Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
Trial Parameters
Brief Summary
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.
Eligibility Criteria
Inclusion Criteria: * Patient willing and able to provide written informed consent to participate in the study * Confirmed diagnosis of amyloid transthyretin (ATTR) amyloidosis * Aged ≥18 years at the time of signing the informed consent * Patient willing and able to participate in collection of electronic patient reported outcomes (PROs) Exclusion Criteria: * Concurrent participation in any interventional trial for ATTR amyloidosis * Involvement in the planning and/or conduct of the current study * Patients with evidence of primary or light chain amyloidosis (AL) or serum protein A amyloidosis (AA) * Asymptomatic patients with ATTR amyloidosis and asymptomatic ATTR mutation carriers