Study of Ivacaftor in Subjects With Cystic Fibrosis (CF)... | NCT01614470

Name	Class
Cystic Fibrosis Foundation	OTHER

Label	Type	Description	Intervention Names
Part 1: Ivacaftor First, Then Placebo	Experimental	Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.	Drug: Ivacaftor Drug: Placebo
Part 1: Placebo First, Then Ivacaftor	Experimental	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.	Drug: Ivacaftor Drug: Placebo
Part 2: Ivacaftor	Experimental	Ivacaftor 150 mg tablet orally twice daily for 16 weeks.	Drug: Ivacaftor

Name	Type	Description	Arm Group Labels	Other Names
Ivacaftor	Drug	150 mg tablet, oral use, administered twice a day (q12h)	Part 1: Ivacaftor First, Then Placebo Part 1: Placebo First, Then Ivacaftor

Measure	Description	Time Frame
Part 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.	Part 1: Baseline (pre-dose Day 1), Week 8
Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit)	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Absolute change in percent predicted FEV1 over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2, as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.	Baseline (pre-dose Week 12), Week 36

Measure	Description	Time Frame
Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8	BMI was defined as weight in kilogram (kg) divided by height in meters^2 (m^2). Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.	Part 1: Baseline (pre-dose Day 1), Week 8

Name	Affiliation	Role
Christine De Boeck, MD, PhD	University of Leuven	Principal Investigator

ID	Title	Description
FG000	Part 1: Ivacaftor First, Then Placebo	Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
FG001

ID	Title	Description
BG000	Part 1: Ivacaftor First, Then Placebo	Ivacaftor 150 milligram (mg) tablet orally twice daily for 8 weeks in treatment period 1 followed by placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
BG001	Part 1: Placebo First, Then Ivacaftor	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in treatment period 1 followed by ivacaftor 150 mg tablet orally twice daily for 8 weeks in treatment period 2. Washout out period of 4 to 8 weeks was maintained between each treatment period.
BG002	Total	Total of all reporting groups

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 1: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 8

FAS for Part 1: all randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo). Here, "n" signifies those subjects who were evaluable for this measure at given time point for each group, respectively.

Posted

Mean

Standard Deviation

percent predicted of FEV1

Part 1: Baseline (pre-dose Day 1), Week 8

ID	Title	Description
OG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.
OG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.

Units	Counts
Participants	OG00038 OG00137

Title

Denominators

Categories

Baseline (n=38, 37)

Title	Measurements
	OG00076.3659± 20.33450 OG00179.3361± 20.83991

Change Through Week 8 (n=37, 37)

Title	Measurements
	OG000

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		Mixed Models Analysis		<0.0001		Least squares (LS) mean difference	10.6780			2-Sided	95	7.2559	14.1000				No	Superiority or Other

Secondary

Part 1: Change From Baseline in Body Mass Index (BMI) at Week 8

BMI was defined as weight in kilogram (kg) divided by height in meters^2 (m^2). Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during study Part 1.

Posted

Mean

Standard Deviation

kg/m^2

Part 1: Baseline (pre-dose Day 1), Week 8

ID	Title	Description
OG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.
OG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.

Units	Counts
Participants	OG000

Secondary

Part 2: Change From Baseline in Body Mass Index (BMI) at 24 Weeks of Treatment (Week 36 Visit)

BMI was defined as weight in kg divided by height in m^2. Change in BMI over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.

FAS for Part 2: all randomized subjects who received at least 1 dose of study drug (ivacaftor). Only subjects who were randomized to receive ivacaftor during Part 1: Treatment Period 2 were to be analyzed for this measure.

Posted

Mean

Standard Deviation

kg/m^2

Baseline (pre-dose Week 12), Week 36

ID	Title	Description
OG000	Part 1 Treatment Period 2: Ivacaftor, Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 24 weeks (8 weeks in Part 1: Treatment Period 2 and 16 weeks in Part 2).

Units	Counts
Participants	OG000

Secondary

Part 1: Change From Baseline in Sweat Chloride Through Week 8

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 non-missing measurement collected before initial administration of study drug during study Part 1.

Posted

Mean

Standard Deviation

millimole per liter (mmol/L)

Part 1: Baseline (pre-dose Day 1), Week 8

ID	Title	Description
OG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.
OG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.

Units	Counts
Participants

Primary

Part 2: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through 24 Weeks of Treatment (Week 36 Visit)

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years. Absolute change in percent predicted FEV1 over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2, as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.

Posted

Mean

Standard Deviation

percent predicted of FEV1

Baseline (pre-dose Week 12), Week 36

ID	Title	Description
OG000	Part 1 Treatment Period 2: Ivacaftor, Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 24 weeks (8 weeks in Part 1: Treatment Period 2 and 16 weeks in Part 2).

Secondary

Part 2: Change From Baseline in Sweat Chloride Through 24 Weeks of Treatment (Week 36 Visit)

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. Change in sweat chloride over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.

Posted

Mean

Standard Deviation

mmol/L

Baseline (pre-dose Week 12), Week 36

ID	Title	Description
OG000	Part 1 Treatment Period 2: Ivacaftor, Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 24 weeks (8 weeks in Part 1: Treatment Period 2 and 16 weeks in Part 2).

Units	Counts
Participants

Secondary

Part 1: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8

Posted

Mean

Standard Deviation

units on a scale

Part 1: Baseline (pre-dose Day 1), Week 8

ID	Title	Description
OG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.
OG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.

Units

Secondary

Part 2: Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through 24 Weeks of Treatment (Week 36 Visit)

The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Change in CFQ-R respiratory domain score over 24 weeks of ivacaftor treatment (from Week 12 [Part 1: Treatment Period 2] through Week 36 [Part 2]) was reported for subjects who received ivacaftor in Part 1: Treatment Period 2 as per planned analysis. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug during Part 1: Treatment Period 2.

Posted

Mean

Standard Deviation

units on a scale

Baseline (pre-dose Week 12), Week 36

ID	Title	Description
OG000	Part 1 Treatment Period 2: Ivacaftor, Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 24 weeks (8 weeks in Part 1: Treatment Period 2 and 16 weeks in Part 2).

Units	Counts

Secondary

Part 1: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

AE: any adverse change from subject'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.

Safety Set for Part 1 included all subjects who received at least 1 dose of study drug (ivacaftor or placebo).

Posted

Number

participants

Part 1: From signing of informed consent up to Week 20

ID	Title	Description
OG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.
OG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.

Units	Counts

Secondary

Part 2: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

Safety Set for Part 2 included all subjects who received at least 1 dose of study drug (ivacaftor).

Posted

Number

participants

Part 2: Week 20 up to Week 40

ID	Title	Description
OG000	Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 16 weeks.

Units	Counts
Participants	OG000

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Infective pulmonary exacerbation of cystic fibrosis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0007 affected38 at risk EG0016 affected37 at risk EG0024 affected36 at risk
Rhinitis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0003 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Influenza	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0002 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Conjunctivitis infective	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Respiratory tract infection viral	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Sinusitis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0012 affected37 at risk EG0022 affected36 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0012 affected37 at risk EG0023 affected36 at risk
Urinary tract infection	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Bacterial disease carrier	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Bronchitis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Gastroenteritis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Tonsillitis	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Vulvovaginal mycotic infection	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Gastrointestinal viral infection	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Impetigo	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Pharyngitis streptococcal	Infections and infestations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0006 affected38 at risk EG0017 affected37 at risk EG0025 affected36 at risk
Sputum increased	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0003 affected38 at risk EG0013 affected37 at risk EG0022 affected36 at risk
Dysphonia	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Haemoptysis	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0012 affected37 at risk EG0021 affected36 at risk
Lung hyperinflation	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0013 affected37 at risk EG0023 affected36 at risk
Rales	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0013 affected37 at risk EG0021 affected36 at risk
Respiration abnormal	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0022 affected36 at risk
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0023 affected36 at risk
Bronchospasm	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Nasal mucosal disorder	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Paranasal sinus hypersecretion	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Pulmonary congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0012 affected37 at risk EG0022 affected36 at risk
Sputum discoloured	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0022 affected36 at risk
Nasal polyps	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Nasal turbinate hypertrophy	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Pneumonitis	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Vocal cord inflammation	Respiratory, thoracic and mediastinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Pyrexia	General disorders	MedDRA 15.1	Systematic Assessment	EG0003 affected38 at risk EG0011 affected37 at risk EG0022 affected36 at risk
Fatigue	General disorders	MedDRA 15.1	Systematic Assessment	EG0002 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Chest pain	General disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Exercise tolerance decreased	General disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Malaise	General disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Medical device site reaction	General disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Oedema peripheral	General disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Infusion site thrombosis	General disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Injection site pain	General disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Non-cardiac chest pain	General disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Constipation	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0002 affected38 at risk EG0011 affected37 at risk EG0022 affected36 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0014 affected37 at risk EG0023 affected36 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0022 affected36 at risk
Distal ileal obstruction syndrome	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Nausea	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0014 affected37 at risk EG0021 affected36 at risk
Toothache	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Vomiting	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Cheilitis	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Distal intestinal obstruction syndrome	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Flatulence	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0012 affected37 at risk EG0020 affected36 at risk
Gastrooesophageal reflux disease	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0012 affected37 at risk EG0021 affected36 at risk
Abdominal distension	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Anal fissure	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Food poisoning	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Gastritis	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Haemorrhoids	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Pancreatitis	Gastrointestinal disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Headache	Nervous system disorders	MedDRA 15.1	Systematic Assessment	EG0003 affected38 at risk EG0015 affected37 at risk EG0024 affected36 at risk
Dysgeusia	Nervous system disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Sinus headache	Nervous system disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Lethargy	Nervous system disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Benign intracranial hypertension	Nervous system disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Alanine aminotransferase increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Blood creatinine increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
C-reactive protein increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Gamma-glutamyltransferase increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Neutrophil count increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Respiratory rate increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
White blood cell count increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Hepatic enzyme increased	Investigations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Pulmonary function test decreased	Investigations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Breath sounds abnormal	Investigations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Weight decreased	Investigations	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Dermatitis contact	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Dry skin	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0012 affected37 at risk EG0021 affected36 at risk
Rash papular	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Acne	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0002 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Torticollis	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Osteochondrosis	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Pain in jaw	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Tendonitis	Musculoskeletal and connective tissue disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Thyroid disorder	Endocrine disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Ligament sprain	Injury, poisoning and procedural complications	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Arthropod bite	Injury, poisoning and procedural complications	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Sunburn	Injury, poisoning and procedural complications	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Vaccination complication	Injury, poisoning and procedural complications	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Oral papilloma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Malignant melanoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Metrorrhagia	Reproductive system and breast disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Vulvovaginal discomfort	Reproductive system and breast disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Orthostatic hypotension	Vascular disorders	MedDRA 15.1	Systematic Assessment	EG0001 affected38 at risk EG0010 affected37 at risk EG0020 affected36 at risk
Hypertension	Vascular disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Hyperacusis	Ear and labyrinth disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0020 affected36 at risk
Cerumen impaction	Ear and labyrinth disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Hypoacusis	Ear and labyrinth disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Hypoglycaemia	Metabolism and nutrition disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0011 affected37 at risk EG0021 affected36 at risk
Decreased appetite	Metabolism and nutrition disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Hypersensitivity	Immune system disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Seasonal allergy	Immune system disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Conjunctivitis allergic	Eye disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Biliary colic	Hepatobiliary disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk
Anxiety	Psychiatric disorders	MedDRA 15.1	Systematic Assessment	EG0000 affected38 at risk EG0010 affected37 at risk EG0021 affected36 at risk

Facility	Status	City	State	ZIP	Country	Contacts
		Tampa	Florida		United States

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

ID	Title	Description	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Part 1: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.	4	38	27	38
EG001	Part 1: Placebo	Placebo matched to ivacaftor tablet orally twice daily for 8 weeks in either treatment period 1 or treatment period 2.	7	37	30	37
EG002	Part 2: Ivacaftor	Ivacaftor 150 mg tablet orally twice daily for 16 weeks.	3	36	30	36