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| ID | Type | Description | Link |
|---|---|---|---|
| 2014-004827-29 | EudraCT Number |
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This is a Phase 3, multicenter, open-label, 3-part rollover study in subjects with CF who are homozygous or heterozygous for the F508del-CFTR mutation and who participated in studies VX13-661-103 (Study 103, NCT02070744), VX14-661-106 (Study 106, NCT02347657), VX14-661-107 (Study 107, NCT02516410), VX14-661-108 (Study 108, NCT02392234), VX14-661-109 (Study 109, NCT02412111), VX14-661-111 (Study 111, NCT02508207), VX15-661-112 (NCT02730208), and VX16-661-114 (NCT03150719). The study is designed to evaluate the safety and efficacy of long-term treatment of VX-661 in combination with ivacaftor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TEZ/IVA | Experimental | Part A: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 milligram (mg)/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part B: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108,109,112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part C: Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106,108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TEZ/IVA | Drug | Fixed dose tablet for oral administration. |
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| Measure | Description | Time Frame |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 100 | |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs | Day 1 up to Week 100 | |
| Part C: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 196 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. |
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Inclusion Criteria:
Part A:
Participants entering the Treatment Cohort must meet all of the following criteria:
Elect to enroll in the Treatment Cohort
Completed study drug Treatment Period in a parent study (NCT02070744, NCT02347657, NCT02516410, NCT02392234, NCT02412111) or study drug treatment and the Safety Follow up Visit for participants from NCT02508207.
Willing to remain on a stable CF regimen through the Safety Follow-up Visit.
Participants re-enrolling in the Part A Treatment Cohort must meet all of the following criteria:
Participants entering the Part A Observational Cohort must meet the following criteria:
Part B:
Participants who meet all of the following inclusion criteria will be eligible for Part B.
Participants re enrolling in Part B must meet all of the following criteria:
Part C:
Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33581080 | Derived | Flume PA, Biner RF, Downey DG, Brown C, Jain M, Fischer R, De Boeck K, Sawicki GS, Chang P, Paz-Diaz H, Rubin JL, Yang Y, Hu X, Pasta DJ, Millar SJ, Campbell D, Wang X, Ahluwalia N, Owen CA, Wainwright CE; VX14-661-110 study group. Long-term safety and efficacy of tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years or older who are homozygous or heterozygous for Phe508del CFTR (EXTEND): an open-label extension study. Lancet Respir Med. 2021 Jul;9(7):733-746. doi: 10.1016/S2213-2600(20)30510-5. Epub 2021 Feb 10. |
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A total 1131 participants enrolled in the study (1044 in Part A, 464 in Part B and 204 in Part C).
This study consisted of 3 parts: Parts A, B, and C.
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| ID | Title | Description |
|---|---|---|
| FG000 | TEZ/IVA | Part A: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part B: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, 109, 112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part C: Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (Up to 96 Weeks) |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 9, 2017 | May 14, 2020 |
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| IVA | Drug | Tablet for oral administration. |
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| From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Number of Pulmonary Exacerbation (PEx) Events for 106/110 PEx Analysis Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline up to Study 110 Week 96 |
| Part A: Number of Pulmonary Exacerbation (PEx) Events for 108/110 PEx Analysis Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline up to Week 96 |
| Part A: Absolute Change in Body Mass Index (BMI) for 106/110 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Mass Index (BMI) for 108/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Mass Index (BMI) for 103/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Mass Index (BMI) for Study 111/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in BMI Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in BMI Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/110 Efficacy Set | 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. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight for Study 106/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight for 108/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight for 103/110 Efficacy Set | Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight for 111/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Body Weight Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Height Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | From Baseline at Study 110 Week 96 |
| Part A: Absolute Change in Height Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | From Baseline at Study 110 Week 96 |
| Part A: Time-to-first Pulmonary Exacerbation (PEx) for 106/110 PEx Analysis Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. | 96 weeks |
| Part A: Time-to-first Pulmonary Exacerbation (PEx) for 108/110 PEx Analysis Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. | 96 weeks |
| Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA) | Week 24 |
| 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. | From Baseline at Study 110 Week 96 |
| Part B: Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). | From Baseline at Week 96 |
| Part B: Absolute Change in BMI 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. | From Baseline at Week 96 |
| Part B: Number of Pulmonary Exacerbation (PEx) Events | 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. | From Baseline up to Week 96 |
| Tucson |
| Arizona |
| United States |
| Little Rock | Arkansas | United States |
| Long Beach | California | United States |
| Los Angeles | California | United States |
| Oakland | California | United States |
| Palo Alto | California | United States |
| Sacramento | California | United States |
| San Diego | California | United States |
| Aurora | Colorado | United States |
| Denver | Colorado | United States |
| Gainesville | Florida | United States |
| Miami | Florida | United States |
| Orlando | Florida | United States |
| Pensacola | Florida | United States |
| Tampa | Florida | United States |
| Atlanta | Georgia | United States |
| Boise | Idaho | United States |
| Chicago | Illinois | United States |
| Glenview | Illinois | United States |
| Niles | Illinois | United States |
| Peoria | Illinois | United States |
| Indianapolis | Indiana | United States |
| Iowa City | Iowa | United States |
| Lexington | Kentucky | United States |
| New Orleans | Louisiana | United States |
| Baltimore | Maryland | United States |
| Boston | Massachusetts | United States |
| Ann Arbor | Michigan | United States |
| Detroit | Michigan | United States |
| Minneapolis | Minnesota | United States |
| Kansas City | Missouri | United States |
| St Louis | Missouri | United States |
| Omaha | Nebraska | United States |
| Lebanon | New Hampshire | United States |
| Long Branch | New Jersey | United States |
| New Brunswick | New Jersey | United States |
| Albuquerque | New Mexico | United States |
| Albany | New York | United States |
| Buffalo | New York | United States |
| New Hyde Park | New York | United States |
| New York | New York | United States |
| Rochester | New York | United States |
| Syracuse | New York | United States |
| Chapel Hill | North Carolina | United States |
| Akron | Ohio | United States |
| Cincinnati | Ohio | United States |
| Cleveland | Ohio | United States |
| Columbus | Ohio | United States |
| Toledo | Ohio | United States |
| Oklahoma City | Oklahoma | United States |
| Portland | Oregon | United States |
| Philadelphia | Pennsylvania | United States |
| Pittsburgh | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Sioux Falls | South Dakota | United States |
| Knoxville | Tennessee | United States |
| Memphis | Tennessee | United States |
| Nashville | Tennessee | United States |
| Austin | Texas | United States |
| Dallas | Texas | United States |
| Fort Worth | Texas | United States |
| Houston | Texas | United States |
| Tyler | Texas | United States |
| Salt Lake City | Utah | United States |
| Seattle | Washington | United States |
| Spokane | Washington | United States |
| Morgantown | West Virginia | United States |
| Milwaukee | Wisconsin | United States |
| Chermside | Australia |
| Melbourne | Australia |
| South Brisbane | Australia |
| Westmead | Australia |
| Graz | Austria |
| Innsbruck | Austria |
| Salzburg | Austria |
| Brussels | Belgium |
| Ghent | Belgium |
| Leuven | Belgium |
| Vancouver | British Columbia | Canada |
| Toronto | Ontario | Canada |
| Halifax | Canada |
| Montreal | Canada |
| Québec | Canada |
| Copenhagen | Denmark |
| Benite Cedex | France |
| Bordeaux | France |
| Bron | France |
| Lille | France |
| Marseille | France |
| Montpellier | France |
| Paris | France |
| Rennes | France |
| Roscoff | France |
| Rouen | France |
| Berlin | Germany |
| Bochum | Germany |
| Erlangen | Germany |
| Essen | Germany |
| Frankfurt | Germany |
| Giesen | Germany |
| Hannover | Germany |
| Heidelberg | Germany |
| Jena | Germany |
| München | Germany |
| Tübingen | Germany |
| Würzburg | Germany |
| Cork | Ireland |
| Dublin | Ireland |
| Limerick | Ireland |
| Haifa | NAP | Israel |
| Haifa | Israel |
| Jerusalem | Israel |
| Petah Tikva | Israel |
| Tel Litwinsky | Israel |
| Ancona | Italy |
| Genova | Italy |
| Milan | Italy |
| Orbassano | Italy |
| Palermo | Italy |
| Potenza | Italy |
| Roma | Italy |
| Rome | Italy |
| Torino | Italy |
| Verona | Italy |
| Amsterdam | Netherlands |
| Heidelberglaan | Netherlands |
| Nijmegen | Netherlands |
| Rotterdam | Netherlands |
| The Hague | Netherlands |
| Barcelona | Spain |
| Madrid | Spain |
| Sabadell | Spain |
| Seville | Spain |
| Valencia | Spain |
| Gothenburg | Sweden |
| Stockholm | Sweden |
| Uppsala | Sweden |
| Bern | Switzerland |
| Zurich | Switzerland |
| Leeds | West Yorkshire | United Kingdom |
| Belfast | United Kingdom |
| Birmingham | United Kingdom |
| Exeter | United Kingdom |
| Glasgow | United Kingdom |
| Leeds | United Kingdom |
| Liverpool | United Kingdom |
| London | United Kingdom |
| Manchester | United Kingdom |
| Newcastle upon Tyne | United Kingdom |
| Penarth | United Kingdom |
| Sheffield | United Kingdom |
| Southampton | United Kingdom |
| Stoke-on-Trent | United Kingdom |
| Safety Set | Received at least 1 dose of TEZ/IVA |
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| COMPLETED |
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| NOT COMPLETED |
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| Part B (Up to 96 Weeks) |
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| Part C (Up to 192 Weeks) |
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Baseline data is based on the parent study baseline, which is defined as the most recent non-missing measurements collected before the first dose of the study drug in the treatment period of parent studies. Baseline data is presented for all participants who signed informed consent and enrolled, or dosed, in Part A, Part B, or Part C, respectively in this study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | TEZ/IVA | Part A: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part B: Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, 109, 112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. Part C: Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | The Baseline data were planned to be presented separately for Part A , Part B and Part C. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Mean | Standard Deviation | years |
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| Sex: Female, Male | The Baseline data were planned to be presented separately for Part A, Part B and Part C. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants |
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| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A , Part B and Part C. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
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| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A , Part B and Part C. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety Set was defined as all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 up to Week 100 |
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| Primary | Part B: Safety and Tolerability as Assessed by 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. | Posted | Count of Participants | Participants | Day 1 up to Week 100 |
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| Primary | Part C: Safety and Tolerability as Assessed by 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. | Posted | Count of Participants | Participants | No | Day 1 up to Week 196 |
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| Secondary | Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | percentage points | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | percentage points | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | The 103/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 103 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | percentage points | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 111/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 111 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | percentage points | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 106/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 108/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 103/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | The 103/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 103 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | percent change | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Relative Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for 111/110 Efficacy Set | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 111/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 111 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | percent change | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Number of Pulmonary Exacerbation (PEx) Events for 106/110 PEx Analysis Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 PEx analysis set included study 106 participants who received TEZ/IVA in Study 106 or Study 110. | Posted | Number | PEx events | From Baseline up to Study 110 Week 96 |
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| Secondary | Part A: Number of Pulmonary Exacerbation (PEx) Events for 108/110 PEx Analysis Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 PEx analysis set included study 108 participants who received TEZ/IVA in Study 108 or Study 110. | Posted | Number | PEx events | From Baseline up to Week 96 |
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| Secondary | Part A: Absolute Change in Body Mass Index (BMI) for 106/110 Efficacy Set | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Mass Index (BMI) for 108/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Mass Index (BMI) for 103/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | The 103/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 103 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | kg/m^2 | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Mass Index (BMI) for Study 111/110 Efficacy Set | BMI was defined as weight in kg divided by height in square meter (m^2). Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 111/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 111 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | kg/m^2 | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in BMI Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in BMI Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 103/110 Efficacy Set | 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. Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | The 103/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 103 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | units on a scale | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight for Study 106/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | kg | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight for 108/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | kg | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight for 103/110 Efficacy Set | Data are reported for TEZ/IVA-TEZ/IVA group (participants who received TEZ/IVA in both parent study 103 and in current study 110). Baseline was defined as the parent study baseline. | The 103/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 103 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | kg | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight for 111/110 Efficacy Set | Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 111 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 111 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 111/110 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part A and rolled over from parent study 111 and had pre-defined CFTR genotypes. | Posted | Mean | Standard Deviation | kg | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Body Weight Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Height Z-score for 106/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline except for Placebo-TEZ/IVA category, for which baseline was study 110 baseline. | The 106/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 106 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Absolute Change in Height Z-score for 108/110 Efficacy Set | 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. Baseline was defined as the parent study baseline. | The 108/110 efficacy set included all enrolled participants <20 years of age at Screening who received at least 1 dose of study drug in Part A and rolled over from parent study 108 and had pre-defined CFTR genotypes. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Study 110 Week 96 |
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| Secondary | Part A: Time-to-first Pulmonary Exacerbation (PEx) for 106/110 PEx Analysis Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 106 and TEZ/IVA in current study 110) and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 106 and in current study 110) as per pre-specified analysis plan. | The 106/110 PEx analysis set included study 106 participants who received TEZ/IVA in Study 106 or Study 110. | Posted | Number | 95% Confidence Interval | event-free probability | 96 weeks |
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| Secondary | Part A: Time-to-first Pulmonary Exacerbation (PEx) for 108/110 PEx Analysis Set | Time-to-first pulmonary exacerbation was analyzed using Kaplan-Meier estimates and expressed in terms of event-free probability. PEx 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. Data are reported separately for Placebo-TEZ/IVA category (participants who received placebo in parent study 108 and TEZ/IVA in current study 110); IVA-TEZ/IVA category (participants who received IVA monotherapy in parent study 108 and TEZ/IVA in current study 110); and TEZ/IVA-TEZ/IVA category (participants who received TEZ/IVA in both parent study 108 and in current study 110) as per pre-specified analysis plan. | The 108/110 PEx analysis set included study 108 participants who received TEZ/IVA in Study 108 or Study 110. | Posted | Number | 95% Confidence Interval | event-free probability | 96 weeks |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Plasma Concentrations of TEZ, TEZ Metabolite (M1-TEZ), Ivacaftor (IVA) and Ivacaftor Metabolite (M1-IVA) | The Pharmacokinetic set included data for all participants who received TEZ/IVA treatment and met PK data inclusion and exclusion criteria. | Posted | Mean | Standard Deviation | nanogram/milliliter (ng/mL) | Week 24 |
|
| ||||||||||||||||||||||||||||||||||
| 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. | The efficacy set included all enrolled participants who received at least 1 dose of study drug in Part B and rolled over from parent studies and had pre-defined CFTR genotypes. It was performed on each of the CFTR mutation groups separately (F508del/F508del and F508del/Residual Function). Here, "Number Analyzed" signifies participants who were evaluable for the specific category of CFTR genotypes. | Posted | Mean | Standard Deviation | percentage points | From Baseline at Study 110 Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). | The efficacy set included all enrolled participants who received at least 1 dose of study drug in Part B and rolled over from parent studies and had pre-defined CFTR genotypes. It was performed on each of the CFTR mutation groups separately (F508del/F508del and F508del/Residual Function). Here, "Number Analyzed" signifies participants who were evaluable for the specific category of CFTR genotypes. | Posted | Mean | Standard Deviation | kg/m^2 | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change in BMI 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. | The efficacy set included all enrolled participants who received at least 1 dose of study drug in Part B and rolled over from parent studies and had pre-defined CFTR genotypes. It was performed on each of the CFTR mutation groups separately (F508del/F508del and F508del/Residual Function).Here, "Number Analyzed" signifies participants who were evaluable for the specific category of CFTR genotypes. | Posted | Mean | Standard Deviation | z-score | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part B: Number of Pulmonary Exacerbation (PEx) Events | 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 efficacy set included all enrolled participants who received at least 1 dose of study drug in Part B and rolled over from parent studies and had pre-defined CFTR genotypes. It was performed on each of the CFTR mutation groups separately (F508del/F508del and F508del/Residual Function). Here, "Number Analyzed" signifies participants who were evaluable for the specific category of CFTR genotypes. | Posted | Number | PEx events | From Baseline up to Week 96 |
|
|
Day 1 Through Safety Follow-up Visit (up to Week 100 for Part A, up to Week 100 for Part B and up to Week 196 for Part C)
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: TEZ/IVA | Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 103,106,107,108,109 and 111 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. | 1 | 1,042 | 351 | 1,042 | 938 | 1,042 |
| EG001 | Part B: TEZ/IVA | Participants who received either TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, 109, 112 and 114 were administered TEZ 100 mg/IVA 150 mg fixed-dose tablet in the morning and IVA 150 mg mono tablet in the evening for 96 weeks. | 0 | 463 | 136 | 463 | 391 | 463 |
| EG002 | Part C: TEZ/IVA | Participants who received TEZ/IVA, IVA monotherapy or Placebo in parent studies 106, 108, and 114 were administered TEZ 100 mg/IVA 150 mg fixed dose tablet in the morning and IVA 150 mg mono tablet in the evening for 192 weeks. | 0 | 204 | 44 | 204 | 149 | 204 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Bone marrow failure | Blood and lymphatic system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Lymphadenitis | Blood and lymphatic system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Thrombocytopenia | Blood and lymphatic system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | Meddra 25.1 | Systematic Assessment |
| |
| Left ventricular dysfunction | Cardiac disorders | Meddra 25.1 | Systematic Assessment |
| |
| Myocarditis | Cardiac disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cystic fibrosis lung | Congenital, familial and genetic disorders | Meddra 25.1 | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pituitary enlargement | Endocrine disorders | Meddra 25.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Appendiceal mucocoele | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Colitis | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cyclic vomiting syndrome | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Enteritis | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Enterocolitis haemorrhagic | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Ileus | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Large intestinal obstruction | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Melaena | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Obstructive pancreatitis | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Oesophageal achalasia | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Oesophageal varices haemorrhage | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pancreatitis chronic | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Subileus | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Volvulus | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Asthenia | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Chest pain | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Device related thrombosis | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| General physical health deterioration | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Medical device site erythema | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | Meddra 25.1 | Systematic Assessment |
| |
| Biliary colic | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Biliary dilatation | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cholecystitis | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cholecystitis chronic | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Gallbladder rupture | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hepatic mass | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hepatitis toxic | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Anaphylactic reaction | Immune system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Food allergy | Immune system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Type IV hypersensitivity reaction | Immune system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Anal abscess | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Atypical mycobacterial lower respiratory tract infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Atypical mycobacterial pneumonia | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Diverticulitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Erysipelas | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Groin abscess | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Herpes simplex | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Infectious mononucleosis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Lower respiratory tract infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Lung infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Lung infection pseudomonal | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Meningitis viral | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Pneumonia pseudomonal | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Salpingitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Typhoid fever | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection bacterial | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | Meddra 25.1 | Systematic Assessment |
| |
| Anaesthetic complication cardiac | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Ear injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Foot fracture | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Foreign body in eye | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Hand fracture | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Ligament injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Ligament rupture | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Patella fracture | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Peroneal nerve injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Procedural nausea | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Radius fracture | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Tendon injury | Injury, poisoning and procedural complications | Meddra 25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Blood creatinine increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Chest X-ray abnormal | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Computerised tomogram thorax abnormal | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Influenza A virus test positive | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Influenza B virus test positive | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Respiratory syncytial virus test positive | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Urine amphetamine positive | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | Meddra 25.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypocalcaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypomagnesaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hypovolaemia | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Malnutrition | Metabolism and nutrition disorders | Meddra 25.1 | Systematic Assessment |
| |
| Diastasis recti abdominis | Musculoskeletal and connective tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Neuropathic arthropathy | Musculoskeletal and connective tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Adenocarcinoma of colon | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Benign male reproductive tract neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Malignant glioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Oesophageal adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Oesophageal carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Schwannoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Meddra 25.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Facial paralysis | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Lumbar radiculopathy | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Quadrantanopia | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Toxic encephalopathy | Nervous system disorders | Meddra 25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Bipolar I disorder | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Disruptive mood dysregulation disorder | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Panic attack | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Paranoia | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Suicide attempt | Psychiatric disorders | Meddra 25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Hydronephrosis | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Renal impairment | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Renal infarct | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | Meddra 25.1 | Systematic Assessment |
| |
| Gynaecomastia | Reproductive system and breast disorders | Meddra 25.1 | Systematic Assessment |
| |
| Ovarian cyst | Reproductive system and breast disorders | Meddra 25.1 | Systematic Assessment |
| |
| Bronchial wall thickening | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Sinus polyp | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | Meddra 25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Urticarial vasculitis | Skin and subcutaneous tissue disorders | Meddra 25.1 | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | Meddra 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Immunisation reaction | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 20, 2019 | May 14, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000654124 | tezacaftor, ivacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Adverse Event |
|
| Sponsor Decision |
|
| Rolled over into another study |
|
| Withdrawal of consent (not due to AE) |
|
| Commercial drug is available for participant |
|
| Rolled over into another study |
|
| Withdrawal of consent (not due to AE) |
|
| Commercial drug is available for participant |
|
|
| Part C |
|
|
| Male |
|
| Part B |
|
|
| Part C |
|
|
| Not Hispanic or Latino |
|
| Not collected per local regulations |
|
| Part B |
|
|
| Part C |
|
|
| Black or African American |
|
| Asian |
|
| American Indian or Alaska Native |
|
| Native Hawaiian or Other Pacific Islander |
|
| Not collected per local regulations |
|
| Other |
|
| Part B |
|
|
| Part C |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
| Participants |
|
|
| Participants |
|
|
| Participants |
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
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|
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| Participants |
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| Participants |
|
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| Participants |
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| Participants |
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