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
A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
2 other identifiers
interventional
86
6 countries
29
Brief Summary
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Aug 2017
Longer than P75 for phase_3
29 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
August 16, 2017
CompletedFirst Submitted
Initial submission to the registry
September 6, 2017
CompletedFirst Posted
Study publicly available on registry
September 8, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 2, 2023
CompletedStudy Completion
Last participant's last visit for all outcomes
October 2, 2023
CompletedResults Posted
Study results publicly available
October 23, 2024
CompletedOctober 23, 2024
October 1, 2024
6.1 years
September 6, 2017
October 1, 2024
October 1, 2024
Conditions
Outcome Measures
Primary Outcomes (1)
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Day 1 up to Week 120
Secondary Outcomes (1)
Absolute Change in Sweat Chloride
From Baseline at Week 96
Study Arms (2)
Ivacaftor Arm
EXPERIMENTALParticipants 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.
Observational Arm
NO INTERVENTIONInterventions
Eligibility Criteria
You may qualify if:
- Ivacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B:
- 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.
You may not qualify if:
- Ivacaftor Arm: Participants 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.
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (29)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Stanford University
Palo Alto, California, 94034, United States
Alfred I DuPont Hospital for Children
Wilmington, Delaware, 19803, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Center for Advanced Pediatrics
Atlanta, Georgia, 30329, United States
Northwestern University
Chicago, Illinois, 60611, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
John Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Childrens's Hospitals and Clinics of Minnnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Billings Clinic Hospital
Billings, Montana, 59101, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin
Madison, Wisconsin, 53792, 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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 6, 2017
First Posted
September 8, 2017
Study Start
August 16, 2017
Primary Completion
October 2, 2023
Study Completion
October 2, 2023
Last Updated
October 23, 2024
Results First Posted
October 23, 2024
Record last verified: 2024-10
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing