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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-005320-38 | EudraCT Number |
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This study evaluated the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. |
|
| ELX/TEZ/IVA | Experimental | Participants 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed-dose combination (FDC) tablets for oral administration. |
|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 (RD) Score |
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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 |
|---|---|---|---|---|---|---|
| Medizinische Universität Innsbruck | Innsbruck | Austria | ||||
| Cliniques Universitaires de Bruxelles Hopital Erasme |
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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This study was conducted in cystic fibrosis (CF) participants aged 6 years and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. |
| FG001 | ELX/TEZ/IVA | Participants 6 to less than (<) 12 years of age and weighing <30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 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 | Apr 21, 2022 | Jul 2, 2024 |
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| IVA | Drug | Tablet for oral administration. |
|
|
| Placebo (matched to ELX/TEZ/IVA) | Other | Placebo matched to ELX/TEZ/IVA for oral administration. |
|
| Placebo (matched to IVA) | Other | Placebo matched to IVA for oral administration. |
|
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 Weight | From Baseline at Week 24 |
| 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. | From Baseline Through Week 24 |
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 28 |
| Brussels |
| Belgium |
| Universitair Ziekenhuis Brussel - Campus Jette | Brussels | Belgium |
| Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital | Edegem | Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| Cliniques Universitaires Saint-Luc | Woluwe-Saint-Lambert | Belgium |
| Stollery Children's Hospital | Edmonton | Canada |
| Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu | Montreal | Canada |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| St. Paul's Hospital | Vancouver | Canada |
| Klinika Detskych Infekcnich Nemoci | Brno | Czechia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| CHU Lyon - Hopital Femme Mere-Enfant | Bron | France |
| Centre Hospitalier Intercommunal Creteil | Créteil | France |
| Institut Cœur Poumon, CHU de Lille | Lille | France |
| CHU Marseille - Hopital Nord | Marseille | France |
| Hopital Arnaud de Villeneuve | Montpellier | France |
| Centre Hospitalier Universitaire De Nantes - G. R. Laennec | Nantes | France |
| Centre Hospitalier Universitaire (CHU) de Nice - Hopital Pasteur | Nice | France |
| Hopital Cochin | Paris | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Hopital Robert Debre | Paris | France |
| Hopital Haut-Leveque - CRCM Adulte | Pessac | France |
| Centre Hospitalier Lyon Sud | Pierre-Bénite | France |
| Centre Hospitalier Universitaire De Reims, Hopital Sebastopol | Reims | France |
| CHU de Rennes - Hôpital Sud | Rennes | France |
| Centre de Perharidy | Roscoff | France |
| CHU de Toulouse - Hopital Larre | Toulouse | France |
| Hopital Bretonneau | Tours | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Friedrich-Alexander University of Erlangen-Nuremberg, University Children's Hospital | Erlangen | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Johann Wolfgang Goethe University | Frankfurt | Germany |
| Justus-Liebig-Universität Gießen Zentrum für Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Universitätsklinikum Halle (Saale) / Universitätsklinik und Poliklinik für Innere Medizin, Schwerpunkt Pneumologie | Halle | Germany |
| Hannover Medical School | Hanover | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Universitaetsklinikum Jena, Mukoviszidose-Zentrum | Jena | Germany |
| Johannes Gutenberg-Universitaet | Mainz | Germany |
| Dr. von Haunersches Kinderspital | München | Germany |
| Klinikum Innenstadt, University of Munich | München | Germany |
| Pneumologisches Studienzentrum Muenchen-West | München | Germany |
| Klinik fur Kinder- und Jugendmedizin, Universitatsklinikum Munster | Münster | Germany |
| Klinikum Westbrandenburg (CF) | Potsdam | Germany |
| Universitätsklinikum Tübingen Klinik für Kinder- und Jugendmedizin | Tübingen | Germany |
| Universitätsklinikum Ulm, Klinik für Kinder- und Jugendmedizin (CF) | Ulm | Germany |
| Universitätsklinikum Würzburg | Würzburg | Germany |
| National Koranyi Institute for TBC and Pulmonology | Budapest | Hungary |
| Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona | 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 Ospedale Maggiore Policlinico, Mangiagalli e Regina Elena | Milan | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Azienda Ospedaliera Universitaria Federico II (Adults) | Naples | Italy |
| Malattie Apparato Respiratorio 2 - Centro Fibrosi Cistica | Orbassano | Italy |
| Centro Regionale Fibrosi Cistica, A.O. Ospedale San Carlo | Potenza | Italy |
| Ospedale Pediatrico Bambino Gesu | Rome | Italy |
| Azienda Ospedaliera di Verona - Ospedale Civile Maggiore | Verona | Italy |
| Academisch Medisch Centrum (Academic Medical Centre) | Amsterdam | Netherlands |
| UMC St. Radboud | Nijmegen | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| Universitair Medisch Centrum Utrecht | Utrecht | Netherlands |
| Oslo University Hospital, Department of Paediatric Medicine | Oslo | Norway |
| Pediatric Hospital Polanki named of Maciej Płażyński | Gdansk | Poland |
| Institute of Tuberculosis and Lung Diseases | Rabka-Zdrój | Poland |
| Instytut Matki i Dziecka, Klinika Mukowiscydozy IMiD, Oddział Chorób Płuc SZPZOZ im.Dzieci Warszawy w Dziekanowie Leśnym | Łomianki | Poland |
| Hospital de Santa Maria | Lisbon | Portugal |
| Hospital Sao Joao | Porto | Portugal |
| Hospital de Cruces | Barakaldo | Spain |
| Hospital Saint Joan de Deu | Barcelona | Spain |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Universitari Vall d´Hebron Servicio de Broncoscopia | Barcelona | Spain |
| Hospital Universitario de Jerez de la Frontera | Jerez de la Frontera | Spain |
| Hospital Infantil Universitario Nino Jesus | Madrid | Spain |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Infantil La Paz | Madrid | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Corporacio Sanitaria Parc Tauli - Sabadell Hospital Universitari | Sabadell | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Hospital Universitario y Politecnico La Fe | Valencia | Spain |
| Sahlgrenska Universitetssjukhuset | Gothenburg | Sweden |
| Karolinska Universitetssjukhuset, Huddinge | Stockholm | Sweden |
| Kinderspital Zuerich | Zurich | Switzerland |
| COMPLETED |
|
| NOT COMPLETED |
|
|
All participants who received at least one dose of the study drug were included in the baseline analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. |
| BG001 | ELX/TEZ/IVA | Participants 6 to <12 years of age and weighing <30kg at Day 1 received ELX 100 mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, 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 |
| |||||||||||||||
| 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. | Mean | Standard Deviation | percent predicted FEV1 |
|
| 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. | The Full Analysis Set (FAS) will include all randomized participants who carry the intended mutation and received at least 1 dose of study drug. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | percent predicted FEV1 | From Baseline Through Week 24 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | millimole per liter (mmol/L) | From Baseline Through Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) 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. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | units on a scale | From Baseline Through Week 24 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | kg/m^2 | From Baseline at Week 24 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in Weight | FAS. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | kilogram (kg) | From Baseline at Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| 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. Here "Overall Number of Participants Analyzed" signifies those participants who were evaluated for this outcome measure. | Posted | Number | PEx events | From Baseline Through Week 24 |
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | Safety Set was defined as all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | Day 1 up to Week 28 |
|
|
Day 1 up to Week 28
Safety Set was defined as all participants who received at least 1 dose of study drug.
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 received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. | 0 | 102 | 15 | 102 | 86 | 102 |
| EG001 | ELX/TEZ/IVA | Participants 6 to <12 years of age and weighing <30 kg at Day 1 received ELX 100mg/TEZ 50 mg /IVA 75 mg as FDC tablets in the morning and IVA as mono tablet in the evening and those weighing ≥30 kg at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 24 weeks. | 1 | 205 | 18 | 205 | 164 | 205 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Stress cardiomyopathy | Cardiac disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pancreatitis | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Subileus | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Drug intolerance | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cholecystitis acute | Hepatobiliary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Bronchopulmonary aspergillosis allergic | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Hepatitis viral | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Vestibular neuronitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Polyarthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Lung adenocarcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 26.0 | Systematic Assessment |
| |
| Anxiety disorder | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Post-traumatic stress disorder | Psychiatric disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 26.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 26.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 26.0 | 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 | Jun 8, 2023 | Jul 2, 2024 | 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 |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
Not provided
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Not Collected per Local Regulations |
|
| Asian |
|
| Other |
|
| Not Collected per Local Regulations |
|
| White, Asian |
|
|
|
| Units | Counts |
|---|---|
| Participants |
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