Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (LICA2025)
10 августа 2026 г. обновлено: Gianluca Di Bella, University of Messina
Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (ATTR-CA): New Horizons in Disease Staging and Follow-Up
Transthyretin cardiac amyloidosis (ATTR-CA) is a progressive infiltrative cardiomyopathy caused by the deposition of misfolded transthyretin protein within the myocardium. Current disease staging and follow-up strategies mainly rely on cardiac biomarkers and renal function; however, the systemic nature of ATTR suggests that additional organ involvement may provide valuable prognostic information.
The purpose of this prospective observational study is to investigate liver dysfunction and coagulation abnormalities in patients with wild-type or hereditary ATTR-CA and to evaluate their potential role as novel markers of disease severity and progression. Patients with ATTR-CA will be compared with an age-matched control population with non-amyloid hypertrophic cardiomyopathy.
Clinical, laboratory, echocardiographic, hepatic ultrasound, liver stiffness, and coagulation parameters will be assessed at baseline and during follow-up. The study will also evaluate changes in these parameters after 6 and 12 months of treatment with tafamidis.
The results may improve the understanding of cardio-hepatic interactions in ATTR-CA and identify new tools for disease staging and longitudinal monitoring.
Обзор исследования
Статус
Рекрутинг
Подробное описание
Transthyretin cardiac amyloidosis (ATTR-CA) is an increasingly recognized cause of heart failure and left ventricular hypertrophy in older adults. Although several prognostic models have been developed for ATTR-CA, most currently available staging systems are based primarily on cardiac biomarkers and renal function. These approaches may not fully capture the systemic nature of the disease.
Emerging evidence suggests that liver dysfunction and coagulation abnormalities may represent underexplored manifestations of ATTR-CA. Liver involvement may result from direct amyloid deposition, chronic venous congestion related to heart failure, or a combination of both mechanisms. Similarly, alterations in coagulation pathways may reflect hepatic dysfunction and systemic disease burden.
LICA2025 is a single-center, prospective, observational study designed to evaluate biochemical and instrumental markers of liver function and coagulation in patients with wild-type or hereditary ATTR-CA followed at the University Hospital G. Martino of Messina, Italy. A control population with non-amyloid hypertrophic cardiomyopathy will be enrolled for comparison.
The primary objective is to compare liver and coagulation parameters between ATTR-CA patients and controls. Secondary objectives include evaluating the relationship between hepatic/coagulation abnormalities and cardiac disease severity, assessing changes after 6±1 and 12±1 months of tafamidis therapy, and exploring potential interactions between liver dysfunction and coagulation disturbances.
Participants will undergo clinical evaluation, laboratory testing, electrocardiography, transthoracic echocardiography, hepatic ultrasound, liver elastography (FibroScan), and coagulation assessment according to the study protocol. Follow-up evaluations will be performed at baseline, 6 months, and 12 months.
The study aims to identify novel biomarkers and imaging parameters that may improve disease staging, risk stratification, and longitudinal monitoring in transthyretin cardiac amyloidosis.
Тип исследования
Наблюдательный
Регистрация (Оцененный)
70
Контакты и местонахождение
В этом разделе приведены контактные данные лиц, проводящих исследование, и информация о том, где проводится это исследование.
Контакты исследования
- Имя: Luigi Colarusso, MD, PhD Candidate
- Номер телефона: +390902212341
- Электронная почта: luigi.colarusso@polime.it
Места учебы
-
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Sicily
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Messina, Sicily, Италия, 98124
- Рекрутинг
- AOU Policlinico G. Martino, UOC Cardiologia con UTIC
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Контакт:
- Luigi Colarusso, MD, PhD Candidate
- Номер телефона: +390902212341
- Электронная почта: luigi.colarusso@polime.it
-
Младший исследователь:
- Roberto Licordari, MD, PhD
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Главный следователь:
- Luigi Colarusso, MD, PhD Candidate
-
-
Критерии участия
Исследователи ищут людей, которые соответствуют определенному описанию, называемому критериям приемлемости. Некоторыми примерами этих критериев являются общее состояние здоровья человека или предшествующее лечение.
Критерии приемлемости
Возраст, подходящий для обучения
- Взрослый
- Пожилой взрослый
Принимает здоровых добровольцев
Нет
Метод выборки
Невероятностная выборка
Исследуемая популяция
Adult patients with wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) followed at the University Hospital G. Martino, Messina, Italy, and an age-matched control population with non-amyloid hypertrophic phenotype cardiomyopathy.
Описание
Inclusion Criteria:
- Written informed consent obtained prior to study participation.
- Diagnosis of wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) according to current European recommendations.
- Ability to comply with study procedures and follow-up visits.
Exclusion Criteria:
- Age younger than 18 years.
- Severe liver dysfunction due to causes other than amyloidosis.
- Inability to comply with study procedures because of language barriers, cognitive impairment, or severe psychiatric disorders.
- Comorbidities associated with life expectancy less than 12 months.
- Active alcohol or substance abuse.
- For coagulation analyses: congenital coagulation disorders, thrombotic disorders, active malignancy, or sepsis.
- Pregnancy or breastfeeding.
Учебный план
В этом разделе представлена подробная информация о плане исследования, в том числе о том, как планируется исследование и что оно измеряет.
Как устроено исследование?
Детали дизайна
Когорты и вмешательства
Группа / когорта |
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ATTR-CA Patients
Patients with wild-type or hereditary transthyretin cardiac amyloidosis (ATTR-CA) diagnosed according to current European recommendations and followed at the University Hospital G. Martino, Messina.
Participants will undergo clinical, laboratory, echocardiographic, hepatic ultrasound, liver elastography, and coagulation assessments at baseline and during follow-up.
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Hypertrophic Phenotype Controls
Age-matched patients with non-amyloid hypertrophic phenotype cardiomyopathy serving as a control population.
Participants will undergo the same clinical, laboratory, echocardiographic, hepatic, and coagulation evaluations as the ATTR-CA group.
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Что измеряет исследование?
Первичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
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Liver Stiffness Measured by Transient Elastography (FibroScan)
Временное ограничение: Baseline, 6 Months, 12 Months
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Liver stiffness expressed in kilopascals (kPa) measured by transient elastography (FibroScan) in ATTR-CA patients and controls.
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Baseline, 6 Months, 12 Months
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Controlled Attenuation Parameter (CAP) Measured by FibroScan
Временное ограничение: Baseline, 6 months, 12 months
|
Controlled attenuation parameter (CAP) measured by FibroScan as an instrumental measure of hepatic steatosis, expressed in decibels per meter (dB/m), compared between patients with ATTR-CA and controls.
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Baseline, 6 months, 12 months
|
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Serum Aspartate Aminotransferase (AST/GOT)
Временное ограничение: Baseline, 6 months, 12 months
|
Serum AST/GOT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
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Baseline, 6 months, 12 months
|
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Serum Alanine Aminotransferase (ALT/GPT)
Временное ограничение: baseline, 6 months, 12 months
|
Serum ALT/GPT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Serum Gamma-Glutamyl Transferase (GGT)
Временное ограничение: baseline, 6 months, 12 months
|
Serum gamma-glutamyl transferase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Serum Alkaline Phosphatase (ALP)
Временное ограничение: baseline, 6 months, 12 months
|
Serum alkaline phosphatase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Serum Bile Acids
Временное ограничение: baseline, 6 months, 12 months
|
Serum bile acid concentration (mcmol/L) measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Total Bilirubin (mg/dL)
Временное ограничение: baseline, 6 months, 12 months
|
Serum total bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Direct Bilirubin (mg/dL)
Временное ограничение: baseline, 6 months, 12 months
|
Serum direct bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Serum Albumin (g/dL)
Временное ограничение: baseline, 6 months, 12 months
|
Serum albumin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Serum Gamma Globulins (g/dL)
Временное ограничение: baseline, 6 months, 12 months
|
Serum gamma-globulin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Serum Immunoglobulin M (IgM) (g/L)
Временное ограничение: baseline, 6 months, 12 months
|
Serum IgM concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Serum Ferritin (ng/mL)
Временное ограничение: baseline, 6 months, 12 months
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Serum ferritin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Anti-Mitochondrial Antibodies
Временное ограничение: baseline, 6 months, 12 months
|
U/ml
|
baseline, 6 months, 12 months
|
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Fibrosis-4 (FIB-4) Index
Временное ограничение: baseline, 6 months, 12 months
|
FIB-4 index calculated from age, AST, ALT, and platelet count, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Portal Vein Diameter Assessed by Liver Ultrasound
Временное ограничение: baseline, 6 months, 12 months
|
Portal vein diameter (mm) measured by liver ultrasound, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Portal Vein Flow Velocity Assessed by Doppler Ultrasound (cm/s)
Временное ограничение: baseline, 6 months, 12 months
|
Portal vein blood flow velocity measured by Doppler ultrasound, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
|
Spleen Diameter Assessed by Ultrasound (mm)
Временное ограничение: baseline, 6 months, 12 months
|
Spleen diameter measured by abdominal ultrasound, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Prothrombin Time (PT) (s)
Временное ограничение: baseline, 6 months, 12 months
|
Prothrombin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Activated Partial Thromboplastin Time (aPTT) (s)
Временное ограничение: baseline, 6 months, 12 months
|
Activated partial thromboplastin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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International Normalized Ratio (INR)
Временное ограничение: baseline, 6 months, 12 months
|
International normalized ratio (INR) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Plasma Fibrinogen
Временное ограничение: baseline, 6 months, 12 months
|
Plasma fibrinogen concentration (g/L) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Fibrin/Fibrinogen Degradation Products
Временное ограничение: Baseline, 6 months, 12 months
|
Fibrin/fibrinogen degradation products concentration (mcg/Lm) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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Baseline, 6 months, 12 months
|
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D-Dimer
Временное ограничение: baseline, 6 months, 12 months
|
D-dimer concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Alpha-2-Antiplasmin
Временное ограничение: baseline, 6 months, 12 months
|
Alpha-2-antiplasmin concentration (UI/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Antithrombin
Временное ограничение: baseline, 6 months, 12 months
|
Antithrombin concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Plasminogen
Временное ограничение: baseline, 6 months, 12 months
|
Plasminogen concentration (IU/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Thrombin-Antithrombin Complex
Временное ограничение: baseline, 6 months, 12 months
|
Plasma fibrinogen concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Plasmin-Alpha-2-Antiplasmin Complex
Временное ограничение: baseline, 6 months, 12 months
|
Plasmin-Alpha-2-Antiplasmin Complex concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
|
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Prothrombin Fragment 1+2
Временное ограничение: baseline, 6 months, 12 months
|
Prothrombin Fragment 1+2 concentration (pmol/L) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
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Coagulation Factor X
Временное ограничение: baseline, 6 months, 12 months
|
Coagulation Factor X concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
|
|
von Willebrand Factor Antigen
Временное ограничение: baseline, 6 months, 12 months
|
von Willebrand Factor Antigen concentration (IU/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
|
baseline, 6 months, 12 months
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Plasminogen Activator Inhibitor-1 (PAI-1)
Временное ограничение: baseline, 6 months, 12 months
|
Plasminogen Activator Inhibitor-1 concentration (U/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Вторичные показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
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Correlation Between Liver Stiffness (kPa) and NT-proBNP (ng/L)
Временное ограничение: Baseline
|
Correlation between liver stiffness measured by FibroScan (kPa) and plasma NT-proBNP concentration (ng/L) in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
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Correlation Between Liver Stiffness and Interventricular Septal Thickness (mm)
Временное ограничение: Baseline
|
Correlation between liver stiffness measured by FibroScan (kPa) and interventricular septal thickness expressed in mm measured by transthoracic echocardiography in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
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Correlation Between Liver Stiffness (kPa) and Left Ventricular Global Longitudinal Strain (GLS %)
Временное ограничение: Baseline
|
Correlation between liver stiffness measured by FibroScan (kPa) and Global Longitudinal Strain (expressed in %) measured by transthoracic echocardiography in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
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Correlation Between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L)
Временное ограничение: Baseline
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Correlation between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L) measured by standard laboratory testing in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
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Change in Liver Stiffness During Disease-Modifying Treatment
Временное ограничение: Baseline, 6±1 months, and 12±1 months
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Change in liver stiffness measured by FibroScan and expressed in kPa from baseline to 6±1 months and 12±1 months after treatment initiation.
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Baseline, 6±1 months, and 12±1 months
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Change in Controlled Attenuation Parameter During Disease-Modifying Treatment
Временное ограничение: Baseline, 6±1 months, and 12±1 months
|
Change in controlled attenuation parameter (CAP) measured by FibroScan and expressed in dB/m from baseline to 6±1 months and 12±1 months after treatment initiation.
|
Baseline, 6±1 months, and 12±1 months
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Другие показатели результатов
Мера результата |
Мера Описание |
Временное ограничение |
|---|---|---|
|
Correlation Between Liver Stiffness and Prothrombin Fragment 1+2
Временное ограничение: Baseline
|
Correlation between liver stiffness measured by transient elastography (FibroScan), expressed in kPa, and plasma prothrombin fragment 1+2 concentration in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
|
Correlation Between FIB-4 Index and Prothrombin Fragment 1+2
Временное ограничение: Baseline
|
Correlation between FIB-4 and plasma prothrombin fragment 1+2 concentration in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
|
Correlation Between Liver Stiffness and Fibrinogen
Временное ограничение: Baseline
|
Correlation between liver stiffness measured by transient elastography (FibroScan), expressed in kPa, and plasma fibrinogen concentration in patients with ATTR-CA.
Correlation will be assessed using Pearson or Spearman correlation coefficient, as appropriate.
|
Baseline
|
Соавторы и исследователи
Здесь вы найдете людей и организации, участвующие в этом исследовании.
Спонсор
Следователи
- Учебный стул: Gianluca Di Bella, MD, PhD, University of Messina and AOU Policlinico G. Martino
- Главный следователь: Luigi Colarusso, MD, PhD Candidate, AOU Policlinico G. Martino
Даты записи исследования
Эти даты отслеживают ход отправки отчетов об исследованиях и сводных результатов на сайт ClinicalTrials.gov. Записи исследований и сообщаемые результаты проверяются Национальной медицинской библиотекой (NLM), чтобы убедиться, что они соответствуют определенным стандартам контроля качества, прежде чем публиковать их на общедоступном веб-сайте.
Изучение основных дат
Начало исследования (Действительный)
30 января 2026 г.
Первичное завершение (Оцененный)
1 января 2028 г.
Завершение исследования (Оцененный)
1 января 2028 г.
Даты регистрации исследования
Первый отправленный
12 июня 2026 г.
Впервые представлено, что соответствует критериям контроля качества
10 августа 2026 г.
Первый опубликованный (Действительный)
14 августа 2026 г.
Обновления учебных записей
Последнее опубликованное обновление (Действительный)
14 августа 2026 г.
Последнее отправленное обновление, отвечающее критериям контроля качества
10 августа 2026 г.
Последняя проверка
1 июня 2026 г.
Дополнительная информация
Термины, связанные с этим исследованием
Ключевые слова
Дополнительные соответствующие термины MeSH
- Заболевания нервной системы
- Сосудистые заболевания
- Сердечно-сосудистые заболевания
- Сердечные заболевания
- Нервно-мышечные заболевания
- Метаболизм, врожденные ошибки
- Генетические заболевания, врожденные
- Метаболические заболевания
- Заболевания периферической нервной системы
- Заболевания пищеварительной системы
- Гематологические заболевания
- Нейродегенеративные заболевания
- Наследственные дегенеративные заболевания, нервная система
- Геморрагические расстройства
- Недостатки протеостаза
- Амилоидные невропатии
- Амилоидоз, семейный
- Амилоидоз
- Врожденные, наследственные и неонатальные заболевания и аномалии
- Пищевые и метаболические заболевания
- Гемики и лимфатические заболевания
- Сердечная недостаточность
- Заболевания печени
- Кардиомиопатии
- Нарушения гемостаза
- Амилоидные невропатии, семейные
- Амилоидоз, наследственный, связанный с транстиретином
Другие идентификационные номера исследования
- CEL Messina Prot. 28-25
- Delibera n. 63/2026 (Другой идентификатор: AOU Policlinico G. Martino)
Планирование данных отдельных участников (IPD)
Планируете делиться данными об отдельных участниках (IPD)?
НЕ РЕШЕНО
Описание плана IPD
The decision regarding IPD sharing has not yet been finalized.
Any future data sharing will be considered in accordance with applicable ethical approvals, participant consent, institutional policies, and data protection regulations.
Информация о лекарствах и устройствах, исследовательские документы
Изучает лекарственный продукт, регулируемый FDA США.
Нет
Изучает продукт устройства, регулируемый Управлением по санитарному надзору за качеством пищевых продуктов и медикаментов США.
Нет
Эта информация была получена непосредственно с веб-сайта clinicaltrials.gov без каких-либо изменений. Если у вас есть запросы на изменение, удаление или обновление сведений об исследовании, обращайтесь по адресу register@clinicaltrials.gov. Как только изменение будет реализовано на clinicaltrials.gov, оно будет автоматически обновлено и на нашем веб-сайте. .