A Non-interventional, Prospective, Multi-country Study Collecting Real-world Data on the Characteristics, Treatment Patterns, and Outcomes of Patients With Transthyretin (ATTR) Amyloidosis

AstraZeneca

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.

Description

MaesTTRo is an international, longitudinal, non-interventional study of adult patients with transthyretin (ATTR) amyloidosis. The study plans to enroll a minimum of 1850 patients with ATTR amyloidosis, including a minimum of 850 patients with ATTR cardiomyopathy (ATTR-CM), and a minimum of 100 patients with ATTRv-PN hereditary polyneuropathy. The enrollment period is expected to last approximately 4 years. The duration of follow-up for each patient will be at least 3 years and up to 7 years depending on the date when the patient is enrolled. This study design will include both primary and s…

Eligibility

Age range: 18–130 years
Sex: All
Healthy volunteers: No

Detailed 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 amyloid…

Interventions

Drug
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Data will be collected on patients with ATTR amyloidosis in a real-world setting

Locations (60)

Research Site
La Jolla, California
Research Site
Los Angeles, California
Research Site
San Francisco, California
Research Site
San Francisco, California
Research Site
New Haven, Connecticut
Research Site
Washington D.C., District of Columbia