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

Post-Authorisation Safety Study: A Cohort Event Monitoring Study to Characterise the Use of Eplontersen in Patients With Prior Liver Transplant and Pre-existing Severe Hepatic Impairment and to Assess Long-term Safety Among All New Users of Eplontersen (sTTRing)

Who can join

Ages 18 and older · All sexes

Inclusion Criteria:

1. D8450R00003 participants who consented to have their data used for future related research studies.
2. D8450R00003 participants who initiated eplontersen treatment up to 1-year prior to enrolment into D8450R00003 study observation period, irrespective of ATTR phenotype or genotype. For the comparative analyses, patients unexposed to eplontersen treatment and who initiated another ATTR treatment during D8450R00003 study observation period will be included

Exclusion Criteria:

1. Patients with exposure to eplontersen more than 1-year prior to enrolment into D8450R00003 study.
2. Patients who participated in an interventional ATTR study in the 12-months prior to enrolment into D8450R00003 study.

About the study

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.

What is being tested

• None ( observational study ) (other)

Sponsor: AstraZeneca · Participants: 320 · Started: Sep 1, 2026

Contact the study team

• AstraZeneca Clinical Study Information Center · Phone: 1-877-240-9479

Official record on ClinicalTrials.gov — NCT07654855

Locations in the U.S.

Conditions

• Transthyretin-Related Hereditary Amyloidosis

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.