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| ID | Type | Description | Link |
|---|---|---|---|
| 2018-000183-28 | EudraCT Number |
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This study will evaluate the efficacy of VX-445 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-445/TEZ/IVA for 24 weeks in the TC treatment period. |
|
| VX-445/TEZ/IVA TC | Experimental | Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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-445/TEZ/IVA | Drug | Participants received VX-445/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 |
|---|---|---|---|---|---|---|
| Banner University of Arizona Medical Center | Tucson | Arizona | 85724 | United States | ||
| University of Arkansas for Medical Sciences |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37983082 | Derived | Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4. | |
| 31697873 | Derived |
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This study was conducted in participants with cystic fibrosis (CF) aged 12 years or older.
A total of 405 participants were enrolled in the study, of which 2 participants were enrolled but were not dosed in the triple combination (TC) treatment period. Results are presented for 403 participants dosed in the TC treatment period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants who received placebo matched to VX-445/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. |
| FG001 | VX-445/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 | Jul 19, 2018 | Apr 22, 2020 |
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| IVA | Drug | Participants received IVA orally once daily in the evening |
|
|
| Placebo | Drug | Participants received placebo matched VX-445/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-445, TEZ, M1-TEZ, and IVA | Pre-dose on Week 4, 8, 12, and 16 |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Miller Children's Hospital/ Long Beach Memorial | Long Beach | California | 90806 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| Valley Children's Hospital/ Children's Hospital of Central California | Madera | California | 93636 | United States |
| Kaiser Permanente | Oakland | California | 94611 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| UCSF Gateway Medical Center | San Francisco | California | 94143 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| University of Florida, Shands Hospital | Gainesville | Florida | 32610 | United States |
| Joe DiMaggio Cystic Fibrosis & Pulmonary Center/ Joe DiMaggio Children's Hospital/ Memorial Regional Hospital | Hollywood | Florida | 33021 | United States |
| Nemours Children's Specialty Care | Jacksonville | Florida | 32207 | United States |
| Central Florida Pulmonary Group | Orlando | Florida | 32803 | United States |
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States |
| Tampa General Hospital Cardiac and Lung Transplant Clinic | Tampa | Florida | 33606 | United States |
| Children's Speciality Services at North Druid Hills | Atlanta | Georgia | 30324 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| Ann & Robert Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Northwestern Memorial Hospital | Chicago | Illinois | 60611 | United States |
| Saint Francis Medical Center/ Children's Hospital of Illinois/OSF | Peoria | Illinois | 61637 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Tulane Medical Center | New Orleans | Louisiana | 70112 | United States |
| Maine Medical Partners | Portland | Maine | 04102 | United States |
| Massachusetts General Hospital Cystic Fibrosis Center | Boston | Massachusetts | 02114 | United States |
| Harper University Hospital | Detroit | Michigan | 48201 | United States |
| Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Billings Clinic | Billings | Montana | 59101 | United States |
| Nebraska Medical Center | Omaha | Nebraska | 68198 | United States |
| Morristown Medical Center | Morristown | New Jersey | 07960 | United States |
| University of New Mexico Clinical & Translational Science Center | Albuquerque | New Mexico | 87131 | United States |
| Mount Sinai Beth Israel | New York | New York | 10003 | United States |
| New York Medical College | Valhalla | New York | 10595 | United States |
| UNC Marsico Clinical Research Center | Chapel Hill | North Carolina | 27517 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| UC Health Holmes | Cincinnati | Ohio | 45220 | United States |
| University Hospitals Cleveland Medical Center/ Rainbow Babies and Children's Hospital | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | United States |
| Dayton Children's Hospital | Dayton | Ohio | 45404 | United States |
| ProMedica Toledo Hospital/ Toledo Children's Hospital/ Pediatric Pulmonary & Cystic Fibrosis Center | Toledo | Ohio | 43606 | United States |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Austin Children's Chest Associates | Austin | Texas | 78723 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| The University of Texas Health Science Center at Tyler | Tyler | Texas | 75708 | United States |
| Vermont Lung Center | Colchester | Vermont | 05446 | United States |
| University of Virginia Primary Care Center | Charlottesville | Virginia | 22908 | United States |
| Children's Hospital of the King's Daughters | Norfolk | Virginia | 23507 | United States |
| Children's Hospital of Richmond at VCU, Children's Pavilion | Richmond | Virginia | 23298 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| University of Wisconsin Hospitals and Clinics | Madison | Wisconsin | 53792 | United States |
| CTSI Adult Translational Research Unit/Medical College of Wisconsin/Froedtert Hospital | Milwaukee | Wisconsin | 53226 | United States |
| Women & Children's Hospital | North Adelaide | Australia |
| The Royal Children's Hospital | Parkville | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| The Children's Hospital at Westmead | Westmead | Australia |
| Westmead Hospital | Westmead | Australia |
| University of Graz | Graz | Austria |
| Medizinische Universitat Innsbruck | Innsbruck | Austria |
| LKH - Universitätsklinikum der PMU Salzburg | Salzburg | Austria |
| Medizinische Universitat Wien | Vienna | Austria |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| UZ Antwerpen | Edegem | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gathuisberg | Leuven | Belgium |
| British Columbia's Children's Hospital | Vancouver | British Columbia | Canada |
| St. Paul's Hospital | Vancouver | British Columbia | Canada |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Quebec | Canada |
| University of Calgary Medical Clinic of the Foothills Medical Centre | Calgary | Canada |
| Centre Hospitalier De L'Universite Laval | Qubec | Canada |
| Saint John Regional Hospital | Saint John | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| Vancouver Island Health Authority | Victoria | Canada |
| Fakultni Nemocnice Brno | Brno | Czechia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Centre Hospitalier Lyon Sud | Benite Cedex | France |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHU Marseille - Hopital Nord | Marseille | France |
| CHU de Nice - Hopital Pasteur | Nice | France |
| Hopital Cochin | Paris | France |
| CHU de Rouen - Hopital Charles Nicolle | Rouen Cedex, Seine Maritime | France |
| Hopital Foch (Suresnes), Hopital Foch, Adultes | Suresnes | France |
| Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital | Erlangen | Germany |
| Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Hannover Medical School | Hanover | Germany |
| Heidelberg Cystic Fibrosis Center | Heidelberg | Germany |
| Johannes Gutenberg-Universitaet | Mainz | Germany |
| Dr. von Haunersches Kinderspital | München | Germany |
| Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin | Tübingen | Germany |
| University Hospital Wuerzburg | Würzburg | Germany |
| General Hospital of Attika "Sismanoglio"(Adult CF center, NHS) | Marousi | Greece |
| Azienda Ospedaliero Universitaria Ospedale Riuniti | Ancona | Italy |
| Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer | Florence | Italy |
| IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | Italy |
| Azienda Ospedaliera Universitaria Policlinico G. Martino | Messina | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo | Potenza | Italy |
| Azienda Ospedaliera di Verona-Ospedale Civile Maggiore | Verona | Italy |
| Academic Medical Center | Amsterdam | Netherlands |
| University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Heidelberglaan | Netherlands |
| UMC St. Radboud | Nijmegen | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| Karolinska Univeritetssjukhuset, Huddinge | Stockholm | Sweden |
| The Royal Belfast Hospital for Sick Children | Belfast | United Kingdom |
| Heart of England NHS Foundation Trust, Birmingham Heartlands Hospital | Brimingham | United Kingdom |
| Royal Hospital for Sick Children | Edinburgh | United Kingdom |
| Western General Hospital | Edinburgh | United Kingdom |
| Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital | Exeter | United Kingdom |
| Leeds General Infirmary | Leeds | United Kingdom |
| King's College Hospital | London | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | United Kingdom |
| Southampton General Hospital | Southampton | United Kingdom |
| Middleton PG, Mall MA, Drevinek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R; VX17-445-102 Study Group. Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele. N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31. |
Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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 |
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| 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-445/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. |
| BG001 | VX-445/TEZ/IVA TC | Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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 |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| 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. | FAS. | 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 |
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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 at Week 4 |
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| 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 | z-score | 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 2 participants assigned to Placebo group who inadvertently received one or more doses of VX-445/TEZ/IVA TC regimen were included in VX-445/TEZ/IVA TC group for the purpose of safety analysis. | Posted | Count of Participants | 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-445, 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 | microgram per milliliter (mcg/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 2 participants assigned to Placebo group who inadvertently received one or more doses of VX-445/TEZ/IVA TC regimen were included in VX-445/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-445/TEZ/IVA in the morning and placebo matched to IVA in the evening for 24 weeks in the TC treatment period. | 0 | 201 | 42 | 201 | 180 | 201 |
| EG001 | VX-445/TEZ/IVA TC | Participants who received VX-445 200 mg/TEZ 100 mg/IVA150 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 | 202 | 28 | 202 | 168 | 202 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Atypical mycobacterial lower respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Coccidioidomycosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Genital herpes simplex | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Lung infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral sinusitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Diaphragmatic paralysis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Painful respiration | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pleuritic pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Hypersensitivity vasculitis | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Distal intestinal obstruction syndrome | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cholangitis | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gallbladder enlargement | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Portal hypertension | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Axonal neuropathy | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Mental impairment | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Neuroglycopenia | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Adverse drug reaction | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Medical device site inflammation | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Suicidal ideation | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
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| Acute kidney injury | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
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| Renal colic | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
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| Oral herpes | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Bacterial test positive | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 22.0 | 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 | Mar 21, 2019 | Apr 22, 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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| Not Collected per Local Regulations |
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| Australian Aboriginal |
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| Not Otherwise Specified |
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| Other- Unknown Mixed Heritage |
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| White |
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| White, Asian |
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| White, Black or African American |
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