PL_GNT01_ISR_Grant 53234273

Launched by NATIONAL INSTITUTE OF CARDIOLOGY, WARSAW, POLAND · Nov 27, 2024

Keywords

ClinConnect Summary

This is a national registry in Poland that observes transthyretin amyloidosis (TTR amyloidosis) to learn more about how common it is, what genetic mutations are involved, and how the disease tends to progress. It’s a prospective, non-interventional study, meaning researchers watch people over time without giving them new treatments. At the start they’ll collect basic information (like age, gender, TTR genotype, medical and family history, and transplant history), and on follow-up visits they’ll check symptoms, do general exams, run labs, assess heart and nerve function, and measure quality of life. The main goal is to describe the disease in the country and better understand factors that influence its course.

Who can participate: adults aged 18 or older who have a confirmed diagnosis of TTR amyloidosis or who have a confirmed TTR mutation (with or without a diagnosed disease). Participants must be willing to give informed consent. People with light-chain (AL) amyloidosis are not eligible. The study plans to enroll about 100 people across multiple centers (center in Warsaw), starting in 2019, with follow-up for at least 12 months. There is no experimental treatment involved, and results are not yet published. The registry is sponsored by the National Institute of Cardiology in Warsaw, with Pfizer as a collaborator.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Adults over 18 years old with confirmed diagnosis of TTR amyloidosis
• Exclusion Criteria:
• • Refusal to participate in the study. Light-chain amyloidosis.