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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-003455-11 | EudraCT Number |
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This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 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 Day 1 up to Week 52 | |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 86 |
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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 |
|---|---|---|---|---|---|---|
| The Prince Charles Hospital | Chermside | Australia | ||||
| Institute for Respiratory Health |
| 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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The study was conducted in 2 parts, Part A and Part B. Participants from Part A also participated in Part B. The Participant flow was planned to be presented for the overall treatment arm.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part A (48 Weeks) |
|
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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 | Dec 21, 2021 | Jun 7, 2023 |
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| IVA | Drug | Tablet for oral administration. |
|
|
| Nedlands |
| Australia |
| Telethon Kids Institute, Perth Children's Hospital | Nedlands | Australia |
| The Royal Children's Hospital | Parkville, VIC | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| Universitair Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Universitaetsklinkum Koeln, CF-Studienzentrum | Cologne | Germany |
| Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen | Essen | Germany |
| Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie | Essen | Germany |
| Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin | Jena | Germany |
| Klinikum Innenstadt, University of Munich | München | Germany |
| Belfast City Hospital | Belfast | United Kingdom |
| University Hospitals Birmingham NHS Foundation Trust | Birmingham | United Kingdom |
| University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital | Bristol | United Kingdom |
| Royal Papworth Hospital NHS Foundation Trust | Cambridge | United Kingdom |
| Western General Hospital | Edinburgh | 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 |
| Leeds General Infirmary | Leeds | United Kingdom |
| St. James University Hospital | Leeds | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | United Kingdom |
| Great Ormond Street Hospital for Sick Children | London | United Kingdom |
| London and St Bartholomew's Hospital | London | United Kingdom |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary | Newcastle upon Tyne | United Kingdom |
| Nottingham University Hospitals NHS Trust, Queens Medical Center | Nottingham | United Kingdom |
| All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough | Penarth | 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 |
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| NOT COMPLETED |
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| Part B (86 Weeks) |
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Baseline data is based on the parent study baseline, which is defined as the most recent non-missing measurements collected before the first dose of the study drug in the treatment period of parent studies. Baseline data is presented for participants who received at least 1 dose of study drug in this study.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Part A: Participants received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 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 | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Mean | Standard Deviation | years |
| ||||||||||||||||
| Sex: Female, Male | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
| ||||||||||||||||
| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
| ||||||||||||||||
| Race/Ethnicity, Customized | The Baseline data were planned to be presented separately for Part A and Part B. Here "Number Analyzed" signifies participants who were evaluable for the specified part of the study. | Count of Participants | Participants | No |
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| 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) | Safety set included all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | From Day 1 up to Week 52 |
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| Primary | Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Safety Set included all participants who received at least 1 dose of study drug. | Posted | Count of Participants | Participants | From Day 1 up to Week 86 |
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Day 1 up to Week 52 for Part A, Day 1 up to Week 86 for Part B
MedDRA 24.0 for Part A and MedDRA 25.1 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 qd in the treatment period for 48 weeks. | 0 | 172 | 26 | 172 | 127 | 172 |
| EG001 | Part B: ELX/TEZ/IVA | Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg qd in the treatment period up to 86 weeks. | 0 | 50 | 8 | 50 | 47 | 50 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Atrial fibrillation | Cardiac disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Myocardial infarction | Cardiac disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Sinus arrest | Cardiac disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Ileus | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Vascular device occlusion | General disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Cholecystitis | Hepatobiliary disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Chronic sinusitis | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Pneumonia pseudomonal | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Limb traumatic amputation | Injury, poisoning and procedural complications | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Rib fracture | Injury, poisoning and procedural complications | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Blood bilirubin increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Forced expiratory volume decreased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Gamma-glutamyltransferase increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Electrolyte imbalance | Metabolism and nutrition disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Dupuytren's contracture | Musculoskeletal and connective tissue disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Loss of consciousness | Nervous system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Aggression | Psychiatric disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Dissociative disorder | Psychiatric disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Nephrolithiasis | Renal and urinary disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Uterine polyp | Reproductive system and breast disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Erythema nodosum | Skin and subcutaneous tissue disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Immunisation reaction | Immune system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Vaccination complication | Injury, poisoning and procedural complications | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Blood bilirubin increased | Investigations | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0, 25.1 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0, 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 | Jan 18, 2022 | Jun 7, 2023 | 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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| Male |
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| Part B |
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| Not Hispanic or Latino |
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| Not collected per local regulations |
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| Part B |
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| Black or African American |
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| Asian |
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| American Indian or Alaska Native |
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| Native Hawaiian or Other Pacific Islander |
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| White, Asian |
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| Not collected per local regulations |
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| Part B |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Participants with TEAEs |
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| Participants with SAEs |
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