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Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

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: NCT01705145
Recruitment Status : Completed
First Posted : October 12, 2012
Results First Posted : April 24, 2015
Last Update Posted : April 5, 2016
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Intervention Drug: Ivacaftor
Enrollment 35
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Ivacaftor
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Part A: Ivacaftor 50 milligram (mg) (for participants weighing less than [<] 14 kilograms [kg]) or 75 mg (for participants weighing greater than or equal to [>=] 14 kg) every 12 hours (q12h) from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.

Part B: Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.

Period Title: Part A
Started 9
Completed 9
Not Completed 0
Period Title: Part B
Started 34 [1]
Completed 33
Not Completed 1
Reason Not Completed
Adverse Event             1
[1]
Eight participants from Part A continued in Part B of the study.
Arm/Group Title Ivacaftor
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Part A: Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.

Part B: Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.

Overall Number of Baseline Participants 35
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Safety set included all participants who received at least 1 dose of study drug.
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 35 participants
Part A (n = 9) 3.1  (1.17)
Part B (n = 34) 3.2  (0.96)
[1]
Measure Description: Data was planned to be reported separately for Part A and Part B of the study. Here “n” signifies participants who were evaluable for specified part of the study.
Sex/Gender, Customized   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 35 participants
Part A (n = 9): Female 3
Part A (n = 9): Male 6
Part B (n = 34): Female 6
Part B (n = 34): Male 28
[1]
Measure Description: Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies participants who were evaluable for specified part of the study.
1.Primary Outcome
Title Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
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AE: any adverse change from participant's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.

Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. Data was reported as per the dose received and for overall participants.

Time Frame Part A: Up to 93 Days
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Part A Safety set included all participants who received at least 1 dose of study drug in part A.
Arm/Group Title Part A: Ivacaftor 50 mg Part A: Ivacaftor 75 mg Part A: Overall Ivacaftor
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Ivacaftor 50 mg (for participants weighing <14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.
Overall Number of Participants Analyzed 4 5 9
Measure Type: Number
Unit of Measure: participants
AEs 3 5 8
SAEs 0 0 0
Related AEs 1 3 4
2.Primary Outcome
Title Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
Hide Description

AE: any adverse change from participant's baseline (pre-treatment) condition, including any adverse experience, abnormal recording/clinical laboratory assessment which occurs during course of study, whether it is considered related to study drug or not. AE includes both serious and non-serious AE. SAE: medical event or condition, which falls into any of following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event.

Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. Data was reported as per the dose received.

Time Frame Part B: Up to 28 Weeks
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Part B Safety set included all participants who received at least 1 dose of study drug in part B.
Arm/Group Title Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description:
Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 10 24 34
Measure Type: Number
Unit of Measure: participants
AEs 10 23 33
SAEs 3 3 6
Related AEs 3 8 11
3.Primary Outcome
Title Part A: Plasma Concentration of Ivacaftor and Its Metabolites
Hide Description Plasma concentration was reported for ivacaftor and its metabolites (hydroxymethyl ivacaftor [M1] and ivacaftor carboxylate [M6]) up to 24 hours post-dose on Day 4 (Hour 0 [pre-dose] on Day 1 and Day 4; 2, 3, 6, 24 hours post-dose on Day 4). Data was planned to be reported for overall participants in the period.
Time Frame Part A: up to 24 hours post-dose on Day 4
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Part A Safety set included all participants who received at least 1 dose of study drug in part A.
Arm/Group Title Part A: Overall Ivacaftor
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Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: nanogram per milliliter (ng/mL)
Ivacaftor: Hour 0 on Day 1 0.00  (0.00)
Ivacaftor: Hour 0 on Day 4 396  (337)
Ivacaftor: 2 Hours Post-Dose on Day 4 726  (284)
Ivacaftor: 3 Hours Post-Dose on Day 4 957  (283)
Ivacaftor: 6 Hours Post-Dose on Day 4 542  (241)
Ivacaftor: 24 Hours Post-Dose on Day 4 124  (149)
M1: Hour 0 on Day 1 0.00  (0.00)
M1: Hour 0 on Day 4 1240  (723)
M1: 2 Hours Post-Dose on Day 4 1540  (578)
M1: 3 Hours Post-Dose on Day 4 2310  (820)
M1: 6 Hours Post-Dose on Day 4 1580  (622)
M1: 24 Hours Post-Dose on Day 4 389  (336)
M6: Hour 0 on Day 1 0.00  (0.00)
M6: Hour 0 on Day 4 1150  (709)
M6: 2 Hours Post-Dose on Day 4 1050  (606)
M6: 3 Hours Post-Dose on Day 4 1300  (614)
M6: 6 Hours Post-Dose on Day 4 1390  (532)
M6: 24 Hours Post-Dose on Day 4 439  (355)
4.Secondary Outcome
Title Part B: Plasma Concentration of Ivacaftor and Its Metabolites
Hide Description Plasma concentration was reported for ivacaftor and its metabolites (M1 and M6) up to 24 hours post-dose on Day 168 (Hour 0 [predose] on Day 1, 14, 56, 112, and 168; 2, 3, 6 hours post-dose on Day 14; 1 hour post-dose on Day 56; 4, 6 hours post-dose on Day 112; 24 hours post-dose on Day 168). Data was planned to be reported for overall participants in the period.
Time Frame Part B: up to 24 hours post-dose on Day 168
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Hide Analysis Population Description
Part B Safety set included all participants who received at least 1 dose of study drug in part B.
Arm/Group Title Part B: Overall Ivacaftor
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Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 34
Mean (Standard Deviation)
Unit of Measure: ng/mL
Ivacaftor: Hour 0 on Day 1 0.00  (0.00)
Ivacaftor: Hour 0 on Day 14 614  (590)
Ivacaftor: 2 Hours Post-Dose on Day 14 932  (541)
Ivacaftor: 3 Hours Post-Dose on Day 14 1080  (587)
Ivacaftor: 6 Hours Post-Dose on Day 14 1140  (627)
Ivacaftor: Hour 0 on Day 56 448  (455)
Ivacaftor: 1 Hour Post-Dose on Day 56 514  (421)
Ivacaftor: Hour 0 on Day 112 596  (747)
Ivacaftor: 4 Hours Post-Dose on Day 112 1080  (835)
Ivacaftor: 6 Hours Post-Dose on Day 112 1010  (885)
Ivacaftor: Hour 0 on Day 168 500  (545)
Ivacaftor: 24 Hours Post-Dose on Day 168 207  (372)
M1: Hour 0 on Day 1 0.00  (0.00)
M1: Hour 0 on Day 14 1580  (1030)
M1: 2 Hours Post-Dose on Day 14 1870  (924)
M1: 3 Hours Post-Dose on Day 14 2280  (1140)
M1: 6 Hours Post-Dose on Day 14 2670  (1080)
M1: Hour 0 on Day 56 1340  (880)
M1: 1 Hour Post-Dose on Day 56 1170  (698)
M1: Hour 0 on Day 112 1680  (1360)
M1: 4 Hours Post-Dose on Day 112 2450  (1510)
M1: 6 Hours Post-Dose on Day 112 2500  (1520)
M1: Hour 0 on Day 168 1460  (1200)
M1: 24 Hours Post-Dose on Day 168 602  (647)
M6: Hour 0 on Day 1 0.00  (0.00)
M6: Hour 0 on Day 14 1520  (1130)
M6: 2 Hours Post-Dose on Day 14 1430  (989)
M6: 3 Hours Post-Dose on Day 14 1630  (1130)
M6: 6 Hours Post-Dose on Day 14 2090  (1350)
M6: Hour 0 on Day 56 1510  (1080)
M6: 1 Hour Post-Dose on Day 56 1310  (974)
M6: Hour 0 on Day 112 1660  (1130)
M6: 4 Hours Post-Dose on Day 112 1810  (1230)
M6: 6 Hours Post-Dose on Day 112 2130  (1380)
M6: Hour 0 on Day 168 1520  (1130)
M6: 24 Hours Post-Dose on Day 168 632  (465)
5.Secondary Outcome
Title Part B: Absolute Change From Baseline in Sweat Chloride at Week 24
Hide Description Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Data was reported as per the dose received and for overall participants.
Time Frame Part B: Baseline, Week 24
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Part B Safety set included all participants who received at least 1 dose of study drug in part B. Number of participants analyzed is for participants who were evaluable for this outcome measure.
Arm/Group Title Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description:
Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 7 18 25
Mean (Standard Deviation)
Unit of Measure: millimole per liter (mmol/L)
-47.07  (24.256) -46.78  (27.584) -46.86  (26.193)
6.Secondary Outcome
Title Part B: Absolute Change From Baseline in Weight at Week 24
Hide Description Data was reported as per the dose received and for overall participants.
Time Frame Part B: Baseline, Week 24
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Hide Analysis Population Description
Part B Safety set included all participants who received at least 1 dose of study drug in part B. Number of participants analyzed is for participants who were evaluable for this outcome measure.
Arm/Group Title Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description:
Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 9 24 33
Mean (Standard Deviation)
Unit of Measure: kilograms (kg)
1.00  (0.418) 1.50  (0.552) 1.36  (0.561)
7.Secondary Outcome
Title Part B: Absolute Change From Baseline in Stature at Week 24
Hide Description Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Data was reported as per the dose received and for overall participants.
Time Frame Part B: Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Part B Safety set included all participants who received at least 1 dose of study drug in part B. Number of participants analyzed is for participants who were evaluable for this outcome measure.
Arm/Group Title Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description:
Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 9 23 32
Mean (Standard Deviation)
Unit of Measure: centimeters (cm)
2.5  (1.45) 3.5  (0.93) 3.3  (1.17)
8.Secondary Outcome
Title Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
Hide Description BMI = (Weight [in kg]) divided by (Stature [in meters])^2. Data was reported as per the dose received and for overall participants.
Time Frame Baseline, Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Part B Safety set included all participants who received at least 1 dose of study drug in part B. Number of participants analyzed is for participants who were evaluable for this outcome measure.
Arm/Group Title Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description:
Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
Overall Number of Participants Analyzed 9 23 32
Mean (Standard Deviation)
Unit of Measure: kilogram per square meter (kg/m^2)
0.332  (0.5393) 0.314  (0.5492) 0.319  (0.5378)
Time Frame Part A: up to 93 days; Part B: up to 28 weeks
Adverse Event Reporting Description Adverse events were reported separately for each part and as per the dose received and for overall participants in each part.
 
Arm/Group Title Part A: Ivacaftor 50 mg Part A: Ivacaftor 75 mg Part A: Overall Ivacaftor Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Hide Arm/Group Description Ivacaftor 50 mg (for participants weighing <14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study. Ivacaftor 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study. Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h from Day 1 through Day 3 and 1 morning dose on Day 4 during Part A of the study. Ivacaftor 50 mg (for participants weighing <14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants. Ivacaftor 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants. Ivacaftor 50 mg (for participants weighing <14 kg) or 75 mg (for participants weighing >=14 kg) q12h for 24 weeks during Part B of the study. Part B included participants from Part A and newly enrolled participants.
All-Cause Mortality
Part A: Ivacaftor 50 mg Part A: Ivacaftor 75 mg Part A: Overall Ivacaftor Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Part A: Ivacaftor 50 mg Part A: Ivacaftor 75 mg Part A: Overall Ivacaftor Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)   0/5 (0.00%)   0/9 (0.00%)   3/10 (30.00%)   3/24 (12.50%)   6/34 (17.65%) 
Gastrointestinal disorders             
Vomiting  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Infections and infestations             
Infective pulmonary exacerbation of cystic fibrosis  1  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  1/24 (4.17%)  2/34 (5.88%) 
Device related sepsis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Investigations             
Pseudomonas test positive  1  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Transaminases increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Nervous system disorders             
Convulsion  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (15.1)
2
Term from vocabulary, MedDRA 15.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Part A: Ivacaftor 50 mg Part A: Ivacaftor 75 mg Part A: Overall Ivacaftor Part B: Ivacaftor 50 mg Part B: Ivacaftor 75 mg Part B: Overall Ivacaftor
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/4 (75.00%)   5/5 (100.00%)   8/9 (88.89%)   9/10 (90.00%)   23/24 (95.83%)   32/34 (94.12%) 
Blood and lymphatic system disorders             
Lymphadenopathy  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Eye disorders             
Amblyopia  1  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Anisometropia  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Eye inflammation  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Gastrointestinal disorders             
Vomiting  2  0/4 (0.00%)  2/5 (40.00%)  2/9 (22.22%)  3/10 (30.00%)  6/24 (25.00%)  9/34 (26.47%) 
Constipation  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  4/24 (16.67%)  4/34 (11.76%) 
Abdominal distension  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Abdominal pain  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Eructation  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Lip blister  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Nausea  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Oral mucosal erythema  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Retching  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Teething  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Diarrhoea  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  0/24 (0.00%)  0/34 (0.00%) 
General disorders             
Pyrexia  2  2/4 (50.00%)  2/5 (40.00%)  4/9 (44.44%)  4/10 (40.00%)  2/24 (8.33%)  6/34 (17.65%) 
Application site rash  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Fatigue  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Product taste abnormal  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Infections and infestations             
Upper respiratory tract infection  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  7/24 (29.17%)  8/34 (23.53%) 
Croup infectious  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  2/10 (20.00%)  1/24 (4.17%)  3/34 (8.82%) 
Infective pulmonary exacerbation of cystic fibrosis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  3/24 (12.50%)  3/34 (8.82%) 
Otitis media  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  2/10 (20.00%)  1/24 (4.17%)  3/34 (8.82%) 
Sinusitis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  2/10 (20.00%)  1/24 (4.17%)  3/34 (8.82%) 
Gastroentritis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  2/24 (8.33%)  2/34 (5.88%) 
Rhinitis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  2/24 (8.33%)  2/34 (5.88%) 
Bacterial disease carrier  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Genital candidiasis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Hand-foot-and-mouth disease  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Infectious mononucleosis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Lung infection  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Nasopharyngitis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Pharyngitis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Pharyngitis streptococcal  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Respiratory tract infection viral  1  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Viral rash  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Viral upper respiratory tract infection  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Bronchitis viral  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  0/24 (0.00%)  0/34 (0.00%) 
Injury, poisoning and procedural complications             
Contusion  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  2/24 (8.33%)  2/34 (5.88%) 
Face injury  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Fall  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Mouth injury  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Open wound  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Traumatic haemorrhage  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Upper limb fracture  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Investigations             
Bacterial test positive  1  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  3/24 (12.50%)  3/34 (8.82%) 
Haemophilus test positive  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  3/24 (12.50%)  3/34 (8.82%) 
Hepatic enzyme increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  2/10 (20.00%)  0/24 (0.00%)  2/34 (5.88%) 
Activated partial thromboplastin time prolonged  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Alanine aminotransferase increased  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Antibiotic resistant Staphylococcus test positive  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Aspartate aminotransferase increased  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Blood creatine increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Blood creatinine increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Liver function test abnormal  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Lymphocyte count increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Respiratory rate increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Pancreatic enzyme increased  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  0/24 (0.00%)  0/34 (0.00%) 
Staphylococcus test positive  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
White blood cell count increased  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Metabolism and nutrition disorders             
Decreased appetite  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  1/24 (4.17%)  2/34 (5.88%) 
Weight gain poor  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Musculoskeletal and connective tissue disorders             
Arthralgia  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Pain in extremity  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Nervous system disorders             
Headache  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  2/24 (8.33%)  2/34 (5.88%) 
Drooling  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Sinus headache  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Renal and urinary disorders             
Chromaturia  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Pollakiuria  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  4/10 (40.00%)  15/24 (62.50%)  19/34 (55.88%) 
Nasal congestion  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  4/10 (40.00%)  5/24 (20.83%)  9/34 (26.47%) 
Rhinorrhoea  2  0/4 (0.00%)  2/5 (40.00%)  2/9 (22.22%)  2/10 (20.00%)  5/24 (20.83%)  7/34 (20.59%) 
Productive cough  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  3/24 (12.50%)  3/34 (8.82%) 
Dysponea exertional  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Epistaxis  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Nasal inflamation  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Oropharyngeal pain  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Snoring  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Tonsillar hypertrophy  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Upper respiratory track congestion  2  0/4 (0.00%)  1/5 (20.00%)  1/9 (11.11%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Skin and subcutaneous tissue disorders             
Rash  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  2/10 (20.00%)  2/24 (8.33%)  4/34 (11.76%) 
Acne  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Dermatitis contact  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Petechiae  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  0/10 (0.00%)  1/24 (4.17%)  1/34 (2.94%) 
Skin irritation  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Urticaria  2  0/4 (0.00%)  0/5 (0.00%)  0/9 (0.00%)  1/10 (10.00%)  0/24 (0.00%)  1/34 (2.94%) 
Ecchymosis  2  0/4 (0.00%)  2/5 (40.00%)  2/9 (22.22%)  0/10 (0.00%)  0/24 (0.00%)  0/34 (0.00%) 
Rash macular  2  1/4 (25.00%)  0/5 (0.00%)  1/9 (11.11%)  0/10 (0.00%)  0/24 (0.00%)  0/34 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (15.1)
2
Term from vocabulary, MedDRA 15.1
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: NCT01705145     History of Changes
Other Study ID Numbers: VX11-770-108
KIWI
First Submitted: October 8, 2012
First Posted: October 12, 2012
Results First Submitted: April 2, 2015
Results First Posted: April 24, 2015
Last Update Posted: April 5, 2016