Cystic Fibrosis
Conditions
Brief summary
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.
Interventions
DRUGLUM/IVA
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).
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Subjects entering the Treatment Cohort must meet both of the following criteria: * Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment * Willing to remain on a stable CF medication through the Safety Follow-up Visit. Subjects entering the Observational Cohort must meet 1 of the following criteria: * Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B. * Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.
Exclusion criteria
(Treatment Cohort Only): * History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject (e.g., cirrhosis with portal hypertension). * Pregnant and nursing females. * Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements. * History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator * History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator. * Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Absolute Change in Lung Clearance Index (LCI) 2.5 | From Parent Study Baseline at Week 96 | 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. |
| Absolute Change in Body Mass Index (BMI) | From Parent Study Baseline at Week 96 | BMI was defined as weight in kilograms divided by height in square meter (m\^2). |
| Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | From Parent Study Baseline at Week 96 | 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. |
| Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) | Day 1 up to Week 100 | — |
| Absolute Change in LCI 5.0 | From Parent Study Baseline at Week 96 | 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. |
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | From Parent Study Baseline at Week 96 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Relative Change in ppFEV1 | From Parent Study Baseline at Week 96 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. |
| Absolute Change in BMI-for-age Z-score | From Parent Study Baseline at Week 96 | 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. |
| Absolute Change in Weight | From Parent Study Baseline at Week 96 | — |
| Absolute Change in Weight-for-age Z-score | From Parent Study Baseline at Week 96 | 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. |
| Absolute Change in Sweat Chloride | From Parent Study Baseline at Week 96 | Sweat samples were collected using an approved collection device. |
| Absolute Change in Height-for-age Z-score | From Parent Study Baseline at Week 96 | 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. |
| Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score | From Parent Study Baseline at Week 96 | 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. |
| Time-to-first Pulmonary Exacerbation | From Parent Study Baseline through Week 96 | 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. |
| Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event | From Parent Study Baseline through Week 96 | 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. |
| Number of Pulmonary Exacerbation Events Per Patient-year | From Parent Study Baseline through Week 96 | 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. |
| Rate of Change in LCI 2.5 | Day 15 after first dose of LUM/IVA through Week 96 | 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. |
| Rate of Change in LCI 5.0 | Day 15 after first dose of LUM/IVA through Week 96 | 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. |
| Rate of Change in ppFEV1 | Day 15 after first dose of LUM/IVA through Week 96 | 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. |
| 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 | — |
| Absolute Change in Height | From Parent Study Baseline at Week 96 | — |
Countries
Australia, Belgium, Canada, Denmark, France, Germany, Sweden, United Kingdom, United States
Participant flow
Recruitment details
This study consists of 2 Treatment Periods: Treatment Period 1 and Treatment Period 2. Treatment Period 1 had Treatment Cohorts and an Observational Cohort.
Pre-assignment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 143 |
| PBO to LUM/IVA Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. | 96 |
| 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. | 6 |
| Total | 245 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Treatment Period 1 (96 Weeks) | Adverse Event | 1 | 6 | 0 | 0 |
| Treatment Period 1 (96 Weeks) | Lost to Follow-up | 2 | 0 | 0 | 0 |
| Treatment Period 1 (96 Weeks) | Other | 4 | 4 | 0 | 0 |
| Treatment Period 1 (96 Weeks) | Physician Decision | 4 | 0 | 0 | 0 |
| Treatment Period 1 (96 Weeks) | Withdrawal of consent (not due to AE) | 3 | 2 | 1 | 0 |
| Treatment Period 2 (168 Weeks) | Commercial drug is available for subject | 0 | 0 | 0 | 10 |
Baseline characteristics
| Characteristic | Total | LUM/IVA to LUM/IVA | PBO to LUM/IVA | Observational Cohort |
|---|---|---|---|---|
| Age, Customized Greater than or equal to 9 years | 144 Participants | 85 Participants | 58 Participants | 1 Participants |
| Age, Customized Less than 9 Years | 101 Participants | 58 Participants | 38 Participants | 5 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 2 Participants | 2 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 238 Participants | 139 Participants | 93 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 3 Participants | 2 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 0 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 6 Participants | 3 Participants | 3 Participants | 0 Participants |
| Race (NIH/OMB) White | 238 Participants | 140 Participants | 92 Participants | 6 Participants |
| Sex: Female, Male Female | 141 Participants | 83 Participants | 56 Participants | 2 Participants |
| Sex: Female, Male Male | 104 Participants | 60 Participants | 40 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 143 | 0 / 96 | 0 / 6 | 0 / 10 |
| other Total, other adverse events | 141 / 143 | 93 / 96 | 0 / 0 | 9 / 10 |
| serious Total, serious adverse events | 43 / 143 | 29 / 96 | 1 / 6 | 0 / 10 |
Outcome results
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)
Time frame: Day 1 up to Week 100
Population: Safety set included all participants who received at least 1 dose of study drug in Treatment Period 1.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| LUM/IVA to LUM/IVA | 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) | Participants with any AEs | 142 participants |
| LUM/IVA to LUM/IVA | 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) | Participants with SAEs | 43 participants |
| PBO to LUM/IVA | 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) | Participants with any AEs | 94 participants |
| PBO to LUM/IVA | 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) | Participants with SAEs | 29 participants |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in BMI-for-age Z-score | 0.17 z-score |
| PBO to LUM/IVA | Absolute Change in BMI-for-age Z-score | 0.31 z-score |
Secondary
Absolute Change in Body Mass Index (BMI)
BMI was defined as weight in kilograms divided by height in square meter (m\^2).
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Body Mass Index (BMI) | 1.78 kg/m^2 |
| PBO to LUM/IVA | Absolute Change in Body Mass Index (BMI) | 2.04 kg/m^2 |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | 7.4 units on a scale |
| PBO to LUM/IVA | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score | 6.6 units on a scale |
Secondary
Absolute Change in Height
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Height | 13.4 centimeter (cm) |
| PBO to LUM/IVA | Absolute Change in Height | 13.5 centimeter (cm) |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Height-for-age Z-score | -0.01 z-score |
| PBO to LUM/IVA | Absolute Change in Height-for-age Z-score | 0.02 z-score |
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.
Time frame: From Parent Study Baseline at Week 96
Population: LCI set.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in LCI 5.0 | -0.21 lung clearance index |
| PBO to LUM/IVA | Absolute Change in LCI 5.0 | -0.31 lung clearance index |
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.
Time frame: From Parent Study Baseline at Week 96
Population: 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.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Lung Clearance Index (LCI) 2.5 | -0.85 lung clearance index |
| PBO to LUM/IVA | Absolute Change in Lung Clearance Index (LCI) 2.5 | -0.86 lung clearance index |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | 3.1 percent predicted of FEV1 |
| PBO to LUM/IVA | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | 0.0 percent predicted of FEV1 |
Secondary
Absolute Change in Sweat Chloride
Sweat samples were collected using an approved collection device.
Time frame: From Parent Study Baseline at Week 96
Population: Full Analysis Set (FAS) includes all participants enrolled and dosed in either parent study. Analysis period includes both parent study and current study.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Sweat Chloride | -22.9 millimole per liter (mmol/L) |
| PBO to LUM/IVA | Absolute Change in Sweat Chloride | -22.8 millimole per liter (mmol/L) |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score | 5.1 units on a scale |
| PBO to LUM/IVA | Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score | 3.9 units on a scale |
Secondary
Absolute Change in Weight
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Weight | 10.3 kg |
| PBO to LUM/IVA | Absolute Change in Weight | 11.0 kg |
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.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Absolute Change in Weight-for-age Z-score | 0.12 z-score |
| PBO to LUM/IVA | Absolute Change in Weight-for-age Z-score | 0.24 z-score |
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.
Time frame: From Parent Study Baseline through Week 96
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Number of Pulmonary Exacerbation Events Per Patient-year | 0.45 events per patient-year |
| PBO to LUM/IVA | Number of Pulmonary Exacerbation Events Per Patient-year | 0.30 events per patient-year |
Secondary
Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 100
Population: All participants included in the observational cohort.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) | 1 participants |
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.
Time frame: From Parent Study Baseline through Week 96
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event | 49.5 percentage of participants |
| PBO to LUM/IVA | Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event | 32.3 percentage of participants |
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.
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Rate of Change in LCI 2.5 | -0.01 slope |
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.
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Rate of Change in LCI 5.0 | 0.00 slope |
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.
Time frame: Day 15 after first dose of LUM/IVA through Week 96
Population: 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.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| LUM/IVA to LUM/IVA | Rate of Change in ppFEV1 | 0.58 slope |
Secondary
Relative Change in ppFEV1
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time frame: From Parent Study Baseline at Week 96
Population: FAS.
| Arm | Measure | Value (LEAST_SQUARES_MEAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Relative Change in ppFEV1 | 4.9 percent change |
| PBO to LUM/IVA | Relative Change in ppFEV1 | 0.5 percent change |
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.
Time frame: From Parent Study Baseline through Week 96
Population: 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.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| LUM/IVA to LUM/IVA | Time-to-first Pulmonary Exacerbation | 720.00 days |
| PBO to LUM/IVA | Time-to-first Pulmonary Exacerbation | NA days |
Secondary
Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 up to Week 168
Population: Safety set included all participants who received at least 1 dose of study drug in Treatment Period 2.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| LUM/IVA to LUM/IVA | Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Participants with any AEs | 9 participants |
| LUM/IVA to LUM/IVA | Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Participants with SAEs | 0 participants |