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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004132-11 | EudraCT Number |
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This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants who received placebo matched to VX-659/TEZ/IVA for 24 weeks in the TC treatment period. |
|
| VX-659/TEZ/IVA TC | Experimental | Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-659/TEZ/IVA | Drug | Participants received VX-659/TEZ/IVA orally once daily in the morning. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline at Week 4 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | From Baseline through Week 24 |
| Number of Pulmonary Exacerbations (PEx) |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35233 | United States | ||
| Stanford University |
This study was conducted in participants with cystic fibrosis (CF) aged 12 years or older.
A total of 385 participants were enrolled in the study, of which 3 participants were enrolled but were not dosed in the TC treatment period. Results are presented for 382 participants dosed in the TC treatment period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants who received placebo matched to VX-659/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. |
| FG001 | VX-659/TEZ/IVA TC |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 27, 2018 | Feb 5, 2020 |
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| IVA | Drug | Participants received IVA orally once daily in the evening. |
|
|
| Placebo | Drug | Participants received placebo matched VX-659/TEZ/IVA orally once daily in the morning and placebo matched to IVA orally once daily in the evening. |
|
Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. |
| From Baseline through Week 24 |
| Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Baseline through Week 24 |
| Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | From Baseline through Week 24 |
| Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). | From Baseline at Week 24 |
| Absolute Change in Sweat Chloride | Sweat samples were collected using an approved collection device. | From Baseline at Week 4 |
| Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | From Baseline at Week 4 |
| Time-to-first Pulmonary Exacerbation (PEx) | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | From Baseline through Week 24 |
| Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline | BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. | From Baseline at Week 24 |
| Absolute Change in Body Weight | From Baseline at Week 24 |
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks) |
| Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA | Pre-dose on Week 4, 8, 12, and 16 |
| Palo Alto |
| California |
| 94304 |
| United States |
| Children's Hospital Colorado | Aurora | Colorado | 80045 | United States |
| Hartford Health | Hartford | Connecticut | 06106 | United States |
| Yale New Haven Medical Center | New Haven | Connecticut | 06511 | United States |
| University of Miami/ Miller School of Medicine | Miami | Florida | 33136 | United States |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | United States |
| Arnold Palmer Hospital | Orlando | Florida | 32806 | United States |
| Johns Hopkins All Children's Hospital Outpatient Care Center | St. Petersburg | Florida | 33701 | United States |
| St. Luke's CF Center of Idaho | Boise | Idaho | 83712 | United States |
| Cystic Fibrosis Center of Chicago | Glenview | Illinois | 60025 | United States |
| Advocate Children's Hospital - Park Ridge/ North Suburban Pulmonary and Critical Care Consultants | Niles | Illinois | 60714 | United States |
| Indiana Clinical Research Center, IU Health University Hospital | Indianapolis | Indiana | 46202 | United States |
| The University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| Kentucky Clinic | Lexington | Kentucky | 40536 | United States |
| Kosair Charities Pediatric Clinical Research Unit | Louisville | Kentucky | 40202 | United States |
| The Johns Hopkins Hospital | Baltimore | Maryland | 21287 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| University of Massachusetts Memorial Medical Center | Worcester | Massachusetts | 01655 | United States |
| University of Michigan Health System | Ann Arbor | Michigan | 48109 | United States |
| Helen DeVos Children's Hospital CF Center | Grand Rapids | Michigan | 49503 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Children's Mercy Hospital | Kansas City | Missouri | 64108 | United States |
| Washington University School of Medicine/ St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Dartmouth Hitchcock Medical Center, Lebanon | Lebanon | New Hampshire | 03756 | United States |
| Rutgers-Robert Wood Johnson Medical School | New Brunswick | New Jersey | 80901 | United States |
| Albany Medical College | Albany | New York | 12208 | United States |
| Lung and Cystic Fibrosis Center at Women and Children's Hospital of Buffalo | Buffalo | New York | 14222 | United States |
| Northwell Health, Long Island Jewish Medical Center | New Hyde Park | New York | 11040 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| SUNY Upstate Medical University | Syracuse | New York | 13210 | United States |
| Clinical Research of Charlotte | Charlotte | North Carolina | 28277 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Respiratory Diseases of Children and Adolescents | Oklahoma City | Oklahoma | 73112 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center | Philadelphia | Pennsylvania | 19017 | United States |
| Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15224 | United States |
| Sanford Research/ USD | Sioux Falls | South Dakota | 57105 | United States |
| University of Tennessee Medical Center - Adult Cystic Fibrosis Clinic | Knoxville | Tennessee | 37920 | United States |
| Children's Foundation Research Center/ Le Bonheur Children's Hospital | Memphis | Tennessee | 38103 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37920 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| University of Utah / Primary Children's Medical Center | Salt Lake City | Utah | 84132 | United States |
| Seattle Children's Hospital | Seattle | Washington | 98105 | United States |
| Providence Pediatric Pulmonary & Allergy/Immunology Clinic | Spokane | Washington | 99204 | United States |
| The Alfred Hospital | Melbourne | Victoria | Australia |
| Royal Adelaide Hospital | Adelaide | Australia |
| Prince Charles Hospital | Chermside | Australia |
| Royal Brisbane & Women's Hospital | Herston | Australia |
| Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital | Nedlands | Australia |
| John Hunter Hospital & Hunter Medical Research Institute | New Lambton Heights | Australia |
| Sydney Children's Hospital, Randwick | Randwick | Australia |
| Princess Margaret Hospital for Children | Subiaco | Australia |
| Stollery Children's Hospital | Edmonton | Alberta | Canada |
| Queen Elizabeth II Health Sciences Center | Halifax | Nova Scotia | Canada |
| St. Michael's Hospital | Toronto | Canada |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| University Hospital Cologne | Cologne | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Clinic of J.W Goethe University | Frankfurt | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder - und Jugendmedizin | Jena | Germany |
| Universitatsklinikum Schleswig-Holstein, Klinik fur Kinder- und Jugendmedizin | Lübeck | Germany |
| Klinikum Innenstadt, University of Munich | München | Germany |
| Pneumologische Praxis Pasing | München | Germany |
| Beaumont Hospital | Dublin | Ireland |
| Children's University Hospital Temple Street | Dublin | Ireland |
| Cork University Hospital | Dublin | Ireland |
| Our Lady's Children's Hospital | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| National University of Ireland | Galway | Ireland |
| University Hospital Limerick | Limerick | Ireland |
| Lady Davis Carmel Medical Center | Haifa | Israel |
| Pediatric Pulmonary Unit Rambam Medical Center | Haifa | Israel |
| Hadassah Medical Organization | Jerusalem | Israel |
| Schneider Children's Medical Center | Petah Tikva | Israel |
| Sheba Medical Center | Tel Litwinsky | Israel |
| Instytut Matki i Dziecka | Warsaw | Poland |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Universitari Vall d'Hebron Servicio de Broncoscopia | Barcelona | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Infantil La Paz | Madrid | Spain |
| Coporacio Sanitaria Parc Tauli | Sabadell | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Hospital Universitario y Politecnico La Fe | Valencia | Spain |
| Lindenhofspital - Quartier Bleu | Bern | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| Universitaetsspital Zuerich | Zurich | Switzerland |
| Papworth Hospital NHS Foundation Trust, Papworth Everard | Cambridge | United Kingdom |
| Queen Elizabeth University Hospital | Glasgow | United Kingdom |
| The Leeds Teaching Hospitals NHS Trust, St. James University Hospital | Leeds | United Kingdom |
| Liverpool Heart and Chest Hospital | Liverpool | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | United Kingdom |
| Wythenshawe Hospital | Manchester | United Kingdom |
| The Newcastle Upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |
| Nottingham University Hospitals NHS Trust, Queens Medical Center | Nottingham | United Kingdom |
| University Hospital Llandough | Penarth | United Kingdom |
Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as fixed-dose combination (FDC) tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period.
| Safety Set | Based on actual treatment received. |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
All randomized participants who carried the intended CF transmembrane conductance regulator gene (CFTR) allele mutation and received at least 1 dose of study drug in the TC Treatment Period.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants who received placebo matched to VX-659/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. |
| BG001 | VX-659/TEZ/IVA TC | Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period. |
| 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, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race | Count of Participants | Participants |
| ||||||||||||||||
| Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Mean | Standard Deviation | percentage points |
|
| 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 Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Analysis population included all participants in the Full Analysis Set (all randomized participants who carried the intended CFTR allele mutation and received at least 1 dose of study drug) who completed the Week 4 Visit or were randomized at least 28 days before the data cutoff date. | Posted | Least Squares Mean | Standard Error | percentage points | From Baseline at Week 4 |
|
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| Secondary | Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) | FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. | Full analysis set (FAS) included all randomized participants who carried the intended CFTR allele mutation and received at least 1 dose of study drug in the TC Treatment Period. | Posted | Least Squares Mean | Standard Error | percentage points | From Baseline through Week 24 |
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| Secondary | Number of Pulmonary Exacerbations (PEx) | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | FAS. | Posted | Number | pulmonary exacerbation events | From Baseline through Week 24 |
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| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | FAS. | Posted | Least Squares Mean | Standard Error | millimole per liter (mmol/L) | From Baseline through Week 24 |
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| Secondary | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | FAS. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline through Week 24 |
|
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| Secondary | Absolute Change in Body Mass Index (BMI) | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). | FAS. | Posted | Least Squares Mean | Standard Error | kilogram per meter square (kg/m^2) | From Baseline at Week 24 |
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| Secondary | Absolute Change in Sweat Chloride | Sweat samples were collected using an approved collection device. | FAS. | Posted | Least Squares Mean | Standard Error | mmol/L | From Baseline at Week 4 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire Revised (CFQ-R) Respiratory Domain Score | 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, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. | FAS. | Posted | Least Squares Mean | Standard Error | units on a scale | From Baseline at Week 4 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Time-to-first Pulmonary Exacerbation (PEx) | Pulmonary exacerbation was defined as the treatment with new or changed antibiotic therapy (intravenous, inhaled, or oral) for greater than or equal to 4 sinopulmonary signs/symptoms. | FAS. | Posted | Median | 95% Confidence Interval | days | From Baseline through Week 24 |
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| Secondary | Absolute Change in BMI Z-score for Participants <=20 Years of Age at Baseline | BMI was defined as weight in kg divided by height in m^2. z-score is a statistical measure to describe whether a mean was above or below the standard. BMI, adjusted for age and sex, was analyzed as BMI-for-age z-score. A z-score of 0 is equal to the mean and is considered normal. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Higher values are indicative of higher BMI. | FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were <=20 years of age at Baseline. | Posted | Least Squares Mean | Standard Error | kg/m^2 | From Baseline at Week 24 |
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| Secondary | Absolute Change in Body Weight | FAS. | Posted | Least Squares Mean | Standard Error | kg | From Baseline at Week 24 |
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| Secondary | Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | Adverse events are presented as per Safety Set. Group assignments for participants in the Safety Set were based on actual treatment received, such that 1 participant assigned to Placebo group who inadvertently received one or more doses of VX-659/TEZ/IVA TC regimen was included in VX-659/TEZ/IVA TC group for the purpose of safety analysis. | Posted | Number | participants | From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks) |
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| Secondary | Observed Pre-dose Concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA | Pharmacokinetic (PK) set included all randomized participants who carried the intended CFTR allele mutation and received at least 1 dose of study drug in the TC Treatment Period. Here "Number Analyzed" signifies those participants who were evaluable for this outcome measure at specified time points. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Pre-dose on Week 4, 8, 12, and 16 |
|
|
From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 28 weeks)
Adverse events are presented as per Safety Set. Group assignments for participants in the Safety Set were based on actual treatment received, such that 1 participant assigned to Placebo group who inadvertently received one or more doses of VX-659/TEZ/IVA TC regimen was included in VX-659/TEZ/IVA TC group for the purpose of safety analysis.
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 | Participants who received placebo matched to VX-659/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. | 0 | 189 | 58 | 189 | 166 | 189 |
| EG001 | VX-659/TEZ/IVA TC | Participants who received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 24 weeks in the TC treatment period. | 0 | 193 | 11 | 193 | 141 | 193 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Intentional self-injury | Psychiatric disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Intentional overdose | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Pneumothorax traumatic | Injury, poisoning and procedural complications | MedDRA 21.1 | Systematic Assessment |
| |
| Forced expiratory volume decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Pericardial effusion | Cardiac disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Cystic fibrosis related diabetes | Congenital, familial and genetic disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Right-to-left cardiac shunt | Congenital, familial and genetic disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Bile duct stone | Hepatobiliary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Device damage | Product Issues | MedDRA 21.1 | Systematic Assessment |
| |
| Axillary mass | Musculoskeletal and connective tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Lower respiratory tract infection bacterial | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Dysentery | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Respiratory tract infection bacterial | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Vascular device infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Alanine aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 21.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Respiration abnormal | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 21.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 21.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 21.1 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Monitor | Vertex Pharmaceuticals Incorporated | 617-341-6777 | medicalinfo@vrtx.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 22, 2019 | Feb 5, 2020 | SAP_001.pdf |
Not provided
| 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
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Black or African American |
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| Both White and Black/African American |
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