Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (LICA2025)
2026년 8월 10일 업데이트: 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.
연구 개요
상세 설명
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.
연구 유형
관찰
등록 (추정된)
70
연락처 및 위치
이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.
연구 연락처
- 이름: Luigi Colarusso, MD, PhD Candidate
- 전화번호: +390902212341
- 이메일: luigi.colarusso@polime.it
연구 장소
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Sicily
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Messina, Sicily, 이탈리아, 98124
- 모병
- AOU Policlinico G. Martino, UOC Cardiologia con UTIC
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연락하다:
- Luigi Colarusso, MD, PhD Candidate
- 전화번호: +390902212341
- 이메일: luigi.colarusso@polime.it
-
부수사관:
- Roberto Licordari, MD, PhD
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수석 연구원:
- Luigi Colarusso, MD, PhD Candidate
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참여기준
연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.
자격 기준
공부할 수 있는 나이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
아니
샘플링 방법
비확률 샘플
연구 인구
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.
설명
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.
공부 계획
이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.
연구는 어떻게 설계됩니까?
디자인 세부사항
코호트 및 개입
그룹/코호트 |
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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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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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Liver Stiffness Measured by Transient Elastography (FibroScan)
기간: 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
기간: Baseline, 6 months, 12 months
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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)
기간: Baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: 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
기간: baseline, 6 months, 12 months
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U/ml
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baseline, 6 months, 12 months
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Fibrosis-4 (FIB-4) Index
기간: baseline, 6 months, 12 months
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FIB-4 index calculated from age, AST, ALT, and platelet count, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Portal Vein Diameter Assessed by Liver Ultrasound
기간: baseline, 6 months, 12 months
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Portal vein diameter (mm) measured by liver ultrasound, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Portal Vein Flow Velocity Assessed by Doppler Ultrasound (cm/s)
기간: baseline, 6 months, 12 months
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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
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Spleen Diameter Assessed by Ultrasound (mm)
기간: baseline, 6 months, 12 months
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Spleen diameter measured by abdominal ultrasound, compared between patients with ATTR-CA and controls.
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baseline, 6 months, 12 months
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Prothrombin Time (PT) (s)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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)
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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Plasma fibrinogen concentration (g/L) 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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Fibrin/Fibrinogen Degradation Products
기간: Baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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D-dimer 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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Alpha-2-Antiplasmin
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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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
기간: baseline, 6 months, 12 months
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Prothrombin Fragment 1+2 concentration (pmol/L) 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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Coagulation Factor X
기간: baseline, 6 months, 12 months
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Coagulation Factor X 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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von Willebrand Factor Antigen
기간: baseline, 6 months, 12 months
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von Willebrand Factor Antigen 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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Plasminogen Activator Inhibitor-1 (PAI-1)
기간: baseline, 6 months, 12 months
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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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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Correlation Between Liver Stiffness (kPa) and NT-proBNP (ng/L)
기간: Baseline
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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.
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Baseline
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Correlation Between Liver Stiffness and Interventricular Septal Thickness (mm)
기간: Baseline
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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.
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Baseline
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Correlation Between Liver Stiffness (kPa) and Left Ventricular Global Longitudinal Strain (GLS %)
기간: Baseline
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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.
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Baseline
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Correlation Between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L)
기간: 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.
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Baseline
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Change in Liver Stiffness During Disease-Modifying Treatment
기간: 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
기간: Baseline, 6±1 months, and 12±1 months
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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.
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Baseline, 6±1 months, and 12±1 months
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기타 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Correlation Between Liver Stiffness and Prothrombin Fragment 1+2
기간: Baseline
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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.
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Baseline
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Correlation Between FIB-4 Index and Prothrombin Fragment 1+2
기간: Baseline
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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.
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Baseline
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Correlation Between Liver Stiffness and Fibrinogen
기간: Baseline
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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.
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Baseline
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공동 작업자 및 조사자
여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.
수사관
- 연구 의자: Gianluca Di Bella, MD, PhD, University of Messina and AOU Policlinico G. Martino
- 수석 연구원: Luigi Colarusso, MD, PhD Candidate, AOU Policlinico G. Martino
연구 기록 날짜
이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.
연구 주요 날짜
연구 시작 (실제)
2026년 1월 30일
기본 완료 (추정된)
2028년 1월 1일
연구 완료 (추정된)
2028년 1월 1일
연구 등록 날짜
최초 제출
2026년 6월 12일
QC 기준을 충족하는 최초 제출
2026년 8월 10일
처음 게시됨 (실제)
2026년 8월 14일
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
2026년 8월 14일
QC 기준을 충족하는 마지막 업데이트 제출
2026년 8월 10일
마지막으로 확인됨
2026년 6월 1일
추가 정보
이 연구와 관련된 용어
키워드
추가 관련 MeSH 약관
기타 연구 ID 번호
- CEL Messina Prot. 28-25
- Delibera n. 63/2026 (기타 식별자: AOU Policlinico G. Martino)
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
미정
IPD 계획 설명
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.
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
아니
미국 FDA 규제 기기 제품 연구
아니
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .