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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
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Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucus, chronic bacterial infection of the airways, and inflammatory response are all thought to play a role in causing lung damage. Through its function as a chloride channel, CFTR is believed to be integral in epithelial ion and water transport and hence, maintaining the normal hydration of lung secretions.
VX-770 (ivacaftor) is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR. Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, VX-770 has been selected for clinical development as a possible treatment for patients with CF.
Hyperpolarized noble gas magnetic resonance imaging (HG-MRI) is a promising new means of assessing lung function by direct imaging of certain non-radioactive isotopes of an inert noble gas, such as helium or xenon. Through this technique, high-resolution 3-dimensional images of lung ventilation can be obtained in both pediatric and adult patients during a single short breath-hold following inhalation of the gas.
This is a 2-part study to evaluate the effect of VX-770 on hyperpolarized helium-3 magnetic resonance imaging (3He-MRI), and to evaluate the safety and efficacy of VX-770 in subjects aged 12 years and older with CF who have the G551D-CFTR mutation. Part A is a single-blind, placebo-controlled study that includes 4 weeks of VX-770 treatment and 4 weeks of placebo treatment. Part B is an open-label, 48 week study of long-term effect of VX 770 on hyperpolarized 3He-MRI.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| VX-770 | Experimental | Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study. Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-770 | Drug | Tablet. |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Day 43 | Subjects inhaled hyperpolarized helium-3 (3He) gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid magnetic resonance imaging (MRI) was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He-MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | Part A: Baseline (pre-dose Day 15), Day 43 |
| Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Week 48 | Subjects were asked to inhale hyperpolarized 3 He gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid MRI was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | Part B: Baseline (Day -1), Week 48 |
| 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 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. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Talissa Altes, MD | University of Virginia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Charlottesville | Virginia | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28132845 | Derived | Altes TA, Johnson M, Fidler M, Botfield M, Tustison NJ, Leiva-Salinas C, de Lange EE, Froh D, Mugler JP 3rd. Use of hyperpolarized helium-3 MRI to assess response to ivacaftor treatment in patients with cystic fibrosis. J Cyst Fibros. 2017 Mar;16(2):267-274. doi: 10.1016/j.jcf.2016.12.004. Epub 2017 Jan 26. |
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All results were planned to be reported separately for Part A and Part B of the study.
Study was initiated on October 10, 2010 after first eligible subject signed informed consent form and enrolled in study.
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| ID | Title | Description |
|---|---|---|
| FG000 | VX-770 | Part A: Subjects received placebo tablets matched to VX-770 150 milligram (mg) orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study. Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A |
|
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| Placebo |
| Drug |
Tablet. |
|
| Part A: Day 1 up to Day 57 |
| Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | Part A: Baseline (pre-dose Day 15), Day 43 |
| Part A: Absolute Change From Baseline in Sweat Chloride at Day 43 | 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 in VX-770 treatment phase (Day 15 to 42). | Part A: Baseline (pre-dose Day 15), Day 43 |
| Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day 43 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | Baseline (pre-dose Day 15), Day 43 |
| Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs | 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. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. | Part B: Day 1 up to Week 48 |
| Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | Part B: Baseline (Day -1), Week 48 |
| Part B: Absolute Change From Baseline in Sweat Chloride at Week 48 | 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 in Part B (48 weeks). | Part B: Baseline (Day -1), Week 48 |
| Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Week 48 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | Part B: Baseline (Day -1), Week 48 |
| COMPLETED |
|
| NOT COMPLETED |
|
| Part B |
|
|
Full Analysis Set (FAS) = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo in Part A and VX-770 in Part B).
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| ID | Title | Description |
|---|---|---|
| BG000 | VX-770 | Part A: Subjects received placebo tablets matched to VX-770 150 mg orally twice daily from Day 1 to 14 (Placebo run-in period), followed by VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), and then placebo tablets matched to VX-770 150 mg orally twice daily from Day 43 to 57 (Placebo washout period) during Part A of the study. Part B: Subjects received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study. Part B included subjects from Part A and newly enrolled subjects. |
| Units | Counts |
|---|---|
| Participants |
|
| 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 |
| |||||||||||||||||||||
| Sex/Gender, Customized | Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. Subjects may be counted in more than one category. | Number | participants |
| ||||||||||||||||||||||
| Race/Ethnicity, Customized | Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. Subject may be counted in more than one category. | Number | participants |
| ||||||||||||||||||||||
| Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) | 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. Number of subjects in each percent predicted FEV1 category (less than [<] 70%, greater than or equal to [>=] 70%-<90%, and >=90%) are reported. Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. Subjects may be reported in more than one category. | Number | participants |
| ||||||||||||||||||||||
| Body Weight | Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. | Mean | Standard Deviation | kilogram (kg) |
| |||||||||||||||||||||
| Height | Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. | Mean | Standard Deviation | centimeter (cm) |
| |||||||||||||||||||||
| Body Mass Index (BMI) | BMI = (Weight [in kg]) divided by (Height [in meters])^2. Data was planned to be reported separately for Part A and Part B of the study. Here "n" signifies subjects who were evaluable for specified part of the study. | Mean | Standard Deviation | kilogram per square meter (kg/m^2) |
|
| 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: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Day 43 | Subjects inhaled hyperpolarized helium-3 (3He) gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid magnetic resonance imaging (MRI) was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He-MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | FAS for Part A = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part A. | Posted | Mean | Standard Deviation | percentage of total lung volume | Part A: Baseline (pre-dose Day 15), Day 43 |
|
|
| |||||||||||||||||||||||||
| Primary | Part B: Change From Baseline in Total Ventilation Defect Defined by Hyperpolarized Helium 3 Magnetic Resonance Imaging (3He-MRI) at Week 48 | Subjects were asked to inhale hyperpolarized 3 He gas mixed with nitrogen to make a total volume of approximately one-third forced vital capacity (FVC) to a maximum of 1 liter and hold their breath for 20 seconds or less. Rapid MRI was performed during inhalation/exhalation and/or breath-hold. Areas of decreased ventilation were observed as ventilation defects that are visualized as decreased (and/or absent) 3He intensity in 3He MRI. The total ventilation defect was defined as the ratio of total ventilation defect volume (L) to total lung volume (L), expressed as a percentage. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | FAS for Part B = all enrolled subjects who received at least 1 dose of study drug (VX-770) in Part B. | Posted | Mean | Standard Deviation | percentage of total lung volume | Part B: Baseline (Day -1), Week 48 |
| |||||||||||||||||||||||||||
| Secondary | Part A: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs | 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. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. | Safety Set for Part A = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part A. Data was reported as per the intervention received (Placebo [Placebo Run in/Washout] or VX-770 [VX-770 Treatment]). | Posted | Number | participants | Part A: Day 1 up to Day 57 |
| ||||||||||||||||||||||||||||
| Secondary | Part A: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Day 43 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | FAS for Part A = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part A. | Posted | Mean | Standard Deviation | percent predicted of FEV1 | Part A: Baseline (pre-dose Day 15), Day 43 |
|
| ||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Baseline in Sweat Chloride at Day 43 | 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 in VX-770 treatment phase (Day 15 to 42). | FAS for Part A = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part A. | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | Part A: Baseline (pre-dose Day 15), Day 43 |
|
| ||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Day 43 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in VX-770 treatment phase (Day 15 to 42). | FAS for Part A = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part A. | Posted | Mean | Standard Deviation | units on a scale | Baseline (pre-dose Day 15), Day 43 |
|
| ||||||||||||||||||||||||||
| Secondary | Part B: Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and Related AEs | 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. Related AEs includes all AEs for which the causality was either related to study drug or possibly related to study drug. | Safety Set for Part B = all enrolled subjects who received at least 1 dose of study drug (VX-770 or placebo) in Part B. | Posted | Number | participants | Part B: Day 1 up to Week 48 |
|
| |||||||||||||||||||||||||||
| Secondary | Part B: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 48 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | FAS for Part B = all enrolled subjects who received at least 1 dose of study drug (VX-770) in Part B. Here, "Number of participants analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | percent predicted of FEV1 | Part B: Baseline (Day -1), Week 48 |
|
| ||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in Sweat Chloride at Week 48 | 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 in Part B (48 weeks). | FAS for Part B = all enrolled subjects who received at least 1 dose of study drug (VX-770) in Part B. Here, "Number of participants analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | mmol/L | Part B: Baseline (Day -1), Week 48 |
|
| ||||||||||||||||||||||||||
| Secondary | Part B: Absolute Change From Baseline in in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score At Week 48 | 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. Baseline was defined as the most recent non-missing measurement collected before initial administration of study drug in Part B (48 weeks). | FAS for Part B = all enrolled subjects who received at least 1 dose of study drug (VX-770) in Part B. Here, "Number of participants analyzed" signifies those subjects who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | units on a scale | Part B: Baseline (Day -1), Week 48 |
|
Part A: Day 1 through Day 57; Part B: Day 1 through Week 48
A Participant with multiple events within a system organ class or preferred term was counted only once within the system organ class or preferred term, respectively.
Not provided
| 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 Placebo Run in/Washout | Subjects who received placebo tablets matched to VX-770 150 mg orally twice daily from Day 1 to 14 (Placebo run-in period) and from Day 43 to 57 (Placebo washout period) during Part A of the study were assessed between Day 1 to 14 and Day 43 to 57 of Part A. | 0 | 8 | 2 | 8 | ||
| EG001 | Part A VX-770 Treatment | Subjects who received VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), during Part A of the study were assessed between Day 15 to 42 of Part A. | 0 | 8 | 3 | 8 | ||
| EG002 | Part B VX-770 Treatment | Subjects who received VX-770 150 mg tablets orally twice daily for 48 weeks during Part B of the study were assessed between Day 1 to Week 48 of Part B. Part B included subjects from Part A and newly enrolled subjects. | 1 | 9 | 6 | 9 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| INFECTIVE PULMONARY EXACERBATION OF CYSTIC FIBROSIS | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| CYSTIC FIBROSIS LUNG | Congenital, familial and genetic disorders | MedDRA 12.0 | Systematic Assessment |
| |
| ABDOMINAL DISTENSION | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| ABDOMINAL PAIN | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| NAUSEA | Gastrointestinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| PAIN | General disorders | MedDRA 13.1 | Systematic Assessment |
| |
| PYREXIA | General disorders | MedDRA 13.1 | Systematic Assessment |
| |
| HEADACHE | Nervous system disorders | MedDRA 13.1 | Systematic Assessment |
| |
| UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| INFECTIVE PULMONARY EXACERBATION OF CYSTIC FIBROSIS | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| BRONCHITIS | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| LABYRINTHITIS | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| NASOPHARYNGITIS | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| PHARYNGITIS STREPTOCOCCAL | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| VULVOVAGINAL MYCOTIC INFECTION | Infections and infestations | MedDRA 13.1 | Systematic Assessment |
| |
| BODY MASS INDEX INCREASED | Investigations | MedDRA 13.1 | Systematic Assessment |
| |
| SPIROMETRY ABNORMAL | Investigations | MedDRA 13.1 | Systematic Assessment |
| |
| BACTERIAL TEST POSITIVE | Investigations | MedDRA 13.1 | Systematic Assessment |
| |
| CERUMEN IMPACTION | Ear and labyrinth disorders | MedDRA 13.1 | Systematic Assessment |
| |
| STRESS FRACTURE | Injury, poisoning and procedural complications | MedDRA 13.1 | Systematic Assessment |
| |
| TENDONITIS | Musculoskeletal and connective tissue disorders | MedDRA 13.1 | Systematic Assessment |
| |
| COUGH | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| RASH | Skin and subcutaneous tissue disorders | MedDRA 13.1 | Systematic Assessment |
| |
| DRY THROAT | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
| |
| OROPHARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | MedDRA 13.1 | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Part B: Female (n = 9) |
|
| Part B: Male (n = 9) |
|
| Part A, Ethnicity: Not Hispanic or Latino (n = 8) |
|
| Part B, Race: White (n = 9) |
|
| Part B, Ethnicity: Not Hispanic or Latino (n = 9) |
|
| Title | Measurements |
|---|---|
|
| Part A: >=90% (n = 8) |
|
| Part B: <70% (n = 9) |
|
| Part B: >=70%-<90% (n = 9) |
|
| Part B: >=90% (n = 9) |
|
| Units | Counts |
|---|---|
| Participants |
|
|
Subjects who received VX-770 150 mg tablets orally twice daily from Day 15 to 42 (VX-770 treatment period), during Part A of the study were assessed between Day 15 to 42 of Part A.
|
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| Participants |
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| Units |
|---|
| Counts |
|---|
| Participants |
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