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

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-130
SponsorAstraZeneca

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

1,850 Participants
are divided into 3 treatment groups

Sponsors and collaborators

AstraZeneca

Lead sponsor

ICON plc

Collaborator