Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations

The aim of this observational cohort study is to characterise use of eplontersen in patients with prior liver transplant or with pre-existing severe hepatic impairment, as well as to assess long-term safety among all new users of eplontersen; all are areas of missing information Primary objectives are: 1. To describe demographic and clinical characteristics of patients at eplontersen initiation, including the prevalence of prior liver transplant (overall and by reason for liver transplant), and the prevalence of severe hepatic impairment; and to describe patients in these subgroups (prior liver transplant, severe hepatic impairment). 2. To describe long-term safety in patients who initiate eplontersen treatment, including onset of new clinical events, abnormal laboratory values and serious adverse events.

NCT Number	NCT07654855
Lead Sponsor	AstraZeneca Collaborators: ICON Clinical Research
Conditions	Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
Enrollment	320 participants
Start Date	2026-09-01
Primary Completion	2032-01-29 (estimated)
Study Completion	2032-01-29 (estimated)
Updated on ClinicalTrials.gov	2026-06-17