PL_GNT01_ISR_Grant 53234273

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

100 participants

Study Classification

OBSERVATIONAL

Study Start Date

2019-02-11

Study Completion Date

2025-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

We carry out a prospective non-interventional, longitudinal, observational multicentre registry designed to improve our understanding of the epidemiology of TTR amyloidosis in our country. The main objective of the proposed study is to determine the occurrence of TTR amyloidosis and describe clinical profile of patients in the population of our country.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Demographic information, TTR genotype, medical history, family history of the disease, and transplant history are assessed at baseline. On return visits, signs and symptoms of the disease are evaluated, general examinations are conducted, and laboratory data, measures of neurologic and cardiovascular function, and quality of life are assessed according to the standard of care for patients.

Specific Aims and Hypotheses

Our working hypotheses are:

1. TTR amyloidosis affects patients in the population of our country;
2. there are specific TTR mutations in the population of our country;
3. there is genotype-phenotype relationship in hereditary TTR amyloidosis;
4. there are risk factors for TTR amyloidosis in the population of our country. These hypotheses will be tested in our specific aims. In aim 1. we will describe the occurrence of TTR amyloidosis in the population of our country, including the hereditary and acquired forms of the disease.

In aim 2. we will determine and characterize high frequency TTR mutations in the population of our country.

In aim 3. we will determine a clinical profile of patients and we will try to enhance understanding of the natural history of TTR amyloidosis, including the variability, progression of the disease, and predisposing factors. We will evaluate patients' quality of life.

In aim 4. we will search for genotype-phenotype relationship in hereditary TTR amyloidosis.

In aim 5. we will evaluate effects of liver transplantation and other treatments on disease progression in our patients. We will advance knowledge of the disease to optimize the assessment, treatment and monitoring of patients.

In aim 6. we will formulate novel hypotheses for further prospective studies. We will form a community of medical experts on amyloidosis (cardiologists, neurologist, internal medicine physicians, as well as other specialists) to create in the future national centre of amyloidosis in our country that would offer the highest standard of care and gather clinical data on this rare disease.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Transthyretin Cardiomyopathy (TTR-CM) Transthyretin (TTR) Amyloid Cardiomyopathy Transthyretin Amyloidosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Adults over 18 years old with confirmed diagnosis of TTR amyloidosis

Exclusion Criteria

* Refusal to participate in the study. Light-chain amyloidosis.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Monika Gawor-Prokopczyk

PhD

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

National Institute of Cardiology

Warsaw, , Poland

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Poland

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

53234273

Identifier Type: -

Identifier Source: org_study_id