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

Overview

About this study

The purpose of this study is 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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 10/08/2024. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location	Status	Contact
Rochester, Minn. Mayo Clinic principal investigator Martha Grogan, M.D.	Open for enrollment	Contact information: Amyloid Research Team (507) 266-4426 AMYLOIDTEAMRES@mayo.edu

Mayo Clinic Location

Status

Contact

Rochester, Minn.

Mayo Clinic principal investigator

Martha Grogan, M.D.

Open for enrollment

Contact information:

Amyloid Research Team

(507) 266-4426

AMYLOIDTEAMRES@mayo.edu

More information

Publications

Publications are currently not available

Clinical Trials