Study of VX-809 in Cystic Fibrosis Subjects With the ∆F50... | NCT00865904

Label	Type	Description	Intervention Names
Placebo	Placebo Comparator	Placebo matched to VX-809 capsule orally once daily for 28 days.	Drug: Placebo
VX-809, 25 mg	Experimental	VX-809, 25 milligram (mg) capsule orally once daily for 28 days.	Drug: VX-809
VX-809, 50 mg	Experimental	VX-809, 50 mg capsule orally once daily for 28 days.	Drug: VX-809
VX-809, 100 mg	Experimental	VX-809, 100 mg capsule orally once daily for 28 days.	Drug: VX-809
VX-809, 200 mg	Experimental	VX-809, 200 mg capsule orally once daily for 28 days.	Drug: VX-809

Name	Type	Description	Arm Group Labels	Other Names
VX-809	Drug	Capsules	VX-809, 100 mg VX-809, 200 mg VX-809, 25 mg VX-809, 50 mg

Measure	Description	Time Frame
Safety and Tolerability Based on Adverse Events (AEs)	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. Serious adverse event (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, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.	Up to 14 days after last dose (last dose = Day 28)

Measure	Description	Time Frame
Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.	Baseline, Day 28
Change From Baseline in Percent Predicted FEV1 at Day 28

Name	Affiliation	Role
Medical Monitor	Vertex Pharmaceuticals Incorporated	Study Director

PubMed Identifier	Type	Citation	Retractions
37983082	Derived	Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
33331662	Derived

ID	Title	Description
FG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
FG001	VX-809, 25 mg	VX-809, 25 milligram (mg) capsule orally once daily for 28 days.

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

Safety and Tolerability Based on Adverse Events (AEs)

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. Serious adverse event (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, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.

Safety set included all participants who received at least 1 dose of study drug.

Posted

Number

participants

Up to 14 days after last dose (last dose = Day 28)

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.

Units	Counts
Participants	OG00017 OG00118 OG00218 OG003

Title

Denominators

Categories

Participants With Any AE

Title	Measurements
	OG00017 OG00116 OG00215 OG003

Secondary

Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Full Analysis Set (FAS) included all randomized participants who received at least 1 dose of study drug. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Least Squares Mean

95% Confidence Interval

liters

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg

Secondary

Change From Baseline in Percent Predicted FEV1 at Day 28

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method.

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Least Squares Mean

95% Confidence Interval

Percent predicted of FEV1

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg

Secondary

Change From Baseline in Forced Vital Capacity (FVC) at Day 28

FVC is the volume of air that can be forcibly exhaled from the lungs after taking the deepest breath possible.

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Mean

Standard Deviation

liters

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.

Secondary

Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28

FEF25-75 is total volume of air exhaled from the lungs over the middle half of the FVC test, expressed as liters per second (L/sec).

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Mean

Standard Deviation

liters per second (L/sec)

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg

Secondary

Change From Baseline in Sweat Chloride at Day 28

Sweat samples were collected using an approved Macroduct (Wescor) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride.

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Least Squares Mean

95% Confidence Interval

millimole per liter (mmol/L)

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG004	VX-809, 200 mg

Secondary

Change From Baseline in Nasal Potential Difference (NPD) of Zero Chloride Plus Isoproterenol Response at Day 28

Nasal potential difference (NPD) provides a direct and sensitive evaluation of sodium and chloride transport in secretory epithelial cells via assessment of transepithelial bioelectric properties. NPD under conditions of zero chloride concentration perfusion solution in the presence of isoproterenol is reported.

NPDs were performed according to Cystic Fibrosis Foundation Therapeutics Development Network (CFFT TDN) Standard Operating Procedure (SOP) 528.00 "Standardization of Measurement of Nasal Membrane Transepithelial Potential Difference (NPD) - electronic data capture (EDC) and Perfusion or Perfusion-Free Probe".

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Least Squares Mean

95% Confidence Interval

millivolts (mV)

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.

Secondary

Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Domain Scores at Day 28

The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. CFQ-R domains include: Body, Digestion, Eat, Emotion, Health Perceptions, Physical, Respiratory, Role, Social, Treatment Burden, Vitality, and Weight. Individual domain score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

FAS. Number of participants analyzed signifies participants evaluable for this outcome.

Posted

Least Squares Mean

95% Confidence Interval

units on a scale

Baseline, Day 28

ID	Title	Description
OG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
OG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG003	VX-809, 100 mg

Secondary

Maximum Plasma Concentration (Cmax) of VX-809

Only participants who received VX-809 were analyzed for this outcome measure.

FAS. Here, n = participants evaluable for specified category for each arm, respectively.

Posted

Mean

Standard Deviation

nanogram per milliliter (ng/mL)

Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, 24, and 30-60 hours post dose)

ID	Title	Description
OG000	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG001	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG002	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG003	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.

Secondary

Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24) of VX-809

Only participants who received VX-809 were analyzed for this outcome measure.

FAS. Here, n = participants evaluable for specified category for each arm, respectively.

Posted

Mean

Standard Deviation

hour*nanogram per milliliter (hr*ng/mL)

Day 1 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post-dose), Day 28 (pre dose, 0.75, 1.5, 3, 4, 6, 9, 12, and 24 hours post dose)

ID	Title	Description
OG000	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
OG001	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
OG002	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
OG003	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0007 affected17 at risk EG00110 affected18 at risk EG0026 affected18 at risk EG0037 affected17 at risk EG00410 affected19 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0015 affected18 at risk EG0023 affected18 at risk EG003
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0012 affected18 at risk EG0020 affected18 at risk EG003
Rales	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0016 affected18 at risk EG0022 affected18 at risk EG003
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0012 affected18 at risk EG0021 affected18 at risk EG003
Wheezing	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0011 affected18 at risk EG0024 affected18 at risk EG003
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0010 affected18 at risk EG0023 affected18 at risk EG003
Pleuritic pain	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Respiration abnormal	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected17 at risk EG0011 affected18 at risk EG0022 affected18 at risk EG003
Haemoptysis	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Nasal oedema	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Increased viscosity of bronchial secretion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Lung hyperinflation	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Nasal turbinate abnormality	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Paranasal sinus hypersecretion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0012 affected18 at risk EG0020 affected18 at risk EG003
Postnasal drip	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Rhonchi	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Bronchospasm	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Nasal mucosal disorder	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Nasal ulcer	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Painful respiration	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Pharyngeal erythema	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Sputum discoloured	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Tonsillar hypertrophy	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Tonsillar inflammation	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Upper respiratory tract congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0013 affected18 at risk EG0021 affected18 at risk EG003
Abdominal pain	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0013 affected18 at risk EG0021 affected18 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0012 affected18 at risk EG0022 affected18 at risk EG003
Abdominal pain upper	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0013 affected18 at risk EG0020 affected18 at risk EG003
Post-tussive vomiting	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0022 affected18 at risk EG003
Flatulence	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Abdominal pain lower	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Aphthous stomatitis	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Dyspepsia	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Gastrointestinal sounds abnormal	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Abdominal discomfort	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Abdominal distension	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Abnormal faeces	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Dry mouth	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Frequent bowel movements	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Gingival pain	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Infrequent bowel movements	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Rectal haemorrhage	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Fatigue	General disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected17 at risk EG0013 affected18 at risk EG0023 affected18 at risk EG003
Pyrexia	General disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected17 at risk EG0012 affected18 at risk EG0021 affected18 at risk EG003
Pain	General disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Chest discomfort	General disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Catheter site haematoma	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Catheter site rash	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Chest pain	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Chills	General disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Hunger	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Irritability	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Nodule	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Oedema peripheral	General disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Breath sounds abnormal	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Forced expiratory volume decreased	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Pulmonary function test decreased	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
C-reactive protein increased	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Prothrombin time prolonged	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Sputum abnormal	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0012 affected18 at risk EG0020 affected18 at risk EG003
Blood glucose increased	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Neutrophil count increased	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
White blood cell count increased	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Activated partial thromboplastin time prolonged	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Alanine aminotransferase increased	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Blood urine present	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Electrocardiogram QT prolonged	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Glucose urine	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Protein urine present	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Urine leukocyte esterase positive	Investigations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Weight decreased	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Headache	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0003 affected17 at risk EG0014 affected18 at risk EG0025 affected18 at risk EG003
Dizziness	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Tension headache	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Hypogeusia	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Migraine	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Sinus headache	Investigations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Somnolence	Nervous system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0023 affected18 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0002 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0012 affected18 at risk EG0021 affected18 at risk EG003
Neck pain	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Arthritis	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Clubbing	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Flank pain	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Joint effusion	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Joint range of motion decreased	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Medial tibial stress syndrome	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Musculoskeletal discomfort	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Musculoskeletal pain	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Tendonitis	Musculoskeletal and connective tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0012 affected18 at risk EG0021 affected18 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Rhinitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Influenza	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Oral candidiasis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Pharyngitis streptococcal	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Sinusitis	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Vulvovaginal mycotic infection	Infections and infestations	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Acne	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Erythema	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Rash	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Blister	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Rash macular	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Dermatitis allergic	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Ecchymosis	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Cystic fibrosis lung	Congenital, familial and genetic disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Cystic fibrosis related diabetes	Congenital, familial and genetic disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0011 affected18 at risk EG0021 affected18 at risk EG003
Arthropod bite	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Post procedural haemorrhage	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Epicondylitis	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Muscle strain	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Sunburn	Injury, poisoning and procedural complications	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Ear pain	Ear and labyrinth disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Tympanic membrane hyperaemia	Ear and labyrinth disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Middle ear effusion	Ear and labyrinth disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Motion sickness	Ear and labyrinth disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Vertigo	Ear and labyrinth disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Hypoglycaemia	Metabolism and nutrition disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Decreased appetite	Metabolism and nutrition disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Gout	Metabolism and nutrition disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Polydipsia	Metabolism and nutrition disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Anxiety	Psychiatric disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0011 affected18 at risk EG0020 affected18 at risk EG003
Agitation	Psychiatric disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Depression	Psychiatric disorders	MedDRA 12.0	Systematic Assessment	EG0001 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Haematuria	Renal and urinary disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Polyuria	Renal and urinary disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Proteinuria	Renal and urinary disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Haematoma	Vascular disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Hypertension	Vascular disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Lymphadenopathy	Blood and lymphatic system disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003
Palpitations	Cardiac disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0020 affected18 at risk EG003
Conjunctival hyperaemia	Eye disorders	MedDRA 12.0	Systematic Assessment	EG0000 affected17 at risk EG0010 affected18 at risk EG0021 affected18 at risk EG003

Facility	Status	City	State	ZIP	Country	Contacts
		Birmingham	Alabama		United States

ID	Term
D003550	Cystic Fibrosis
D010182	Pancreatic Diseases
D008171	Lung Diseases
D030342	Genetic Diseases, Inborn
D007232	Infant, Newborn, Diseases

ID	Term
D004066	Digestive System Diseases
D012140	Respiratory Tract Diseases
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

ID	Title	Description
BG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.
BG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.
BG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.
BG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.
BG004	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.
BG005	Total	Total of all reporting groups

ID	Title	Description	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo	Placebo matched to VX-809 capsule orally once daily for 28 days.	1	17	17	17
EG001	VX-809, 25 mg	VX-809, 25 mg capsule orally once daily for 28 days.	5	18	16	18
EG002	VX-809, 50 mg	VX-809, 50 mg capsule orally once daily for 28 days.	1	18	15	18
EG003	VX-809, 100 mg	VX-809, 100 mg capsule orally once daily for 28 days.	0	17	16	17
EG004	VX-809, 200 mg	VX-809, 200 mg capsule orally once daily for 28 days.	3	19	18	19