| Primary | Part A: Maximum Observed Concentration (Cmax) of TEZ and IVA | | Pharmacokinetic (PK) set included participants who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | | Geometric Mean | Geometric Coefficient of Variation | nanogram per milliliter (ng/mL) | | Day 1 and Day 14 | | | | ID | Title | Description |
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| OG000 | Part A | Participants weighing <25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days. |
| | | Title | Denominators | Categories |
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| Day 1: TEZ (<25 Kg) | | | | Day 1: TEZ (≥25 Kg) | |
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| Primary | Part A: Area Under the Concentration Versus Time Curve During Dosing Interval (AUCtau) of TEZ and IVA | | PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points."Number Analyzed=0" signified no subjects were evaluated for the specified parameter at that time point. | Posted | | Geometric Mean | Geometric Coefficient of Variation | hour*nanogram per milliliter (hr*ng/mL) | | Day 1 and Day 14 | | | | ID | Title | Description |
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| OG000 | Part A | Participants weighing <25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days. |
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| Primary | Part B: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | | Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm. | Posted | | Number | | participants | | Day 1 up to Week 28 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part A: Cmax of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA) | | PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | | Geometric Mean | Geometric Coefficient of Variation | ng/mL | | Day 1 and Day 14 | | | | ID | Title | Description |
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| OG000 | Part A | Participants weighing <25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days. |
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| Secondary | Part A: AUCtau of TEZ Metabolites (M1-TEZ, M2-TEZ) and IVA Metabolites (M1-IVA, M6-IVA) | | PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. "Number Analyzed=0" signified no subjects were evaluated for the specified parameter at that time point. | Posted | | Geometric Mean | Geometric Coefficient of Variation | hr*ng/mL | | Day 1 and Day 14 | | | | ID | Title | Description |
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| OG000 | Part A | Participants weighing <25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days. |
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| Secondary | Part A: Number of Participants With AEs and SAEs | | Safety set: included all participants who received at least 1 dose of study drug. The planned analysis was designed to assess overall treatment arm, irrespective of weight-based dosing regimen. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus analysis is presented for the single treatment arm. | Posted | | Number | | participants | | Day 1 up to Day 28 | | | | ID | Title | Description |
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| OG000 | Part A | Participants weighing <25 kg received TEZ 50 mg/IVA 75 mg for 14 days. Participants weighing ≥25 kg received TEZ 50 mg/IVA 150 mg for 14 days. |
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| Secondary | Part B: Cmax of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA) | | PK set. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | | Geometric Mean | Geometric Coefficient of Variation | ng/mL | | Week 16 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: AUCtau of TEZ, TEZ Metabolites (M1-TEZ, M2-TEZ), IVA, and IVA Metabolites (M1-IVA, M6-IVA ) | | PK set. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | | Geometric Mean | Geometric Coefficient of Variation | hr*ng/mL | | Week 16 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | 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. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | percentage points | | From Baseline through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Relative Change in ppFEV1 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | percent change | | From Baseline through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Absolute Change in Weight | | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | kg | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | 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. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Absolute Change in Height | | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | centimeter (cm) | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | 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. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kg divided by height in square meter (m^2). | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | kg/m^2 | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | 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. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Absolute Change in Sweat Chloride | Sweat samples were collected using an approved collection device. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | | From Baseline through Week 4 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | Part B: Absolute Change in Sweat Chloride | Sweat samples were collected using an approved collection device. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | mmol/L | | From Baseline through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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| Secondary | 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. | FAS: participants who carry the intended CFTR mutations and received at least 1 dose of study drug. The aim of weight based dosing is to achieve similar exposures in children of different weights, thus the planned analysis is presented for the single treatment arm irrespective of weight-based dosing regimen. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | From Baseline through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B | Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination in the morning and IVA 75 mg in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination in the morning and IVA 150 mg in the evening for 24 weeks. |
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