This is a Phase 3, 2-part (Part A and Part B), open label, multicenter study evaluating the pharmacokinetic (PK), safety, and tolerability of multiple doses of tezacaftor (TEZ) in combination with ivacaftor (IVA) in subjects 6 through 11 years of age with CF who are homozygous or heterozygous for the F508del- CF transmembrane conductance regulator protein (CFTR) mutation.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
83
Unnamed facility
Birmingham, Alabama, United States
Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA
Time frame: Day 1 and Day 14
Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA
Time frame: Day 1 and Day 14
Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 28
Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Time frame: Day 1 and Day 14
Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA)
Time frame: Day 1 and Day 14
Part A: Number of Participants With AEs and SAEs
Time frame: Day 1 up to Day 28
Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA)
Time frame: Week 16
Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA )
Time frame: Week 16
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline through Week 24
Part B: Relative Change in ppFEV1
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Unnamed facility
Anchorage, Alaska, United States
Unnamed facility
Little Rock, Arkansas, United States
Unnamed facility
Los Angeles, California, United States
Unnamed facility
Palo Alto, California, United States
Unnamed facility
Aurora, Colorado, United States
Unnamed facility
Wilmington, Delaware, United States
Unnamed facility
Orlando, Florida, United States
Unnamed facility
St. Petersburg, Florida, United States
Unnamed facility
Atlanta, Georgia, United States
...and 23 more locations
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Baseline through Week 24
Part B: Absolute Change in Weight
Time frame: From Baseline at Week 24
Part B: Absolute Change in Weight-for-age Z-Score
z-score is a statistical measure to describe whether a mean was above or below the standard. Weight, adjusted for age and sex, was analyzed as weight-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher weight.
Time frame: From Baseline at Week 24
Part B: Absolute Change in Height
Time frame: From Baseline at Week 24
Part B: Absolute Change in Height-for-age z-Score
z-score is a statistical measure to describe whether a mean was above or below the standard. Height, adjusted for age and sex, was analyzed as height-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher height.
Time frame: From Baseline at Week 24
Part B: Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kg divided by height in square meter (m\^2).
Time frame: From Baseline at Week 24
Part B: Absolute Change in BMI-for-age z-Score
BMI was defined as weight in kg divided by height in m\^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI.
Time frame: From Baseline at Week 24
Part B: Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Time frame: From Baseline through Week 4
Part B: Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Time frame: From Baseline through Week 24
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time frame: From Baseline through Week 24