ATTR Amyloid Cardiomyopathy: Characterization of Extracellular Vesicles as Potential Disease Stratifiers and Prognostic Biomarkers
NCT ID: NCT07314268
Last Updated: 2026-01-02
Study Results
Results pending
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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Recruitment Status
RECRUITING
Total Enrollment
70 participants
Study Classification
OBSERVATIONAL
Study Start Date
2024-10-18
Study Completion Date
2026-12-31
Brief Summary
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This study explores whether extracellular vesicles (EVs) tiny particles released into the bloodstream by cells can serve as early and minimally invasive biomarkers for transthyretin amyloid cardiomyopathy (ATTR-CM). Because ATTR-CM is often diagnosed only after significant heart damage has occurred, there is an urgent need for earlier detection methods.
The study will enroll individuals with different clinical presentations of transthyretin amyloidosis, along with healthy controls. Participants will undergo blood sampling, cardiac imaging (including echocardiography, cardiac MRI, and scintigraphy when indicated), and molecular EV analysis.
By comparing EV profiles across groups, the study aims to determine whether these vesicles reflect early cardiac involvement, track disease progression, and support more accurate and timely diagnosis. Ultimately, this research seeks to improve clinical decision-making and patient outcomes in ATTR cardiomyopathy.
The study will enroll individuals with different clinical presentations of transthyretin amyloidosis, along with healthy controls. Participants will undergo blood sampling, cardiac imaging (including echocardiography, cardiac MRI, and scintigraphy when indicated), and molecular EV analysis.
By comparing EV profiles across groups, the study aims to determine whether these vesicles reflect early cardiac involvement, track disease progression, and support more accurate and timely diagnosis. Ultimately, this research seeks to improve clinical decision-making and patient outcomes in ATTR cardiomyopathy.
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Detailed Description
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Transthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive infiltrative disease caused by the deposition of misfolded transthyretin protein in the myocardium. Despite advances in imaging and treatment, early detection and accurate risk stratification remain major challenges, often resulting in delayed diagnosis and worse clinical outcomes.
This prospective observational study will enroll 70 adult participants, distributed into four predefined groups:
* ATTR-CM with myocardial dysfunction (n = 20)
* Hereditary ATTR with predominant neurologic involvement (n = 20)
* Genotype-positive individuals without clinical manifestation (n = 10)
* Healthy controls (n = 20)
All participants will undergo blood collection for extracellular vesicle (EV) isolation and molecular profiling. EVs are nanoscale particles released by cells that carry proteins, lipids, and nucleic acids reflective of their cellular origin and physiological state, potentially serving as minimally invasive biomarkers.
Participants with cardiac involvement will additionally undergo standardized cardiac evaluations, including echocardiography, cardiac MRI, and nuclear imaging when indicated. Clinical data, functional status, and laboratory parameters will be correlated with EV characteristics to assess their association with disease severity and progression.
By integrating EV profiling with clinical and imaging findings across different phenotypes and disease stages, this study aims to identify biomarkers capable of improving diagnostic accuracy, tracking disease evolution, and supporting personalized care strategies in ATTR amyloidosis.
This prospective observational study will enroll 70 adult participants, distributed into four predefined groups:
* ATTR-CM with myocardial dysfunction (n = 20)
* Hereditary ATTR with predominant neurologic involvement (n = 20)
* Genotype-positive individuals without clinical manifestation (n = 10)
* Healthy controls (n = 20)
All participants will undergo blood collection for extracellular vesicle (EV) isolation and molecular profiling. EVs are nanoscale particles released by cells that carry proteins, lipids, and nucleic acids reflective of their cellular origin and physiological state, potentially serving as minimally invasive biomarkers.
Participants with cardiac involvement will additionally undergo standardized cardiac evaluations, including echocardiography, cardiac MRI, and nuclear imaging when indicated. Clinical data, functional status, and laboratory parameters will be correlated with EV characteristics to assess their association with disease severity and progression.
By integrating EV profiling with clinical and imaging findings across different phenotypes and disease stages, this study aims to identify biomarkers capable of improving diagnostic accuracy, tracking disease evolution, and supporting personalized care strategies in ATTR amyloidosis.
Conditions
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Wild-type ATTR Amyloidosis
Amyloidosis Cardiac
Amyloidosis, Hereditary
Amyloidosis Transthyretin
Study Design
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Observational Model Type
COHORT
Study Time Perspective
PROSPECTIVE
Study Groups
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TTR-CA + DM
TTR amyloid cardiomyopathy and myocardial dysfunction
No interventions assigned to this group
TTR-A
TTR amyloidosis and neurological involvement
No interventions assigned to this group
TTR-Gen
TTR amyloid genotype without phenotypic expression
No interventions assigned to this group
CTL
healthy controls
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* Adult patients aged 18 years or older;
* Confirmed diagnosis of transthyretin cardiac amyloidosis (TTR-CA), with or without myocardial dysfunction, according to established diagnostic criteria;
* Willingness to comply with study procedures and requirements;
* Ability to provide written informed consent.
* Confirmed diagnosis of transthyretin cardiac amyloidosis (TTR-CA), with or without myocardial dysfunction, according to established diagnostic criteria;
* Willingness to comply with study procedures and requirements;
* Ability to provide written informed consent.
Exclusion Criteria
* Presence of other significant cardiac conditions that may interfere with study outcomes, such as severe coronary artery disease or major valvular disease;
* Inability to provide informed consent or to participate in the required clinical assessments and examinations.
* Inability to provide informed consent or to participate in the required clinical assessments and examinations.
Minimum Eligible Age
18 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
Yes
Sponsors
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InCor Heart Institute
OTHER
Sponsor Role
collaborator
University of Sao Paulo General Hospital
OTHER
Sponsor Role
lead
Responsible Party
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FELIX JOSÉ ALVAREZ RAMIRES
Principal Investigator
Responsibility Role
PRINCIPAL_INVESTIGATOR
Locations
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Instituto do Coracao, HCFMUSP
São Paulo, São Paulo, Brazil
Site Status
RECRUITING
Countries
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Brazil
Central Contacts
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Facility Contacts
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Camila R Moreno, PhD
Role: backup
5511994849265
References
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Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Document Type: Informed Consent Form
Other Identifiers
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83160124.2.0000.0068
Identifier Type: -
Identifier Source: org_study_id
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