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A Study to Evaluate the Safety and Efficacy of VX-121 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: NCT03912233
Recruitment Status : Completed
First Posted : April 11, 2019
Results First Posted : April 20, 2023
Last Update Posted : April 20, 2023
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Sequential Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: VX-121
Drug: TEZ
Drug: VX-561
Drug: TEZ/IVA
Drug: IVA
Drug: Placebo
Enrollment 87
Recruitment Details Three parts were planned for this study, only Parts 1 (participants heterozygous for F508del and a minimal function mutation [F/MF genotypes]) and 2 (participants homozygous for F508del [F/F genotypes]) were conducted. Part 3 was optional and not conducted at sponsor's discretion.
Pre-assignment Details A total of 87 participants were enrolled in this study (58 participants in Part 1 and 29 participants in Part 2 run-in Period), 1 participant in Part 2 run-in period discontinued from the study and was not randomized in the treatment period. Therefore, results are presented for 86 participants in this study.
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description Participants received placebo matched to VX-121/tezacaftor (TEZ)/VX-561 triple combination (TC) for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period. Participants received VX-121 5 milligram (mg) once daily (qd)/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Following run-in period with TEZ 100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period. Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Period Title: Overall Study
Started 10 9 19 20 10 18
Completed 8 9 19 20 3 6
Not Completed 2 0 0 0 7 12
Reason Not Completed
Adverse Event             1             0             0             0             0             0
Physician Decision             1             0             0             0             0             0
Other             0             0             0             0             7             12
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose Total
Hide Arm/Group Description Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period. Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period. Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period. Total of all reporting groups
Overall Number of Baseline Participants 10 9 19 20 10 18 86
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants 20 participants 10 participants 18 participants 86 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
10
 100.0%
9
 100.0%
19
 100.0%
20
 100.0%
10
 100.0%
18
 100.0%
86
 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 10 participants 9 participants 19 participants 20 participants 10 participants 18 participants 86 participants
Female
2
  20.0%
4
  44.4%
3
  15.8%
9
  45.0%
2
  20.0%
7
  38.9%
27
  31.4%
Male
8
  80.0%
5
  55.6%
16
  84.2%
11
  55.0%
8
  80.0%
11
  61.1%
59
  68.6%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants 20 participants 10 participants 18 participants 86 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
2
  10.0%
1
  10.0%
0
   0.0%
3
   3.5%
Not Hispanic or Latino
10
 100.0%
8
  88.9%
19
 100.0%
17
  85.0%
8
  80.0%
18
 100.0%
80
  93.0%
Unknown or Not Reported
0
   0.0%
1
  11.1%
0
   0.0%
1
   5.0%
1
  10.0%
0
   0.0%
3
   3.5%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants 20 participants 10 participants 18 participants 86 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%
1
   5.3%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.2%
White
9
  90.0%
8
  88.9%
18
  94.7%
17
  85.0%
9
  90.0%
18
 100.0%
79
  91.9%
More than one race
1
  10.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.2%
Unknown or Not Reported
0
   0.0%
1
  11.1%
0
   0.0%
3
  15.0%
1
  10.0%
0
   0.0%
5
   5.8%
Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants 9 participants 19 participants 20 participants 10 participants 18 participants 86 participants
<40 percent
1
  10.0%
1
  11.1%
1
   5.3%
0
   0.0%
1
  10.0%
2
  11.1%
6
   7.0%
>=40 to <70 percent
9
  90.0%
6
  66.7%
14
  73.7%
17
  85.0%
6
  60.0%
11
  61.1%
63
  73.3%
>=70 to <=90 percent
0
   0.0%
2
  22.2%
4
  21.1%
3
  15.0%
3
  30.0%
5
  27.8%
17
  19.8%
[1]
Measure Description: FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
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 Day 1 Through Safety Follow-up (up to Day 75 for Part 1 and up to 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 Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description:
Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.
Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Overall Number of Participants Analyzed 10 9 19 20 10 18
Measure Type: Number
Unit of Measure: participants
Participants With AEs 9 8 16 20 8 16
Participants With SAEs 2 1 1 0 0 0
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 carry the intended cystic fibrosis transmembrane conductance regulator gene (CFTR) allele mutation(s) and received at least 1 dose of study drug in the treatment period.
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description:
Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.
Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Overall Number of Participants Analyzed 10 9 19 20 10 18
Least Squares Mean (95% Confidence Interval)
Unit of Measure: percentage points
1.9
(-4.1 to 8.0)
4.6
(-1.3 to 10.6)
14.2
(10.0 to 18.4)
9.8
(5.7 to 13.8)
-0.1
(-6.4 to 6.1)
15.9
(11.3 to 20.6)
3.Secondary Outcome
Title Absolute Change in Sweat Chloride (SwCl) 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.
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description:
Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.
Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Overall Number of Participants Analyzed 10 9 19 20 10 18
Least Squares Mean (95% Confidence Interval)
Unit of Measure: millimole per liter (mmol/L)
2.3
(-7.0 to 11.6)
-42.8
(-51.7 to -34.0)
-45.8
(-51.9 to -39.7)
-49.5
(-55.9 to -43.1)
-2.6
(-8.2 to 3.1)
-45.5
(-49.7 to -41.3)
4.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.
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description:
Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period.
Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Overall Number of Participants Analyzed 10 9 19 20 10 18
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
3.3
(-10.1 to 16.6)
17.6
(3.5 to 31.6)
21.2
(11.9 to 30.6)
29.8
(21.0 to 38.7)
-5.0
(-16.9 to 7.0)
19.4
(10.5 to 28.3)
5.Secondary Outcome
Title Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ and Its Metabolite (M1-TEZ) and, VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561)
Hide Description [Not Specified]
Time Frame Pre-dose at Day 15 and Day 29
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetic (PK) set included all participants who received at least 1 dose study drug in the treatment period and for whom the PK data are considered sufficient and interpretable. Participants who received VX-121/TEZ/VX-561 TC in Parts 1 or 2 were to be analyzed for Ctrough. Overall participants in Part 1 were assessed for Ctrough, therefore data are reported in single Part 1: TC combined arm. The "number analyzed" signifies participants who were evaluable at specified time point.
Arm/Group Title Part 1: VX-121/TEZ/VX-561 TC - Combined Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description:
Participants who either received VX-121 5 mg, 10 mg or 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period.
Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
Overall Number of Participants Analyzed 48 18
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter (ng/mL)
Day 15: VX-121 5 mg Number Analyzed 8 participants 0 participants
317  (119)
Day 29: VX-121 5 mg Number Analyzed 7 participants 0 participants
366  (130)
Day 15: VX-121 10 mg Number Analyzed 18 participants 0 participants
520  (214)
Day 29: VX-121 10 mg Number Analyzed 14 participants 0 participants
582  (342)
Day 15: VX-121 20 mg Number Analyzed 20 participants 17 participants
974  (500) 1050  (414)
Day 29: VX-121 20 mg Number Analyzed 18 participants 16 participants
1160  (592) 1030  (371)
Day 15: TEZ Number Analyzed 46 participants 17 participants
1890  (925) 1870  (675)
Day 29: TEZ Number Analyzed 46 participants 16 participants
1920  (994) 2070  (1340)
Day 15: M1-TEZ Number Analyzed 46 participants 17 participants
4500  (1290) 4550  (1200)
Day 29: M1-TEZ Number Analyzed 46 participants 16 participants
4640  (1730) 4440  (1680)
Day 15: VX-561 Number Analyzed 46 participants 17 participants
475  (247) 457  (264)
Day 29: VX-561 Number Analyzed 46 participants 16 participants
510  (285) 434  (257)
Day 15: M1-VX-561 Number Analyzed 46 participants 17 participants
311  (141) 326  (175)
Day 29: M1-VX-561 Number Analyzed 46 participants 16 participants
336  (173) 316  (188)
Day 15: M6-VX-561 Number Analyzed 46 participants 17 participants
148  (98.0) 174  (128)
Day 29: M6-VX-561 Number Analyzed 46 participants 16 participants
163  (128) 159  (94.6)
Time Frame From Day 1 Through Safety Follow-up (up to Day 75 for Part 1 and up to Day 85 for Part 2)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Hide Arm/Group Description Participants received placebo matched to VX-121/TEZ/VX-561 TC for 4 weeks in the treatment period and placebo matched to TEZ/VX-561 for 18 days in the washout period. Participants received VX-121 5 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 10 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/VX-561 150 mg qd for 18 days in the washout period. Following run-in period with TEZ 100 mg/IVA 150 mg q12h for 4 weeks, participants received TEZ 100 mg/IVA 150 mg q12h for 4 weeks in the treatment period and TEZ 100 mg/IVA 150 mg q12h for 4 weeks in the washout period. Following run-in period with TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks, participants received VX-121 20 mg qd/TEZ 100 mg qd/VX-561 150 mg qd TC for 4 weeks in the treatment period and TEZ 100 mg qd/IVA 150 mg q12h for 4 weeks in the washout period.
All-Cause Mortality
Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/10 (0.00%)   0/9 (0.00%)   0/19 (0.00%)   0/20 (0.00%)   0/10 (0.00%)   0/18 (0.00%) 
Hide Serious Adverse Events
Part 1: Placebo Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/10 (20.00%)   1/9 (11.11%)   1/19 (5.26%)   0/20 (0.00%)   0/10 (0.00%)   0/18 (0.00%) 
Infections and infestations             
Infective pulmonary exacerbation of cystic fibrosis † 1  1/10 (10.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Breast cancer † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Skin and subcutaneous tissue disorders             
Urticaria † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
1
Term from vocabulary, MedDRA 22.1
†
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 Part 1: VX-121/TEZ/VX-561 TC - Low Dose Part 1: VX-121/TEZ/VX-561 TC - Medium Dose Part 1: VX-121/TEZ/VX-561 TC - High Dose Part 2: TEZ/IVA Part 2: VX-121/TEZ/VX-561 TC - High Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   9/10 (90.00%)   8/9 (88.89%)   16/19 (84.21%)   20/20 (100.00%)   8/10 (80.00%)   16/18 (88.89%) 
Blood and lymphatic system disorders             
Anaemia † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Lymphadenopathy † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Thrombocytopenia † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Cardiac disorders             
Palpitations † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Ear and labyrinth disorders             
Ear pain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Eye disorders             
Swelling of eyelid † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Gastrointestinal disorders             
Abdominal discomfort † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Abdominal distension † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  1/20 (5.00%)  1/10 (10.00%)  0/18 (0.00%) 
Abdominal pain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  2/20 (10.00%)  1/10 (10.00%)  1/18 (5.56%) 
Abdominal pain upper † 1  0/10 (0.00%)  1/9 (11.11%)  1/19 (5.26%)  2/20 (10.00%)  0/10 (0.00%)  0/18 (0.00%) 
Constipation † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  2/20 (10.00%)  0/10 (0.00%)  0/18 (0.00%) 
Diarrhoea † 1  0/10 (0.00%)  0/9 (0.00%)  4/19 (21.05%)  5/20 (25.00%)  1/10 (10.00%)  2/18 (11.11%) 
Dyspepsia † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Flatulence † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  2/20 (10.00%)  0/10 (0.00%)  0/18 (0.00%) 
Frequent bowel movements † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Gastritis † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Nausea † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  1/18 (5.56%) 
Post-tussive vomiting † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Salivary hypersecretion † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  1/18 (5.56%) 
General disorders             
Asthenia † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Chest pain † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Chills † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  3/10 (30.00%)  0/18 (0.00%) 
Fatigue † 1  0/10 (0.00%)  2/9 (22.22%)  5/19 (26.32%)  2/20 (10.00%)  2/10 (20.00%)  0/18 (0.00%) 
Feeling abnormal † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Influenza like illness † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Malaise † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Oedema peripheral † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Pain † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Pyrexia † 1  2/10 (20.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  2/10 (20.00%)  0/18 (0.00%) 
Immune system disorders             
Seasonal allergy † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Infections and infestations             
Bronchopulmonary aspergillosis † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Bronchopulmonary aspergillosis allergic † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Conjunctivitis † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Infective pulmonary exacerbation of cystic fibrosis † 1  4/10 (40.00%)  3/9 (33.33%)  0/19 (0.00%)  3/20 (15.00%)  2/10 (20.00%)  0/18 (0.00%) 
Nasopharyngitis † 1  1/10 (10.00%)  2/9 (22.22%)  2/19 (10.53%)  2/20 (10.00%)  2/10 (20.00%)  2/18 (11.11%) 
Oral candidiasis † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Rhinitis † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  1/18 (5.56%) 
Sinusitis † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Tooth infection † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Upper respiratory tract infection † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  1/18 (5.56%) 
Viral rash † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Viral upper respiratory tract infection † 1  0/10 (0.00%)  0/9 (0.00%)  3/19 (15.79%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Vulvovaginal mycotic infection † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Injury, poisoning and procedural complications             
Arthropod bite † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Contusion † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Ligament sprain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Limb injury † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Skin laceration † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Investigations             
Activated partial thromboplastin time prolonged † 1  1/10 (10.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Alanine aminotransferase increased † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Aspartate aminotransferase increased † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood bilirubin increased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood bilirubin unconjugated increased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood creatine phosphokinase increased † 1  0/10 (0.00%)  1/9 (11.11%)  3/19 (15.79%)  1/20 (5.00%)  0/10 (0.00%)  2/18 (11.11%) 
Blood glucose increased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood potassium increased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood pressure diastolic increased † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Blood pressure increased † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Blood sodium decreased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Crystal urine present † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Eosinophil count increased † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Forced expiratory volume decreased † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Gamma-glutamyltransferase increased † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Glucose urine present † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
International normalised ratio increased † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Weight increased † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
White blood cell count increased † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Metabolism and nutrition disorders             
Decreased appetite † 1  0/10 (0.00%)  0/9 (0.00%)  2/19 (10.53%)  1/20 (5.00%)  2/10 (20.00%)  0/18 (0.00%) 
Hyperglycaemia † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Hypoglycaemia † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  2/18 (11.11%) 
Polydipsia † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Musculoskeletal and connective tissue disorders             
Arthralgia † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Back pain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Intervertebral disc protrusion † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Musculoskeletal chest pain † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Musculoskeletal pain † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Myalgia † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  2/10 (20.00%)  0/18 (0.00%) 
Neck pain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Pain in extremity † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Tendon pain † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Nervous system disorders             
Dizziness † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  2/20 (10.00%)  0/10 (0.00%)  1/18 (5.56%) 
Dysgeusia † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Headache † 1  1/10 (10.00%)  2/9 (22.22%)  4/19 (21.05%)  6/20 (30.00%)  1/10 (10.00%)  2/18 (11.11%) 
Paraesthesia † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Psychiatric disorders             
Abnormal dreams † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Anxiety † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Insomnia † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  1/10 (10.00%)  0/18 (0.00%) 
Mood swings † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Panic attack † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Sleep disorder † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Somnambulism † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Renal and urinary disorders             
Nephrolithiasis † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Pollakiuria † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Respiratory, thoracic and mediastinal disorders             
Cough † 1  5/10 (50.00%)  4/9 (44.44%)  5/19 (26.32%)  9/20 (45.00%)  7/10 (70.00%)  5/18 (27.78%) 
Dysphonia † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Dyspnoea † 1  1/10 (10.00%)  1/9 (11.11%)  2/19 (10.53%)  3/20 (15.00%)  2/10 (20.00%)  0/18 (0.00%) 
Epistaxis † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Haemoptysis † 1  1/10 (10.00%)  0/9 (0.00%)  0/19 (0.00%)  2/20 (10.00%)  1/10 (10.00%)  0/18 (0.00%) 
Lower respiratory tract congestion † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Nasal congestion † 1  0/10 (0.00%)  1/9 (11.11%)  1/19 (5.26%)  3/20 (15.00%)  2/10 (20.00%)  1/18 (5.56%) 
Nasal obstruction † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Oropharyngeal pain † 1  0/10 (0.00%)  2/9 (22.22%)  3/19 (15.79%)  2/20 (10.00%)  0/10 (0.00%)  3/18 (16.67%) 
Painful respiration † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  1/10 (10.00%)  1/18 (5.56%) 
Paranasal sinus hypersecretion † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Pleuritic pain † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  1/10 (10.00%)  0/18 (0.00%) 
Productive cough † 1  3/10 (30.00%)  0/9 (0.00%)  2/19 (10.53%)  3/20 (15.00%)  0/10 (0.00%)  0/18 (0.00%) 
Rales † 1  1/10 (10.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Respiration abnormal † 1  0/10 (0.00%)  1/9 (11.11%)  1/19 (5.26%)  0/20 (0.00%)  1/10 (10.00%)  0/18 (0.00%) 
Rhinorrhoea † 1  0/10 (0.00%)  0/9 (0.00%)  2/19 (10.53%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Sinus congestion † 1  0/10 (0.00%)  1/9 (11.11%)  1/19 (5.26%)  1/20 (5.00%)  1/10 (10.00%)  0/18 (0.00%) 
Sneezing † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Sputum increased † 1  3/10 (30.00%)  6/9 (66.67%)  3/19 (15.79%)  4/20 (20.00%)  3/10 (30.00%)  5/18 (27.78%) 
Throat clearing † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Wheezing † 1  0/10 (0.00%)  1/9 (11.11%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Skin and subcutaneous tissue disorders             
Acne † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
Exfoliative rash † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Hyperhidrosis † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  0/20 (0.00%)  0/10 (0.00%)  1/18 (5.56%) 
Night sweats † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Pruritus † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Rash † 1  1/10 (10.00%)  1/9 (11.11%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  3/18 (16.67%) 
Rash pruritic † 1  0/10 (0.00%)  0/9 (0.00%)  0/19 (0.00%)  1/20 (5.00%)  0/10 (0.00%)  0/18 (0.00%) 
Skin lesion † 1  0/10 (0.00%)  0/9 (0.00%)  1/19 (5.26%)  0/20 (0.00%)  0/10 (0.00%)  0/18 (0.00%) 
1
Term from vocabulary, MedDRA 22.1
†
Indicates events were collected by systematic assessment
[Not Specified]
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: NCT03912233    
Other Study ID Numbers: VX18-121-101
First Submitted: April 10, 2019
First Posted: April 11, 2019
Results First Submitted: October 13, 2021
Results First Posted: April 20, 2023
Last Update Posted: April 20, 2023