The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an ivacaftor-responsive CF transmembrane conductance regulator (CFTR) gene mutation.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
57
Granules in sachet for oral administration.
Unnamed facility
Birmingham, Alabama, United States
Unnamed facility
Palo Alto, California, United States
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)
Time frame: Day 1 through Day 70
Part A: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)
Time frame: Pre-dose, 2-4 hours, 6-8 hours, 24-60 hours post-dose
Part B +A/B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious (TEAEs)
Time frame: Day 1 through Week 38
Part A/B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)
Time frame: Day 4 (pre-dose, 2-4 hours, 6-8 hours post-dose); Day 15 (pre-dose); Week 4 (pre-dose); Week 8 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 12 (pre-dose); Week 18 (pre-dose) and Week 24 (pre-dose)
Part B: Observed Plasma Concentration of IVA and Their Metabolites (M1-IVA and M6-IVA)
Time frame: Week 2 (pre-dose, 2-4 hours, 6-8 hours post-dose); Week 8 (pre-dose,1 hour, 4-hour post-dose); Week 24 (pre-dose, 2-4 hours post dose)
Part B + A/B: Absolute Change From Baseline in Sweat Chloride
Sweat samples were collected using an approved collection device.
Time frame: From Baseline at Week 24
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Unnamed facility
Atlanta, Georgia, United States
Unnamed facility
Chicago, Illinois, United States
Unnamed facility
Indianapolis, Indiana, United States
Unnamed facility
Baltimore, Maryland, United States
Unnamed facility
Boston, Massachusetts, United States
Unnamed facility
Kansas City, Missouri, United States
Unnamed facility
Columbus, Ohio, United States
Unnamed facility
Philadelphia, Pennsylvania, United States
...and 13 more locations