International Observational Study of Adults With Transthyretin ATTR Amyloidosis Tracking Real-World Treatment and Outcomes

What this Trial Is About

Researchers are conducting the MaesTTRo study, an international, observational research project focusing on adult patients with transthyretin ATTR amyloidosis. The study aims to follow at least 1850 patients worldwide, including those with ATTR cardiomyopathy ATTR-CM and hereditary polyneuropathy ATTRv-PN, to understand the natural progression of the disease and document real-world treatment patterns and outcomes. Special attention is given to treatments such as eplontersen, an antisense oligonucleotide therapy targeting the TTR protein. Participants will be observed over a period ranging from at least 3 to up to 7 years, depending on enrollment timing. Data collected includes patient-reported outcomes via electronic questionnaires completed at enrollment and every six months during routine clinic visits. Clinical information, treatment details, and diagnostic results such as biopsy findings, imaging scans, nerve function tests, and laboratory results will be gathered from medical records as per usual care. In the United States, additional de-identified healthcare data may be collected through a tokenization process, with patient consent. During the study, participants will continue their regular medical visits with no extra site visits required specifically for the study. Researchers will monitor various health aspects including symptom progression, quality of life scores, cardiac and neurological function, and healthcare resource use. The study will also track mortality and other clinical outcomes over the follow-up period. This information will help describe treatment effectiveness and disease characteristics in real-world settings.

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis