| Primary | Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA | | Pharmacokinetic (PK) set for Part A included all participants who have received at least 1 dose of study drug in Part A. Here, the "number analyzed" signifies participants who were evaluable at the specified time point. This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | microgram per milliliter (mcg/mL) | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
| | | Title | Denominators | Categories |
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| ELX | | | | TEZ | |
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| Primary | Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA | | PK set (Part A). Here, the "number analyzed" signifies participants who were evaluable at the specified time point. This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | mcg/mL | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Primary | Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA | | PK set (Part A). This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | hour*microgram per milliliter (h*mcg/mL) | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Primary | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | | Safety set for Part B included all participants who received at least 1 dose of study drug in Part B. The safety and tolerability analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Number | | participants | | Part B: Day 1 Through Safety Follow-up Visit (up to Week 28) | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part A: Cmax of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) | | PK set (Part A). Here, the "number analyzed" signifies participants who were evaluable at the specified time point. This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | mcg/mL | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Secondary | Part A: Ctrough of ELX Metabolite (M23-ELX), TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA) | | PK set (Part A). Here, the "number analyzed" signifies participants who were evaluable at the specified time point. This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | mcg/mL | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Secondary | Part A: AUC0-24h of ELX Metabolite (M23-ELX) and TEZ Metabolite (M1-TEZ) | | PK set (Part A). This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | h*mcg/mL | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Secondary | Part A: Area Under the Concentration Versus Time Curve From 0 to 6 Hours (AUC0-6h) of IVA Metabolite (M1-IVA) | The AUC data was analyzed for up to 6 hours for IVA metabolite (M1-IVA). Therefore, AUC0-6h is reported for M1-IVA metabolite. | PK set (Part A). Here "overall number of participants analyzed" signifies participants who were evaluable at the specified time points. This outcome measure was planned only for Part A arm. | Posted | | Mean | Standard Deviation | h*mcg/mL | | Part A: Day 15 | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in Part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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| Secondary | Part A: Safety and Tolerability as Assessed by Number of Participants With TEAEs and SAEs | | Safety set for Part A included all participants who received at least 1 dose of study drug in Part A. This outcome measure was planned only for Part A arm. | Posted | | Number | | participants | | Part A: Day 1 Through Safety Follow-up Visit (up to Day 43) | | | | ID | Title | Description |
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| OG000 | Part A: ELX/TEZ/IVA | Participants in part A received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h in the treatment period for 15 days. |
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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. | Full analysis set (FAS) for Part B included all enrolled participants who carry the intended CFTR allele mutation and received at least 1 dose of study drug in Part B. The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | percentage points | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period 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 (Part B). The efficacy analysis for Part B was planned for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period 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 squared height in meters (m^2). | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | kg/m^2 | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period 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 squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Weight | | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | kg | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Weight-for-age Z-Score | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Height | | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | centimeters (cm) | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Height-for-Age Z-Score | The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | Part B: From Baseline at Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale | The study drug acceptability (participant reaction) was assessed by a visual analog scale that incorporates a 5 point facial hedonic scale (Liked it Very Much, Liked it a Little, Not sure, Disliked it a Little, Disliked it Very Much). Number of participants with the indicated categorical response in the drug acceptability assessment were reported. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome. | Posted | | Number | | participants | | Part B: At Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Number of Pulmonary Exacerbations Events | Pulmonary exacerbation was defined as new or changed treatment with oral, inhaled, or intravenous antibiotics and fulfillment of pre-specified protocol defined criteria. The total number of pulmonary exacerbations events across all participants were reported. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Number | | pulmonary exacerbations events | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Number of CF Related Hospitalizations | The total number of CF related hospitalization events across all participants were reported. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Number | | hospitalizations | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Ctrough of ELX, ELX Metabolite (M23-ELX), TEZ, TEZ Metabolite (M1-TEZ), IVA and IVA Metabolite (M1-IVA) | | The PK set for Part B included all participants who have received at least 1 dose of study drug in Part B. Here "number analyzed" signifies those participants who were evaluable at specified time points. | Posted | | Mean | Standard Deviation | mcg/mL | | Part B: At Week 4 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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| Secondary | Part B: Absolute Change in Lung Clearance Index 2.5 (LCI2.5) | LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | FAS (Part B). The efficacy analysis for Part B was assessed for the overall treatment arm, irrespective of weight based dose regimen. Therefore, the analysis is reported for the single triple combination (Part B: ELX/TEZ/IVA) arm. | Posted | | Least Squares Mean | 95% Confidence Interval | lung clearance index | | Part B: From Baseline Through Week 24 | | | | ID | Title | Description |
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| OG000 | Part B: ELX/TEZ/IVA | Participants in Part B weighing <30 kg at Day 1 received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h and participants weighing >=30 kg at Day 1 received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks. |
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