Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Completed
CT.gov ID
NCT02544451
Collaborator
(none)
246
61
4
56
4
0.1
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
246 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 6 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Actual Study Start Date
:
Aug 1, 2015
Actual Primary Completion Date
:
Aug 1, 2018
Actual Study Completion Date
:
Apr 1, 2020
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Treatment Period 1: LUM/IVA to LUM/IVA
|
Drug: LUM/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).
Other Names:
|
| Experimental: Treatment Period 1: Placebo (PBO) to LUM/IVA
|
Drug: LUM/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).
Other Names:
|
| No Intervention: Treatment Period 1: Observational Cohort
|
|
| Experimental: Treatment Period 2: LUM/IVA
|
Drug: LUM/IVA
LUM 200 mg q12h/IVA 250 mg q12h (for 6 through 11 years of age).
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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 Outcome Measures
- 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 Sweat Chloride [From Parent Study Baseline at Week 96]
Sweat samples were collected using an approved collection device.
- 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 Height [From Parent Study Baseline at Week 96]
- 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]
Eligibility Criteria
Criteria
Ages Eligible for Study:
6 Years
and Older
Sexes Eligible for Study:
All
Accepts 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).
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Birmingham | Alabama | United States | ||
| 2 | Tucson | Arizona | United States | ||
| 3 | Long Beach | California | United States | ||
| 4 | Palo Alto | California | United States | ||
| 5 | Aurora | Colorado | United States | ||
| 6 | Wilmington | Delaware | United States | ||
| 7 | Jacksonville | Florida | United States | ||
| 8 | Orlando | Florida | United States | ||
| 9 | Atlanta | Georgia | United States | ||
| 10 | Chicago | Illinois | United States | ||
| 11 | Indianapolis | Indiana | United States | ||
| 12 | Iowa City | Iowa | United States | ||
| 13 | Boston | Massachusetts | United States | ||
| 14 | Minneapolis | Minnesota | United States | ||
| 15 | Kansas City | Missouri | United States | ||
| 16 | Saint Louis | Missouri | United States | ||
| 17 | Omaha | Nebraska | United States | ||
| 18 | Lebanon | New Hampshire | United States | ||
| 19 | Buffalo | New York | United States | ||
| 20 | Syracuse | New York | United States | ||
| 21 | Chapel Hill | North Carolina | United States | ||
| 22 | Cincinnati | Ohio | United States | ||
| 23 | Cleveland | Ohio | United States | ||
| 24 | Dayton | Ohio | United States | ||
| 25 | Portland | Oregon | United States | ||
| 26 | Pittsburgh | Pennsylvania | United States | ||
| 27 | Charleston | South Carolina | United States | ||
| 28 | Austin | Texas | United States | ||
| 29 | Houston | Texas | United States | ||
| 30 | Salt Lake City | Utah | United States | ||
| 31 | Colchester | Vermont | United States | ||
| 32 | Charlottesville | Virginia | United States | ||
| 33 | Norfolk | Virginia | United States | ||
| 34 | Richmond | Virginia | United States | ||
| 35 | Seattle | Washington | United States | ||
| 36 | Madison | Wisconsin | United States | ||
| 37 | Milwaukee | Wisconsin | United States | ||
| 38 | Parkville | Victoria | Australia | ||
| 39 | Herston | Australia | |||
| 40 | New Lambton Heights | Australia | |||
| 41 | Subiaco | Australia | |||
| 42 | Westmead | Australia | |||
| 43 | Brussels | Belgium | |||
| 44 | Leuven | Belgium | |||
| 45 | Vancouver | British Columbia | Canada | ||
| 46 | Toronto | Ontario | Canada | ||
| 47 | Montreal | Quebec | Canada | ||
| 48 | Copenhagen | Denmark | |||
| 49 | Bron | Cedex | France | ||
| 50 | Bordeaux Cedex | France | |||
| 51 | Paris Cedex 15 | France | |||
| 52 | Paris | France | |||
| 53 | Berlin | Germany | |||
| 54 | Giessen | Germany | |||
| 55 | Hanover | Germany | |||
| 56 | Koeln | Germany | |||
| 57 | Stockholm | Sweden | |||
| 58 | Leeds | West Yorkshire | United Kingdom | ||
| 59 | Belfast | United Kingdom | |||
| 60 | Edinburgh | United Kingdom | |||
| 61 | London | United Kingdom |
Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02544451
Other Study ID Numbers:
- VX15-809-110
First Posted:
Sep 9, 2015
Last Update Posted:
May 24, 2021
Last Verified:
Feb 1, 2021
Study Results
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 Detail | 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. |
| Arm/Group Title | Treatment Period 1: LUM/IVA to LUM/IVA | Treatment Period 1: PBO to LUM/IVA | Treatment Period 1: Observational Cohort | Treatment Period 2: LUM/IVA |
|---|---|---|---|---|
| Arm/Group Description | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. | Participants received placebo (PBO) in parent study (109) and then received LUM/IVA for 96 weeks in the current study. | 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. | Eligible subjects from Treatment Period 1 received LUM/IVA for up to approximately 168 weeks. |
| Period Title: Treatment Period 1 (96 Weeks) | ||||
| STARTED | 143 | 96 | 6 | 0 |
| COMPLETED | 129 | 84 | 5 | 0 |
| NOT COMPLETED | 14 | 12 | 1 | 0 |
| Period Title: Treatment Period 1 (96 Weeks) | ||||
| STARTED | 0 | 0 | 0 | 10 |
| COMPLETED | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 0 | 0 | 0 | 10 |
Baseline Characteristics
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA | Observational Cohort | Total |
|---|---|---|---|---|
| Arm/Group Description | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. | Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. | 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. | Total of all reporting groups |
| Overall Participants | 143 | 96 | 6 | 245 |
| Age, Customized (Count of Participants) | ||||
| Less than 9 Years |
58
40.6%
|
38
39.6%
|
5
83.3%
|
101
41.2%
|
| Greater than or equal to 9 years |
85
59.4%
|
58
60.4%
|
1
16.7%
|
144
58.8%
|
| Sex: Female, Male (Count of Participants) | ||||
| Female |
83
58%
|
56
58.3%
|
2
33.3%
|
141
57.6%
|
| Male |
60
42%
|
40
41.7%
|
4
66.7%
|
104
42.4%
|
| Ethnicity (NIH/OMB) (Count of Participants) | ||||
| Hispanic or Latino |
2
1.4%
|
2
2.1%
|
0
0%
|
4
1.6%
|
| Not Hispanic or Latino |
139
97.2%
|
93
96.9%
|
6
100%
|
238
97.1%
|
| Unknown or Not Reported |
2
1.4%
|
1
1%
|
0
0%
|
3
1.2%
|
| Race (NIH/OMB) (Count of Participants) | ||||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Asian |
0
0%
|
1
1%
|
0
0%
|
1
0.4%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| White |
140
97.9%
|
92
95.8%
|
6
100%
|
238
97.1%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
3
2.1%
|
3
3.1%
|
0
0%
|
6
2.4%
|
Outcome Measures
| Title | 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) |
|---|---|
| Description | |
| Time Frame | Day 1 up to Week 100 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set included all participants who received at least 1 dose of study drug in Treatment Period 1. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. | Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. |
| Measure Participants | 143 | 96 |
| Participants with any AEs |
142
99.3%
|
94
97.9%
|
| Participants with SAEs |
43
30.1%
|
29
30.2%
|
| Title | Absolute Change in Lung Clearance Index (LCI) 2.5 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the LCI set who received LUM/IVA in the parent study. | All participants in the LCI set who received PBO in the parent study. |
| Measure Participants | 133 | 101 |
| Least Squares Mean (95% Confidence Interval) [lung clearance index] |
-0.85
|
-0.86
|
| Title | Absolute Change in Sweat Chloride |
|---|---|
| Description | Sweat samples were collected using an approved collection device. |
| Time Frame | From Parent Study Baseline at Week 96 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Full Analysis Set (FAS) includes all participants enrolled and dosed in either parent study. Analysis period includes both parent study and current study. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [millimole per liter (mmol/L)] |
-22.9
|
-22.8
|
| Title | Absolute Change in Body Mass Index (BMI) |
|---|---|
| Description | BMI was defined as weight in kilograms divided by height in square meter (m^2). |
| Time Frame | From Parent Study Baseline at Week 96 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [kg/m^2] |
1.78
|
2.04
|
| Title | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [units on a scale] |
7.4
|
6.6
|
| Title | Observational Cohort: Safety as Assessed by Serious Adverse Events (SAEs) |
|---|---|
| Description | |
| Time Frame | Day 1 up to Week 100 |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants included in the observational cohort. |
| Arm/Group Title | Observational Cohort |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 6 |
| Number [participants] |
1
0.7%
|
| Title | Absolute Change in LCI 5.0 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| LCI set. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the LCI set who received LUM/IVA in the parent study. | All participants in the LCI set who received PBO in the parent study. |
| Measure Participants | 133 | 101 |
| Least Squares Mean (95% Confidence Interval) [lung clearance index] |
-0.21
|
-0.31
|
| Title | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [percent predicted of FEV1] |
3.1
|
0.0
|
| Title | Relative Change in ppFEV1 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [percent change] |
4.9
|
0.5
|
| Title | Absolute Change in BMI-for-age Z-score |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [z-score] |
0.17
|
0.31
|
| Title | Absolute Change in Weight |
|---|---|
| Description | |
| Time Frame | From Parent Study Baseline at Week 96 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [kg] |
10.3
|
11.0
|
| Title | Absolute Change in Weight-for-age Z-score |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [z-score] |
0.12
|
0.24
|
| Title | Absolute Change in Height |
|---|---|
| Description | |
| Time Frame | From Parent Study Baseline at Week 96 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [centimeter (cm)] |
13.4
|
13.5
|
| Title | Absolute Change in Height-for-age Z-score |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [z-score] |
-0.01
|
0.02
|
| Title | Absolute Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Total Domain Score |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| FAS. |
| Arm/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants in the FAS who received LUM/IVA in the parent study. | All participants in the FAS who received PBO in the parent study. |
| Measure Participants | 161 | 101 |
| Least Squares Mean (95% Confidence Interval) [units on a scale] |
5.1
|
3.9
|
| Title | Time-to-first Pulmonary Exacerbation |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants who received LUM/IVA in parent study 109. | All participants who received PBO in parent study 109. |
| Measure Participants | 103 | 96 |
| Median (Inter-Quartile Range) [days] |
720.00
|
NA
|
| Title | Percentage of Participants Having At Least 1 Pulmonary Exacerbation Event |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants who received LUM/IVA in parent study 109. | All participants who received PBO in parent study 109. |
| Measure Participants | 103 | 96 |
| Number [percentage of participants] |
49.5
34.6%
|
32.3
33.6%
|
| Title | Number of Pulmonary Exacerbation Events Per Patient-year |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA to LUM/IVA | PBO to LUM/IVA |
|---|---|---|
| Arm/Group Description | All participants who received LUM/IVA in parent study 109. | All participants who received PBO in parent study 109. |
| Measure Participants | 103 | 96 |
| Number (95% Confidence Interval) [events per patient-year] |
0.45
|
0.30
|
| Title | Rate of Change in LCI 2.5 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA Overall |
|---|---|
| Arm/Group Description | All participants who received LUM/IVA in parent study 109, 011B LCI sub-study, or current study. |
| Measure Participants | 229 |
| Number (95% Confidence Interval) [slope] |
-0.01
|
| Title | Rate of Change in LCI 5.0 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA Overall |
|---|---|
| Arm/Group Description | All participants who received LUM/IVA in parent study 109, 011B LCI sub-study, or current study. |
| Measure Participants | 229 |
| Number (95% Confidence Interval) [slope] |
0.00
|
| Title | Rate of Change in ppFEV1 |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| 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/Group Title | LUM/IVA Overall |
|---|---|
| Arm/Group Description | All participants who received LUM/IVA in either parent study or current study. |
| Measure Participants | 257 |
| Number (95% Confidence Interval) [slope] |
0.58
|
| Title | Treatment Period 2: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) |
|---|---|
| Description | |
| Time Frame | Day 1 up to Week 168 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set included all participants who received at least 1 dose of study drug in Treatment Period 2. |
| Arm/Group Title | Treatment Period 2: LUM/IVA |
|---|---|
| Arm/Group Description | Eligible subjects from Treatment Period 1 received LUM/IVA for up to approximately 168 weeks. |
| Measure Participants | 10 |
| Participants with any AEs |
9
6.3%
|
| Participants with SAEs |
0
0%
|
Adverse Events
| Time Frame | Treatment Period 1: Day 1 up to Week 100, Treatment Period 2: Day 1 up to Week 168 | |||||||
|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | 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. | |||||||
| Arm/Group Title | Treatment Period 1: LUM/IVA to LUM/IVA | Treatment Period 1: PBO to LUM/IVA | Treatment Period 1: Observational Cohort | Treatment Period 2: LUM/IVA | ||||
| Arm/Group Description | Participants received LUM/IVA in parent studies (109, 011B) and continued to receive LUM/IVA for 96 weeks in the current study. | Participants received PBO in parent study (109) and then received LUM/IVA for 96 weeks in the current study. | 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. | Eligible subjects from Treatment Period 1 received LUM/IVA for up to approximately 168 weeks. | ||||
All Cause Mortality |
||||||||
| Treatment Period 1: LUM/IVA to LUM/IVA | Treatment Period 1: PBO to LUM/IVA | Treatment Period 1: Observational Cohort | Treatment Period 2: LUM/IVA | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/143 (0%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
Serious Adverse Events |
||||||||
| Treatment Period 1: LUM/IVA to LUM/IVA | Treatment Period 1: PBO to LUM/IVA | Treatment Period 1: Observational Cohort | Treatment Period 2: LUM/IVA | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 43/143 (30.1%) | 29/96 (30.2%) | 1/6 (16.7%) | 0/10 (0%) | ||||
| Blood and lymphatic system disorders | ||||||||
| Lymphadenitis | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Congenital, familial and genetic disorders | ||||||||
| Cystic fibrosis hepatic disease | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Cystic fibrosis lung | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Gastrointestinal disorders | ||||||||
| Constipation | 4/143 (2.8%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Distal intestinal obstruction syndrome | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Oesophagitis | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| General disorders | ||||||||
| Pyrexia | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Hepatobiliary disorders | ||||||||
| Autoimmune hepatitis | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Infections and infestations | ||||||||
| Infective pulmonary exacerbation of cystic fibrosis | 34/143 (23.8%) | 15/96 (15.6%) | 1/6 (16.7%) | 0/10 (0%) | ||||
| Bronchopulmonary aspergillosis allergic | 2/143 (1.4%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Pneumonia | 1/143 (0.7%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Appendicitis | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Atypical mycobacterial lower respiratory tract infection | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Gastroenteritis viral | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Lower respiratory tract infection bacterial | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Viral upper respiratory tract infection | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Injury, poisoning and procedural complications | ||||||||
| Intentional overdose | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Investigations | ||||||||
| Pulmonary function test decreased | 2/143 (1.4%) | 2/96 (2.1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Alanine aminotransferase increased | 0/143 (0%) | 2/96 (2.1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Aspartate aminotransferase increased | 0/143 (0%) | 2/96 (2.1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Pseudomonas test positive | 1/143 (0.7%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Atypical mycobacterium test positive | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Oxygen saturation decreased | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Metabolism and nutrition disorders | ||||||||
| Diabetic ketoacidosis | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Hyperglycaemia | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Renal and urinary disorders | ||||||||
| Nephrolithiasis | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Haemoptysis | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Pleuritic pain | 1/143 (0.7%) | 0/96 (0%) | 0/6 (0%) | 0/10 (0%) | ||||
| Sinus disorder | 0/143 (0%) | 1/96 (1%) | 0/6 (0%) | 0/10 (0%) | ||||
Other (Not Including Serious) Adverse Events |
||||||||
| Treatment Period 1: LUM/IVA to LUM/IVA | Treatment Period 1: PBO to LUM/IVA | Treatment Period 1: Observational Cohort | Treatment Period 2: LUM/IVA | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 141/143 (98.6%) | 93/96 (96.9%) | 0/0 (NaN) | 9/10 (90%) | ||||
| Ear and labyrinth disorders | ||||||||
| Ear pain | 12/143 (8.4%) | 4/96 (4.2%) | 4/0 (Infinity) | 0/10 (0%) | ||||
| Eye disorders | ||||||||
| Eyelid oedema | 0/143 (0%) | 0/96 (0%) | 0/0 (NaN) | 1/10 (10%) | ||||
| Gastrointestinal disorders | ||||||||
| Vomiting | 30/143 (21%) | 15/96 (15.6%) | 15/0 (Infinity) | 0/10 (0%) | ||||
| Abdominal pain upper | 22/143 (15.4%) | 20/96 (20.8%) | 20/0 (Infinity) | 0/10 (0%) | ||||
| Abdominal pain | 17/143 (11.9%) | 19/96 (19.8%) | 19/0 (Infinity) | 1/10 (10%) | ||||
| Diarrhoea | 16/143 (11.2%) | 8/96 (8.3%) | 8/0 (Infinity) | 1/10 (10%) | ||||
| Nausea | 13/143 (9.1%) | 11/96 (11.5%) | 11/0 (Infinity) | 1/10 (10%) | ||||
| Constipation | 11/143 (7.7%) | 11/96 (11.5%) | 11/0 (Infinity) | 1/10 (10%) | ||||
| Flatulence | 4/143 (2.8%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Dyspepsia | 1/143 (0.7%) | 0/96 (0%) | 0/0 (NaN) | 1/10 (10%) | ||||
| Gastritis | 0/143 (0%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| General disorders | ||||||||
| Pyrexia | 45/143 (31.5%) | 27/96 (28.1%) | 27/0 (Infinity) | 1/10 (10%) | ||||
| Fatigue | 10/143 (7%) | 11/96 (11.5%) | 11/0 (Infinity) | 0/10 (0%) | ||||
| Chest pain | 2/143 (1.4%) | 5/96 (5.2%) | 5/0 (Infinity) | 0/10 (0%) | ||||
| Hepatobiliary disorders | ||||||||
| Hepatomegaly | 1/143 (0.7%) | 0/96 (0%) | 0/0 (NaN) | 1/10 (10%) | ||||
| Immune system disorders | ||||||||
| Seasonal allergy | 8/143 (5.6%) | 5/96 (5.2%) | 5/0 (Infinity) | 0/10 (0%) | ||||
| Infections and infestations | ||||||||
| Infective pulmonary exacerbation of cystic fibrosis | 59/143 (41.3%) | 34/96 (35.4%) | 34/0 (Infinity) | 4/10 (40%) | ||||
| Upper respiratory tract infection | 36/143 (25.2%) | 13/96 (13.5%) | 13/0 (Infinity) | 0/10 (0%) | ||||
| Nasopharyngitis | 21/143 (14.7%) | 16/96 (16.7%) | 16/0 (Infinity) | 3/10 (30%) | ||||
| Viral upper respiratory tract infection | 21/143 (14.7%) | 14/96 (14.6%) | 14/0 (Infinity) | 0/10 (0%) | ||||
| Sinusitis | 17/143 (11.9%) | 8/96 (8.3%) | 8/0 (Infinity) | 0/10 (0%) | ||||
| Otitis media | 13/143 (9.1%) | 8/96 (8.3%) | 8/0 (Infinity) | 0/10 (0%) | ||||
| Pharyngitis streptococcal | 12/143 (8.4%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Bacterial disease carrier | 11/143 (7.7%) | 6/96 (6.3%) | 6/0 (Infinity) | 2/10 (20%) | ||||
| Influenza | 11/143 (7.7%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Ear infection | 9/143 (6.3%) | 7/96 (7.3%) | 7/0 (Infinity) | 1/10 (10%) | ||||
| Rhinitis | 9/143 (6.3%) | 7/96 (7.3%) | 7/0 (Infinity) | 6/10 (60%) | ||||
| Upper respiratory tract infection bacterial | 8/143 (5.6%) | 1/96 (1%) | 1/0 (Infinity) | 0/10 (0%) | ||||
| Bronchitis | 7/143 (4.9%) | 7/96 (7.3%) | 7/0 (Infinity) | 2/10 (20%) | ||||
| Pharyngitis | 5/143 (3.5%) | 7/96 (7.3%) | 7/0 (Infinity) | 0/10 (0%) | ||||
| Gastroenteritis | 7/143 (4.9%) | 4/96 (4.2%) | 4/0 (Infinity) | 1/10 (10%) | ||||
| Oral fungal infection | 2/143 (1.4%) | 0/96 (0%) | 0/0 (NaN) | 1/10 (10%) | ||||
| Respiratory tract infection bacterial | 3/143 (2.1%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| Injury, poisoning and procedural complications | ||||||||
| Ligament sprain | 4/143 (2.8%) | 2/96 (2.1%) | 2/0 (Infinity) | 1/10 (10%) | ||||
| Investigations | ||||||||
| Bacterial test positive | 30/143 (21%) | 16/96 (16.7%) | 16/0 (Infinity) | 0/10 (0%) | ||||
| Alanine aminotransferase increased | 22/143 (15.4%) | 21/96 (21.9%) | 21/0 (Infinity) | 0/10 (0%) | ||||
| Aspartate aminotransferase increased | 17/143 (11.9%) | 13/96 (13.5%) | 13/0 (Infinity) | 0/10 (0%) | ||||
| Pseudomonas test positive | 10/143 (7%) | 3/96 (3.1%) | 3/0 (Infinity) | 0/10 (0%) | ||||
| Pulmonary function test decreased | 8/143 (5.6%) | 11/96 (11.5%) | 11/0 (Infinity) | 0/10 (0%) | ||||
| Activated partial thromboplastin time prolonged | 6/143 (4.2%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Forced expiratory volume decreased | 6/143 (4.2%) | 13/96 (13.5%) | 13/0 (Infinity) | 1/10 (10%) | ||||
| International normalised ratio increased | 4/143 (2.8%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Prothrombin time prolonged | 3/143 (2.1%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Weight decreased | 1/143 (0.7%) | 5/96 (5.2%) | 5/0 (Infinity) | 1/10 (10%) | ||||
| Pulmonary imaging procedure abnormal | 0/143 (0%) | 0/96 (0%) | 0/0 (NaN) | 1/10 (10%) | ||||
| Metabolism and nutrition disorders | ||||||||
| Decreased appetite | 3/143 (2.1%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Glucose tolerance impaired | 1/143 (0.7%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| Musculoskeletal and connective tissue disorders | ||||||||
| Arthralgia | 5/143 (3.5%) | 5/96 (5.2%) | 5/0 (Infinity) | 1/10 (10%) | ||||
| Nervous system disorders | ||||||||
| Headache | 29/143 (20.3%) | 26/96 (27.1%) | 26/0 (Infinity) | 1/10 (10%) | ||||
| Dizziness | 6/143 (4.2%) | 5/96 (5.2%) | 5/0 (Infinity) | 0/10 (0%) | ||||
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Cough | 91/143 (63.6%) | 64/96 (66.7%) | 64/0 (Infinity) | 2/10 (20%) | ||||
| Nasal congestion | 34/143 (23.8%) | 21/96 (21.9%) | 21/0 (Infinity) | 0/10 (0%) | ||||
| Oropharyngeal pain | 32/143 (22.4%) | 18/96 (18.8%) | 18/0 (Infinity) | 0/10 (0%) | ||||
| Rhinorrhoea | 24/143 (16.8%) | 13/96 (13.5%) | 13/0 (Infinity) | 0/10 (0%) | ||||
| Productive cough | 19/143 (13.3%) | 15/96 (15.6%) | 15/0 (Infinity) | 0/10 (0%) | ||||
| Sputum increased | 18/143 (12.6%) | 7/96 (7.3%) | 7/0 (Infinity) | 0/10 (0%) | ||||
| Sinus congestion | 12/143 (8.4%) | 4/96 (4.2%) | 4/0 (Infinity) | 0/10 (0%) | ||||
| Nasal polyps | 9/143 (6.3%) | 3/96 (3.1%) | 3/0 (Infinity) | 0/10 (0%) | ||||
| Haemoptysis | 8/143 (5.6%) | 1/96 (1%) | 1/0 (Infinity) | 0/10 (0%) | ||||
| Wheezing | 8/143 (5.6%) | 4/96 (4.2%) | 4/0 (Infinity) | 1/10 (10%) | ||||
| Respiration abnormal | 7/143 (4.9%) | 7/96 (7.3%) | 7/0 (Infinity) | 0/10 (0%) | ||||
| Asthma | 5/143 (3.5%) | 5/96 (5.2%) | 5/0 (Infinity) | 0/10 (0%) | ||||
| Dyspnoea | 4/143 (2.8%) | 6/96 (6.3%) | 6/0 (Infinity) | 0/10 (0%) | ||||
| Bronchiectasis | 0/143 (0%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| Epistaxis | 5/143 (3.5%) | 2/96 (2.1%) | 2/0 (Infinity) | 2/10 (20%) | ||||
| Lower respiratory tract congestion | 1/143 (0.7%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| Paranasal sinus hypersecretion | 4/143 (2.8%) | 2/96 (2.1%) | 2/0 (Infinity) | 1/10 (10%) | ||||
| Rales | 2/143 (1.4%) | 1/96 (1%) | 1/0 (Infinity) | 1/10 (10%) | ||||
| Rhinitis allergic | 4/143 (2.8%) | 4/96 (4.2%) | 4/0 (Infinity) | 1/10 (10%) | ||||
| Skin and subcutaneous tissue disorders | ||||||||
| Rash | 10/143 (7%) | 10/96 (10.4%) | 10/0 (Infinity) | 0/10 (0%) | ||||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Results Point of Contact
| Name/Title | Medical Monitor |
|---|---|
| Organization | Vertex Pharmaceuticals Incorporated |
| Phone | 617-341-6777 |
| medicalinfo@vrtx.com |
Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT02544451
Other Study ID Numbers:
- VX15-809-110
First Posted:
Sep 9, 2015
Last Update Posted:
May 24, 2021
Last Verified:
Feb 1, 2021