Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (LICA2025)
10 agosto 2026 aggiornato da: 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.
Panoramica dello studio
Stato
Reclutamento
Descrizione dettagliata
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.
Tipo di studio
Osservativo
Iscrizione (Stimato)
70
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Luigi Colarusso, MD, PhD Candidate
- Numero di telefono: +390902212341
- Email: luigi.colarusso@polime.it
Luoghi di studio
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Sicily
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Messina, Sicily, Italia, 98124
- Reclutamento
- AOU Policlinico G. Martino, UOC Cardiologia con UTIC
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Contatto:
- Luigi Colarusso, MD, PhD Candidate
- Numero di telefono: +390902212341
- Email: luigi.colarusso@polime.it
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Sub-investigatore:
- Roberto Licordari, MD, PhD
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Investigatore principale:
- Luigi Colarusso, MD, PhD Candidate
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Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Adulto
- Adulto più anziano
Accetta volontari sani
No
Metodo di campionamento
Campione non probabilistico
Popolazione di studio
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.
Descrizione
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.
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
Coorti e interventi
Gruppo / Coorte |
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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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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Liver Stiffness Measured by Transient Elastography (FibroScan)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Correlation Between Liver Stiffness (kPa) and NT-proBNP (ng/L)
Lasso di tempo: 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)
Lasso di tempo: 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 %)
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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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Altre misure di risultato
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Correlation Between Liver Stiffness and Prothrombin Fragment 1+2
Lasso di tempo: 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
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Correlation Between FIB-4 Index and Prothrombin Fragment 1+2
Lasso di tempo: 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
Lasso di tempo: 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.
|
Baseline
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Sponsor
Investigatori
- Cattedra di studio: Gianluca Di Bella, MD, PhD, University of Messina and AOU Policlinico G. Martino
- Investigatore principale: Luigi Colarusso, MD, PhD Candidate, AOU Policlinico G. Martino
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
30 gennaio 2026
Completamento primario (Stimato)
1 gennaio 2028
Completamento dello studio (Stimato)
1 gennaio 2028
Date di iscrizione allo studio
Primo inviato
12 giugno 2026
Primo inviato che soddisfa i criteri di controllo qualità
10 agosto 2026
Primo Inserito (Effettivo)
14 agosto 2026
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
14 agosto 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
10 agosto 2026
Ultimo verificato
1 giugno 2026
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
- Arresto cardiaco
- Emostasi
- Biomarcatori
- Ecocardiografia
- Disfunzione epatica
- ATTR
- Fibroscan
- Amiloidosi da transtiretina
- Amiloidosi cardiaca
- Tafamidis
- Rigidità del fegato
- Acoramidis
- Disturbo della coagulazione
- Stadiazione della malattia
- ATTR-CA
- Hepatic Congestion
- Cardiohepatic Syndrome
- Wild-Type ATTR
- Hereditary ATTR
Termini MeSH pertinenti aggiuntivi
- Malattie del sistema nervoso
- Malattie vascolari
- Malattia cardiovascolare
- Malattie cardiache
- Malattie neuromuscolari
- Metabolismo, errori congeniti
- Malattie genetiche, congenite
- Malattie metaboliche
- Malattie del sistema nervoso periferico
- Malattie dell'apparato digerente
- Malattie ematologiche
- Malattie Neurodegenerative
- Malattie Eredodegenerative, Sistema Nervoso
- Disturbi emorragici
- Carenze di proteostasi
- Neuropatie amiloidi
- Amiloidosi familiare
- Amiloidosi
- Malattie e anomalie congenite, ereditarie e neonatali
- Malattie nutrizionali e metaboliche
- Malattie emiche e linfatiche
- Arresto cardiaco
- Malattie del fegato
- Cardiomiopatie
- Disturbi emostatici
- Neuropatie amiloidi, familiari
- Amiloidosi, ereditaria, correlata alla transtiretina
Altri numeri di identificazione dello studio
- CEL Messina Prot. 28-25
- Delibera n. 63/2026 (Altro identificatore: AOU Policlinico G. Martino)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
INDECISO
Descrizione del piano 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.
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
No
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .