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Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Period 1: LUM/IVA to LUM/IVA | Experimental |
| |
| Treatment Period 1: Placebo (PBO) to LUM/IVA | Experimental |
| |
| Treatment Period 1: Observational Cohort | No Intervention | ||
| Treatment Period 2: LUM/IVA | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LUM/IVA | Drug | Lumacaftor (LUM) 200 mg every 12 hours (q12h)/ivacaftor (IVA) 250 mg q12h (for 6 through 11 years of age). LUM 400 mg q12h/IVA 250 mg q12h (for 12 years and older). |
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Period 1 (Treatment Cohorts): 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Lung Clearance Index (LCI) 2.5 | LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | From Parent Study Baseline at Week 96 |
| Absolute Change in Sweat Chloride |
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Inclusion Criteria:
Subjects entering the Treatment Cohort must meet both of the following criteria:
Subjects entering the Observational Cohort must meet 1 of the following criteria:
Exclusion Criteria (Treatment Cohort Only):
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33516285 | Derived | Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28. |
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Participants from Parent Studies 109 (NCT02514473) and 011B (NCT01897233) were enrolled in this study. A total of 240 participants were enrolled in Treatment Period 1 Treatment Cohorts, out of which 1 participant was enrolled but never dosed. Participants enrolled in the Observational and Treatment Period 2 Cohorts were followed for safety endpoint only, no efficacy data were collected.
This study consists of 2 Treatment Periods: Treatment Period 1 and Treatment Period 2. Treatment Period 1 had Treatment Cohorts and an Observational Cohort.
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| ID | Title | Description |
|---|---|---|
| FG000 | Treatment Period 1: LUM/IVA to LUM/IVA | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. |
| FG001 | Treatment Period 1: PBO to LUM/IVA |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period 1 (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 | Sep 15, 2015 | Aug 23, 2019 |
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| LUM/IVA | Drug | LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age). |
|
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Sweat samples were collected using an approved collection device. |
| From Parent Study Baseline at Week 96 |
| Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilograms divided by height in square meter (m^2). | From Parent Study Baseline at Week 96 |
| Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | From Parent Study Baseline at Week 96 |
| Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) | Day 1 up to Week 100 |
| Absolute Change in LCI 5.0 | LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value. | From Parent Study Baseline at Week 96 |
| 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 Parent Study Baseline at Week 96 |
| Relative Change in ppFEV1 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Parent Study Baseline at Week 96 |
| Absolute Change in BMI-for-age Z-score | BMI was defined as weight in kilograms divided by height in m^2. 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 Parent Study Baseline at Week 96 |
| Absolute Change in Weight | From Parent Study Baseline at Week 96 |
| Absolute Change in Weight-for-age Z-score | z-score is a statistical measure to describe whether a mean was above or below the standard. 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 Parent Study Baseline at Week 96 |
| Absolute Change in Height | From Parent Study Baseline at Week 96 |
| Absolute Change in Height-for-age Z-score | z-score is a statistical measure to describe whether a mean was above or below the standard. 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 Parent Study Baseline at Week 96 |
| Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score | The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction. | From Parent Study Baseline at Week 96 |
| Time-to-first Pulmonary Exacerbation | 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 Parent Study Baseline through Week 96 |
| Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event | 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 Parent Study Baseline through Week 96 |
| Number of Pulmonary Exacerbation Events Per Patient-year | 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 Parent Study Baseline through Week 96 |
| Rate of Change in LCI 2.5 | Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | Day 15 after first dose of LUM/IVA through Week 96 |
| Rate of Change in LCI 5.0 | Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | Day 15 after first dose of LUM/IVA through Week 96 |
| Rate of Change in ppFEV1 | Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | Day 15 after first dose of LUM/IVA through Week 96 |
| Treatment Period 2: 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 168 |
| Tucson |
| Arizona |
| United States |
| Long Beach | California | United States |
| Palo Alto | California | United States |
| Aurora | Colorado | United States |
| Wilmington | Delaware | United States |
| Jacksonville | Florida | United States |
| Orlando | Florida | United States |
| Atlanta | Georgia | United States |
| Chicago | Illinois | United States |
| Indianapolis | Indiana | United States |
| Iowa City | Iowa | United States |
| Boston | Massachusetts | 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 |
| Buffalo | New York | United States |
| Syracuse | New York | United States |
| Chapel Hill | North Carolina | United States |
| Cincinnati | Ohio | United States |
| Cleveland | Ohio | United States |
| Dayton | Ohio | United States |
| Portland | Oregon | United States |
| Pittsburgh | Pennsylvania | United States |
| Charleston | South Carolina | United States |
| Austin | Texas | United States |
| Houston | Texas | United States |
| Salt Lake City | Utah | United States |
| Colchester | Vermont | United States |
| Charlottesville | Virginia | United States |
| Norfolk | Virginia | United States |
| Richmond | Virginia | United States |
| Seattle | Washington | United States |
| Madison | Wisconsin | United States |
| Milwaukee | Wisconsin | United States |
| Parkville | Victoria | Australia |
| Herston | Australia |
| New Lambton Heights | Australia |
| Subiaco | Australia |
| Westmead | Australia |
| Brussels | Belgium |
| Leuven | Belgium |
| Vancouver | British Columbia | Canada |
| Toronto | Ontario | Canada |
| Montreal | Quebec | Canada |
| Copenhagen | Denmark |
| Bron | Cedex | France |
| Bordeaux | France |
| Paris | France |
| Berlin | Germany |
| Cologne | Germany |
| Giessen | Germany |
| Hanover | Germany |
| Stockholm | Sweden |
| Leeds | West Yorkshire | United Kingdom |
| Belfast | United Kingdom |
| Edinburgh | United Kingdom |
| London | United Kingdom |
Participants received placebo (PBO) in parent study (109) and then received LUM/IVA for 96 weeks in the current study.
| FG002 | Treatment Period 1: Observational Cohort | Participants completed a parent study (109, 011B) but were not eligible or elected to not receive LUM/IVA for 96 weeks in the current study. |
| FG003 | Treatment Period 2: LUM/IVA | Eligible subjects from Treatment Period 1 received LUM/IVA for up to approximately 168 weeks. |
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| NOT COMPLETED |
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| Treatment Period 2 (168 Weeks) |
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Treatment Cohort: All participants dosed in the parent studies and dosed in the current study.
Observational Cohort: All participants enrolled in the observational cohort of the current study.
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| ID | Title | Description |
|---|---|---|
| BG000 | LUM/IVA to LUM/IVA | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. |
| BG001 | PBO to LUM/IVA | Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. |
| BG002 | Observational Cohort | Participants completed a parent study (109, 011B) but were not eligible or elected to not receive LUM/IVA for 96 weeks in the current study. |
| BG003 | Total | Total of all reporting groups |
| 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, Customized | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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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 | Treatment Period 1 (Treatment Cohorts): 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 in Treatment Period 1. | Posted | Number | participants | Day 1 up to Week 100 |
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| Secondary | Absolute Change in Lung Clearance Index (LCI) 2.5 | LCI 2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value. | LCI set includes all participants enrolled and dosed in either parent study 109 or 011B LCI sub-study. Analysis period includes both parent study and current study. | Posted | Least Squares Mean | 95% Confidence Interval | lung clearance index | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Sweat Chloride | Sweat samples were collected using an approved collection device. | Full Analysis Set (FAS) includes all participants enrolled and dosed in either parent study. Analysis period includes both parent study and current study. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilograms divided by height in square meter (m^2). | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Parent Study Baseline at Week 96 |
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| Secondary | Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) | All participants included in the observational cohort. | Posted | Number | participants | Day 1 up to Week 100 |
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| Secondary | Absolute Change in LCI 5.0 | LCI 5.0 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/20th of its starting value. | LCI set. | Posted | Least Squares Mean | 95% Confidence Interval | lung clearance index | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | percent predicted of FEV1 | From Parent Study Baseline at Week 96 |
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| Secondary | Relative Change in ppFEV1 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | percent change | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in BMI-for-age Z-score | BMI was defined as weight in kilograms divided by height in m^2. 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. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Weight | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | kg | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Weight-for-age Z-score | z-score is a statistical measure to describe whether a mean was above or below the standard. 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. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Height | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | centimeter (cm) | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Height-for-age Z-score | z-score is a statistical measure to describe whether a mean was above or below the standard. 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. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Parent Study Baseline at Week 96 |
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| Secondary | Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score | The TSQM measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed in the total domain score, which ranges from 0 to 100, where higher scores indicate greater satisfaction. | FAS. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Parent Study Baseline at Week 96 |
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| Secondary | Time-to-first Pulmonary Exacerbation | 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. | Analysis included all participants dosed in parent study 109. LUM/IVA to LUM/IVA analysis period includes both parent study and current study. PBO to LUM/IVA analysis period includes the current study only. | Posted | Median | Inter-Quartile Range | days | From Parent Study Baseline through Week 96 |
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| Secondary | Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event | 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. | Analysis included all participants dosed in parent study 109. LUM/IVA to LUM/IVA analysis period includes both parent study and current study. PBO to LUM/IVA analysis period includes the current study only. | Posted | Number | percentage of participants | From Parent Study Baseline through Week 96 |
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| Secondary | Number of Pulmonary Exacerbation Events Per Patient-year | 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. | Analysis included all participants dosed in parent study 109. LUM/IVA to LUM/IVA analysis period includes both parent study and current study. PBO to LUM/IVA analysis period includes the current study only. | Posted | Number | 95% Confidence Interval | events per patient-year | From Parent Study Baseline through Week 96 |
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| Secondary | Rate of Change in LCI 2.5 | Rate of change analysis evaluates the change in LCI 2.5 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | As pre-specified in the SAP, this analysis was conducted in the LUM/IVA Overall group because of sample size. Analysis period is 15 days after first dose of LUM/IVA in parent study or current study (if assigned to placebo in study 109) through the end of current study. | Posted | Number | 95% Confidence Interval | slope | Day 15 after first dose of LUM/IVA through Week 96 |
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| Secondary | Rate of Change in LCI 5.0 | Rate of change analysis evaluates the change in LCI 5.0 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | As pre-specified in the SAP, this analysis was conducted in the LUM/IVA Overall group because of sample size. Analysis period is 15 days after first dose of LUM/IVA in parent study or current study (if assigned to placebo in study 109) through the end of current study. | Posted | Number | 95% Confidence Interval | slope | Day 15 after first dose of LUM/IVA through Week 96 |
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| Secondary | Rate of Change in ppFEV1 | Rate of change analysis evaluates the change in ppFEV1 after long term treatment with LUM/IVA. A rate of change equal to zero would indicate that treatment effects were stable. | As pre-specified in the SAP, this analysis was conducted in the LUM/IVA Overall group because of sample size. Analysis period is 15 days after first dose of LUM/IVA in parent study or current study (if assigned to placebo in study 109) through the end of current study. | Posted | Number | 95% Confidence Interval | slope | Day 15 after first dose of LUM/IVA through Week 96 |
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| Secondary | Treatment Period 2: 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 in Treatment Period 2. | Posted | Number | participants | Day 1 up to Week 168 |
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Treatment Period 1: Day 1 up to Week 100, Treatment Period 2: Day 1 up to Week 168
Only serious adverse events were collected for the observational cohort. Non-serious AEs were not collected and are not reported for the observational cohort. Adverse events reported based on MedDRA version 21.0 for Treatment Period 1 and MedDRA version 22.1 for Treatment Period 2.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Treatment Period 1: LUM/IVA to LUM/IVA | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. | 0 | 143 | 43 | 143 | 141 | 143 |
| EG001 | Treatment Period 1: PBO to LUM/IVA | Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. | 0 | 96 | 29 | 96 | 93 | 96 |
| EG002 | Treatment Period 1: Observational Cohort | Participants completed a parent study (109, 011B) but were not eligible or elected to not receive LUM/IVA for 96 weeks in the current study. | 0 | 6 | 1 | 6 | 0 | 0 |
| EG003 | Treatment Period 2: LUM/IVA | Eligible subjects from Treatment Period 1 received LUM/IVA for up to approximately 168 weeks. | 0 | 10 | 0 | 10 | 9 | 10 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Bronchopulmonary aspergillosis allergic | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Appendicitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Atypical mycobacterial lower respiratory tract infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Gastroenteritis viral | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Lower respiratory tract infection bacterial | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Pulmonary function test decreased | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Pseudomonas test positive | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Atypical mycobacterium test positive | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Oxygen saturation decreased | Investigations | 21.0, 22.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Distal intestinal obstruction syndrome | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Sinus disorder | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Cystic fibrosis hepatic disease | Congenital, familial and genetic disorders | 21.0, 22.1 | Systematic Assessment |
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| Cystic fibrosis lung | Congenital, familial and genetic disorders | 21.0, 22.1 | Systematic Assessment |
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| Pyrexia | General disorders | 21.0, 22.1 | Systematic Assessment |
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| Intentional overdose | Injury, poisoning and procedural complications | 21.0, 22.1 | Systematic Assessment |
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| Diabetic ketoacidosis | Metabolism and nutrition disorders | 21.0, 22.1 | Systematic Assessment |
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| Lymphadenitis | Blood and lymphatic system disorders | 21.0, 22.1 | Systematic Assessment |
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| Autoimmune hepatitis | Hepatobiliary disorders | 21.0, 22.1 | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | 21.0, 22.1 | Systematic Assessment |
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| Oesophagitis | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Hyperglycaemia | Metabolism and nutrition disorders | 21.0, 22.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Sinusitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Otitis media | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Bacterial disease carrier | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Influenza | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Ear infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Rhinitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Upper respiratory tract infection bacterial | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Bronchitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Pharyngitis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Activated partial thromboplastin time prolonged | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| International normalised ratio increased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Prothrombin time prolonged | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Fatigue | General disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Chest pain | General disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | 21.0, 22.1 | Systematic Assessment |
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| Seasonal allergy | Immune system disorders | 21.0, 22.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | 21.0, 22.1 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | 21.0, 22.1 | Systematic Assessment |
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| Ligament sprain | Injury, poisoning and procedural complications | 21.0, 22.1 | Systematic Assessment |
| |
| Pulmonary imaging procedure abnormal | Investigations | 21.0, 22.1 | Systematic Assessment |
| |
| Bronchiectasis | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Eyelid oedema | Eye disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Hepatomegaly | Hepatobiliary disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Glucose tolerance impaired | Metabolism and nutrition disorders | 21.0, 22.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
| |
| Oral fungal infection | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
| |
| Respiratory tract infection bacterial | Infections and infestations | 21.0, 22.1 | Systematic Assessment |
|
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 | Jul 20, 2018 | Aug 23, 2019 | 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 |
|---|---|
| C000599212 | lumacaftor, ivacaftor drug combination |
Not provided
Not provided
Not provided
| Greater than or equal to 9 years |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
|
| Unknown or Not Reported |
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