Trial Outcomes & Findings for A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation (NCT NCT02875366)
NCT ID: NCT02875366
Last Updated: 2019-06-17
Results Overview
CPET was used to assess change in exercise tolerance, as measured by VO2max.
COMPLETED
PHASE4
70 participants
Baseline, Week 24
2019-06-17
Participant Flow
Participant milestones
| Measure |
Placebo
Participants received placebo matched to lumacaftor (LUM)/ivacaftor (IVA) fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
|
LUM/IVA
Participants received LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
36
|
34
|
|
Overall Study
COMPLETED
|
36
|
31
|
|
Overall Study
NOT COMPLETED
|
0
|
3
|
Reasons for withdrawal
| Measure |
Placebo
Participants received placebo matched to lumacaftor (LUM)/ivacaftor (IVA) fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.
|
LUM/IVA
Participants received LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Overall Study
Adverse Event
|
0
|
2
|
|
Overall Study
Noncompliance
|
0
|
1
|
Baseline Characteristics
A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation
Baseline characteristics by cohort
| Measure |
Placebo
n=36 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
Total
n=70 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
26.1 years
STANDARD_DEVIATION 10.58 • n=5 Participants
|
24.9 years
STANDARD_DEVIATION 10.17 • n=7 Participants
|
25.5 years
STANDARD_DEVIATION 10.33 • n=5 Participants
|
|
Sex: Female, Male
Female
|
18 Participants
n=5 Participants
|
13 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
18 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
39 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
36 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
70 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
36 Participants
n=5 Participants
|
34 Participants
n=7 Participants
|
70 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: The Full Analysis Set (FAS) included all randomized participants who received any amount of study drug. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Maximal Oxygen Consumption (VO2max) During Cardiopulmonary Exercise Testing (CPET) at Week 24
|
-3.5 percent change
Interval -7.7 to 0.8
|
-6.6 percent change
Interval -11.3 to -2.0
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Exercise Duration During CPET at Week 24
|
0.4 percent change
Interval -3.0 to 3.8
|
-2.8 percent change
Interval -6.5 to 1.0
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Exercise duration is defined as the time at the termination of CPET exercise minus the corresponding time when CPET starts for each CPET exercise.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Exercise Duration During CPET at Week 24
|
-2.1 seconds
Interval -20.0 to 15.9
|
-17.4 seconds
Interval -37.2 to 2.4
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
CPET was used to assess change in exercise tolerance, as measured by VO2max.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in VO2max During CPET at Week 24
|
-1.3 milliliter per kilogram per minute
Interval -2.5 to -0.1
|
-2.7 milliliter per kilogram per minute
Interval -4.0 to -1.3
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=25 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Oxygen Consumption (VO2) at Anaerobic Threshold at Week 24
|
94.6 milliliter per minute
Interval -5.8 to 195.0
|
-55.1 milliliter per minute
Interval -168.0 to 57.9
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Anaerobic threshold was defined as the exercise intensity at which lactate starts to accumulate.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=25 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in VO2 at Anaerobic Threshold at Week 24
|
9.4 percent change
Interval 0.9 to 17.8
|
1.8 percent change
Interval -7.7 to 11.3
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Functional VO2 Gain at Week 24
|
0.07 milliliter per minute per watt
Interval -0.29 to 0.43
|
-0.53 milliliter per minute per watt
Interval -0.93 to -0.14
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Functional VO2 Gain at Week 24
|
1.46 percent change
Interval -3.1 to 6.03
|
-4.85 percent change
Interval -9.93 to 0.22
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Outcome measures
| Measure |
Placebo
n=31 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
|
0.5 ratio
Interval -0.3 to 1.4
|
0.8 ratio
Interval -0.1 to 1.7
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Outcome measures
| Measure |
Placebo
n=31 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=26 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Pulmonary Ventilation (VE) Versus Carbon Dioxide Production (VCO2) Slope at Week 24
|
2.0 percent change
Interval -0.7 to 4.6
|
3.0 percent change
Interval 0.1 to 5.8
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=30 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
|
-4.0 percentage of predicted FEV1
Interval -7.3 to -0.7
|
-0.6 percentage of predicted FEV1
Interval -4.0 to 2.9
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Outcome measures
| Measure |
Placebo
n=32 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=30 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Week 24
|
-5.4 percent change
Interval -10.3 to -0.5
|
-1.8 percent change
Interval -6.9 to 3.2
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
BMI was defined as weight in kilograms (kg) divided by height in square meter (m\^2).
Outcome measures
| Measure |
Placebo
n=33 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=30 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
|
0.3 kg/m^2
Interval 0.0 to 0.6
|
0.5 kg/m^2
Interval 0.1 to 0.8
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
BMI was defined as weight in kg divided by height in m\^2.
Outcome measures
| Measure |
Placebo
n=33 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=30 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in BMI at Week 24
|
1.5 percent change
Interval 0.0 to 3.1
|
2.5 percent change
Interval 0.9 to 4.1
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
The CFQ-R assessed respiratory symptoms on a scale with scores ranging from 0 to 100; where higher scores indicated fewer symptoms and better health-related quality of life.
Outcome measures
| Measure |
Placebo
n=33 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=30 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24
|
-6.1 units on a scale
Interval -11.7 to -0.5
|
0.1 units on a scale
Interval -5.9 to 6.1
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Number Analyzed" signifies those participants who were evaluated for this outcome at the specified time point.
The PHQ-8 is an eight item self-reported measure of depression. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual eight items and ranges from 0 to 24, with higher scores indicating more severe depression symptoms. Total score of 0 to 5 indicates none to minimal depression, 6 to 10 indicates mild depression, 11 to 15 indicates moderate depression, 16 to 20 indicates moderately severe depression and 21 to 24 indicates severe depression.
Outcome measures
| Measure |
Placebo
n=36 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Baseline: None to minimal
|
27 participants
|
28 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Baseline: Mild
|
8 participants
|
5 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Baseline: Moderate
|
0 participants
|
1 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Baseline: Moderately severe
|
1 participants
|
0 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Baseline: Severe
|
0 participants
|
0 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Week 24: None to minimal
|
23 participants
|
24 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Week 24: Mild
|
8 participants
|
3 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Week 24: Moderate
|
1 participants
|
3 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Week 24: Moderately severe
|
1 participants
|
0 participants
|
|
Number of Participants in Each Severity Category of Patient Health Questionnaire (PHQ-8)
Week 24: Severe
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Number Analyzed" signifies those participants who were evaluated for this outcome at the specified time point.
The GAD-7 is a seven item, self-reported measurement of GAD severity. Each item is rated on a scale ranging from 0 (not at all) to 3 (nearly every day). Total score is the sum of individual seven items and ranges from 0 to 21, with higher scores indicating more severe anxiety symptoms. Total score of 0 to 5 indicates none to minimal anxiety, 6 to 10 indicates mild anxiety, 11 to 15 indicates moderate anxiety, 16 to 21 indicates severe anxiety.
Outcome measures
| Measure |
Placebo
n=36 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Baseline: None to minimal
|
31 participants
|
29 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Week 24: None to minimal
|
26 participants
|
26 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Week 24: Mild
|
6 participants
|
3 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Week 24: Moderate
|
1 participants
|
1 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Week 24: Severe
|
0 participants
|
0 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Baseline: Mild
|
5 participants
|
5 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Baseline: Moderate
|
0 participants
|
0 participants
|
|
Number of Participants in Each Severity Category of Generalized Anxiety Disorder (GAD-7) Scores
Baseline: Severe
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activity counts.
Outcome measures
| Measure |
Placebo
n=19 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=20 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Daily Physical Activity Counts as Determined by Actigraphy at Week 24
|
-23239 physical activity counts per day
Standard Deviation 73936.4
|
-22409 physical activity counts per day
Standard Deviation 52450.1
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily physical activities.
Outcome measures
| Measure |
Placebo
n=19 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=20 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Physical Activity as Determined by Actigraphy at Week 24
|
-5 percent change
Standard Deviation 26.9
|
-7 percent change
Standard Deviation 24.3
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Outcome measures
| Measure |
Placebo
n=18 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=18 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Duration of Sleep Time at Week 24
|
-0.4 hours
Standard Deviation 0.88
|
0.1 hours
Standard Deviation 0.74
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about sleep duration and quality.
Outcome measures
| Measure |
Placebo
n=18 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=18 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Duration of Sleep Time at Week 24
|
-5.7 percent change
Standard Deviation 12.30
|
1.5 percent change
Standard Deviation 9.79
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Outcome measures
| Measure |
Placebo
n=15 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=21 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Absolute Change From Baseline in Time Above Sedentary Duration at Week 24
|
-0.7 hours
Standard Deviation 1.48
|
-0.7 hours
Standard Deviation 2.09
|
SECONDARY outcome
Timeframe: Baseline, Week 24Population: FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome.
Participants were provided with a wrist-worn actigraphy device which continuously collected data about daily activities and sleep duration and quality.
Outcome measures
| Measure |
Placebo
n=15 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=21 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Relative (Percent) Change From Baseline in Time Above Sedentary Duration at Week 24
|
0.2 percent change
Standard Deviation 64.76
|
2.3 percent change
Standard Deviation 77.18
|
SECONDARY outcome
Timeframe: Day 1 up to Week 28Population: The Safety Set was defined as all participants who received at least 1 dose of study drug.
Outcome measures
| Measure |
Placebo
n=36 Participants
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 Participants
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants With AEs
|
35 participants
|
30 participants
|
|
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Participants With SAEs
|
9 participants
|
15 participants
|
Adverse Events
Placebo
LUM/IVA
Serious adverse events
| Measure |
Placebo
n=36 participants at risk
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 participants at risk
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
16.7%
6/36 • Day 1 up to Week 28
|
23.5%
8/34 • Day 1 up to Week 28
|
|
Infections and infestations
Lower respiratory tract infection bacterial
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Infections and infestations
Cytomegalovirus infection
|
2.8%
1/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Distal intestinal obstruction syndrome
|
0.00%
0/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Inguinal hernia
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Cardiac disorders
Tachycardia
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
General disorders
Fatigue
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Reproductive system and breast disorders
Testicular torsion
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
2.8%
1/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
2.8%
1/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Infections and infestations
Infection
|
0.00%
0/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
Other adverse events
| Measure |
Placebo
n=36 participants at risk
Participants received placebo matched to LUM/IVA fixed-dose combination tablet orally q12h for 24 weeks.
|
LUM/IVA
n=34 participants at risk
Participants received LUM 400 mg/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.
|
|---|---|---|
|
Infections and infestations
Infective pulmonary exacerbation of cystic fibrosis
|
16.7%
6/36 • Day 1 up to Week 28
|
23.5%
8/34 • Day 1 up to Week 28
|
|
Infections and infestations
Upper respiratory tract infection
|
11.1%
4/36 • Day 1 up to Week 28
|
11.8%
4/34 • Day 1 up to Week 28
|
|
Infections and infestations
Nasopharyngitis
|
8.3%
3/36 • Day 1 up to Week 28
|
8.8%
3/34 • Day 1 up to Week 28
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.6%
2/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Infections and infestations
Sinusitis
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Infections and infestations
Vulvovaginal candidiasis
|
8.3%
3/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
22.2%
8/36 • Day 1 up to Week 28
|
14.7%
5/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Respiration abnormal
|
25.0%
9/36 • Day 1 up to Week 28
|
14.7%
5/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
2.8%
1/36 • Day 1 up to Week 28
|
8.8%
3/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Increased viscosity of bronchial secretion
|
0.00%
0/36 • Day 1 up to Week 28
|
8.8%
3/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Sputum increased
|
13.9%
5/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
11.1%
4/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
5.6%
2/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
8.3%
3/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/36 • Day 1 up to Week 28
|
8.8%
3/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Nausea
|
13.9%
5/36 • Day 1 up to Week 28
|
8.8%
3/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
3/36 • Day 1 up to Week 28
|
2.9%
1/34 • Day 1 up to Week 28
|
|
Gastrointestinal disorders
Constipation
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
General disorders
Pyrexia
|
0.00%
0/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Investigations
Blood creatine phosphokinase increased
|
2.8%
1/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Investigations
Bacterial test positive
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Investigations
Blood iron decreased
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Investigations
Fungal test positive
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Psychiatric disorders
Anxiety
|
0.00%
0/36 • Day 1 up to Week 28
|
5.9%
2/34 • Day 1 up to Week 28
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Nervous system disorders
Headache
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.6%
2/36 • Day 1 up to Week 28
|
0.00%
0/34 • Day 1 up to Week 28
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
- Publication restrictions are in place
Restriction type: OTHER