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The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lumacaftor/Ivacaftor combination | Experimental | Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Lumacaftor | Drug |
|
| |
| Ivacaftor |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; event does not necessarily have a causal relationship with treatment. This includes any newly occurring event/previous condition that has increased in severity/frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event, which falls into any of the following categories, regardless of its relationship to 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. TEAEs: AEs that started/ worsened on/after the start of study drug through the Safety Follow up Visit (4 weeks after the last dose of study drug). Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Day 1 up to Week 28 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Denver | Colorado | United States | ||||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29126871 | Derived | Taylor-Cousar JL, Jain M, Barto TL, Haddad T, Atkinson J, Tian S, Tang R, Marigowda G, Waltz D, Pilewski J; VX14-809-106 Investigator Group. Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. J Cyst Fibros. 2018 Mar;17(2):228-235. doi: 10.1016/j.jcf.2017.09.012. Epub 2017 Nov 8. |
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A total of 46 participants were enrolled and treated in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | LUM/IVA | Participants received lumacaftor (LUM) 400 milligram (mg) in combination with ivacaftor (IVA) 250 mg as fixed-dose combination (FDC) tablet orally every 12 hours (q12h) for 24 weeks. A reduced initial dose of LUM 200 mg in combination with IVA 125 mg FDC tablet orally q12h was permitted. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Drug |
|
|
| Baseline, Up to Week 24 |
| Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Baseline, Up to Week 24 |
| Duration For Which Participants Received Intravenous (IV) Antibiotics | The duration for which participants received IV antibiotics for sinopulmonary signs and symptoms were reported. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Baseline through Week 24 |
| Number of Hospitalizations | Number of hospitalizations (all causes) through Week 24 was summarized. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Baseline through Week 24 |
| Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4 | Sweat samples were collected using an approved collection device. Baseline was defined as the average of the measurements at screening and on Day 1 pre-dose. The average absolute change from baseline in sweat chloride was derived as: (Average of Day 15 and Week 4 value) minus Baseline value. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Baseline, Day 15 and Week 4 |
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score Through Week 24 | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Baseline, Through Week 24 |
| Tampa |
| Florida |
| United States |
| Chicago | Illinois | United States |
| St Louis | Missouri | United States |
| Pittsburgh | Pennsylvania | United States |
| Houston | Texas | United States |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | LUM/IVA | Participants received LUM 400 mg in combination with IVA 250 mg as FDC tablet orally q12h for 24 weeks. A reduced initial dose of LUM 200 mg in combination with IVA 125 mg FDC tablet orally q12h was permitted. |
| 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 |
| |||||||||||||||||
| Sex: Female, Male | 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) or Serious Adverse Events (SAEs) | AE: any untoward medical occurrence in a participant during the study; event does not necessarily have a causal relationship with treatment. This includes any newly occurring event/previous condition that has increased in severity/frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event, which falls into any of the following categories, regardless of its relationship to 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. TEAEs: AEs that started/ worsened on/after the start of study drug through the Safety Follow up Visit (4 weeks after the last dose of study drug). Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Safety Set included all participants who were exposed to any amount of study drug. | Posted | Count of Participants | Participants | No | Day 1 up to Week 28 |
|
|
| ||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | Full Analysis Set (FAS) included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Percent predicted of FEV1 | Baseline, Up to Week 24 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24 | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate FEV1 (for age, gender, race, and height). The Hankinson standard was used for male participants 18 years and older and female participants 16 years and older. The Wang standard was used for male participants aged 12 to 17 years and for female participants aged 12 to 15 years. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | FAS included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Liter (L) | Baseline, Up to Week 24 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Duration For Which Participants Received Intravenous (IV) Antibiotics | The duration for which participants received IV antibiotics for sinopulmonary signs and symptoms were reported. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | FAS included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who received at least one IV antibiotic for sinopulmonary signs and symptoms. | Posted | Mean | Standard Deviation | Days | Baseline through Week 24 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Number of Hospitalizations | Number of hospitalizations (all causes) through Week 24 was summarized. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | FAS included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Number | Hospitalizations | Baseline through Week 24 |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4 | Sweat samples were collected using an approved collection device. Baseline was defined as the average of the measurements at screening and on Day 1 pre-dose. The average absolute change from baseline in sweat chloride was derived as: (Average of Day 15 and Week 4 value) minus Baseline value. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | FAS included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Error | Millimoles per litre (mmol/L) | Baseline, Day 15 and Week 4 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score Through Week 24 | The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), the scaled score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification. | FAS included all participants who were enrolled and administered any amount of study drug. Here, "Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Units on a scale | Baseline, Through Week 24 |
|
|
Day 1 up to Week 28
AEs were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification.
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 | LUM/IVA | Participants received LUM 400 mg in combination with IVA 250 mg as FDC tablet orally q12h for 24 weeks. A reduced initial dose of LUM 200 mg in combination with IVA 125 mg FDC tablet orally q12h was permitted. | 1 | 46 | 18 | 46 | 43 | 46 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Neuralgia | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Bacteraemia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hot flush | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Affect lability | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Irritability | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Breast pain | Reproductive system and breast disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Menorrhagia | Reproductive system and breast disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood glucose decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood immunoglobulin E increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood phosphorus decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Blood pressure diastolic increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Fungal test positive | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Oxygen consumption increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Prostatic specific antigen increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Sputum abnormal | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| White blood cell count increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Increased viscosity of bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Painful respiration | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paranasal sinus discomfort | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Sputum retention | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Upper-airway cough syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Transient ischaemic attack | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Lacrimation increased | Eye disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrointestinal tract mucosal discolouration | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nephrocalcinosis | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Night sweats | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Musculoskeletal discomfort | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Vulvovaginal mycotic infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Chronic sinusitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Clostridium difficile colitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Clostridium difficile infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Labyrinthitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Tooth abscess | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
|
Restriction Description: PI is free to publish results of the study after (1)first multi-center publication, (2)if 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 |
|---|---|
| C569105 | lumacaftor |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
|
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