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
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: IVA
- Registration Number
- NCT03277196
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 86
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.
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
Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Ivacaftor Arm IVA 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.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method Absolute Change in Sweat Chloride From Baseline at Week 96 Sweat samples were collected using an approved collection device.
Trial Locations
- Locations (29)
Center for Advanced Pediatrics
🇺🇸Atlanta, Georgia, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Childrens's Hospitals and Clinics of Minnnesota
🇺🇸Minneapolis, Minnesota, United States
Children's Health Ireland at Crumlin
🇮🇪Dublin, Ireland
Alfred I DuPont Hospital for Children
🇺🇸Wilmington, Delaware, United States
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
Children's University Hospital Temple Street
🇮🇪Dublin, Ireland
University Hospital Limerick
🇮🇪Limerick, Ireland
Billings Clinic Hospital
🇺🇸Billings, Montana, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Nemours Children's Hospital
🇺🇸Orlando, Florida, United States
John Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Riley Hospital for Children at Indiana University Health
🇺🇸Indianapolis, Indiana, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
University of Wisconsin
🇺🇸Madison, Wisconsin, United States
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
🇩🇪Heidelberg, Germany
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
Oxford University Hospitals NHS Trust, John Radcliffe Hospital
🇬🇧Oxford, United Kingdom
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom
Stanford University
🇺🇸Palo Alto, California, United States