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A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02951182
Recruitment Status : Completed
First Posted : November 1, 2016
Results First Posted : August 28, 2020
Last Update Posted : August 28, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: TEZ
Drug: IVA
Drug: VX-440
Drug: Matched Placebo
Enrollment 74
Recruitment Details Of the 74 participants enrolled (47 participants in Part 1 and 27 participants in Part 2), 1 participant in Part 2 discontinued in the Run-in Period because continuation criteria were not met and was not randomized in the Treatment Period. Therefore, only 73 participants are included in the results below.
Pre-assignment Details Four parts were originally planned for the study; only Parts 1 and 2 were conducted. Part 3 was removed in protocol Version 2.0. Part 4 was not conducted at the Sponsor's discretion.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description Participants received placebo matched to VX-440/tezacaftor (TEZ; VX-661)/ivacaftor (IVA, VX-770) as triple combination for 4 weeks. Participants received VX-440 200 milligram (mg) every 12 hours (q12h)/TEZ 100 mg once daily (qd)/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Period Title: Overall Study
Started 11 9 9 18 6 20
Completed 11 9 9 18 6 20
Not Completed 0 0 0 0 0 0
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2 Total
Hide Arm/Group Description Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks. Participants received VX-440 200 mg q12h/TEZ 100 mg qd/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. Total of all reporting groups
Overall Number of Baseline Participants 11 9 9 18 6 20 73
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 9 participants 18 participants 6 participants 20 participants 73 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
11
 100.0%
9
 100.0%
9
 100.0%
18
 100.0%
6
 100.0%
20
 100.0%
73
 100.0%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 9 participants 18 participants 6 participants 20 participants 73 participants
Female
2
  18.2%
1
  11.1%
3
  33.3%
1
   5.6%
0
   0.0%
4
  20.0%
11
  15.1%
Male
9
  81.8%
8
  88.9%
6
  66.7%
17
  94.4%
6
 100.0%
16
  80.0%
62
  84.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 9 participants 18 participants 6 participants 20 participants 73 participants
Hispanic or Latino
1
   9.1%
0
   0.0%
1
  11.1%
0
   0.0%
0
   0.0%
1
   5.0%
3
   4.1%
Not Hispanic or Latino
10
  90.9%
9
 100.0%
8
  88.9%
17
  94.4%
6
 100.0%
18
  90.0%
68
  93.2%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
1
   5.6%
0
   0.0%
1
   5.0%
2
   2.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 9 participants 18 participants 6 participants 20 participants 73 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
10
  90.9%
9
 100.0%
9
 100.0%
18
 100.0%
6
 100.0%
20
 100.0%
72
  98.6%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
1
   9.1%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.4%
1.Primary Outcome
Title Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description [Not Specified]
Time Frame From first dose of Study Drug in the Treatment Period through Safety Follow-up Visit (Up to Day 57 for Part 1 and Day 85 for Part 2)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set included all participants who received at least 1 dose of study drug in the Treatment Period.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
Participants received VX-440 200 mg q12h/TEZ 100 mg qd/IVA 150 mg q12h as triple combination for 4 weeks.
Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 11 9 9 18 6 20
Measure Type: Number
Unit of Measure: participants
Participants with AEs 9 9 9 15 6 15
Participants with SAEs 0 0 0 2 2 1
2.Primary Outcome
Title Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame From Baseline through Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) included all randomized participants who have received at least 1 dose of study drug in the Treatment Period. As per pre-specified planned analysis, reporting group "Part 1 Cohort 1A: TC" and "Part 1 Cohort 1B: TC Low Dose" were pooled for the purpose of efficacy analysis.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1: TC-1A/ TC-1B-low Pooled Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
All participants pooled together for Part 1: TC-1A arm and Part 1: TC-1B low dose arm.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 11 18 18 6 20
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percentage points
1.4
(-2.7 to 5.5)
10.0
(6.9 to 13.2)
12.0
(8.8 to 15.2)
-2.5
(-7.2 to 2.2)
9.5
(6.9 to 12.2)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1: TC-1A/ TC-1B-low Pooled
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0016
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 8.6
Confidence Interval (2-Sided) 95%
3.5 to 13.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1 Cohort 1B: TC High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 10.6
Confidence Interval (2-Sided) 95%
5.5 to 15.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Part 2: TEZ/IVA, Part 2: TC-2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Mixed-effects Model for Repeated Measure
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 12.0
Confidence Interval (2-Sided) 95%
6.7 to 17.4
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Absolute Change in Sweat Chloride Concentrations
Hide Description Sweat samples were collected using an approved collection device.
Time Frame From Baseline through Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. As per pre-specified planned analysis, reporting group "Part 1 Cohort 1A: TC" and "Part 1 Cohort 1B: TC Low Dose" were pooled for the purpose of efficacy analysis.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1: TC-1A/ TC-1B-low Pooled Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
All participants pooled together for Part 1: TC-1A arm and Part 1: TC-1B low dose arm.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 11 18 18 6 20
Least Squares Mean (95% Confidence Interval)
Unit of Measure: millimole per liter (mmol/L)
1.6
(-6.2 to 9.4)
-20.7
(-26.6 to -14.7)
-33.1
(-39.1 to -27.1)
2.1
(-10.9 to 15.1)
-31.3
(-38.6 to -24.1)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1: TC-1A/ TC-1B-low Pooled
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value -22.3
Confidence Interval (2-Sided) 95%
-32.1 to -12.4
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1 Cohort 1B: TC High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value -34.7
Confidence Interval (2-Sided) 95%
-44.7 to -24.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Part 2: TEZ/IVA, Part 2: TC-2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value -33.4
Confidence Interval (2-Sided) 95%
-48.5 to -18.3
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Relative Change in ppFEV1
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Time Frame From Baseline through Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. As per pre-specified planned analysis, reporting group "Part 1 Cohort 1A: TC" and "Part 1 Cohort 1B: TC Low Dose" were pooled for the purpose of efficacy analysis.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1: TC-1A/ TC-1B-low Pooled Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
All participants pooled together for Part 1: TC-1A arm and Part 1: TC-1B low dose arm.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 11 18 18 6 20
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percent change
2.6
(-4.9 to 10.0)
17.3
(11.5 to 23.2)
21.7
(15.9 to 27.5)
-3.4
(-11.4 to 4.6)
16.6
(12.1 to 21.1)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1: TC-1A/ TC-1B-low Pooled
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 14.8
Confidence Interval (2-Sided) 95%
5.3 to 24.2
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1 Cohort 1B: TC High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 19.1
Confidence Interval (2-Sided) 95%
9.7 to 28.6
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Part 2: TEZ/IVA, Part 2: TC-2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 20.0
Confidence Interval (2-Sided) 95%
10.8 to 29.1
Estimation Comments [Not Specified]
5.Secondary Outcome
Title Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
Hide 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 Baseline at Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
FAS. As per pre-specified planned analysis, reporting group "Part 1 Cohort 1A: TC" and "Part 1 Cohort 1B: TC Low Dose" were pooled for the purpose of efficacy analysis.
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1: TC-1A/ TC-1B-low Pooled Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks.
All participants pooled together for Part 1: TC-1A arm and Part 1: TC-1B low dose arm.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 11 18 18 6 20
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
2.2
(-6.2 to 10.6)
18.3
(11.7 to 24.9)
20.7
(14.2 to 27.1)
-7.8
(-14.8 to -0.8)
12.3
(8.7 to 16.0)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1: TC-1A/ TC-1B-low Pooled
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 16.1
Confidence Interval (2-Sided) 95%
5.4 to 26.8
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Part 1: Placebo - Cohort 1A and 1B Combined, Part 1 Cohort 1B: TC High Dose
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 18.5
Confidence Interval (2-Sided) 95%
7.9 to 29.1
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Part 2: TEZ/IVA, Part 2: TC-2
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Method of Estimation Estimation Parameter Least Squares (LS) Mean Difference
Estimated Value 20.2
Confidence Interval (2-Sided) 95%
11.9 to 28.4
Estimation Comments [Not Specified]
6.Secondary Outcome
Title Pre-dose Plasma Concentration (Ctrough) of VX-440, TEZ, M1-TEZ, IVA and M1-IVA
Hide Description [Not Specified]
Time Frame Predose at Day 8, Day 15 and Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic Set (PK) included all participants who have received at least 1 dose of study drug in Treatment Period. Here "Number Analyzed" signifies those participants who were evaluable at specified time points. Day 8 assessment was planned for only TC-1A arm and VX-440 Ctrough category was not applicable for TEZ/IVA arm.
Arm/Group Title Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description:
Participants received VX-440 200 mg q12h/TEZ 100 mg qd/IVA 150 mg q12h as triple combination for 4 weeks.
Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks.
Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
Overall Number of Participants Analyzed 9 9 18 6 19
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter (ng/mL)
VX-440: Day 8 Number Analyzed 9 participants 0 participants 0 participants 0 participants 0 participants
1670  (1910)
VX-440: Day 15 Number Analyzed 9 participants 8 participants 16 participants 0 participants 18 participants
1840  (1460) 1120  (707) 8540  (6850) 12900  (9940)
VX-440: Day 29 Number Analyzed 9 participants 9 participants 17 participants 0 participants 17 participants
1090  (1010) 1440  (1870) 6650  (3500) 10300  (7340)
TEZ: Day 8 Number Analyzed 8 participants 0 participants 0 participants 0 participants 0 participants
810  (323)
TEZ: Day 15 Number Analyzed 9 participants 8 participants 16 participants 6 participants 18 participants
749  (271) 1070  (422) 928  (558) 2240  (1010) 1250  (837)
TEZ: Day 29 Number Analyzed 9 participants 9 participants 17 participants 6 participants 17 participants
761  (320) 1040  (659) 846  (514) 1420  (565) 893  (579)
M1-TEZ: Day 8 Number Analyzed 8 participants 0 participants 0 participants 0 participants 0 participants
3160  (645)
M1-TEZ: Day 15 Number Analyzed 9 participants 8 participants 16 participants 6 participants 18 participants
3490  (486) 4610  (1170) 3400  (1270) 4640  (1730) 3940  (1200)
M1-TEZ: Day 29 Number Analyzed 9 participants 9 participants 17 participants 6 participants 17 participants
3560  (378) 4180  (1950) 3050  (1260) 4060  (1560) 3280  (1230)
IVA: Day 8 Number Analyzed 8 participants 0 participants 0 participants 0 participants 0 participants
281  (167)
IVA: Day 15 Number Analyzed 9 participants 8 participants 16 participants 6 participants 18 participants
245  (92.7) 192  (97.0) 233  (149) 1040  (352) 380  (312)
IVA: Day 29 Number Analyzed 9 participants 9 participants 17 participants 6 participants 17 participants
209  (112) 164  (108) 219  (139) 902  (344) 290  (263)
M1-IVA: Day 8 Number Analyzed 8 participants 0 participants 0 participants 0 participants 0 participants
795  (327)
M1-IVA: Day 15 Number Analyzed 9 participants 8 participants 16 participants 6 participants 18 participants
854  (427) 566  (245) 850  (546) 1640  (218) 1220  (796)
M1-IVA: Day 29 Number Analyzed 9 participants 9 participants 17 participants 6 participants 17 participants
702  (372) 520  (338) 792  (466) 1580  (764) 1080  (732)
Time Frame From first dose of Study Drug in the Treatment Period through Safety Follow-up Visit (Up to Day 57 for Part 1 and Day 85 for Part 2)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Hide Arm/Group Description Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks. Participants received VX-440 200 mg q12/TEZ 100 mg qd/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks. Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period.
All-Cause Mortality
Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)   0/9 (0.00%)   0/9 (0.00%)   0/18 (0.00%)   0/6 (0.00%)   0/20 (0.00%) 
Hide Serious Adverse Events
Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)   0/9 (0.00%)   0/9 (0.00%)   2/18 (11.11%)   2/6 (33.33%)   1/20 (5.00%) 
Gastrointestinal disorders             
Distal intestinal obstruction syndrome  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Infections and infestations             
Infective pulmonary exacerbation of cystic fibrosis  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/6 (16.67%)  1/20 (5.00%) 
Psychiatric disorders             
Adjustment disorder  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
1
Term from vocabulary, MedDRA 20.0
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Part 1: Placebo - Cohort 1A and 1B Combined Part 1 Cohort 1A: Triple Combination (TC) Part 1 Cohort 1B: TC Low Dose Part 1 Cohort 1B: TC High Dose Part 2: TEZ/IVA Part 2: TC-2
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   9/11 (81.82%)   9/9 (100.00%)   9/9 (100.00%)   15/18 (83.33%)   5/6 (83.33%)   15/20 (75.00%) 
Ear and labyrinth disorders             
Ear pain  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Eye disorders             
Eye pruritus  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Gastrointestinal disorders             
Diarrhoea  1  1/11 (9.09%)  1/9 (11.11%)  1/9 (11.11%)  2/18 (11.11%)  0/6 (0.00%)  4/20 (20.00%) 
Abdominal pain  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  2/18 (11.11%)  1/6 (16.67%)  0/20 (0.00%) 
Abdominal pain upper  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Abnormal faeces  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Flatulence  1  1/11 (9.09%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Anorectal discomfort  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
Dyspepsia  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Faeces pale  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Nausea  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
General disorders             
Fatigue  1  0/11 (0.00%)  0/9 (0.00%)  2/9 (22.22%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Chills  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Influenza like illness  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Pain  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Pyrexia  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Immune system disorders             
Anaphylactic reaction  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Hypersensitivity  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Infections and infestations             
Infective pulmonary exacerbation of cystic fibrosis  1  2/11 (18.18%)  6/9 (66.67%)  0/9 (0.00%)  2/18 (11.11%)  0/6 (0.00%)  2/20 (10.00%) 
Viral upper respiratory tract infection  1  1/11 (9.09%)  0/9 (0.00%)  2/9 (22.22%)  2/18 (11.11%)  2/6 (33.33%)  1/20 (5.00%) 
Upper respiratory tract infection  1  0/11 (0.00%)  2/9 (22.22%)  0/9 (0.00%)  2/18 (11.11%)  0/6 (0.00%)  2/20 (10.00%) 
Folliculitis  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
Gastroenteritis  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Influenza  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Pharyngitis  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
Injury, poisoning and procedural complications             
Anaesthetic complication neurological  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Contusion  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Joint injury  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Ligament rupture  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Post procedural swelling  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Post-traumatic neck syndrome  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Procedural nausea  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Procedural pain  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Sunburn  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Investigations             
Alanine aminotransferase increased  1  1/11 (9.09%)  0/9 (0.00%)  1/9 (11.11%)  2/18 (11.11%)  0/6 (0.00%)  2/20 (10.00%) 
Aspartate aminotransferase increased  1  1/11 (9.09%)  0/9 (0.00%)  1/9 (11.11%)  2/18 (11.11%)  0/6 (0.00%)  1/20 (5.00%) 
Blood urine present  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
International normalised ratio increased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Activated partial thromboplastin time prolonged  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Blood bicarbonate decreased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Blood bilirubin increased  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Blood bilirubin unconjugated increased  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Blood chloride increased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Blood creatinine increased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Blood glucose increased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Blood pressure increased  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Crystal urine present  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Lipase decreased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Monocyte count increased  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Prothrombin time prolonged  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Pulmonary function test decreased  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Weight decreased  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Metabolism and nutrition disorders             
Hypoglycaemia  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Hyperglycaemia  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Increased appetite  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Musculoskeletal and connective tissue disorders             
Musculoskeletal chest pain  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Arthralgia  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Back pain  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Flank pain  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Muscle twitching  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Myalgia  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Neck pain  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Tendon discomfort  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
Tendonitis  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Nervous system disorders             
Headache  1  1/11 (9.09%)  1/9 (11.11%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Dizziness  1  2/11 (18.18%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Lethargy  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Presyncope  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Syncope  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Psychiatric disorders             
Anxiety  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Insomnia  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  1/6 (16.67%)  0/20 (0.00%) 
Mood swings  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Renal and urinary disorders             
Nephrolithiasis  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Reproductive system and breast disorders             
Testicular cyst  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  4/11 (36.36%)  3/9 (33.33%)  1/9 (11.11%)  1/18 (5.56%)  0/6 (0.00%)  5/20 (25.00%) 
Sputum increased  1  3/11 (27.27%)  1/9 (11.11%)  1/9 (11.11%)  1/18 (5.56%)  1/6 (16.67%)  3/20 (15.00%) 
Haemoptysis  1  1/11 (9.09%)  0/9 (0.00%)  1/9 (11.11%)  1/18 (5.56%)  0/6 (0.00%)  1/20 (5.00%) 
Dyspnoea  1  2/11 (18.18%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Nasal congestion  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  2/20 (10.00%) 
Oropharyngeal pain  1  2/11 (18.18%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Paranasal sinus hypersecretion  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Respiration abnormal  1  2/11 (18.18%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Dysphonia  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Lower respiratory tract congestion  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  2/20 (10.00%) 
Productive cough  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  0/6 (0.00%)  0/20 (0.00%) 
Rhinorrhoea  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Wheezing  1  0/11 (0.00%)  1/9 (11.11%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Dyspnoea exertional  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Increased viscosity of bronchial secretion  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Paranasal sinus discomfort  1  0/11 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Pulmonary pain  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Rales  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Sinus disorder  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Sinus pain  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Throat irritation  1  0/11 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  1/20 (5.00%) 
Skin and subcutaneous tissue disorders             
Acne  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Alopecia  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Blister  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Hyperhidrosis  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Night sweats  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Pruritus  1  1/11 (9.09%)  0/9 (0.00%)  0/9 (0.00%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Skin disorder  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
Vascular disorders             
Hot flush  1  0/11 (0.00%)  0/9 (0.00%)  1/9 (11.11%)  0/18 (0.00%)  0/6 (0.00%)  0/20 (0.00%) 
1
Term from vocabulary, MedDRA 20.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
 
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
Phone: 617-341-6777
EMail: medicalinfo@vrtx.com
Layout table for additonal information
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT02951182    
Other Study ID Numbers: VX15-440-101
2016-000454-36 ( EudraCT Number )
First Submitted: October 26, 2016
First Posted: November 1, 2016
Results First Submitted: August 11, 2020
Results First Posted: August 28, 2020
Last Update Posted: August 28, 2020