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Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

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ClinicalTrials.gov Identifier: NCT01946412
Recruitment Status : Completed
First Posted : September 19, 2013
Results First Posted : February 1, 2017
Last Update Posted : February 1, 2017
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Intervention Drug: Ivacaftor
Enrollment 33
Recruitment Details This was a Phase 3, multicenter, 2 arm study in participants who received at least 1 dose of study drug in parent study VX11-770-108 (study 108) (NCT01705145).
Pre-assignment Details In study VX11-770-109 (study 109) (NCT01946412), participants were to be enrolled in either ivacaftor arm or observational arm. However, there were no participants enrolled in the observational arm. A total of 33 participants were enrolled in the ivacaftor arm.
Arm/Group Title Ivacaftor
Hide Arm/Group Description Participants received ivacaftor 50 milligram (mg) or 75 mg or 150 mg based on body weight and age. Ivacaftor 50 mg administered every 12 hours (q12h) for participants aged 2 to less than (<) 6 years and weighing <14 kilograms (kg), ivacaftor 75 mg q12h for participants aged 2 to <6 years and weighing greater than or equal to (>=) 14 kg and ivacaftor 150 mg q12h for participants >=6 years.
Period Title: Overall Study
Started 33
Completed 28
Not Completed 5
Reason Not Completed
Adverse Event             1
Non-Compliance             1
Continued with commercial Kalydeco             2
Difficulty in swallowing             1
Arm/Group Title Ivacaftor
Hide Arm/Group Description Participants received ivacaftor 50 mg or 75 mg or 150 mg based on body weight and age. Ivacaftor 50 mg administered q12h for participants aged 2 to < 6 years and weighing <14 kg, ivacaftor 75 mg q12h for participants aged 2 to <6 years and weighing >= 14 kg and ivacaftor 150 mg q12h for participants >=6 years.
Overall Number of Baseline Participants 33
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 33 participants
3.7  (1.04)
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
Female
6
  18.2%
Male
27
  81.8%
1.Primary Outcome
Title 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 was considered treatment-emergent.
Time Frame Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set included all participants who received at least 1 dose of study drug in study 109 (NCT01946412). As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Measure Type: Number
Unit of Measure: participants
AEs 9 24
SAEs 6 5
2.Secondary Outcome
Title Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84
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. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
Time Frame Baseline (study 108), Week 24, 48, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively.As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: millimole per liter (mmol/L)
Baseline (n=7,22) 93.1  (16.2) 99.6  (13.6)
Absolute Change at Week 24 (n=6,18) -62.1  (12.4) -48.5  (18.4)
Absolute Change at Week 48 (n=6,15) -29.3  (37.8) -51.8  (27.1)
Absolute Change at Week 72 (n=7,14) -46.4  (16.0) -52.9  (26.7)
Absolute Change at Week 84 (n=6,14) -46.5  (31.0) -58.1  (23.9)
3.Secondary Outcome
Title Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84
Hide Description Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of >=15 microliter was required for determination of sweat chloride. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
Time Frame Baseline (study 109), Week 24, 48, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: mmol/L
Baseline (n= 8,23) 47.8  (23.3) 52.9  (23.1)
Absolute Change at Week 24 (n=7,18) -4.3  (31.6) 3.4  (15.9)
Absolute Change at Week 48 (n=7,17) 18.1  (39.5) -4.5  (22.6)
Absolute Change at Week 72 (n=8,15) -1.5  (24.3) -6.0  (20.8)
Absolute Change at Week 84 (n=7,16) -2.4  (44.2) -11.2  (25.4)
4.Secondary Outcome
Title Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145)
Time Frame Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 09 24
Mean (Standard Deviation)
Unit of Measure: kilogram (kg)
Baseline (n=9, 24) 12.5  (1.1) 16.8  (1.8)
Absolute Change at Week 12 (n= 9, 23) 1.3  (0.4) 1.9  (0.7)
Absolute Change at Week 24 (n=9, 23) 2.0  (0.6) 2.5  (0.9)
Absolute Change at Week 36 (n=9, 23) 2.4  (0.7) 3.1  (1.0)
Absolute Change at Week 48 (n=9, 22) 2.6  (0.9) 3.4  (1.1)
Absolute Change at Week 60 (n=9, 22) 3.2  (1.0) 4.0  (1.3)
Absolute Change at Week 72 (n=9, 20) 3.4  (0.9) 4.8  (1.6)
Absolute Change at Week 84 (n=9, 19) 4.0  (1.2) 5.7  (1.9)
5.Secondary Outcome
Title Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
Time Frame Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: Kg
Baseline (n= 9, 24) 13.5  (1.0) 18.3  (2.0)
Absolute Change at Week 12 (n=9, 23) 0.3  (0.4) 0.4  (0.5)
Absolute Change at Week 24 (n=9, 23) 1.0  (0.5) 1.0  (0.7)
Absolute Change at Week 36 (n=9, 23) 1.4  (0.6) 1.6  (0.8)
Absolute Change at Week 48 (n=9, 22) 1.6  (0.7) 1.9  (0.8)
Absolute Change at Week 60 (n=9, 22) 2.2  (0.8) 2.5  (1.1)
Absolute Change at Week 72 (n=9, 20) 2.4  (0.7) 3.3  (1.4)
Absolute Change at Week 84 (n=9, 19) 3.0  (1.0) 4.2  (1.7)
6.Secondary Outcome
Title Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
Time Frame Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: Centimeters (cm)
Baseline (n=9, 24) 89.1  (4.3) 102.3  (6.4)
Absolute Change in Week 12 (n=9, 23) 4.6  (1.2) 5.4  (1.1)
Absolute Change in Week 24 (n=9, 23) 6.0  (1.7) 7.7  (3.3)
Absolute Change in Week 36 (n=9, 23) 7.8  (1.8) 8.8  (1.4)
Absolute Change in Week 48 (n=9, 21) 9.7  (2.0) 10.4  (1.7)
Absolute Change in Week 60 (n=9, 22) 11.0  (2.2) 11.6  (2.0)
Absolute Change in Week 72 (n=9, 20) 12.5  (2.6) 13.4  (2.0)
Absolute Change in Week 84 (n=9, 19) 13.6  (2.3) 15.0  (2.3)
7.Secondary Outcome
Title Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
Time Frame Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: cm
Baseline (n=9, 24) 91.7  (4.3) 105.8  (6.6)
Absolute Change at Week 12 (n=9, 23) 2.0  (1.1) 1.8  (0.9)
Absolute Change at Week 24 (n=9, 23) 3.4  (1.2) 4.1  (3.2)
Absolute Change at Week 36 (n=9, 23) 5.2  (1.5) 5.2  (1.1)
Absolute Change at Week 48 (n=9, 21) 7.2  (1.3) 6.8  (1.3)
Absolute Change at Week 60 (n=9, 22) 8.4  (1.4) 8.0  (1.5)
Absolute Change at Week 72 (n=9, 20) 10.0  (2.1) 9.8  (1.6)
Absolute Change at Week 84 (n=9, 19) 11.1  (1.7) 11.4  (1.9)
8.Secondary Outcome
Title Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145).
Time Frame Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: Kilogram per square meter (kg/m^2)
Baseline (n=9, 24) 15.74  (0.69) 16.06  (1.15)
Absolute Change at Week 12 (n=9, 23) 0.03  (0.40) 0.09  (0.60)
Absolute Change at Week 24 (n=9, 23) 0.31  (0.57) -0.12  (0.90)
Absolute Change at Week 36 (n=9, 23) 0.15  (0.52) 0.09  (0.73)
Absolute Change at Week 48 (n=9, 22) -0.31  (0.60) -0.13  (0.77)
Absolute Change at Week 60 (n=9, 22) -0.12  (0.70) -0.06  (0.90)
Absolute Change at Week 72 (n=9, 20) -0.38  (0.68) 0.09  (0.97)
Absolute Change at Week 84 (n=9, 19) -0.16  (0.96) 0.28  (0.97)
9.Secondary Outcome
Title Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84
Hide Description BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412).
Time Frame Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)
Hide Outcome Measure Data
Hide Analysis Population Description
Safety set. Here “n” signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description:
Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
Overall Number of Participants Analyzed 9 24
Mean (Standard Deviation)
Unit of Measure: kg/m^2
Baseline (n= 9, 24) 16.07  (0.55) 16.33  (1.12)
Absolute Change at Week 12 (n= 9, 23) -0.30  (0.60) -0.16  (0.48)
Absolute Change at Week 24 (n= 9, 23) -0.02  (0.70) -0.36  (1.03)
Absolute Change at Week 36 (n= 9, 23) -0.18  (0.83) -0.16  (0.63)
Absolute Change at Week 48 (n= 9, 22) -0.64  (0.81) -0.35  (0.57)
Absolute Change at Week 60 (n= 9, 22) -0.45  (0.82) -0.29  (0.72)
Absolute Change at Week 72 (n= 9, 20) -0.71  (0.90) -0.21  (0.78)
Absolute Change at Week 84 (n= 9, 19) -0.49  (1.09) -0.01  (0.87)
Time Frame Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing)
Adverse Event Reporting Description As per the planned analysis for this study, participants were to be reported based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
 
Arm/Group Title Ivacaftor 50 mg Ivacaftor 75 mg
Hide Arm/Group Description Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412). Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412).
All-Cause Mortality
Ivacaftor 50 mg Ivacaftor 75 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Ivacaftor 50 mg Ivacaftor 75 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   6/9 (66.67%)   5/24 (20.83%) 
General disorders     
Pyrexia  1  2/9 (22.22%)  0/24 (0.00%) 
Infections and infestations     
Infective pulmonary exacerbation of cystic fibrosis  1  3/9 (33.33%)  3/24 (12.50%) 
Enterovirus infection  1  0/9 (0.00%)  1/24 (4.17%) 
Respiratory syncytial virus infection  1  1/9 (11.11%)  0/24 (0.00%) 
Staphylococcal infection  1  0/9 (0.00%)  1/24 (4.17%) 
Investigations     
Alanine aminotransferase increased  1  2/9 (22.22%)  0/24 (0.00%) 
Aspartate aminotransferase increased  1  2/9 (22.22%)  0/24 (0.00%) 
Adenovirus test positive  1  1/9 (11.11%)  0/24 (0.00%) 
Metabolism and nutrition disorders     
Dehydration  1  1/9 (11.11%)  0/24 (0.00%) 
Nervous system disorders     
Seizure anoxic  1  0/9 (0.00%)  1/24 (4.17%) 
Indicates events were collected by systematic assessment
1
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%
Ivacaftor 50 mg Ivacaftor 75 mg
Affected / at Risk (%) Affected / at Risk (%)
Total   9/9 (100.00%)   24/24 (100.00%) 
Blood and lymphatic system disorders     
Anaemia  1  1/9 (11.11%)  0/24 (0.00%) 
Lymphadenopathy  1  1/9 (11.11%)  0/24 (0.00%) 
Congenital, familial and genetic disorders     
Phimosis  1  0/9 (0.00%)  1/24 (4.17%) 
Ear and labyrinth disorders     
Ear pain  1  1/9 (11.11%)  1/24 (4.17%) 
Cerumen impaction  1  0/9 (0.00%)  1/24 (4.17%) 
Eye disorders     
Conjunctivitis  1  2/9 (22.22%)  0/24 (0.00%) 
Amblyopia  1  0/9 (0.00%)  1/24 (4.17%) 
Cataract cortical  1  0/9 (0.00%)  1/24 (4.17%) 
Eye pruritus  1  0/9 (0.00%)  1/24 (4.17%) 
Lacrimation increased  1  0/9 (0.00%)  1/24 (4.17%) 
Gastrointestinal disorders     
Vomiting  1  6/9 (66.67%)  7/24 (29.17%) 
Abdominal pain  1  0/9 (0.00%)  5/24 (20.83%) 
Constipation  1  1/9 (11.11%)  2/24 (8.33%) 
Abdominal pain upper  1  0/9 (0.00%)  1/24 (4.17%) 
Diarrhoea  1  1/9 (11.11%)  0/24 (0.00%) 
Gastrooesophageal reflux disease  1  0/9 (0.00%)  1/24 (4.17%) 
Nausea  1  0/9 (0.00%)  1/24 (4.17%) 
Stomatitis  1  1/9 (11.11%)  0/24 (0.00%) 
Tooth discolouration  1  0/9 (0.00%)  1/24 (4.17%) 
General disorders     
Pyrexia  1  7/9 (77.78%)  6/24 (25.00%) 
Fatigue  1  0/9 (0.00%)  1/24 (4.17%) 
Hepatobiliary disorders     
Cholelithiasis  1  1/9 (11.11%)  0/24 (0.00%) 
Immune system disorders     
Hypersensitivity  1  0/9 (0.00%)  1/24 (4.17%) 
Infections and infestations     
Otitis media  1  2/9 (22.22%)  4/24 (16.67%) 
Sinusitis  1  2/9 (22.22%)  3/24 (12.50%) 
Viral upper respiratory tract infection  1  0/9 (0.00%)  5/24 (20.83%) 
Gastroenteritis viral  1  0/9 (0.00%)  4/24 (16.67%) 
Infective pulmonary exacerbation of cystic fibrosis  1  0/9 (0.00%)  4/24 (16.67%) 
Pharyngitis streptococcal  1  1/9 (11.11%)  3/24 (12.50%) 
Nasopharyngitis  1  1/9 (11.11%)  2/24 (8.33%) 
Rhinitis  1  2/9 (22.22%)  1/24 (4.17%) 
Bronchitis  1  1/9 (11.11%)  1/24 (4.17%) 
Respiratory tract infection viral  1  0/9 (0.00%)  2/24 (8.33%) 
Upper respiratory tract infection  1  1/9 (11.11%)  1/24 (4.17%) 
Varicella  1  0/9 (0.00%)  2/24 (8.33%) 
Ear infection  1  0/9 (0.00%)  1/24 (4.17%) 
Gastroenteritis  1  0/9 (0.00%)  1/24 (4.17%) 
Hand-foot-and-mouth disease  1  0/9 (0.00%)  1/24 (4.17%) 
Herpangina  1  0/9 (0.00%)  1/24 (4.17%) 
Influenza  1  0/9 (0.00%)  1/24 (4.17%) 
Lower respiratory tract infection bacterial  1  0/9 (0.00%)  1/24 (4.17%) 
Lung infection pseudomonal  1  0/9 (0.00%)  1/24 (4.17%) 
Molluscum contagiosum  1  0/9 (0.00%)  1/24 (4.17%) 
Myringitis  1  0/9 (0.00%)  1/24 (4.17%) 
Parainfluenzae virus infection  1  0/9 (0.00%)  1/24 (4.17%) 
Pneumonia  1  0/9 (0.00%)  1/24 (4.17%) 
Rhinovirus infection  1  0/9 (0.00%)  1/24 (4.17%) 
Staphylococcal skin infection  1  0/9 (0.00%)  1/24 (4.17%) 
Streptococcal infection  1  1/9 (11.11%)  0/24 (0.00%) 
Tonsillitis  1  0/9 (0.00%)  1/24 (4.17%) 
Upper respiratory tract infection bacterial  1  0/9 (0.00%)  1/24 (4.17%) 
Viral infection  1  0/9 (0.00%)  1/24 (4.17%) 
Injury, poisoning and procedural complications     
Laceration  1  1/9 (11.11%)  1/24 (4.17%) 
Arthropod bite  1  0/9 (0.00%)  1/24 (4.17%) 
Contusion  1  0/9 (0.00%)  1/24 (4.17%) 
Eye injury  1  0/9 (0.00%)  1/24 (4.17%) 
Investigations     
Alanine aminotransferase increased  1  1/9 (11.11%)  4/24 (16.67%) 
Aspartate aminotransferase increased  1  1/9 (11.11%)  3/24 (12.50%) 
Bacterial test positive  1  1/9 (11.11%)  1/24 (4.17%) 
Gamma-glutamyltransferase increased  1  1/9 (11.11%)  1/24 (4.17%) 
Haemophilus test positive  1  0/9 (0.00%)  2/24 (8.33%) 
Respiratory rate increased  1  1/9 (11.11%)  1/24 (4.17%) 
Adenovirus test positive  1  1/9 (11.11%)  0/24 (0.00%) 
Antibiotic resistant Staphylococcus test positive  1  0/9 (0.00%)  1/24 (4.17%) 
Blood alkaline phosphatase increased  1  0/9 (0.00%)  1/24 (4.17%) 
Influenza A virus test positive  1  0/9 (0.00%)  1/24 (4.17%) 
Lymph node palpable  1  0/9 (0.00%)  1/24 (4.17%) 
Neutrophil count decreased  1  0/9 (0.00%)  1/24 (4.17%) 
Pseudomonas test positive  1  1/9 (11.11%)  0/24 (0.00%) 
Respiratory syncytial virus test positive  1  1/9 (11.11%)  0/24 (0.00%) 
Staphylococcus test positive  1  1/9 (11.11%)  0/24 (0.00%) 
Metabolism and nutrition disorders     
Decreased appetite  1  0/9 (0.00%)  1/24 (4.17%) 
Musculoskeletal and connective tissue disorders     
Arthralgia  1  0/9 (0.00%)  1/24 (4.17%) 
Pain in extremity  1  0/9 (0.00%)  1/24 (4.17%) 
Nervous system disorders     
Convulsion  1  0/9 (0.00%)  1/24 (4.17%) 
Headache  1  0/9 (0.00%)  1/24 (4.17%) 
Lethargy  1  1/9 (11.11%)  0/24 (0.00%) 
Migraine  1  0/9 (0.00%)  1/24 (4.17%) 
Psychiatric disorders     
Attention deficit/hyperactivity disorder  1  0/9 (0.00%)  1/24 (4.17%) 
Encopresis  1  0/9 (0.00%)  1/24 (4.17%) 
Onychophagia  1  0/9 (0.00%)  1/24 (4.17%) 
Renal and urinary disorders     
Enuresis  1  0/9 (0.00%)  1/24 (4.17%) 
Micturition urgency  1  1/9 (11.11%)  0/24 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Cough  1  8/9 (88.89%)  16/24 (66.67%) 
Nasal congestion  1  4/9 (44.44%)  3/24 (12.50%) 
Rhinorrhoea  1  4/9 (44.44%)  2/24 (8.33%) 
Productive cough  1  1/9 (11.11%)  2/24 (8.33%) 
Asthma  1  0/9 (0.00%)  2/24 (8.33%) 
Dyspnoea  1  1/9 (11.11%)  1/24 (4.17%) 
Allergic sinusitis  1  0/9 (0.00%)  1/24 (4.17%) 
Paranasal sinus hypersecretion  1  1/9 (11.11%)  0/24 (0.00%) 
Respiration abnormal  1  0/9 (0.00%)  1/24 (4.17%) 
Respiratory tract congestion  1  0/9 (0.00%)  1/24 (4.17%) 
Snoring  1  0/9 (0.00%)  1/24 (4.17%) 
Upper respiratory tract congestion  1  1/9 (11.11%)  0/24 (0.00%) 
Skin and subcutaneous tissue disorders     
Rash  1  3/9 (33.33%)  1/24 (4.17%) 
Dry skin  1  0/9 (0.00%)  1/24 (4.17%) 
Eczema  1  0/9 (0.00%)  1/24 (4.17%) 
Nail disorder  1  0/9 (0.00%)  1/24 (4.17%) 
Red man syndrome  1  0/9 (0.00%)  1/24 (4.17%) 
Indicates events were collected by systematic assessment
1
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: NCT01946412     History of Changes
Other Study ID Numbers: VX11-770-109
First Submitted: September 16, 2013
First Posted: September 19, 2013
Results First Submitted: December 6, 2016
Results First Posted: February 1, 2017
Last Update Posted: February 1, 2017