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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-000833-37 | EudraCT Number |
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This study evaluated the long-term safety, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation and a gating or residual function mutation (F/G and F/RF genotypes).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed-dose combination (FDC) tablet for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 100 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Absolute Change From Parent Study Baseline 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 96 |
| Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) |
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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 | ||
| Miller Children's Hospital / Long Beach Memorial |
| 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. | |
| 33331662 | Derived |
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Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.
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Participants from parent study VX18-445-104 (NCT04058353) were enrolled in this study. A total of 251 participants were enrolled in this study.
The study was conducted as a single part study (Part A) in countries including United States (US), and the protocol for other countries was amended as a regional protocol so that participants in these countries had the opportunity to participate for up to an additional 48 weeks in Part B.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Part A: Participants received ELX (elexacaftor) 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. |
| 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 29, 2020 | Dec 13, 2023 |
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| IVA | Drug | Tablet for oral administration. |
|
|
Sweat samples were collected using an approved collection device. |
| From Baseline at Week 96 |
| Part A: Absolute Change From Parent Study Baseline 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 96 |
| Part A: Absolute Change From Parent Study Baseline in BMI Z-score | BMI was defined as weight in kilogram (kg) divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | From Baseline at Week 96 |
| Part A: Absolute Change From Parent Study Baseline in Body Weight | From Baseline at Week 96 |
| Part A: Absolute Change From Parent Study Baseline 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 96 |
| Long Beach |
| California |
| 90806 |
| United States |
| Stanford University | Palo Alto | California | 94304 | United States |
| University of California Davis Medical Center | Sacramento | California | 95817 | United States |
| University of California San Francisco, Lung Transplant Program | San Francisco | California | 94143 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| University of Miami Miller School of Medicine | Miami | Florida | 33136 | United States |
| Central Florida Pulmonary Group, PA | Orlando | Florida | 32803 | United States |
| Indiana University | Indianapolis | Indiana | 46202 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University of Kentucky | Lexington | Kentucky | 40536 | United States |
| Massachusetts General Hospital Cystic Fibrosis Center Clinical Rsearch Center | Boston | Massachusetts | 02114 | United States |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | United States |
| Michigan Medicine | Ann Arbor | Michigan | 48109-5212 | United States |
| University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Washington University School of Medicine / St. Louis Children's Hospital | St Louis | Missouri | 63110 | United States |
| Mount Sinai Beth Israel | New York | New York | 10003 | United States |
| Columbia University Medical Center | New York | New York | 10032 | United States |
| University of North Carolina Hospitals | Chapel Hill | North Carolina | 27514 | United States |
| UC Health Holmes | Cincinnati | Ohio | 45220 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | 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 |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Children's Hospital of Pittsburgh of UPMC | Pittsburgh | Pennsylvania | 15224 | United States |
| Dell Children's Medical Group | Austin | Texas | 78723 | United States |
| The University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Texas Children's Hospital | Houston | Texas | 77030 | United States |
| University of Utah / Primary Children's Medical Center | Salt Lake City | Utah | 84132 | United States |
| University of Washington Medical Center | Seattle | Washington | 98195 | United States |
| University of Wisconsin Hospital and Clinics | Madison | Wisconsin | 53792 | United States |
| The Prince Charles Hospital | Chermside | Australia |
| Alfred Hospital | Melbourne | Australia |
| Telethon Kids Institute | Nedlands | Australia |
| The Royal Children's Hospital | Parkville | Australia |
| Mater Adult Hospital | South Brisbane | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| Westmead Hospital | Westmead | Australia |
| Cliniques Universitaires de Bruxelles Hopital Erasme | Brussels | Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| McGill University Health Center | Montreal | Canada |
| St. Paul's Hospital | Vancouver | Canada |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Centre Hospitalier Lyon Sud | Benite Cedex | France |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHRU de Lille - Hopital Albert Calmette | Lille | France |
| CHU Marseille - Hopital Nord | Marseille | France |
| CHU de Montpellier - Hopital Arnaud de Villeneuve | Montpellier | France |
| Hopital Cochin | Paris | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Hopital Pontchaillou CHU de Rennes | Rennes | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Justus-Liebig-Universitaet Gießen Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie | Halle | Germany |
| Pneumologisches Studienzentrum Muenchen-West | München | Germany |
| University Hospital Wuerzburg | Würzburg | Germany |
| Beaumont Hospital | Dublin | Ireland |
| Children's Health Ireland at Crumlin | Dublin | Ireland |
| St. Vincent's University Hospital | Dublin | Ireland |
| University Hospital Limerick (Adults) | Limerick | Ireland |
| Azienda Ospedaliero Universitaria Ospedale Riuniti | Ancona | Italy |
| IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Ospedale Pediatrico Bambino Gesu | Rome | 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 |
| Hospital Universitari Vall d´Hebron Servicio de Broncoscopia | Barcelona | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Hospital Universitario y Politecnico La Fe | Valencia | Spain |
| Papworth Hospital NHS Foundation Trust, Papworth Everard | Cambridge | United Kingdom |
| Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital | Exeter | United Kingdom |
| Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | United Kingdom |
| 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 |
| Southampton General Hospital | Southampton | United Kingdom |
| 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. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
The open label Full Analysis Set (OL-FAS) is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Part A: Participants received ELX 200mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. |
| 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 | No |
| ||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
| ||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants | No |
|
| 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 | Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | The open label (OL) Safety Set for Part A was defined as all participants who received at least 1 dose of study drug in the OLS Part A (participants were assigned based on parent study actual treatment). | Posted | Count of Participants | Participants | No | From Baseline up to Week 100 |
|
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline 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. | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | percentage points | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here, "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline in Body Mass Index (BMI) | BMI was defined as weight in kilogram (kg) divided by height in square meter (m^2). | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here, "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline in BMI Z-score | BMI was defined as weight in kilogram (kg) divided by squared height in meters (m^2). The z-score is a statistical measure to describe whether a value was above or below the standard. A z-score of 0 is equal to the standard. Lower numbers indicate values lower than the standard and higher numbers indicate values higher than the standard. | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here, "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | z-score | From Baseline at Week 96 |
|
| |||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline in Body Weight | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled subjects who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here, "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | Kilogram (Kg) | From Baseline at Week 96 |
|
| ||||||||||||||||||||||||||||||||||
| Secondary | Part A: Absolute Change From Parent Study Baseline 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. | The OL Full Analysis Set (OL-FAS) for Part A is defined as all enrolled participants who have received at least 1 dose of study drug in the open-label study. This analysis set included study 104 parent study participants who received Control IVA or TEZ/IVA and ELX/TEZ/IVA. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome. Here, "Number Analyzed" signifies participants who were evaluable for the specified category. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Baseline at Week 96 |
|
|
Day 1 through Safety follow-up (up to Week 100 for Part A and up to Week 52 for Part B)
The study was conducted as a single part study (Part A) in countries including United States (US) and the protocol for other countries was amended so that participants in these countries had the opportunity to participate for up to an additional 48 weeks in Part B. The adverse events data has been reported for both the parts separately. MedDRA 24.1 applied for Part A and MedDRA 25.1 applied for Part B.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Part A: ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 96 weeks. | 1 | 251 | 38 | 251 | 223 | 251 |
| EG001 | Part B: ELX/TEZ/IVA | Part B: Participants from certain countries participated in Part B and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. | 0 | 84 | 3 | 84 | 48 | 84 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Vision blurred | Eye disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Aspergilloma | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Tendon rupture | Injury, poisoning and procedural complications | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| C-reactive protein increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Colon cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Intraductal proliferative breast lesion | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Epididymal cyst | Reproductive system and breast disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Pneumothorax spontaneous | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Rash erythematous | Skin and subcutaneous tissue disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.1,25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Vaccination complication | Injury, poisoning and procedural complications | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Blood creatine phosphokinase increased | Investigations | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 24.1,25.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.1,25.1 | 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 2, 2022 | Dec 14, 2023 | SAP_001.pdf |
| 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 |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Not collected per local regulations |
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| Other |
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| Not collected per local regulations |
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| More than one race |
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