Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
MaesTTRo
A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin (ATTR) Amyloidosis
1 other identifier
observational
1,850
7 countries
67
Brief Summary
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for all trials
Started Jun 2024
Longer than P75 for all trials
67 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 13, 2024
CompletedFirst Posted
Study publicly available on registry
June 20, 2024
CompletedStudy Start
First participant enrolled
June 25, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 29, 2031
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 29, 2031
May 26, 2026
May 1, 2026
7.5 years
May 13, 2024
May 22, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (26)
Demographic characteristics (overall and in patients initiating a treatment with eplontersen)
* Age * Sex as determined by the investigator (male/female) * Race and ethnicity, where allowed
From time of enrollment for up to 7 years
Treatment patterns (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following treatments will be assessed: * ATTR amyloidosis treatment: Tafamidis, Diflunisal, Acoramidis, Vutrisiran, Patisiran, Inotersen, Eplontersen, Doxycycline and taurodesoxycholic acid, Liver transplant * Heart failure/arrhythmia-related treatment: Diuretics, Angiotensin converting enzyme inhibitor, Angiotensin receptor blocker, Angiotensin receptor-neprilysin inhibitor, Anticoagulation, Beta-blockers, Sodium-glucose co-transporter-2 inhibitor, Mineralocorticoid receptor antagonist, Digoxin, Pacemaker use, Implantable cardioverter-defibrillator, Left ventricular assist device, Cardiac transplant, Transcatheter aortic valve replacement, Surgical aortic valve replacement * Polyneuropathy-related treatment: Antiepileptics (gabapentin, pregabalin, carbamazepine, phenytoin), Antidepressants, Topical pain treatments, Opioids, Tetrahydrocannabinol * Other: Medications for gastrointestinal symptoms, Vitamin A supplementation, Dialysis
From time of enrollment for up to 7 years
Clinical characteristics (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following clinical characteristics will be assessed: * Modified body mass index (mBMI) * Medical history * TTR genetic test results * Family history of ATTR * Time period between the first symptoms to date of diagnosis of ATTR * Time since diagnosis of ATTR
From time of enrollment for up to 7 years
Findings from biopsy (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following biopsy information will be collected: * Type of biopsy * Amyloid identification result (Positive, Negative, Inconclusive for amyloid) * Method of amyloid typing * Result of the biopsy (Normal/Abnormal) * Reason for considering the biopsy result abnormal
From time of enrollment for up to 7 years
Findings from Cardiovascular magnetic resonance imaging (CMR) (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following information will be collected: extracellular volume (ECV), contrast use, left ventricular (LV) end-diastolic volume, LV end-systolic volume, LV ejection fraction, LV Mass Index, interventricular wall thickness, right ventricular Free Wall Thickness, LV Free Wall Thickness, left Atrial Volume Index, native T1 mapping, CMR result (Normal/Abnormal), reason for considering the result abnormal.
From time of enrollment for up to 7 years
Findings from Bone tracer cardiac scintigraphy (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following information will be collected: Type of tracer, Heart to contralateral lung ratio (H/CL), Perugini grade, scintigraphy result (Normal/Abnormal), reason for considering the result abnormal.
From time of enrollment for up to 7 years
Findings from Echocardiography (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following information will be collected: LV ejection fraction, LV End Diastolic Volume, LV End Systolic Volume LV End Diastolic Dimension, LV End Systolic Dimension, Interventricular Septal Thickness End Diastole, Posterial Wall Thickness End Diastole, Left Ventricular Mass Index, Left Atrial Volume, Left Atrial Volume Index, Mitral valve regurgitation, Aortic valve regurgitation, Tricuspid valve regurgitation, Pulmonic valve regurgitation, LV Outflow Gradient, Stroke Volume, Lateral early diastolic myocardial velocity (e' lateral), Medial early diastolic myocardial velocity (e' medial), Mitral E/e' Ratio, Early diastolic mitral inflow velocity (E), Late diastolic mitral inflow velocity (A), Mitral Peak E/A Ratio, Global LV longitudinal strain, Pulmonary artery systolic pressure, RV Free Wall Thickness Severity of Aortic stenosis, Severity of Mitral stenosis.
From time of enrollment for up to 7 years
ECG variables (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following ECG information will be collected: * Interpretation of the ECG (Normal/Abnormal/Borderline) * Heart rhythm * Presence of extrasystoles * Presence of conduction abnormalities * Evidence of left ventricular hypertrophy (LVH)
From time of enrollment for up to 7 years
Sural nerve and tibial nerve amplitude (overall and in patients initiating a treatment with eplontersen)
Sural nerve and tibial nerve amplitude will be measured in overall and in patients initiating a treatment with eplontersen
From time of enrollment for up to 7 years
Biomarker results (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following biomarker results will be collected: * Serum TTR levels * Complete blood count * Hemoglobin * Troponin I * Cystatin * Creatinine * Reported glomerular filtration rate (GFR) * Albumin * Liver enzymes: alkaline phosphatase (ALP), alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma glutamyl transferase (GGT), total bilirubin * N-terminal pro B-type natriuretic peptide (NT-proBNP) * Vitamin A level * Neurofilament light chain (NfL) * International normalized ratio (INR) test
From time of enrollment for up to 7 years
Urine test results (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following test results will be collected: * Urine albumin-creatinine ratio (UACR) * Urine protein creatinine ratio (UPCR)
From time of enrollment for up to 7 years
Clinical manifestations (signs and symptoms) of ATTR amyloidosis (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following clinical manifestations will be assessed: ischemic heart disease, acute myocardial infarction, heart failure, atrial fibrillation, arrhythmias, conduction system disease, aortic valve stenosis polyneuropathy, carpal tunnel syndrome, autonomic neuropathy, nephrotic syndrome, subnephrotic proteinuria, gastrointestinal dysfunction, chronic kidney disease / acute kidney injury, spinal stenosis, spinal stenosis surgery, hepatomegaly, ascites, oedema, other amyloidosis related manifestations (e.g., Popeye's sign, tendon rupture)
From time of enrollment for up to 7 years
36-Item Short Form Health Survey Version 2 (SF-36v2) Physical Component Summary score (overall and in patients initiating a treatment with eplontersen)
The SF-36v2 is a 36-item, generic health survey that provides scores for eight health domains (physical functioning, role-physical, bodily pain, general health, vitality, social functioning, role-emotional, and mental health) and two summary scores; the physical component summary (PCS) score and the mental component summary (MCS) score. Higher scores indicate a better health state.
From time of enrollment for up to 7 years
Norfolk Quality of Life-Diabetic Neuropathy total score (overall and in patients initiating a treatment with eplontersen)
The Norfolk QOL-DN is a 35-item, disease-specific instrument that provides scores for five domains (symptoms, large fiber neuropathy, small fiber neuropathy, autonomic neuropathy, and activities of daily living) and a total score. Higher scores indicate a worse health state.
From time of enrollment for up to 7 years
Kansas City Cardiomyopathy Questionnaire (KCCQ) overall summary score (overall and in patients initiating a treatment with eplontersen)
The KCCQ is a 23-item, disease-specific questionnaire that assesses seven domains (physical limitations, symptom stability, symptom frequency, symptom burden, self-efficacy, quality of life, and social limitation) and provides three summary scores (total symptom score, clinical summary score, and overall summary score). Higher scores indicate a better health state.
From time of enrollment for up to 7 years
New York Heart Association (NYHA) classification (overall and in patients initiating a treatment with eplontersen)
I=No symptoms; II=Symptoms with ordinary physical activity; III=Symptoms with less than ordinary physical activity; IV=Symptoms at rest.
From time of enrollment for up to 7 years
National Amyloidosis Centre (NAC) ATTR staging or Mayo staging (overall and in patients initiating a treatment with eplontersen)
For NAC staging, Stage I: N-terminal pro-brain natriuretic peptide (NT-proBNP) ≤3000 ng/L and estimated glomerular filtration rate (eGFR) ≥45 ml/min; Stage III: NT-proBNP \>3000 ng/L and eGFR \<45 ml/min; Stage IV:NT-proBNP ≥10,000 ng/L; Stage II: remainder of patients For Mayo staging, Stage I: Both and biomarker values are below the established thresholds; Stage II: Either troponin T or NT-proBNP is above the threshold; Stage III: Both troponin T and NT-proBNP biomarker values are above the threshold. Biomarker Thresholds: Troponin T: \>0.05 mg/mL and NT-proBNP: \>3000 pg/mL.
From time of enrollment for up to 7 years
Familial amyloid polyneuropathy (FAP) (Coutinho) staging (overall and in patients initiating a treatment with eplontersen)
Stage 0: No symptoms; Stage I: Unimpaired ambulation; mostly mild sensory, motor and autonomic neuropathy in the lower limbs; Stage II: Assistance with ambulation required, mostly moderate impairment progression to the lower limbs, upper limbs, and trunk; Stage III: Wheelchair-bound or bedridden; severe sensory, motor, and autonomic involvement of all limbs.
From time of enrollment for up to 7 years
Polyneuropathy disability (PND) score (overall and in patients initiating a treatment with eplontersen)
Stage 0=No symptoms; Stage I=Sensory disturbances but preserved walking capabilities; Stage II=Impaired walking capacity, but ability to walk without a stick or crutches; Stage IIIA=Walking with help of 1 stick or crutch; Stage IIIB=Walking with the help of 2 sticks or crutches; Stage IV=confined to wheel chair or bedridden.
From time of enrollment for up to 7 years
Left Ventricular Ejection Fraction (overall and in patients initiating a treatment with eplontersen)
Left Ventricular Ejection Fraction will be measured in overall and in patients initiating a treatment with eplontersen
From time of enrollment for up to 7 years
6-minute walk test (overall and in patients initiating a treatment with eplontersen)
To address this objective, the distance walked in 6 minutes will be measured.
From time of enrollment for up to 7 years
Charlson comorbidity index (CCI) and CCI components (overall and in patients initiating a treatment with eplontersen)
In order to address CCI, most recent score and all CCI componenet will be measured * Myocardial infarction * Congestive heart failure * Peripheral vascular disease * Cerebrovascular disease * Dementia * Chronic pulmonary disease * Rheumatic disease * Peptic ulcer disease * Diabetes (None, Without chronic complications, With chronic complications) * Hemiplegia or paraplegia * Renal disease * Any malignancy, including lymphoma and leukemia, except malignant neoplasm of skin (None, Localized, Metastatic) * Liver disease (None, Mild, Moderate, Severe) * Metastatic solid tumor * Acquired immune deficiency syndrome (AIDS) / Human immunodeficiency virus (HIV)
From time of enrollment for up to 7 years
Other comorbidities of interest (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following information will be collected: * Percentage of patients with depression * Percentage of patients with fibromyalgia * Percentage of patients with paraproteinemia
From time of enrollment for up to 7 years
Healthcare resource utilization (overall and in patients initiating a treatment with eplontersen)
In order to address this objective, the following information will be collected: * Number of emergency department visits * Number of outpatient visits * Inpatient care/hospitalization general ward (non-intensive): number of inpatient stays, number of bed days * Inpatient care/hospitalization intensive ward: number of inpatient stays, number of bed days
From time of enrollment for up to 7 years
Mortality (overall and in patients initiating a treatment with eplontersen)
Mortality during study follow-up (all-cause, related to ATTR amyloidosis), overall and by NYHA/NAC or Mayo/FAP stage
Throughout study follow-up (up to 7 years)
Liver Disease and Live Transplant
* Liver disease (for patients with mild, moderate or severe liver disease: Child Pugh score, Child Pugh class, ascites, encephalopathy) * Liver transplantation (reason for transplant, type of transplant)
From time of Enrollment for up to 7 Years
Secondary Outcomes (20)
Comparison of demographic and clinical characteristics of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
Comparison of findings from biopsy in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
Comparison of findings from Cardiovascular magnetic resonance imaging (CMR) in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
Comparison of findings from Echocardiography in patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
Comparison of ECG variables of patients prescribed eplontersen at any time during the observation period to patients on other ATTR treatments
Up to 7 years
- +15 more secondary outcomes
Other Outcomes (3)
Risk factors for worsening ATTR progression
up to 7 years
Healthcare costs in patients with ATTR amyloidosis
Up to 7 years
Serious adverse events in patients treated with ATTR amyloidosis treatments
Up to 7 years
Study Arms (3)
ATTR cardiomyopathy (ATTR-CM)
Patients with ATTR-CM at enrollment
Hereditary polyneuropathy (ATTRv-PN)
Patients with ATTRv-PN at enrollment
ATTR-Mixed
Patients with a mixed ATTR amyloidosis phenotype
Interventions
Data will be collected on patients with ATTR amyloidosis in a real-world setting
Eligibility Criteria
Patients with a diagnosis of amyloid transthyretin (ATTR) amyloidosis
You may qualify if:
- Patient willing and able to provide written informed consent to participate in the study
- Confirmed diagnosis of amyloid transthyretin (ATTR) amyloidosis
- Aged ≥18 years at the time of signing the informed consent
- Patient willing and able to participate in collection of electronic patient reported outcomes (PROs)
You may not qualify if:
- Concurrent participation in any interventional trial for ATTR amyloidosis
- Involvement in the planning and/or conduct of the current study
- Patients with evidence of primary or light chain amyloidosis (AL) or serum protein A amyloidosis (AA)
- Asymptomatic patients with ATTR amyloidosis and asymptomatic ATTR mutation carriers
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- AstraZenecalead
- ICON plccollaborator
Study Sites (67)
Research Site
La Jolla, California, 92037, United States
Research Site
Los Angeles, California, 90095, United States
Research Site
San Francisco, California, 94025, United States
Research Site
San Francisco, California, 94143, United States
Research Site
New Haven, Connecticut, 06510, United States
Research Site
Washington D.C., District of Columbia, 20010, United States
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Chicago, Illinois, 60637, United States
Research Site
Indianapolis, Indiana, 06200, United States
Research Site
Boston, Massachusetts, 02111, United States
Research Site
Boston, Massachusetts, 02114, United States
Research Site
Boston, Massachusetts, 02115, United States
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Rochester, Minnesota, 55905, United States
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Kansas City, Missouri, 64111, United States
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St Louis, Missouri, 63110, United States
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New Brunswick, New Jersey, 08901, United States
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Manhasset, New York, 11030, United States
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New York, New York, 10027, United States
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New York, New York, 10029, United States
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Durham, North Carolina, 27710, United States
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Portland, Oregon, 97239, United States
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Danville, Pennsylvania, 17822, United States
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Philadelphia, Pennsylvania, 19104, United States
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Philadelphia, Pennsylvania, 19107, United States
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Greenville, South Carolina, 29607, United States
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Germantown, Tennessee, 38138, United States
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Nashville, Tennessee, 37214, United States
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Dallas, Texas, 75246, United States
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Richmond, Virginia, 23298, United States
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Seattle, Washington, 98915, United States
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Milwaukee, Wisconsin, 53226, United States
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Vancouver, British Columbia, V5Z 1M9, Canada
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Vancouver, British Columbia, V6Z 1Y6, Canada
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Halifax, Nova Scotia, B3RIV9, Canada
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London, Ontario, N6A 5A5, Canada
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Toronto, Ontario, M2J 4W8, Canada
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Rimouski, Quebec, G5L 5T1, Canada
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Beijing, Beijing Municipality, 100034, China
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Beijing, Beijing Municipality, 100191, China
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Beijing, Beijing Municipality, 100730, China
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Fuzhou, Fujian, 350005, China
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Guangzhou, Guangdong, 510080, China
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Guangzhou, Guangdong, 510515, China
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Changsha, Hunan, 410013, China
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Nanchang, Jiangxi, 330000, China
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Shanghai, Shanghai Municipality, 200040, China
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Hebei Sheng, Shijiazhuang, 050000, China
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Shaanxi, Xi'An, 710061, China
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Würzburg, Bavaria, DE-97072, Germany
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Frankfurt am Main, Hesse, 60590, Germany
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Hanover, Lower Saxony, 30625, Germany
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Aachen, North Rhine-Westphalia, 52074, Germany
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Cologne, North Rhine-Westphalia, 50937, Germany
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Mainz, Rhineland-Palatinate, 55131, Germany
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Homburg, Saarland, 66421, Germany
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Berlin, 10117, Germany
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Yamagata, Japan
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Huelva, Andalusia, 21005, Spain
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Cataluna, Barcelona, 08907, Spain
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Bilbao, Basque Country, 48013, Spain
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Las Palmas de Gran Canaria, Canary Islands, 35010, Spain
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Salamanca, Castille and León, 37007, Spain
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Barcelona, Catalu A, 08035, Spain
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Majadahonda, Madrid, 28222, Spain
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Islas Baleares, Palma de Mallorca, 07198, Spain
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Birmingham, West Midlands, B15 2GW, United Kingdom
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Glasgow, G51 4TF, United Kingdom
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London, NW3 2QG, United Kingdom
Related Publications (1)
Gillmore JD, Hahn K, Smith JG, Conceicao I, Tian Z, Grogan M, Pao C, Wittbrodt E, Jarbrink K, Papas MA, Davis MK. Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care. Cardiol Ther. 2025 Sep;14(3):477-490. doi: 10.1007/s40119-025-00402-y. Epub 2025 Mar 19.
PMID: 40108078DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
AstraZeneca Clinical Study Information Center
CONTACT
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 13, 2024
First Posted
June 20, 2024
Study Start
June 25, 2024
Primary Completion (Estimated)
December 29, 2031
Study Completion (Estimated)
December 29, 2031
Last Updated
May 26, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- CSR
- Time Frame
- AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
- Access Criteria
- When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Plan to share only the redacted CSR synopsis