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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003170-44 | EudraCT Number |
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This study was evaluate the effect of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) on glucose tolerance in CF participants, 12 years of age and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ELX/TEZ/IVA | Experimental | Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ELX/TEZ/IVA | Drug | Fixed dose combination (FDC) tablets for oral administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48 | Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours. | Baseline, Week 36 and 48 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48 | Baseline dysglycemia category was defined as the most recent non-missing measurement before the first dose of study drug in the treatment period. Improvement in dysglycemia is a change from cystic fibrosis-related diabetes (CFRD) at baseline to impaired glucose tolerance (IGT)/normal glucose tolerance (NGT) at Week 48 OR change from IGT at baseline to NGT at Week 48. CFRD: 2-hour post-OGTT blood glucose level ≥200 mg/dL or fasting blood glucose level ≥126 mg/dL; IGT: 2-hour post-OGTT blood glucose level ≥140 to <200 mg/dL and fasting blood glucose level <126 mg/dL; NGT: 2 hour post-OGTT blood glucose level <140 mg/dL and fasting blood glucose level <126 mg/dL. |
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Key Inclusion Criteria:
Heterozygous for F508del and an MF mutation (F/MF genotypes)
Forced expiratory volume in 1 second (FEV1) value ≥ 30% of predicted mean for age, sex, and height
Abnormal glucose tolerance determined by an OGTT as either:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Royal Adelaide Hospital | Adelaide | Australia | ||||
| The Prince Charles Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40788823 | Derived | Durieu I, Clements B, Fabrizzi B, Mall MA, McKone E, Ramsey B, Tullis E, Taylor-Cousar JL, van der Meer R, Bachman E, Chin A, Conner S, Jennings M, Weinstock T, Colombo C, Robinson P. Impact of Elexacaftor/Tezacaftor/Ivacaftor on Glucose Tolerance and Abnormal Glucose Metabolism: A Phase 3b, Open-Label Clinical Trial. Am J Respir Crit Care Med. 2025 Oct;211(10):1926-1934. doi: 10.1164/rccm.202411-2312OC. |
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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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This study was conducted in participants with cystic fibrosis (CF) aged 12 years and older who are heterozygous for the F508del mutation and a minimal function mutation (F/MF genotypes), with abnormal glucose metabolism.
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| ID | Title | Description |
|---|---|---|
| FG000 | ELX/TEZ/IVA | Participants received elexacaftor (ELX) 200 mg/tezacaftor (TEZ)100 mg ivacaftor (IVA) 150 mg in the morning and IVA 150 mg in the evening. |
| 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 26, 2021 | Jun 29, 2023 |
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| IVA | Drug | Tablets for oral administration. |
|
|
| Baseline, Week 48 |
| Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 52 |
| Chermside |
| Australia |
| Alfred Hospital | Melbourne | Australia |
| Telethon Kids Institute | Nedlands | Australia |
| The Royal Children's Hospital | Parkville, VIC | Australia |
| Sydney Children's Hospital | Randwick | 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 Gent | Ghent | Belgium |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Leuven | Belgium |
| Klinika Nemoci Plicnich a Tuberkulozy | Brno | Czechia |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Centre Hospitalier Intercommunal Creteil | Créteil | France |
| CHRU de Lille - Hopital Albert Calmette | 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 |
| Centre Hospitalier Lyon Sud | Pierre-Bénite | France |
| CHU de Rouen - Hopital Charles Nicolle | Rouen Cedex, Seine Maritime | France |
| Hopitaux Universitaires de Strasbourg | Strasbourg | France |
| Hopital Foch (Suresnes), Hopital Foch, Adultes | Suresnes | France |
| Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona | Ancona | Italy |
| IRCCS Istituto Giannina Gaslini-Ospedale Pediatrico | Genova | Italy |
| Azienda Ospedaliera Universitaria Policlinico G. Martino | Messina | Italy |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | Italy |
| Azienda Ospedaliero Universitaria Federico II Napoli | Naples | Italy |
| Azienda Ospedaliera di Verona - Ospedale Civile Maggiore | Verona | Italy |
| Academisch Medisch Centrum (Academic Medical Centre) | Amsterdam | Netherlands |
| University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis | Heidelberglaan | Netherlands |
| Erasmus Medical Center | Rotterdam | Netherlands |
| HagaZiekenhuis van den Haag | The Hague | Netherlands |
| 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 12 de Octubre | Madrid | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | Spain |
| Hospital Virgen de la Arrixaca | Murcia | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| COMPLETED |
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| NOT COMPLETED |
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All participants who received at least one dose of the study drug during the treatment period were included in the baseline analysis.
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| ID | Title | Description |
|---|---|---|
| BG000 | ELX/TEZ/IVA | Participants received ELX 200 mg /TEZ 100 mg /IVA 150 mg in the morning and IVA 150 mg in the evening. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| 2-hour Post-OGTT Blood Glucose Levels | Baseline 2-hour post-Oral Glucose Tolerance Test (OGTT) blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours. | Mean | Standard Deviation | milligrams per deciliter (mg/dl) |
|
| 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 | Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48 | Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours. | The Full Analysis Set (FAS) will include all enrolled participants who carry the intended CFTR allele mutation and have received at least 1 dose of study drug. Here, "Overall Number of Participants Analyzed" signifies participants who were evaluable for this outcome. | Posted | Least Squares Mean | 95% Confidence Interval | milligrams per deciliter (mg/dl) | Baseline, Week 36 and 48 |
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| |||||||||||||||||||||||||
| Secondary | Percentage of Participants With Improvement in Dysglycemia Categorization at Week 48 | Baseline dysglycemia category was defined as the most recent non-missing measurement before the first dose of study drug in the treatment period. Improvement in dysglycemia is a change from cystic fibrosis-related diabetes (CFRD) at baseline to impaired glucose tolerance (IGT)/normal glucose tolerance (NGT) at Week 48 OR change from IGT at baseline to NGT at Week 48. CFRD: 2-hour post-OGTT blood glucose level ≥200 mg/dL or fasting blood glucose level ≥126 mg/dL; IGT: 2-hour post-OGTT blood glucose level ≥140 to <200 mg/dL and fasting blood glucose level <126 mg/dL; NGT: 2 hour post-OGTT blood glucose level <140 mg/dL and fasting blood glucose level <126 mg/dL. | FAS. Here, "Overall Number of Participants Analyzed" signifies the number participants with non-missing dysglycemia categorization at Week 48 among participants with abnormal glucose tolerance at baseline. | Posted | Number | 95% Confidence Interval | percentage of participants | Baseline, Week 48 |
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| Secondary | 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 in the treatment period. | Posted | Count of Participants | Participants | No | Day 1 up to Week 52 |
|
|
Day 1 up to Week 52
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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 received ELX 200 mg/TEZ 100 mg/IVA 150 mg in the morning and IVA 150 mg in the evening. | 0 | 69 | 6 | 69 | 62 | 69 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastritis | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Clavicle fracture | Injury, poisoning and procedural complications | MedDRA 25.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
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| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA 25.0 | Systematic Assessment |
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| Pulmonary haemorrhage | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 25.0 | Systematic Assessment |
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| Immunisation reaction | Immune system disorders | MedDRA 25.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Rhinitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Tonsillitis | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 25.0 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Blood bilirubin increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 25.0 | Systematic Assessment |
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| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 25.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 25.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 25.0 | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 25.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 25.0 | 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 | Jul 19, 2022 | Jun 29, 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 |
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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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| Native Hawaiian or Other Pacific Islander |
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| Other |
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| Not collected per local regulations |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Participants with TEAEs |
| |||||
| Participants with SAEs |
|