Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through... | NCT01705145 | Trialant
NCT01705145
Sponsor
Vertex Pharmaceuticals Incorporated
Status
Completed
Last Update Posted
Apr 5, 2016Estimated
Enrollment
35Actual
Phase
Phase 3
Conditions
Cystic Fibrosis
Interventions
Ivacaftor
Countries
United States
Canada
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT01705145
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
VX11-770-108
Secondary IDs
ID
Type
Description
Link
KIWI
Brief Title
Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
Official Title
A Phase 3, 2-Part, Open-Label Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are 2 Through 5 Years of Age and Have a CFTR Gating Mutation
Acronym
Not provided
Organization
Vertex Pharmaceuticals IncorporatedINDUSTRY
Status Module
Record Verification Date
Mar 2016
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Jan 2013
Primary Completion Date
Mar 2014Actual
Completion Date
Mar 2014Actual
First Submitted Date
Oct 8, 2012
First Submission Date that Met QC Criteria
Oct 10, 2012
First Posted Date
Oct 12, 2012Estimated
Results Waived
Not provided
Results First Submitted Date
Apr 2, 2015
Results First Submitted that Met QC Criteria
Apr 23, 2015
Results First Posted Date
Apr 24, 2015Estimated
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Mar 9, 2016
Last Update Posted Date
Apr 5, 2016Estimated
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Vertex Pharmaceuticals IncorporatedINDUSTRY
Collaborators
Name
Class
Cystic Fibrosis Foundation
OTHER
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Not provided
Is FDA Regulated Device
Not provided
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
The purpose of this study is to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD), of ivacaftor in children with cystic fibrosis (CF) who are 2 through 5 years of age and have a CF Transmembrane Conductance Regulator (CFTR) gating mutation in at least 1 allele.
Part A is designed to evaluate the safety and PK of multiple-dose administration of ivacaftor in participants 2 through 5 years of age and to confirm the doses for Part B. Part B is designed to evaluate the safety, PK, PD, and efficacy of ivacaftor in participants 2 through 5 years of age.
Detailed Description
Not provided
Conditions Module
Conditions
Cystic Fibrosis
Keywords
Cystic Fibrosis
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 3
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
35Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Ivacaftor
Experimental
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.
Drug: Ivacaftor
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Ivacaftor
Drug
Ivacaftor
Kalydeco
VX-770
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
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.
Part A: Up to 93 Days
Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
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.
Part B: Up to 28 Weeks
Part A: Plasma Concentration of Ivacaftor and Its Metabolites
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.
Secondary Outcomes
Measure
Description
Time Frame
Part B: Plasma Concentration of Ivacaftor and Its Metabolites
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.
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Male or female with confirmed diagnosis of CF
Must have a CFTR gating mutation in at least 1 allele
Aged 2 through 5 years at screening and Day 1
Weight >= 8 kg at screening and Day 1
Hematology, serum chemistry, coagulation, and vital signs results at screening with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
Exclusion Criteria:
History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the participant
An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1
Abnormal liver function, at screening
History of solid organ or hematological transplantation
Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives before screening
Davies JC, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Robertson S, Green Y, Cooke J, Rosenfeld M; KIWI Study Group. Safety, pharmacokinetics, and pharmacodynamics of ivacaftor in patients aged 2-5 years with cystic fibrosis and a CFTR gating mutation (KIWI): an open-label, single-arm study. Lancet Respir Med. 2016 Feb;4(2):107-15. doi: 10.1016/S2213-2600(15)00545-7. Epub 2016 Jan 21.
See Also Links
Not provided
Available IPD Information
Not provided
IPD Sharing Statement Module
No data available
No data is available for this block.
Results Section
Participant Flow Module
Pre-assignment Details
Not provided
Recruitment Details
Not provided
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Ivacaftor
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.
Periods
Title
Milestones
Reasons Not Completed
Part A
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
0
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
No data available
No data is available for this block.
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
N/A
Intervention Model
Single Group Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
Part A: up to 24 hours post-dose on Day 4
Part B: up to 24 hours post-dose on Day 168
Part B: Absolute Change From Baseline in Sweat Chloride at Week 24
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.
Part B: Baseline, Week 24
Part B: Absolute Change From Baseline in Weight at Week 24
Data was reported as per the dose received and for overall participants.
Part B: Baseline, Week 24
Part B: Absolute Change From Baseline in Stature at Week 24
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.
Part B: Baseline, Week 24
Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
BMI = (Weight [in kg]) divided by (Stature [in meters])^2. Data was reported as per the dose received and for overall participants.
Baseline, Week 24
Aurora
Colorado
United States
Atlanta
Georgia
United States
Indianapolis
Indiana
United States
Lexington
Kentucky
United States
Boston
Massachusetts
United States
Detroit
Michigan
United States
Grand Rapids
Michigan
United States
Minneapolis
Minnesota
United States
Kansas City
Missouri
United States
Omaha
Nebraska
United States
Pittsburgh
Pennsylvania
United States
Salt Lake City
Utah
United States
Charlottesville
Virginia
United States
Richmond
Virginia
United States
Seattle
Washington
United States
Vancouver
Canada
Edinburgh
United Kingdom
Liverpool
United Kingdom
London
United Kingdom
FG0009 subjects
COMPLETED
FG0009 subjects
NOT COMPLETED
FG0000 subjects
Part B
Type
Comment
Milestone Data
STARTED
FG00034 subjectsEight participants from Part A continued in Part B of the study.
COMPLETED
FG00033 subjects
NOT COMPLETED
FG0001 subjects
Type
Comment
Reasons
Adverse Event
FG0001 subjects
Safety set included all participants who received at least 1 dose of study drug.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Ivacaftor
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.
Denominators
Units
Counts
Participants
BG00035
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
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.
Mean
Standard Deviation
years
Title
Denominators
Categories
Part A (n = 9)
Title
Measurements
BG0003.1± 1.17
Part B (n = 34)
Title
Measurements
BG0003.2± 0.96
Sex/Gender, Customized
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.
Number
participants
Title
Denominators
Categories
Part A (n = 9): Female
Title
Measurements
BG0003
Part A (n = 9): Male
Title
Measurements
BG000
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
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.
Part A Safety set included all participants who received at least 1 dose of study drug in part A.
Posted
Number
participants
Part A: Up to 93 Days
ID
Title
Description
OG000
Part A: Ivacaftor 50 mg
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.
OG001
Part A: Ivacaftor 75 mg
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.
OG002
Part A: Overall Ivacaftor
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.
Units
Counts
Participants
OG0004
OG0015
OG0029
Title
Denominators
Categories
AEs
Title
Measurements
OG0003
OG0015
OG0028
SAEs
Primary
Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs
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.
Part B Safety set included all participants who received at least 1 dose of study drug in part B.
Posted
Number
participants
Part B: Up to 28 Weeks
ID
Title
Description
OG000
Part B: Ivacaftor 50 mg
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.
OG001
Part B: Ivacaftor 75 mg
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.
Secondary
Part B: Plasma Concentration of Ivacaftor and Its Metabolites
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.
Part B Safety set included all participants who received at least 1 dose of study drug in part B.
Posted
Mean
Standard Deviation
ng/mL
Part B: up to 24 hours post-dose on Day 168
ID
Title
Description
OG000
Part B: Overall Ivacaftor
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.
Units
Counts
Participants
OG000
Secondary
Part B: Absolute Change From Baseline in Sweat Chloride at Week 24
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.
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.
Posted
Mean
Standard Deviation
millimole per liter (mmol/L)
Part B: Baseline, Week 24
ID
Title
Description
OG000
Part B: Ivacaftor 50 mg
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.
OG001
Part B: Ivacaftor 75 mg
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.
OG002
Part B: Overall Ivacaftor
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.
Secondary
Part B: Absolute Change From Baseline in Weight at Week 24
Data was reported as per the dose received and for overall participants.
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.
Posted
Mean
Standard Deviation
kilograms (kg)
Part B: Baseline, Week 24
ID
Title
Description
OG000
Part B: Ivacaftor 50 mg
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.
OG001
Part B: Ivacaftor 75 mg
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.
OG002
Part B: Overall Ivacaftor
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.
Secondary
Part B: Absolute Change From Baseline in Stature at Week 24
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.
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.
Posted
Mean
Standard Deviation
centimeters (cm)
Part B: Baseline, Week 24
ID
Title
Description
OG000
Part B: Ivacaftor 50 mg
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.
OG001
Part B: Ivacaftor 75 mg
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.
OG002
Part B: Overall Ivacaftor
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.
Primary
Part A: Plasma Concentration of Ivacaftor and Its Metabolites
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.
Part A Safety set included all participants who received at least 1 dose of study drug in part A.
Posted
Mean
Standard Deviation
nanogram per milliliter (ng/mL)
Part A: up to 24 hours post-dose on Day 4
ID
Title
Description
OG000
Part A: Overall Ivacaftor
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.
Units
Counts
Participants
OG000
Secondary
Part B: Absolute Change From Baseline in Body Mass Index (BMI) at Week 24
BMI = (Weight [in kg]) divided by (Stature [in meters])^2. Data was reported as per the dose received and for overall participants.
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.
Posted
Mean
Standard Deviation
kilogram per square meter (kg/m^2)
Baseline, Week 24
ID
Title
Description
OG000
Part B: Ivacaftor 50 mg
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.
OG001
Part B: Ivacaftor 75 mg
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.
OG002
Part B: Overall Ivacaftor
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.
Time Frame
Part A: up to 93 days; Part B: up to 28 weeks
Description
Adverse events were reported separately for each part and as per the dose received and for overall participants in each part.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Part A: Ivacaftor 50 mg
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.
0
4
3
4
EG001
Part A: Ivacaftor 75 mg
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.
0
5
5
5
EG002
Part A: Overall Ivacaftor
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.
0
9
8
9
EG003
Part B: Ivacaftor 50 mg
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.
3
10
9
10
EG004
Part B: Ivacaftor 75 mg
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.
3
24
23
24
EG005
Part B: Overall Ivacaftor
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.
6
34
32
34
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Infective pulmonary exacerbation of cystic fibrosis
Infections and infestations
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG0031 affected10 at risk
EG004
Device related sepsis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pseudomonas test positive
Investigations
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Transaminases increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Convulsion
Nervous system disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG0034 affected10 at risk
EG00415 affected24 at risk
EG00519 affected34 at risk
Nasal congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0012 affected5 at risk
EG0022 affected9 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Dysponea exertional
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Nasal inflamation
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Snoring
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Tonsillar hypertrophy
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Upper respiratory track congestion
Respiratory, thoracic and mediastinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Croup infectious
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Infective pulmonary exacerbation of cystic fibrosis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Otitis media
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Sinusitis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Gastroentritis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Rhinitis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Bacterial disease carrier
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Genital candidiasis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Hand-foot-and-mouth disease
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Infectious mononucleosis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Lung infection
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pharyngitis
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pharyngitis streptococcal
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Respiratory tract infection viral
Infections and infestations
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Viral rash
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Viral upper respiratory tract infection
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0012 affected5 at risk
EG0022 affected9 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Abdominal distension
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Eructation
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Lip blister
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Oral mucosal erythema
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Retching
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Teething
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Bacterial test positive
Investigations
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Haemophilus test positive
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Activated partial thromboplastin time prolonged
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Antibiotic resistant Staphylococcus test positive
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Blood creatine increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Blood creatinine increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Liver function test abnormal
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Lymphocyte count increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Respiratory rate increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pancreatic enzyme increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Staphylococcus test positive
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
White blood cell count increased
Investigations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Acne
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Dermatitis contact
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Petechiae
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Skin irritation
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Ecchymosis
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0012 affected5 at risk
EG0022 affected9 at risk
EG003
Rash macular
Skin and subcutaneous tissue disorders
MedDRA 15.1
Systematic Assessment
EG0001 affected4 at risk
EG0010 affected5 at risk
EG0021 affected9 at risk
EG003
Bronchitis viral
Infections and infestations
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Pyrexia
General disorders
MedDRA 15.1
Systematic Assessment
EG0002 affected4 at risk
EG0012 affected5 at risk
EG0024 affected9 at risk
EG003
Application site rash
General disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Fatigue
General disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Product taste abnormal
General disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Face injury
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Mouth injury
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Open wound
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Traumatic haemorrhage
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Upper limb fracture
Injury, poisoning and procedural complications
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Headache
Nervous system disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0011 affected5 at risk
EG0021 affected9 at risk
EG003
Drooling
Nervous system disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Sinus headache
Nervous system disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Weight gain poor
Metabolism and nutrition disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Amblyopia
Eye disorders
MedDRA (15.1)
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Anisometropia
Eye disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Eye inflammation
Eye disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Chromaturia
Renal and urinary disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Pollakiuria
Renal and urinary disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Lymphadenopathy
Blood and lymphatic system disorders
MedDRA 15.1
Systematic Assessment
EG0000 affected4 at risk
EG0010 affected5 at risk
EG0020 affected9 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
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.
Point of Contact
Title
Organization
Phone
Extension
Email
Medical Monitor
Vertex Pharmaceuticals Incorporated
617-341-6777
medicalinfo@vrtx.com
ID
Term
D003550
Cystic Fibrosis
Ancestor Terms
ID
Term
D010182
Pancreatic Diseases
D004066
Digestive System Diseases
D008171
Lung Diseases
D012140
Respiratory Tract Diseases
D030342
Genetic Diseases, Inborn
D009358
Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D007232
Infant, Newborn, Diseases
Browse Leaves
Not provided
Browse Branches
Not provided
ID
Term
C545203
ivacaftor
Ancestor Terms
Not provided
Browse Leaves
Not provided
Browse Branches
Not provided
6
Part B (n = 34): Female
Title
Measurements
BG0006
Part B (n = 34): Male
Title
Measurements
BG00028
Title
Measurements
OG0000
OG0010
OG0020
Related AEs
Title
Measurements
OG0001
OG0013
OG0024
OG002
Part B: Overall Ivacaftor
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.