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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-001404-42 | EudraCT Number |
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The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants 6 to less than <12 year 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 96 weeks. Doses were adjusted upward with subsequent changes in weight. 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 96 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 |
|---|---|---|
| Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Baseline up to Week 100 |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | From Parent Study Baseline to Week 96 |
| Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5) |
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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 |
|---|---|---|---|---|---|---|
| Telethon Kids Institute | Nedlands | Australia | ||||
| Queensland Children's Hospital |
| 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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A total of 120 participants from the parent study VX19-445-116 (NCT04353817) were enrolled in this study.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Participants 6 to less than <12 year 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 96 weeks. Doses were adjusted upward with subsequent changes in weight. 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 96 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 | Mar 6, 2024 |
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| IVA | Drug | Tablet for oral administration. |
|
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The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. |
| From Parent Study Baseline to Week 96 |
| South Brisbane |
| Australia |
| The Children's Hospital at Westmead | Westmead | Australia |
| 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 |
| Juliane Marie Center, Rigshospitalet | Copenhagen | Denmark |
| Groupe Hospitaler Pellegrin, CHU De Bordeaux | Bordeaux | France |
| CHU Lyon - Hopital Femme Mere-Enfant | Bron | France |
| Hopital Necker, Enfants Malades | Paris | France |
| Hopital Robert Debre | Paris | France |
| Centre de Perharidy | Roscoff | France |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie | Essen | Germany |
| Johann Wolfgang Goethe University | Frankfurt | Germany |
| Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin | Giessen | Germany |
| Medizinische Hochschule Hannover | Hanover | Germany |
| Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin | Heidelberg | Germany |
| Hadassah University Hospital Mount Scopus | Jerusalem | Israel |
| Schneider Children's Medical Center of Israel | Petach Tikvah | Israel |
| Universitair Medisch Centrum Groningen | Groningen | Netherlands |
| Erasmus Medical Center / Sophia Children's Hospital | Rotterdam | Netherlands |
| Hospital Universitari Vall d Hebron | Barcelona | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Inselspital - Universitaetsspital Bern | Bern | Switzerland |
| Kinderspital Zuerich | Zurich | Switzerland |
| University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital | Bristol | United Kingdom |
| Children's Hospital of Wales | Cardiff | United Kingdom |
| Royal Hospital for Sick Children | Edinburgh | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | United Kingdom |
| Great Ormond Street Hospital for Sick Children | London | United Kingdom |
| Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital | London | 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. |
| Placebo-ELX/TEZ/IVA | Participants from the Placebo group of the parent study VX19-445-116, who received ELX/TEZ/IVA during the current study VX20-445-119. |
|
| ELX/TEZ/IVA-ELX/TEZ/IVA | Participants from the ELX/TEZ/IVA group of the parent study VX19-445-116, who continued to receive ELX/TEZ/IVA during the current study VX20-445-119. |
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| COMPLETED |
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| NOT COMPLETED |
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Baseline data for the long-term safety analysis is based on the parent study baseline, which is defined as the most recent non-missing measurement collected before the first dose of study drug in the treatment period of parent study.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Participants 6 to <12 year of age and weighing <30 kg 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 96 weeks. Doses were adjusted upward with subsequent changes in weight. 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 96 weeks. |
| Units | Counts |
|---|---|
| Participants |
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| 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 |
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| Race/Ethnicity, Customized | Count of Participants | Participants | No |
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| 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 | Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | The Open-label Safety Set (OL-SS) included all participants who had received at least 1 dose of study drug in the Open label extension (OLE) study. | Posted | Count of Participants | Participants | No | From Baseline up to Week 100 |
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| |||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl) | Sweat samples were collected using an approved collection device. | OL Full Analysis Set (OL-FAS) included all enrolled participants who received at least 1 dose of study drug in this study. Data were planned to be presented as per parent study reporting groups (i.e. Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA). Here "Overall number of participants analyzed" signifies participants who were evaluable for this outcome at Week 96 and "Number Analyzed" signifies participants who were evaluable in the specified parent study reporting group. | Posted | Least Squares Mean | Standard Error | millimole per liter (mmol/L) | From Parent Study Baseline to Week 96 |
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| Secondary | Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5) | The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry. | OL-FAS. Data were planned to be presented as per parent study reporting groups (i.e. Placebo-ELX/TEZ/IVA and ELX/TEZ/IVA-ELX/TEZ/IVA). Here "Overall number of participants analyzed" signifies participants who were evaluable for this outcome at Week 96 and "Number Analyzed" signifies participants who were evaluable in the specified parent study reporting group. | Posted | Least Squares Mean | Standard Error | Index | From Parent Study Baseline to Week 96 |
|
Day 1 up to Week 100
The OL-SS included all participants who had received at least 1 dose of study drug in the OLE study.
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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 | ELX/TEZ/IVA | Participants 6 to <12 year of age and weighing <30 kg 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 96 weeks. Doses were adjusted upward with subsequent changes in weight. 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 96 weeks. | 0 | 120 | 13 | 120 | 118 | 120 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Enteritis | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Ileus paralytic | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Intestinal obstruction | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Steatorrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| General physical health deterioration | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Bacterial disease carrier | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Pneumonia pseudomonal | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Pneumonia staphylococcal | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA 25.1 | Systematic Assessment |
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| Blood glucose increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Diabetes mellitus inadequate control | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
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| Weight gain poor | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
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| Haematuria | Renal and urinary disorders | MedDRA 25.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ear pain | Ear and labyrinth disorders | MedDRA 25.1 | Systematic Assessment |
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| Conjunctivitis allergic | Eye disorders | MedDRA 25.1 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 25.1 | Systematic Assessment |
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| Immunisation reaction | Immune system disorders | MedDRA 25.1 | Systematic Assessment |
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| Bacterial disease carrier | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Hordeolum | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Viral upper respiratory tract infection | Infections and infestations | MedDRA 25.1 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Bacterial test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
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| SARS-CoV-2 test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Staphylococcus test positive | Investigations | MedDRA 25.1 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 25.1 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 25.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.1 | Systematic Assessment |
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| 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 22, 2023 | Mar 6, 2024 | SAP_001.pdf |
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| 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 |
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| ID | Term |
|---|---|
| C000706587 | elexacaftor, ivacaftor, tezacaftor drug combination |
| C545203 | ivacaftor |
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| Not collected per local regulations |
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| Asian |
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| American Indian or Alaska Native |
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| Other |
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| Not collected per local Regulations |
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| Multiracial |
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| Units | Counts |
|---|---|
| Participants |
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