| Primary | Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | | OLE Safety Set was defined as all subjects who received at least 1 dose of study drug in this OLE study. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA arm irrespective of Part A and B separately. | Posted | | Count of Participants | | Participants | No | From Baseline up to Week 196 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
| | | Title | Denominators | Categories |
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| Participants with TEAEs | | | | Participants with SAEs | | |
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| Secondary | 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. | Open label Extension Full Analysis Set (OLE FAS) included all enrolled participants who have received at least 1 dose of study drug in the OLE study. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | percent predicted FEV1 | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | 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. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | units on a scale | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kg divided by squared height in meters (m^2). | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | kg/m^2 | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | 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 mean was above or below the standard. 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. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Number of Participants With Pulmonary Exacerbations (PEx) for 106/107 | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | The Cumulative TC Set includes participants who enrolled and received at least one dose of study drug during the parent study (445-106 Part B) and/or received at least one dose of study drug during this OLE Study. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who evaluated for this specific outcome measure. | Posted | | Count of Participants | | Participants | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Number of CF-related Hospitalizations for 106/107 | The total number of CF related hospitalization (Planned + Unplanned) events across all participants were reported. | The Cumulative TC Set. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Number | | hospitalizations | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) | The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | index | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Weight | | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | Kilogram (kg) | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Weight-for-age Z-score | The z-score is a statistical measure to describe whether a mean was above or below the standard. 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. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Height | | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | centimeters (cm) | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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| Secondary | Absolute Change in Height-for-age Z-score | The z-score is a statistical measure to describe whether a mean was above or below the standard. 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. | OLE FAS. Data for this outcome measure was planned to be collected and analyzed for the overall ELX/TEZ/IVA TC arm irrespective of Part A and B separately. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this specific outcome measure. | Posted | | Least Squares Mean | 95% Confidence Interval | z-score | | From Baseline up to Week 192 | | | | ID | Title | Description |
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| OG000 | ELX/TEZ/IVA | Participants ≥6 years and <12 years of age and weighing <30kg received ELX 100 mg qd /TEZ 50 mg qd/IVA75 mg q12h and those weighing ≥30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks. |
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