A Phase 3 interventional study of Placebo and Lumacaftor Plus Ivacaftor Combination in Cystic Fibrosis, Homozygous for the F508del CFTR Mutation, sponsored by Vertex Pharmaceuticals Incorporated. Completed at 82 sites in 10 countries. Open to participants aged 12 Years to 65 Years. Per ClinicalTrials.gov, last updated 2016-09-27.
Sponsored by Vertex Pharmaceuticals Incorporated · Phase 3, Interventional, and Treatment
The primary objective of the study was to evaluate the efficacy of lumacaftor in combination with ivacaftor at Week 24 in participants aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter study of orally administered lumacaftor in combination with ivacaftor in participants aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation.
The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1 [first dose of study drug] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7 days after the Week 24 Visit).
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 563 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Vertex Pharmaceuticals Incorporated is the lead sponsor of 243 studies on the registry; 19 are open to participants now.
Of its 102 completed or terminated interventional studies of FDA-regulated products, 49 (48%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.
Drug: Placebo
LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.
Drug: Lumacaftor Plus Ivacaftor Combination · Drug: Ivacaftor
LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.
Drug: Lumacaftor Plus Ivacaftor Combination
Matching placebo tablet
Fixed dose combination tablet
Also known as: VX-809+VX-770, LUM+IVA
Film-coated tablet
Also known as: VX-770, IVA
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24
Absolute change from baseline at week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
Time frame: Baseline, Week 16 and 24
Relative Change From Baseline in Percent Predicted FEV1 at Week 24
Assessed as the average treatment effect at Week 16 and at Week 24. FEV1 and percent predicted FEV1 are defined in Outcome Measure (OM) 1.
Time frame: Baseline, Week 16 and 24
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
BMI was defined as weight in kilogram (kg) divided by height\*height in square meter (m\^2).
Time frame: Baseline, Week 24
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time frame: Baseline, Week 24
Percentage of Participants With Response Based on Percent Predicted FEV1
A participant was considered as a responder if the participant had \>=5% increase from baseline in average percent predicted FEV1 at Week 16 and at Week 24 (relative change). FEV1 and percent predicted FEV1 are defined in OM 1. A participant with a missing average relative change from baseline in percent predicted FEV1 at Week 16 and at Week 24 was considered as a non-responder.
Time frame: Week 16 and 24
Number of Pulmonary Exacerbation Events
The total number of days on study is equal to the Week 24 date or the last dose date (whichever occurred last) minus the first dose date plus 1. The total number of years (48 weeks) on study is equal to the number of days on study divided by 336. Pulmonary exacerbation events per year (48 weeks) are reported.
Time frame: through Week 24
Absolute Change From Baseline in Weight at Week 24
Time frame: Baseline, Week 24
Absolute Change From Baseline in BMI-for-age Z-score at Week 24
Z-Score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is with range from -infinity to +infinity; 0: same mean, \>0: a greater mean, and \<0: a lesser mean than the standard. BMI-for-age z-score was calculated by using centers for disease control and prevention (CDC) growth charts for the pediatric population.
Time frame: Baseline, Week 24
Time-to-First Pulmonary Exacerbation
Time to first pulmonary exacerbation was assessed using Cox Regression method. For participants who completed 24 weeks of treatment, participants without a pulmonary exacerbation before treatment completion were considered censored at the time of treatment completion or at the Week 24 Visit (whichever occurred last). For participants who prematurely discontinued study treatment, participants without a pulmonary exacerbation through the Week 24 Visit were considered censored at the time of the Week 24 Visit.
Time frame: through Week 24
Percentage of Participants With At Least 1 Pulmonary Exacerbation Event
Time frame: through Week 24
Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24
EQ-5D-3L: participant rated questionnaire to assess health-related quality of life. It consists of EQ-5D descriptive system and EQ-5D Visual Analog Scale (VAS). EQ-5D-3L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: no problems (1), some problems (2), and extreme problems (3). The 5 dimensional 3-level systems are converted into a single index utility score. Values for theoretically possible health states are calculated using a regression model and weighted according to the social preferences of the Unites States (US) general population. For this population, the possible EQ-5D-3L index scores ranges from -0.11 (that is, 3 for all 5 dimensions) to 1.0 (that is, 1 for all 5 dimensions), where higher scores indicate a better health state.
Time frame: Baseline, Week 24
Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24
The EQ-5D-3L VAS records the participant's self-rated health on a vertical, visual analogue scale where the best state a participant can imagine is marked 100 and the worst state a participant can imagine is marked 0, higher scores indicates a better health state.
Time frame: Baseline, Week 24
Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Scores at Week 24
The TSQM is a 14-item self-administered questionnaire which measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed to a scale from 0 to 100, where higher scores indicate greater satisfaction.
Time frame: Baseline, Week 24
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or that was newly developed at or after the initial dosing of study drug to 28 days after the last dose of study drug is considered treatment-emergent.
Time frame: up to Week 28
Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg)
Ctrough, Ctrough,avg, C3-6h, and C3-6h,avg for lumacaftor, M28 lumacaftor (lumacaftor metabolite), ivacaftor, M1 ivacaftor (ivacaftor metabolite), and M6 ivacaftor (ivacaftor metabolite) were calculated. C3-6h,avg is average of individual 3 to 6 hours post-dose observed concentrations across Day 15, and Weeks 4 and 8 and Ctrough,avg is average of individual pre-dose observed concentrations across Weeks 4, 8, and 16. This outcome was not planned to be assessed in Placebo arm.
Time frame: For C3-6h: 3 to 6 hours after morning dose on Day 1 and 15, Week 4 and 8; For C3-6h,avg 3 to 6 hours after morning dose on Day 15, Week 4 and 8; For Ctrough and Ctrough,avg: before morning dose on Week 4, 8, and 16
| Milestone | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Started | 187 | 187 | 189 |
| Completed | 185 | 180 | 180 |
| Not completed | 2 | 7 | 9 |
| Withdrew: Adverse event | 1 | 2 | 2 |
| Withdrew: Withdrawal by subject | 1 | 2 | 2 |
| Withdrew: Non-compliance | 0 | 0 | 1 |
| Withdrew: Undefined | 0 | 1 | 2 |
| Withdrew: Randomized but not treated | 0 | 2 | 2 |
Absolute change from baseline at week 24 was assessed as the average treatment effect at Week 16 and at Week 24. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years.
| percent predicted of FEV1 | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24 | -0.15 ± 0.539 | 2.46 ± 0.540 | 2.85 ± 0.540 |
Assessed as the average treatment effect at Week 16 and at Week 24. FEV1 and percent predicted FEV1 are defined in Outcome Measure (OM) 1.
| percent change | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Relative Change From Baseline in Percent Predicted FEV1 at Week 24 | 0.00 ± 0.960 | 4.42 ± 0.961 | 5.25 ± 0.961 |
BMI was defined as weight in kilogram (kg) divided by height\*height in square meter (m\^2).
| kilogram per square meter (kg/m^2) | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in Body Mass Index (BMI) at Week 24 | 0.07 ± 0.066 | 0.48 ± 0.066 | 0.43 ± 0.066 |
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 (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
| units on a scale | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24 | 2.81 ± 1.153 | 5.02 ± 1.166 | 5.66 ± 1.169 |
A participant was considered as a responder if the participant had \>=5% increase from baseline in average percent predicted FEV1 at Week 16 and at Week 24 (relative change). FEV1 and percent predicted FEV1 are defined in OM 1. A participant with a missing average relative change from baseline in percent predicted FEV1 at Week 16 and at Week 24 was considered as a non-responder.
| percentage of participants | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Percentage of Participants With Response Based on Percent Predicted FEV1 | 22.5 | 45.9 | 41.2 |
The total number of days on study is equal to the Week 24 date or the last dose date (whichever occurred last) minus the first dose date plus 1. The total number of years (48 weeks) on study is equal to the number of days on study divided by 336. Pulmonary exacerbation events per year (48 weeks) are reported.
| pulmonary exacerbation events per year | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Number of Pulmonary Exacerbation Events | 1.18 | 0.82 | 0.67 |
| kilograms (kg) | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in Weight at Week 24 | 0.44 ± 0.187 | 1.57 ± 0.188 | 1.38 ± 0.187 |
Z-Score is a statistical measure to evaluate how a single data point compares to a standard. It describes whether a mean was above or below the standard and how unusual the measurement is with range from -infinity to +infinity; 0: same mean, \>0: a greater mean, and \<0: a lesser mean than the standard. BMI-for-age z-score was calculated by using centers for disease control and prevention (CDC) growth charts for the pediatric population.
| z-score | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in BMI-for-age Z-score at Week 24 | -0.0674 ± 0.04706 | 0.1640 ± 0.04652 | 0.1544 ± 0.04513 |
Time to first pulmonary exacerbation was assessed using Cox Regression method. For participants who completed 24 weeks of treatment, participants without a pulmonary exacerbation before treatment completion were considered censored at the time of treatment completion or at the Week 24 Visit (whichever occurred last). For participants who prematurely discontinued study treatment, participants without a pulmonary exacerbation through the Week 24 Visit were considered censored at the time of the Week 24 Visit.
| days | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Time-to-First Pulmonary Exacerbation | NA (NA to NA) | NA (NA to NA) | NA (NA to NA) |
| percentage of participants | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Percentage of Participants With At Least 1 Pulmonary Exacerbation Event | 47.1 | 36.8 | 28.9 |
EQ-5D-3L: participant rated questionnaire to assess health-related quality of life. It consists of EQ-5D descriptive system and EQ-5D Visual Analog Scale (VAS). EQ-5D-3L descriptive system comprises the following 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels: no problems (1), some problems (2), and extreme problems (3). The 5 dimensional 3-level systems are converted into a single index utility score. Values for theoretically possible health states are calculated using a regression model and weighted according to the social preferences of the Unites States (US) general population. For this population, the possible EQ-5D-3L index scores ranges from -0.11 (that is, 3 for all 5 dimensions) to 1.0 (that is, 1 for all 5 dimensions), where higher scores indicate a better health state.
| units on a scale | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24 | 0.0117 ± 0.00673 | 0.0090 ± 0.00682 | 0.0108 ± 0.00683 |
The EQ-5D-3L VAS records the participant's self-rated health on a vertical, visual analogue scale where the best state a participant can imagine is marked 100 and the worst state a participant can imagine is marked 0, higher scores indicates a better health state.
| units on a scale | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24 | 3.3 ± 1.07 | 5.7 ± 1.08 | 6.6 ± 1.08 |
The TSQM is a 14-item self-administered questionnaire which measures participants' experiences with their medication on four dimensions: effectiveness, side effects, convenience and global satisfaction. For each dimension, responses are added and transformed to a scale from 0 to 100, where higher scores indicate greater satisfaction.
| units on a scale | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Effectiveness (n = 159, 161, 161) | -8.49 ± 1.814 | 0.15 ± 1.807 | 3.12 ± 1.793 |
| Side Effects (n = 157, 159, 161) | 2.03 ± 1.144 | -1.14 ± 1.137 | -2.26 ± 1.121 |
| Convenience (n = 158, 160, 161) | 4.57 ± 1.525 | 4.57 ± 1.523 | 4.88 ± 1.501 |
| Global Satisfaction (n= 158, 160, 161) | -9.62 ± 1.841 | -4.98 ± 1.845 | -2.46 ± 1.814 |
AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Any AE that increased in severity or that was newly developed at or after the initial dosing of study drug to 28 days after the last dose of study drug is considered treatment-emergent.
| participants | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Participants With Treatment-Emergent AEs | 181 | 181 | 177 |
| Participants With Treatment-Emergent SAEs | 57 | 51 | 31 |
Ctrough, Ctrough,avg, C3-6h, and C3-6h,avg for lumacaftor, M28 lumacaftor (lumacaftor metabolite), ivacaftor, M1 ivacaftor (ivacaftor metabolite), and M6 ivacaftor (ivacaftor metabolite) were calculated. C3-6h,avg is average of individual 3 to 6 hours post-dose observed concentrations across Day 15, and Weeks 4 and 8 and Ctrough,avg is average of individual pre-dose observed concentrations across Weeks 4, 8, and 16. This outcome was not planned to be assessed in Placebo arm.
| microgram per milliliter (mcg/mL) | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|
| LUM, Day 1: C3-6h (n = 181, 180) | 30.8 ± 12.6 | 20.2 ± 8.33 |
| LUM, Day 15: C3-6h (n = 174, 176) | 30.4 ± 11.2 | 23.9 ± 8.87 |
| LUM, Week 4: Ctrough (n = 174, 179) | 8.11 ± 5.21 | 13.2 ± 6.28 |
| LUM, Week 4: C3-6h (n = 164, 168) | 29.2 ± 12.1 | 24.5 ± 8.75 |
| LUM, Week 8: Ctrough (n = 177, 177) | 7.87 ± 5.71 | 12.4 ± 6.87 |
| LUM, Week 8: C3-6h (n = 174, 170) | 30.4 ± 11.6 | 24.9 ± 9.79 |
| LUM, Week 16: Ctrough (n = 171, 173) | 7.53 ± 6.05 | 12.2 ± 6.87 |
| M-28 LUM, Day 1: C3-6h (n = 181, 180) | 0.226 ± 0.103 | 0.181 ± 0.0790 |
| M-28 LUM, Day 15: C3-6h (n = 174, 176) | 1.43 ± 0.588 | 1.39 ± 0.606 |
| M-28 LUM, Week 4: Ctrough (n = 174, 179) | 1.32 ± 0.680 | 1.42 ± 0.661 |
| M-28 LUM, Week 4: C3-6h (n = 164, 168) | 1.39 ± 0.657 | 1.45 ± 0.675 |
| M-28 LUM, Week 8: Ctrough (n = 177, 177) | 1.34 ± 0.714 | 1.44 ± 0.722 |
| M-28 LUM, Week 8: C3-6h (n = 174, 170) | 1.41 ± 0.702 | 1.48 ± 0.711 |
| M-28 LUM, Week 16: Ctrough (n = 171, 173) | 1.27 ± 0.763 | 1.43 ± 0.775 |
| IVA, Day 1: C3-6h (n = 181, 180) | 1.34 ± 0.643 | 1.36 ± 0.647 |
| IVA, Day 15: C3-6h (n = 174, 176) | 0.647 ± 0.492 | 0.469 ± 0.282 |
| IVA, Week 4: Ctrough (n = 174, 179) | 0.164 ± 0.207 | 0.108 ± 0.112 |
| IVA, Week 4: C3-6h (n = 164, 168) | 0.636 ± 0.380 | 0.473 ± 0.239 |
| IVA, Week 8: Ctrough (n = 177, 177) | 0.182 ± 0.293 | 0.102 ± 0.130 |
| IVA, Week 8: C3-6h (n = 174, 170) | 0.710 ± 0.567 | 0.513 ± 0.277 |
| IVA, Week 16: Ctrough (n = 171, 173) | 0.163 ± 0.237 | 0.113 ± 0.218 |
| M-1 IVA, Day 1: C3-6h (n = 181, 180) | 2.51 ± 1.30 | 2.65 ± 1.29 |
| M-1 IVA, Day 15: C3-6h (n = 174, 176) | 2.21 ± 1.08 | 1.85 ± 0.860 |
| M-1 IVA, Week 4: Ctrough (n = 174, 179) | 0.676 ± 0.612 | 0.501 ± 0.455 |
| M-1 IVA, Week 4: C3-6h (n = 164, 168) | 2.11 ± 1.12 | 1.88 ± 0.953 |
| M-1 IVA, Week 8: Ctrough (n = 177, 177) | 0.672 ± 0.704 | 0.463 ± 0.495 |
| M-1 IVA, Week 8: C3-6h (n = 174, 170) | 2.15 ± 1.19 | 1.91 ± 0.910 |
| M-1 IVA, Week 16: Ctrough (n = 171, 173) | 0.643 ± 0.594 | 0.465 ± 0.449 |
| M-6 IVA, Day 1: C3-6h (n = 181, 180) | 0.993 ± 0.820 | 0.996 ± 0.797 |
| M-6 IVA, Day 15: C3-6h (n = 174, 176) | 3.62 ± 2.18 | 2.99 ± 1.68 |
| M-6 IVA, Week 4: Ctrough (n = 174, 179) | 1.73 ± 1.34 | 1.64 ± 1.20 |
| M-6 IVA, Week 4: C3-6h (n = 164, 168) | 3.07 ± 1.84 | 2.64 ± 1.45 |
| M-6 IVA, Week 8: Ctrough (n = 177, 177) | 1.60 ± 1.22 | 1.47 ± 1.17 |
| M-6 IVA, Week 8: C3-6h (n = 174, 170) | 3.00 ± 2.01 | 2.56 ± 1.48 |
| M-6 IVA, Week 16: Ctrough (n = 171, 173) | 1.51 ± 1.29 | 1.42 ± 1.05 |
| LUM: Ctrough,avg (n = 179, 182) | 7.81 ± 4.26 | 12.7 ± 5.60 |
| LUM: C3-6h,avg (n = 182, 181) | 29.9 ± 9.19 | 24.3 ± 7.72 |
| M-28 LUM: Ctrough,avg (n = 179, 182) | 1.31 ± 0.672 | 1.42 ± 0.676 |
| M-28 LUM: C3-6h,avg (n = 182, 181) | 1.41 ± 0.620 | 1.43 ± 0.640 |
| IVA: Ctrough,avg (n = 179, 182) | 0.170 ± 0.196 | 0.110 ± 0.124 |
| IVA: C3-6h,avg (n = 182, 181) | 0.668 ± 0.392 | 0.484 ± 0.210 |
| M1-IVA: Ctrough,avg (n = 179, 182) | 0.660 ± 0.504 | 0.484 ± 0.391 |
| M1-IVA: C3-6h,avg (n = 182, 181) | 2.14 ± 0.951 | 1.87 ± 0.710 |
| M6-IVA: Ctrough,avg (n = 179, 182) | 1.60 ± 1.08 | 1.50 ± 0.897 |
| M6-IVA: C3-6h,avg (n = 182, 181) | 3.18 ± 1.65 | 2.70 ± 1.23 |
Collected over up to Week 28. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | — | 57/186 (30.6%) | 181/186 (97.3%) |
| LUM 600 mg qd/IVA 250 mg q12h | — | 51/186 (27.4%) | 179/186 (96.2%) |
| LUM 400 mg q12h/ IVA 250 mg q12h | — | 31/187 (16.6%) | 175/187 (93.6%) |
| Event | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 48/186 | 36/186 | 24/187 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 1/186 | 4/186 | 0/187 |
| Distal intestinal obstruction syndromeGastrointestinal disorders | 3/186 | 0/186 | 0/187 |
| BronchitisInfections and infestations | 2/186 | 1/186 | 0/187 |
| Bronchopulmonary aspergillosis allergicInfections and infestations | 0/186 | 2/186 | 0/187 |
| NephrolithiasisRenal and urinary disorders | 2/186 | 1/186 | 0/187 |
| Medical device complicationGeneral disorders | 0/186 | 2/186 | 0/187 |
| Blood creatine phosphokinase increasedInvestigations | 0/186 | 0/186 | 2/187 |
| BronchopneumoniaInfections and infestations | 0/186 | 1/186 | 1/187 |
| AppendicitisInfections and infestations | 0/186 | 1/186 | 0/187 |
| Event | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h |
|---|---|---|---|
| CoughRespiratory, thoracic and mediastinal disorders | 82/186 | 69/186 | 56/187 |
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 74/186 | 58/186 | 50/187 |
| Sputum increasedRespiratory, thoracic and mediastinal disorders | 47/186 | 40/186 | 29/187 |
| HeadacheNervous system disorders | 33/186 | 30/186 | 29/187 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 15/186 | 32/186 | 31/187 |
| NauseaGastrointestinal disorders | 17/186 | 20/186 | 32/187 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 26/186 | 28/186 | 20/187 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 19/186 | 24/186 | 13/187 |
| PyrexiaGeneral disorders | 22/186 | 22/186 | 16/187 |
| NasopharyngitisInfections and infestations | 20/186 | 14/186 | 22/187 |
Full Analysis Set (FAS) included all randomized participants who received any amount of study drug.
| Age, Continuous(years) | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h | Total |
|---|---|---|---|---|
| Mean | 25.7 ± 10.02 | 24.3 ± 8.31 | 25.0 ± 9.03 | 25.0 ± 9.16 |
| Sex: Female, Male(Participants) | Placebo | LUM 600 mg qd/IVA 250 mg q12h | LUM 400 mg q12h/ IVA 250 mg q12h | Total |
|---|---|---|---|---|
| Female | 97 | 96 | 98 | 291 |
| Male | 90 | 89 | 89 | 268 |
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