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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
Not provided
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The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 12 years and older who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is a potent and selective CFTR potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR protein. Potentiators are pharmacological agents that increase the chloride ion transport properties of the channel in the presence of cyclic AMP-dependent protein kinase A (PKA) activation.
This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.
Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect in CF is the G551D mutation.
This study was designed to further evaluate the efficacy of ivacaftor in subjects with CF who have a G551D-CFTR gene mutation and to evaluate safety in this population over a longer period than previously studied.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Subjects who received placebo every 12 hours (q12h) for up to 48 weeks. |
|
| 150 mg Ivacaftor q12h | Experimental | Subjects who received 150 mg of ivacaftor q12h for up to 48 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ivacaftor | Drug | 150-mg tablets given orally q12h for up to 48 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 | Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. | baseline through 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48 | Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. | baseline through 48 weeks |
| Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled) |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
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Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Bonnie W. Ramsey, MD | Children's Hospital and Regional Medical Center, Seattle, Washington, USA | Principal Investigator |
| Stuart Elborn, MD | Respiratory Medicine Group, Queen's University of Belfast, Belfast, Northern Ireland, UK | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233-1711 | United States | ||
| Kaiser Permanente Medical Care Program |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 22047557 | Background | Ramsey BW, Davies J, McElvaney NG, Tullis E, Bell SC, Drevinek P, Griese M, McKone EF, Wainwright CE, Konstan MW, Moss R, Ratjen F, Sermet-Gaudelus I, Rowe SM, Dong Q, Rodriguez S, Yen K, Ordonez C, Elborn JS; VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med. 2011 Nov 3;365(18):1663-72. doi: 10.1056/NEJMoa1105185. | |
| 28651844 |
| Label | URL |
|---|---|
| Genetics Home Reference | View source |
Not provided
A total of 167 subjects were randomized; 161 subjects received at least 1 dose of the study drug. A 2-week run-in period was included to establish the baseline assessments on Day 1 after ensuring that subjects were properly taking their cystic fibrosis (CF) medication regimens.
The study started on 10 June 2009 (signing of first informed consent). After obtaining consent and assent (where applicable), screening evaluations were completed during a period of 2 to 5 weeks (Day -35 to Day -15) before the first dose of study drug.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Oral tablet every 12 hours (q12h) for up to 48 weeks. |
| FG001 | 150 mg Ivacaftor q12h | Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
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| Placebo | Drug | Tablet given orally q12h for up to 48 weeks |
|
The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID). |
| baseline through 24 weeks and 48 weeks |
| Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48 | The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity. | baseline through 24 weeks and 48 weeks |
| Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48 | Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection. | baseline through 24 weeks and 48 weeks |
| Absolute Change From Baseline in Weight at Week 24 and Week 48 | As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status. | baseline to 24 weeks and 48 weeks |
| Oakland |
| California |
| 94611 |
| United States |
| Cystic Fibrosis Research Office, Stanford University | Palo Alto | California | 94304 | United States |
| Rady Children's Hospital | San Diego | California | 92123-5070 | United States |
| National Jewish Medical and Research Center | Denver | Colorado | 80206 | United States |
| Emory Cystic Fibrosis Center | Atlanta | Georgia | 30322 | United States |
| St. Luke's CF Clinic | Boise | Idaho | 83712 | United States |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Iowa | Iowa City | Iowa | 52242 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21205 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Pulmonary, Allergy & Critical Care Medicine, University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| The Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Washington University | St Louis | Missouri | 63110 | United States |
| Adult Pulmonary/ CF, University of Nebraska Medical Center | Omaha | Nebraska | 68198-5300 | United States |
| Monmouth Medical Center | Long Branch | New Jersey | 07740 | United States |
| Women and Children's Hospital of Buffalo | Buffalo | New York | 14222 | United States |
| Long Island Jewish Medical Center | New Hyde Park | New York | 11042 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Pediatric & Pulmonary Division, Rainbow Babies/Case Western | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Toledo Children's Hospital | Toledo | Ohio | 43606 | United States |
| Oregon Health & Sciences University | Portland | Oregon | 97239-3098 | United States |
| Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| East Tennessee Children's Hospital | Knoxville | Tennessee | 37916 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232-5735 | United States |
| University of Utah | Salt Lake City | Utah | 84132 | United States |
| University of Virginia | Charlottesville | Virginia | 22908 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Division of Pulmonary and CCM, University of Washington | Seattle | Washington | 98195-6522 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| The Children's Hospital Westmead | Westmead | New South Wales | 2145 | Australia |
| The Prince Charles Hospital | Chermside | Queensland | 4032 | Australia |
| Royal Children's Hospital Brisbane | Herston | Queensland | 4026 | Australia |
| Mater Adult Hospital | South Brisbane | Queensland | 4101 | Australia |
| Royal Children's Hospital Melbourne | Parkville | Victoria | 3052 | Australia |
| Lung Institute of Western Australia | Nedlands | Western Australia | 6009 | Australia |
| Princess Margaret Hospital for Children | Subiaco | Western Australia | 6008 | Australia |
| Queen Elizabeth II Health Sciences Centre | Halifax | Nova Scotia | B3H 3A7 | Canada |
| St. Michael's Hospital | Toronto | Ontario | M5B 1W8 | Canada |
| CF Center, Hospital for Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| Montreal Children's Hospital - MUHC | Montreal | Quebec | H3H 1P3 | Canada |
| FN Motol | Prague | 15006 | Czechia |
| Hopital Cochin | Paris | 75014 | France |
| Hopital Necker | Paris | 75015 | France |
| Centre de Perharidy | Roscoff | 29684 | France |
| Kinder- und Jugendklinik Universitätsklinikum Erlangen | Erlangen | 91054 | Germany |
| Mukoviszidose-Zentrum am Klinikum der Friedrich-Schiller-Universität Jena, Klinik für Kinder- und Jugendmedizin | Jena | 07740 | Germany |
| Klinikum der LMU München, Dr. von Haunersches Kinderspital (CHA) | Munich | 80337 | Germany |
| Universitäts-Kinderklinik Würzburg | Würzburg | 97080 | Germany |
| Cork University Hospital | Cork | Ireland |
| Our Lady's Children's Hospital | Dublin | 12 | Ireland |
| The National Children's Hospital | Dublin | 24 | Ireland |
| St. Vincent's University Hospital | Dublin | 4 | Ireland |
| Beaumont Hospital | Dublin | 9 | Ireland |
| Belfast City Hospital | Belfast | Northern Ireland | BT9 7AB | United Kingdom |
| Imperial College London | London | SW3 6LR | United Kingdom |
| Derived |
| Flume PA, Wainwright CE, Elizabeth Tullis D, Rodriguez S, Niknian M, Higgins M, Davies JC, Wagener JS. Recovery of lung function following a pulmonary exacerbation in patients with cystic fibrosis and the G551D-CFTR mutation treated with ivacaftor. J Cyst Fibros. 2018 Jan;17(1):83-88. doi: 10.1016/j.jcf.2017.06.002. Epub 2017 Jun 24. |
| 27097977 | Derived | Solem CT, Vera-Llonch M, Liu S, Botteman M, Castiglione B. Impact of pulmonary exacerbations and lung function on generic health-related quality of life in patients with cystic fibrosis. Health Qual Life Outcomes. 2016 Apr 21;14:63. doi: 10.1186/s12955-016-0465-z. |
| 26135562 | Derived | Quittner A, Suthoff E, Rendas-Baum R, Bayliss MS, Sermet-Gaudelus I, Castiglione B, Vera-Llonch M. Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial. Health Qual Life Outcomes. 2015 Jul 2;13:93. doi: 10.1186/s12955-015-0293-6. |
| Medline Plus | View source |
| U.S. FDA Resources | View source |
| Completed Treatment Period, Week 24 |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Oral tablet every 12 hours (q12h) for up to 48 weeks. |
| BG001 | 150 mg Ivacaftor q12h | Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||||
| Age Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race/Ethnicity, Customized | Number | participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
| ||||||||||||||||||
| Percent Predicted Forced Expiratory Volume in 1 Second (FEV1), Continuous | Percent predicted for age, gender, and height. | Mean | Standard Deviation | percentage |
| ||||||||||||||||
| Percent Predicted FEV1, Categorical | Percent predicted for age, gender, and height. | Number | participants |
| |||||||||||||||||
| Weight | Mean | Standard Deviation | kilograms |
| |||||||||||||||||
| Body Mass Index | Mean | Standard Deviation | kilograms per square meter |
| |||||||||||||||||
| Sweat Chloride | Mean | Standard Deviation | millimoles per liter |
|
| 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 | Absolute Mean Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24 | Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame. | Posted | Least Squares Mean | Standard Error | percent of predicted volume (L) | baseline through 24 weeks |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Mean Change From Baseline in Percent Predicted FEV1 Through Week 48 | Spirometry (as measured by FEV1) is a standardized assessment to evaluate lung function that is the most widely used endpoint in cystic fibrosis studies. | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.. | Posted | Least Squares Mean | Standard Error | percent of predicted volume (L) | baseline through 48 weeks |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Score Through Week 24 and Week 48 (Respiratory Domain Score, Pooled) | The CFQ-R is a health-related quality of life measure for subjects with cystic fibrosis. Each domain is scored from 0 (worst) to 100 (best). A difference of at least 4 points in the respiratory domain score of the CFQ-R is considered a minimal clinically important difference (MCID). | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame. | Posted | Least Squares Mean | Standard Error | score on a scale | baseline through 24 weeks and 48 weeks |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Sweat Chloride Concentration Through Week 24 and Week 48 | The sweat chloride (quantitative pilocarpine iontophoresis) test is a standard diagnostic tool for cystic fibrosis (CF), serving as an indicator of cystic fibrosis transmembrane conductance regulator (CFTR) activity. | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame.. | Posted | Least Squares Mean | Standard Error | millimoles per liter | baseline through 24 weeks and 48 weeks |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time-to-first Pulmonary Exacerbation Through Week 24 and Week 48 | Pulmonary exacerbation was defined as a change in antibiotic therapy (intravenous, inhaled, or oral) for any 4 or more of signs/symptoms such as change in sputum; new or increased hemoptysis; increased cough or dyspnea; malaise, fatigue, or lethargy; temperature above 38 degrees C; anorexia or weight loss; sinus pain/tenderness and discharge; change in physical examination of the chest; decreased pulmonary function by 10%; and radiographic changes indicative of pulmonary infection. | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame. | Posted | Number | 95% Confidence Interval | proportion of event-free participants | baseline through 24 weeks and 48 weeks |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Weight at Week 24 and Week 48 | As malnutrition is common in patients with cystic fibrosis (CF) because of increased energy expenditures due to lung disease and fat malabsorption, body weight is an important clinical measure of nutritional status. | All randomized subjects who received at least 1 dose of study drug (ivacaftor or placebo) and had available assessments during the time frame. | Posted | Least Squares Mean | Standard Error | kilograms | baseline to 24 weeks and 48 weeks |
|
|
For enrolled subjects, adverse events were collected through the Follow-up Visit (4 weeks [± 7 days] after the last dose of study drug).
For subjects who were screened but were not subsequently enrolled in the study, non-serious AEs were not collected, but SAEs were reported. For subjects who completed 48 weeks of study drug treatment and enrolled in the open-label extension study, adverse events were only collected through the Week 48 Visit.
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 | Placebo | Oral tablet every 12 hours (q12h) for up to 48 weeks. | 33 | 78 | 78 | 78 | ||
| EG001 | 150 mg Ivacaftor q12h | Oral tablet of 150 mg of ivacaftor q12h for up to 48 weeks. | 20 | 83 | 82 | 83 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrioventricular block complete | Cardiac disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Cystic fibrosis lung | Congenital, familial and genetic disorders | MedDRA 12.0 | Systematic Assessment | CF exacerbations were coded as "cystic fibrosis lung." |
|
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pancreatic pseudocyst | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Catheter related complication | General disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Anaphylactic shock | Immune system disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Implant site infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Lung infection pseudomonal | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Myringitis bullous | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Hepatic enzyme increased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Cervix carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 12.0 | Systematic Assessment |
| |
| Abortion spontaneous | Pregnancy, puerperium and perinatal conditions | MedDRA 12.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
| |
| IgA nephropathy | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Testicular torsion | Reproductive system and breast disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Hemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Rales | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Paranasal sinus hypersecretion | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 12.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Blood glucose increased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Bacteria sputum identified | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Breath sounds abnormal | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 12.0 | Systematic Assessment |
| |
| Cystic fibrosis lung | Congenital, familial and genetic disorders | MedDRA 12.0 | Systematic Assessment | CF exacerbations were coded as "cystic fibrosis lung." |
|
| Headache | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Sinus headache | Nervous system disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 12.0 | Systematic Assessment |
| |
| Joint sprain | Injury, poisoning and procedural complications | MedDRA 12.0 | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 12.0 | Systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex | 617-444-6777 | medicalinfo@vrtx.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D005355 | Fibrosis |
| D010182 | Pancreatic Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D007232 | Infant, Newborn, Diseases |
| D010335 | Pathologic Processes |
| ID | Term |
|---|---|
| D004066 | Digestive System Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Not Allowed to Ask Per Local Regulations |
|
| Europe |
|
| Australia |
|
| ≥ 70% predicted FEV1 |
|
| No |
| Superiority or Other |
|
|
|
|
|
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