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Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion

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ClinicalTrials.gov Identifier: NCT02070744
Recruitment Status : Completed
First Posted : February 25, 2014
Results First Posted : April 13, 2018
Last Update Posted : April 13, 2018
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: VX-661
Drug: Ivacaftor
Drug: Placebo matched to VX-661
Drug: Placebo matched to Ivacaftor
Enrollment 40
Recruitment Details  
Pre-assignment Details The study consisted of 2 phases: a randomized, double-blind, placebo-controlled (PC) phase in which participants received either VX-661 in combination with Ivacaftor (IVA), or matched placebo and an open-label extension (OLE) phase in which participants received VX-661 in combination with IVA.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description Participants received VX-661 50 milligram (mg) tablet plus IVA 150 mg tablet every 12 hours (q12h) for 12 weeks. Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. Participants received two VX-661 50 mg tablets once daily (qd) plus IVA 150 mg tablet q12h for 12 weeks. Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Period Title: PC Phase (12 Weeks)
Started 6 5 15 14 0
Treated 6 5 15 13 0
Completed 6 5 15 13 0
Not Completed 0 0 0 1 0
Reason Not Completed
Randomized, but not treated             0             0             0             1             0
Period Title: OLE Phase (48 Weeks)
Started 0 0 0 0 27 [1]
Completed 0 0 0 0 24
Not Completed 0 0 0 0 3
Reason Not Completed
Withdrawal by Subject             0             0             0             0             2
Other             0             0             0             0             1
[1]
12 participants completed PC phase, but did not enter OLE phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h Total
Hide Arm/Group Description Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks. Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks. Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. Total of all reporting groups
Overall Number of Baseline Participants 6 5 15 13 39
Hide Baseline Analysis Population Description
Full Analysis Set (FAS) was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants 5 participants 15 participants 13 participants 39 participants
33.0  (10.6) 24.2  (2.2) 27.3  (5.2) 30.3  (10.8) 28.8  (8.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants 5 participants 15 participants 13 participants 39 participants
Female
2
  33.3%
2
  40.0%
7
  46.7%
3
  23.1%
14
  35.9%
Male
4
  66.7%
3
  60.0%
8
  53.3%
10
  76.9%
25
  64.1%
1.Primary Outcome
Title PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase) up to 112 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set was defined as all participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Measure Type: Count of Participants
Unit of Measure: Participants
Participants with AEs
6
 100.0%
5
 100.0%
15
 100.0%
13
 100.0%
Participants with SAEs
1
  16.7%
2
  40.0%
4
  26.7%
5
  38.5%
2.Primary Outcome
Title OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs
Hide Description AE: Any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation considered treatment-emergent. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase) up to 364 days
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Set was defined as all participants who received at least 1 dose of study drug in OLE Phase.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Measure Type: Count of Participants
Unit of Measure: Participants
Participants with AEs
25
  92.6%
Participants with SAEs
6
  22.2%
3.Secondary Outcome
Title PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Through Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Percent predicted of FEV1
0.9
(-3.1 to 5.0)
-0.1
(-4.4 to 4.3)
3.0
(0.4 to 5.5)
1.9
(-0.9 to 4.6)
4.Secondary Outcome
Title OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Through Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in OLE phase.
Arm/Group Title OLE Phase:VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Percent predicted of FEV1
2.7
(0.4 to 4.9)
5.Secondary Outcome
Title PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Through Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg + IVA 150 mg PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Percent change
2.6
(-6.0 to 11.1)
1.0
(-8.2 to 10.3)
6.0
(0.6 to 11.3)
4.2
(-1.5 to 10.0)
6.Secondary Outcome
Title OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40
Hide Description FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Through Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Percent change
6.1
(1.7 to 10.5)
7.Secondary Outcome
Title PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12
Hide Description Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Through Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase. Here, “Number of participants analyzed” signifies those participants who were evaluable for this outcome measure.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 5 5 13 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Millimole per liter (mmol/L)
-10.6
(-16.6 to -4.6)
2.9
(-3.0 to 8.9)
-4.7
(-8.4 to -0.9)
0.8
(-3.1 to 4.7)
8.Secondary Outcome
Title OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40
Hide Description Sweat samples were collected using an approved collection device. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Through Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase. Here, “Number of participants analyzed” signifies those participants who were evaluable for this outcome measure.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 25
Least Squares Mean (95% Confidence Interval)
Unit of Measure: mmol/L
-6.6
(-9.7 to -3.5)
9.Secondary Outcome
Title PC Phase: Absolute Change From Baseline in Body Weight at Week 12
Hide Description Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kilogram (kg)
1.0
(-0.4 to 2.3)
1.6
(0.1 to 3.0)
0.5
(-0.3 to 1.4)
0.3
(-0.6 to 1.3)
10.Secondary Outcome
Title OLE Phase: Absolute Change From Baseline in Body Weight at Week 40
Hide Description Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kg
1.0
(-0.2 to 2.2)
11.Secondary Outcome
Title PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12
Hide Description BMI was calculated using following formula: BMI = Weight in kg/height in square meter (m^2). Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: Kilogram per square meter (kg/m^2)
0.38
(-0.08 to 0.84)
0.54
(0.03 to 1.04)
0.18
(-0.11 to 0.47)
0.11
(-0.21 to 0.42)
12.Secondary Outcome
Title OLE Phase: Absolute Change From Baseline BMI at Week 40
Hide Description BMI was calculated using following formula: BMI = Weight in kg/height in m^2. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kg/m^2
0.33
(-0.09 to 0.74)
13.Secondary Outcome
Title PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of PC Phase.
Time Frame Baseline (PC Phase), Through Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
FAS was defined as all randomized participants who received at least 1 dose of study drug in PC Phase.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 5 15 13
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
5.6
(-3.9 to 15.1)
-4.6
(-14.9 to 5.7)
1.0
(-5.1 to 7.0)
0.4
(-6.2 to 7.1)
14.Secondary Outcome
Title OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40
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 (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Baseline was defined as Day 1 of the OLE Phase.
Time Frame Baseline (OLE Phase), Through Week 40
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population was defined as all participants who received at least 1 dose of study drug in the OLE Phase.
Arm/Group Title OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
Overall Number of Participants Analyzed 27
Least Squares Mean (95% Confidence Interval)
Unit of Measure: units on a scale
-0.6
(-5.8 to 4.5)
15.Secondary Outcome
Title PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
Hide Description [Not Specified]
Time Frame Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 15
Mean (Standard Deviation)
Unit of Measure: Nanogram per milliliter (ng/mL)
VX-661 4890  (3150) 6460  (1240)
IVA 1490  (1150) 1210  (585)
16.Secondary Outcome
Title PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661
Hide Description Pharmacokinetic (PK) sampling was performed up to 12 hours post-dose on Day 85. For Arm VX-661 50 mg q12h + IVA 150 mg q12h (VX-661 q12h regimen), area under the concentration versus time curve from time 0 to 12 hours (AUC0-12h) was multiplied by 2 to obtain AUC0-24h.
Time Frame Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. Here, “Number of participants analyzed” signifies those participants who were evaluable for this outcome measure.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 5 15
Mean (Standard Deviation)
Unit of Measure: Hour*nanogram per milliliter (hr*ng/mL)
84900  (55900) 75500  (20300)
17.Secondary Outcome
Title PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
Hide Description [Not Specified]
Time Frame Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses. Here, “Number of participants analyzed” signifies those participants who were evaluable for this outcome measure.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 5 15
Mean (Standard Deviation)
Unit of Measure: hr*ng/mL
14700  (11600) 10100  (4890)
18.Secondary Outcome
Title PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
Hide Description [Not Specified]
Time Frame Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population included all participants who received a dose of VX-661 and IVA, whether the participant completed dosing or not and if the dataset(s) supported the noncompartmental analyses.
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description:
Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks.
Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks.
Overall Number of Participants Analyzed 6 15
Median (Full Range)
Unit of Measure: hour
VX-661
2.48
(1.75 to 4.08)
3.23
(1.92 to 6.03)
IVA
3.59
(1.98 to 6.00)
4.00
(2.98 to 6.03)
Time Frame Baseline (Day 1 of PC Phase) up to 112 days (Placebo control phase); Baseline (Day 1 of OLE Phase) up to 364 days (OLE phase)
Adverse Event Reporting Description Safety Set was defined as all participants who received at least 1 dose of study drug in PC Phase and OLE Phase.
 
Arm/Group Title PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX 661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Hide Arm/Group Description Participants received VX-661 50 mg tablet plus IVA 150 mg tablet q12h for 12 weeks. Participants received placebo matched to VX-661 tablet plus placebo matched to IVA tablet q12h for 12 weeks. Participants received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 12 weeks. Participants received two placebo matched to VX-661 tablets qd plus placebo matched to IVA tablet q12h for 12 weeks. Participants who completed 12 week PC phase underwent a washout period of at least 4 weeks before entering the OLE phase and received two VX-661 50 mg tablets qd plus IVA 150 mg tablet q12h for 48 weeks in OLE phase.
All-Cause Mortality
PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX 661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/6 (0.00%)   0/5 (0.00%)   0/15 (0.00%)   0/13 (0.00%)   0/27 (0.00%) 
Show Serious Adverse Events Hide Serious Adverse Events
PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX 661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   1/6 (16.67%)   2/5 (40.00%)   4/15 (26.67%)   5/13 (38.46%)   6/27 (22.22%) 
Congenital, familial and genetic disorders           
Cystic fibrosis related diabetes  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  1/27 (3.70%) 
Gastrointestinal disorders           
Intestinal obstruction  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Infections and infestations           
Infective pulmonary exacerbation of cystic fibrosis  1  0/6 (0.00%)  2/5 (40.00%)  4/15 (26.67%)  4/13 (30.77%)  5/27 (18.52%) 
Chronic sinusitis  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Pneumonia  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Metabolism and nutrition disorders           
Dehydration  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  1/27 (3.70%) 
Renal and urinary disorders           
Nephrolithiasis  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  1/27 (3.70%) 
Respiratory, thoracic and mediastinal disorders           
Pneumothorax  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
1
Term from vocabulary, MedDRA 19.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
PC Phase: VX-661 50 mg q12h + IVA 150 mg q12h PC Phase: VX 661 Placebo q12h + IVA Placebo q12h PC Phase: VX-661 100 mg qd + IVA 150 mg q12h PC Phase: VX -661 Placebo qd + IVA Placebo q12h OLE Phase: VX-661 100 mg qd + IVA 150 mg q12h
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   6/6 (100.00%)   5/5 (100.00%)   15/15 (100.00%)   13/13 (100.00%)   25/27 (92.59%) 
Ear and labyrinth disorders           
Tinnitus  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Gastrointestinal disorders           
Diarrhoea  1  1/6 (16.67%)  0/5 (0.00%)  3/15 (20.00%)  1/13 (7.69%)  0/27 (0.00%) 
Nausea  1  0/6 (0.00%)  0/5 (0.00%)  2/15 (13.33%)  0/13 (0.00%)  2/27 (7.41%) 
Dyspepsia  1  1/6 (16.67%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Abdominal distension  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Abdominal pain  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Abdominal pain upper  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Haemorrhoids  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Peptic ulcer  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Gastrooesophageal reflux disease  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  3/27 (11.11%) 
Constipation  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  2/27 (7.41%) 
Flatulence  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  2/27 (7.41%) 
General disorders           
Fatigue  1  1/6 (16.67%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  2/27 (7.41%) 
Peripheral swelling  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Non-cardiac chest pain  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Oedema peripheral  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Vaccination site discomfort  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Pyrexia  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  2/27 (7.41%) 
Infections and infestations           
Infective pulmonary exacerbation of cystic fibrosis  1  1/6 (16.67%)  3/5 (60.00%)  5/15 (33.33%)  4/13 (30.77%)  13/27 (48.15%) 
Nasopharyngitis  1  0/6 (0.00%)  0/5 (0.00%)  2/15 (13.33%)  0/13 (0.00%)  3/27 (11.11%) 
Upper respiratory tract infection  1  0/6 (0.00%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  2/27 (7.41%) 
Bacterial vaginosis  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Acute sinusitis  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Bronchitis  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Chronic sinusitis  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Oral candidiasis  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  2/27 (7.41%) 
Viral upper respiratory tract infection  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Vulvovaginal mycotic infection  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Tooth infection  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  2/27 (7.41%) 
Injury, poisoning and procedural complications           
Arthropod bite  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Muscle strain  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Investigations           
Pulmonary function test decreased  1  1/6 (16.67%)  1/5 (20.00%)  2/15 (13.33%)  1/13 (7.69%)  0/27 (0.00%) 
Weight decreased  1  0/6 (0.00%)  0/5 (0.00%)  2/15 (13.33%)  1/13 (7.69%)  3/27 (11.11%) 
Alanine aminotransferase increased  1  0/6 (0.00%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  2/27 (7.41%) 
Aspartate aminotransferase increased  1  0/6 (0.00%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  2/27 (7.41%) 
C-reactive protein increased  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  1/13 (7.69%)  0/27 (0.00%) 
Heart rate increased  1  0/6 (0.00%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Blood bilirubin unconjugated increased  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Blood triglycerides increased  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Breath sounds abnormal  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Gamma-glutamyltransferase increased  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Urine output decreased  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Forced expiratory volume decreased  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  2/13 (15.38%)  0/27 (0.00%) 
Vitamin D decreased  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  2/13 (15.38%)  0/27 (0.00%) 
Activated partial thromboplastin time prolonged  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Oxygen saturation decreased  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Prothrombin time prolonged  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Vitamin A decreased  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Vitamin K decreased  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
White blood cells urine positive  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Metabolism and nutrition disorders           
Decreased appetite  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Gout  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Iron deficiency  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Hypoglycaemia  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Vitamin E deficiency  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Musculoskeletal and connective tissue disorders           
Back pain  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  2/13 (15.38%)  0/27 (0.00%) 
Arthralgia  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  3/27 (11.11%) 
Arthritis  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Muscle spasms  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Musculoskeletal pain  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Pain in extremity  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Myalgia  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  2/27 (7.41%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)           
Lipoma  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Nervous system disorders           
Headache  1  1/6 (16.67%)  0/5 (0.00%)  2/15 (13.33%)  1/13 (7.69%)  4/27 (14.81%) 
Dizziness  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  1/13 (7.69%)  0/27 (0.00%) 
Hyposmia  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Lethargy  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Sinus headache  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Somnolence  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Psychiatric disorders           
Disorientation  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Reproductive system and breast disorders           
Menorrhagia  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Testicular pain  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Amenorrhoea  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Breast tenderness  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Cough  1  3/6 (50.00%)  2/5 (40.00%)  4/15 (26.67%)  5/13 (38.46%)  10/27 (37.04%) 
Dyspnoea  1  1/6 (16.67%)  2/5 (40.00%)  2/15 (13.33%)  1/13 (7.69%)  3/27 (11.11%) 
Haemoptysis  1  0/6 (0.00%)  0/5 (0.00%)  3/15 (20.00%)  3/13 (23.08%)  3/27 (11.11%) 
Respiration abnormal  1  1/6 (16.67%)  1/5 (20.00%)  2/15 (13.33%)  0/13 (0.00%)  3/27 (11.11%) 
Nasal congestion  1  1/6 (16.67%)  0/5 (0.00%)  1/15 (6.67%)  3/13 (23.08%)  2/27 (7.41%) 
Lower respiratory tract congestion  1  1/6 (16.67%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Sputum increased  1  0/6 (0.00%)  2/5 (40.00%)  1/15 (6.67%)  2/13 (15.38%)  4/27 (14.81%) 
Oropharyngeal pain  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  1/13 (7.69%)  2/27 (7.41%) 
Sputum discoloured  1  0/6 (0.00%)  1/5 (20.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Epistaxis  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Nasal oedema  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Painful respiration  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Paranasal sinus hypersecretion  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Productive cough  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
Sinus congestion  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  5/27 (18.52%) 
Wheezing  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  2/27 (7.41%) 
Rales  1  0/6 (0.00%)  1/5 (20.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Pleurisy  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Pleuritic pain  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Pneumothorax  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Respiratory tract congestion  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  3/27 (11.11%) 
Upper respiratory tract congestion  1  0/6 (0.00%)  0/5 (0.00%)  0/15 (0.00%)  1/13 (7.69%)  0/27 (0.00%) 
Skin and subcutaneous tissue disorders           
Acne  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Dermatitis allergic  1  0/6 (0.00%)  0/5 (0.00%)  1/15 (6.67%)  0/13 (0.00%)  0/27 (0.00%) 
Rash  1  1/6 (16.67%)  0/5 (0.00%)  0/15 (0.00%)  0/13 (0.00%)  0/27 (0.00%) 
1
Term from vocabulary, MedDRA 19.0
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.
PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.
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: NCT02070744     History of Changes
Other Study ID Numbers: VX13-661-103
First Submitted: February 21, 2014
First Posted: February 25, 2014
Results First Submitted: March 14, 2018
Results First Posted: April 13, 2018
Last Update Posted: April 13, 2018