
* An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 2 years of age and older.
* As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.
Observational Arm:
* Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.
Exclusion Criteria
* 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 ivacaftor to the participant.
* Participants receiving commercially available ivacaftor treatment
Ivacaftor Arm: Participants Not From Study 124 Part B:
* 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 ivacaftor to the participant
* An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
* Abnormal liver function at screening
* Hemoglobin \<9.5 g/dL at screening
* History of solid organ or hematological transplantation
* Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1
Observational Arm:
* Receiving ivacaftor treatment
0 Months
24 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
Stanford University
Palo Alto, California, United States
Alfred I DuPont Hospital for Children
Wilmington, Delaware, United States
Nemours Children's Hospital
Orlando, Florida, United States
Center for Advanced Pediatrics
Atlanta, Georgia, United States
Northwestern University
Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
John Hopkins Hospital
Baltimore, Maryland, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Childrens's Hospitals and Clinics of Minnnesota
Minneapolis, Minnesota, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Billings Clinic Hospital
Billings, Montana, United States
Nationwide Children's Hospital
Columbus, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Texas Children's Hospital
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Queensland Children's Hospital
South Brisbane, , Australia
The Hospital for Sick Children
Toronto, Ontario, Canada
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
Heidelberg, , Germany
Children's Health Ireland at Crumlin
Dublin, , Ireland
Children's University Hospital Temple Street
Dublin, , Ireland
University Hospital Limerick
Limerick, , Ireland
Paediatric Clinical Research Facility
Edinburgh, , United Kingdom
Alder Hey Children's Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
Great Ormond Street Hospital for Sick Children
London, , United Kingdom
Royal Brompton & Harefield NHS Founcation Trust, Royal BromptonHospital
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Oxford University Hospitals NHS Trust, John Radcliffe Hospital
Oxford, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2017-001379-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX15-770-126
Identifier Type: -
Identifier Source: org_study_id
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