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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
86
Granules for oral administration.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Day 1 up to Week 120
Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Week 96
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
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
...and 19 more locations