About this trial
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
Qualifiers
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)
Disqualifiers
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
Trial design
Treatments tested in this trial
- Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Treatment groups
Sponsors and collaborators
AstraZeneca
Lead sponsor
ICON plc
Collaborator