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Liver and Coagulation Disorders in Cardiac Transthyretin Amyloidosis (LICA2025)

10. August 2026 aktualisiert von: 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.

Studienübersicht

Status

Rekrutierung

Bedingungen

Detaillierte Beschreibung

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.

Studientyp

Beobachtungs

Einschreibung (Geschätzt)

70

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studienorte

  • Italien
    • Sicily
      • Messina, Sicily, Italien, 98124
        • Rekrutierung
        • AOU Policlinico G. Martino, UOC Cardiologia con UTIC
        • Kontakt:
        • Unterermittler:
          • Roberto Licordari, MD, PhD
        • Hauptermittler:
          • Luigi Colarusso, MD, PhD Candidate

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Probenahmeverfahren

Nicht-Wahrscheinlichkeitsprobe

Studienpopulation

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.

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

Kohorten und Interventionen

Gruppe / Kohorte
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.
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.

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Liver Stiffness Measured by Transient Elastography (FibroScan)
Zeitfenster: Baseline, 6 Months, 12 Months
Liver stiffness expressed in kilopascals (kPa) measured by transient elastography (FibroScan) in ATTR-CA patients and controls.
Baseline, 6 Months, 12 Months
Controlled Attenuation Parameter (CAP) Measured by FibroScan
Zeitfenster: 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.
Baseline, 6 months, 12 months
Serum Aspartate Aminotransferase (AST/GOT)
Zeitfenster: Baseline, 6 months, 12 months
Serum AST/GOT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
Baseline, 6 months, 12 months
Serum Alanine Aminotransferase (ALT/GPT)
Zeitfenster: baseline, 6 months, 12 months
Serum ALT/GPT concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Gamma-Glutamyl Transferase (GGT)
Zeitfenster: 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.
baseline, 6 months, 12 months
Serum Alkaline Phosphatase (ALP)
Zeitfenster: baseline, 6 months, 12 months
Serum alkaline phosphatase concentration (U/L) measured by routine laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Bile Acids
Zeitfenster: baseline, 6 months, 12 months
Serum bile acid concentration (mcmol/L) measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Total Bilirubin (mg/dL)
Zeitfenster: baseline, 6 months, 12 months
Serum total bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Direct Bilirubin (mg/dL)
Zeitfenster: baseline, 6 months, 12 months
Serum direct bilirubin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Albumin (g/dL)
Zeitfenster: baseline, 6 months, 12 months
Serum albumin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Gamma Globulins (g/dL)
Zeitfenster: baseline, 6 months, 12 months
Serum gamma-globulin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Immunoglobulin M (IgM) (g/L)
Zeitfenster: baseline, 6 months, 12 months
Serum IgM concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Serum Ferritin (ng/mL)
Zeitfenster: baseline, 6 months, 12 months
Serum ferritin concentration measured by laboratory testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Anti-Mitochondrial Antibodies
Zeitfenster: baseline, 6 months, 12 months
U/ml
baseline, 6 months, 12 months
Fibrosis-4 (FIB-4) Index
Zeitfenster: 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
Portal Vein Diameter Assessed by Liver Ultrasound
Zeitfenster: 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
Portal Vein Flow Velocity Assessed by Doppler Ultrasound (cm/s)
Zeitfenster: baseline, 6 months, 12 months
Portal vein blood flow velocity measured by Doppler ultrasound, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Spleen Diameter Assessed by Ultrasound (mm)
Zeitfenster: baseline, 6 months, 12 months
Spleen diameter measured by abdominal ultrasound, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Prothrombin Time (PT) (s)
Zeitfenster: baseline, 6 months, 12 months
Prothrombin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Activated Partial Thromboplastin Time (aPTT) (s)
Zeitfenster: baseline, 6 months, 12 months
Activated partial thromboplastin time measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
International Normalized Ratio (INR)
Zeitfenster: baseline, 6 months, 12 months
International normalized ratio (INR) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasma Fibrinogen
Zeitfenster: 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
Fibrin/Fibrinogen Degradation Products
Zeitfenster: 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.
Baseline, 6 months, 12 months
D-Dimer
Zeitfenster: 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
Alpha-2-Antiplasmin
Zeitfenster: 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.
baseline, 6 months, 12 months
Antithrombin
Zeitfenster: baseline, 6 months, 12 months
Antithrombin concentration (IU) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasminogen
Zeitfenster: baseline, 6 months, 12 months
Plasminogen concentration (IU/ml) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Thrombin-Antithrombin Complex
Zeitfenster: baseline, 6 months, 12 months
Plasma fibrinogen concentration (ng/mL) measured by standard laboratory coagulation testing, compared between patients with ATTR-CA and controls.
baseline, 6 months, 12 months
Plasmin-Alpha-2-Antiplasmin Complex
Zeitfenster: 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.
baseline, 6 months, 12 months
Prothrombin Fragment 1+2
Zeitfenster: 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
Coagulation Factor X
Zeitfenster: 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
Zeitfenster: 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
Plasminogen Activator Inhibitor-1 (PAI-1)
Zeitfenster: 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.
baseline, 6 months, 12 months

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Correlation Between Liver Stiffness (kPa) and NT-proBNP (ng/L)
Zeitfenster: 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
Correlation Between Liver Stiffness and Interventricular Septal Thickness (mm)
Zeitfenster: 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
Correlation Between Liver Stiffness (kPa) and Left Ventricular Global Longitudinal Strain (GLS %)
Zeitfenster: 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
Correlation Between Prothrombin Fragment 1+2 (pmol/L) and NT-proBNP (ng/L)
Zeitfenster: Baseline
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
Change in Liver Stiffness During Disease-Modifying Treatment
Zeitfenster: Baseline, 6±1 months, and 12±1 months
Change in liver stiffness measured by FibroScan and expressed in kPa from baseline to 6±1 months and 12±1 months after treatment initiation.
Baseline, 6±1 months, and 12±1 months
Change in Controlled Attenuation Parameter During Disease-Modifying Treatment
Zeitfenster: 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

Andere Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Correlation Between Liver Stiffness and Prothrombin Fragment 1+2
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

University of Messina

Mitarbeiter

Azienda Ospedaliera Universitaria Policlinico "G. Martino"

Ermittler

  • Studienstuhl: Gianluca Di Bella, MD, PhD, University of Messina and AOU Policlinico G. Martino
  • Hauptermittler: Luigi Colarusso, MD, PhD Candidate, AOU Policlinico G. Martino

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

30. Januar 2026

Primärer Abschluss (Geschätzt)

1. Januar 2028

Studienabschluss (Geschätzt)

1. Januar 2028

Studienanmeldedaten

Zuerst eingereicht

12. Juni 2026

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

10. August 2026

Zuerst gepostet (Tatsächlich)

14. August 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

14. August 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

10. August 2026

Zuletzt verifiziert

1. Juni 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • CEL Messina Prot. 28-25
  • Delibera n. 63/2026 (Andere Kennung: AOU Policlinico G. Martino)

Plan für individuelle Teilnehmerdaten (IPD)

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Beschreibung des IPD-Plans

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.

Arzneimittel- und Geräteinformationen, Studienunterlagen

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Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

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