Cardiac Amyloidosis in HFpEF

NCT04587648 | Recruiting

• 1 other identifier: 201906065RINA
• Study Type: observational
• Target: 250
• Locations: 1 country
• Sites: 1

Brief Summary

Heart failure with preserved ejection fraction (HFpEF) accounts for half of heart failure cases with heterogenous cause and variable presentations. The diagnosis of HFpEF required clinical signs and symptoms of HF, normal left ventricular ejection fraction (LVEF) and evidence of diastolic dysfunction. No treatment has been shown in recent major clinical trials having benefits in these patients. One major reason of the poor response to medical treatment is the heterogeneity of HFpEF, which contains many different underline causes. To identify the underlying causes of HFpEF may improve the diagnosis and treatment in these patients. Age-related amyloid deposition has first been reported in 1876 and the following autopsy studies showed the prevalence of senile cardiac amyloid is up to 25%. Recently, it has been recognized that the deposits in senile cardiac amyloid are derived from wild-type transthyretin (TTR). Transthyretin amyloidosis cardiac amyloidosis (ATTR CA) is caused by myocardial deposition of misfolded transthyretin protein. There are 2 types of ATTR classified by genetic mutation including wild-type ATTR (ATTRwt) and familial cardiac amyloid caused by TTR mutation (ATTRm). Multimodality techniques have been developed to assist in the diagnosis of the diagnosis of TTR. Among them, 99mTc-3,3-diphosphono-1,2-propanodicarboxylic acid (99mTc-DPD) scintigraphy is a non-invasive test and it can diagnose TTR from other cause diverse form of cardiac amyloidosis and cardiomyopathy. In the study of Gonzalez-Lopez et al, in 120 HFpEF patients, 16 (13.3%) had positive 99mTc-DPD scan. Four patients with positive 99mTc-DPD scan received endomyocardial biopsy and confirmed cardiac amyloid deposition. ATTRwt could be an important cause of HFpEF and it was often under diagnosed. A recent study in Spain reported that 13% of patents over age of 60 years with HFpEF and left ventricular wall thickness of 12mm or more had ATTRwt. However, the prevalence of ATTRwt among patients with HFpEF is not well-established in Taiwan and Asia. The aim of this study is to determine the prevalence, clinical characteristics, risk factors and outcomes of ATTRwt related HFpEF patients in Taiwan.

Conditions

Heart Failure With Preserved Ejection Fraction; Amyloidosis Cardiac

Outcome Measures

Primary Outcomes (1)

• Prevalence of ATTRwt among patients with HFpEF in Taiwan

  At enrollment

Interventions

NA, observational study OTHER

NA, observational study

Eligibility Criteria

• Age: 60 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Eligible HFpEF patients with a LVEF ≥50%, NYHA class I-IV symptoms: Both in-patients or patients in clinics could be enrolled. These patients could be newly diagnosed or has been diagnosed. Study subjects will be pre-stratified according to left ventricular posterior wall thickness (LVPW) ≥12mm or less; number of subjects with LVPW ≥12mm should account for more than 50% of total subjects recruited in the final analysis.

You may qualify if:

• Patient is ≥ 60 years old or 50 y/o with carpal tunnel syndrome or spinal stenosis
• Patient has been diagnosed as HFpEF in their medical history or newly diagnosed as HFpEF. They have HF symptoms with NYHA Classification of I-IV when diagnosis. The criteria of HFpEF is according to our previous studies.
• More than 50% of them have LVPW ≥12mm (when diagnosis).
• Written informed consent could be obtained.

You may not qualify if:

• Patients unwilling to join this projects.
• Patients with unstable coronary artery disease, plan to receive coronary intervention within months.
• Patients has previous history of HFrEF with a LVEF \<40%.

Sponsors & Collaborators

• National Taiwan University Hospital: lead
• National Taiwan University Hospital Jin-Shan branch: collaborator
• National Taiwan University Hospital Hsin-Chu Branch: collaborator

Study Sites (1)

National Taiwan University Hospital

Taipei, 100, Taiwan

RECRUITING

MeSH Terms

Conditions

Amyloid Neuropathies, Familial

Interventions

Sodium; Observation

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Amyloid Neuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Amyloidosis, Familial; Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Amyloidosis; Proteostasis Deficiencies

Intervention Hierarchy (Ancestors)

Metals, Alkali; Elements; Inorganic Chemicals; Metals, Light; Metals; Methods; Investigative Techniques

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 6, 2020
• First Posted: October 14, 2020
• Study Start: August 1, 2019
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2025
• Last Updated: July 5, 2024

Record last verified: 2024-07

Locations

TW (1)