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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
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The purpose of this study is to provide information regarding the long-term safety and pharmacodynamics of ivacaftor treatment in the pediatric population younger than 6 years of age with Cystic Fibrosis (CF) who have a CFTR gating mutation in at least 1 allele and will further explore the efficacy of long-term ivacaftor treatment in this population of patients with CF.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Observational Arm | No Intervention | ||
| Ivacaftor | Experimental | Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the Week 84 Visit. The ivacaftor dose will be:
|
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ivacaftor | Drug |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent. | Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing) |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). |
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Inclusion Criteria (Ivacaftor Arm):
Inclusion Criteria (Observational Arm):
1. Subjects who completed their assigned study drug treatment in the previous study (NCT01705145) and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.
Exclusion Criteria (Ivacaftor Arm):
Exclusion Criteria: (Observational Arm)
1. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
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| Name | Affiliation | Role |
|---|---|---|
| Adebayo Lawal, M.D. | Vertex Pharmaceuticals Incorporated | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31053538 | Derived | Rosenfeld M, Cunningham S, Harris WT, Lapey A, Regelmann WE, Sawicki GS, Southern KW, Chilvers M, Higgins M, Tian S, Cooke J, Davies JC; KLIMB study group. An open-label extension study of ivacaftor in children with CF and a CFTR gating mutation initiating treatment at age 2-5 years (KLIMB). J Cyst Fibros. 2019 Nov;18(6):838-843. doi: 10.1016/j.jcf.2019.03.009. Epub 2019 Apr 30. |
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In study VX11-770-109 (study 109) (NCT01946412), participants were to be enrolled in either ivacaftor arm or observational arm. However, there were no participants enrolled in the observational arm. A total of 33 participants were enrolled in the ivacaftor arm.
This was a Phase 3, multicenter, 2 arm study in participants who received at least 1 dose of study drug in parent study VX11-770-108 (study 108) (NCT01705145).
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| ID | Title | Description |
|---|---|---|
| FG000 | Ivacaftor | Participants received ivacaftor 50 milligram (mg) or 75 mg or 150 mg based on body weight and age. Ivacaftor 50 mg administered every 12 hours (q12h) for participants aged 2 to less than (<) 6 years and weighing <14 kilograms (kg), ivacaftor 75 mg q12h for participants aged 2 to <6 years and weighing greater than or equal to (>=) 14 kg and ivacaftor 150 mg q12h for participants >=6 years. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Baseline (study 108), Week 24, 48, 72 and 84 (study 109) |
| Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of >=15 microliter was required for determination of sweat chloride. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Baseline (study 109), Week 24, 48, 72 and 84 (study 109) |
| Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84 | Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145) | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84 | Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84 | Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84 | Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 | BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 | BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| Denver |
| Colorado |
| United States |
| Atlanta | Georgia | United States |
| Indianapolis | Indiana | United States |
| Boston | Massachusetts | United States |
| Grand Rapids | Michigan | United States |
| Minneapolis | Minnesota | United States |
| Kansas City | Missouri | United States |
| Omaha | Nebraska | United States |
| Salt Lake City | Utah | United States |
| Richmond | Virginia | United States |
| Seattle | Washington | United States |
| Vancouver | British Columbia | Canada |
| Edinburgh | United Kingdom |
| Liverpool | United Kingdom |
| London | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Ivacaftor | Participants received ivacaftor 50 mg or 75 mg or 150 mg based on body weight and age. Ivacaftor 50 mg administered q12h for participants aged 2 to < 6 years and weighing <14 kg, ivacaftor 75 mg q12h for participants aged 2 to <6 years and weighing >= 14 kg and ivacaftor 150 mg q12h for participants >=6 years. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Gender | Count of Participants | Participants |
|
| 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 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. SAE (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, Inpatient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. AEs with start date or increased severity on or after the first dose of study drug through the end of study participation was considered treatment-emergent. | Safety set included all participants who received at least 1 dose of study drug in study 109 (NCT01946412). As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Number | participants | Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing) |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of greater than or equal to (>=) 15 microliter was required for determination of sweat chloride. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively.As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | millimole per liter (mmol/L) | Baseline (study 108), Week 24, 48, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84 | Sweat samples were collected using an approved Macroduct (Wescor, Logan, Utah) collection device. A volume of >=15 microliter was required for determination of sweat chloride. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | mmol/L | Baseline (study 109), Week 24, 48, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84 | Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145) | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | kilogram (kg) | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84 | Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | Kg | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84 | Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | Centimeters (cm) | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84 | Stature was measured as height if children could stand unassisted and follow directions; otherwise, stature was measured as length. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | cm | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 | BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in study 108 Part B (NCT01705145). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | Kilogram per square meter (kg/m^2) | Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 | BMI = (Weight [in kg]) divided by (Stature [in meters]) ^2. Baseline is defined as the most recent measurement prior to intake of the first dose of study drug in study 109 (NCT01946412). | Safety set. Here "n" signifies those participants who were evaluable at the specified time points for each arm, respectively. As per the planned analysis for this study, participants were to be analyzed based on their dosing groups as per parent study VX11-770-108 (NCT01705145). | Posted | Mean | Standard Deviation | kg/m^2 | Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109) |
|
Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing)
As per the planned analysis for this study, participants were to be reported based on their dosing groups as per parent study VX11-770-108 (NCT01705145).
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Ivacaftor 50 mg | Participants who received ivacaftor 50 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412). | 6 | 9 | 9 | 9 | ||
| EG001 | Ivacaftor 75 mg | Participants who received ivacaftor 75 mg q12h in parent study VX11-770-108 (NCT01705145), received either ivacaftor 50 mg q12h for participants aged 2 to <6 years and weighing <14 kg or ivacaftor 75 mg q12h for participants aged 2 to <6 years and >=14 kg or ivacaftor 150 mg q12h for participants >=6 years in this study (NCT01946412). | 5 | 24 | 24 | 24 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Enterovirus infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Staphylococcal infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Seizure anoxic | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Otitis media | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory tract infection viral | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Herpangina | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Lower respiratory tract infection bacterial | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Lung infection pseudomonal | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Molluscum contagiosum | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Myringitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Parainfluenzae virus infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinovirus infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Staphylococcal skin infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Streptococcal infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Upper respiratory tract infection bacterial | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 15.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Allergic sinusitis | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Snoring | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Tooth discolouration | Gastrointestinal disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Haemophilus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory rate increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Adenovirus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Antibiotic resistant Staphylococcus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Blood alkaline phosphatase increased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Influenza A virus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Lymph node palpable | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Neutrophil count decreased | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Pseudomonas test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Respiratory syncytial virus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Staphylococcus test positive | Investigations | MedDRA 15.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Nail disorder | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Red man syndrome | Skin and subcutaneous tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Eye injury | Injury, poisoning and procedural complications | MedDRA 15.1 | Systematic Assessment |
| |
| Conjunctivitis | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Amblyopia | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cataract cortical | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Eye pruritus | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cerumen impaction | Ear and labyrinth disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Convulsion | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Attention deficit/hyperactivity disorder | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Encopresis | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Onychophagia | Psychiatric disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Lymphadenopathy | Blood and lymphatic system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Enuresis | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Micturition urgency | Renal and urinary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Phimosis | Congenital, familial and genetic disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 15.1 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 15.1 | 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
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