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

Condition(s) studied

Investigational drug(s) / intervention(s)

None ( observational study ): Not applicable this is observational study no intervention is planned.

Study summary

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.

Eligibility

Primary outcome measure(s)

• Demographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment — Quarter 2 2026- Quarter 1 2032
  1. Prevalence of liver transplant prior to eplontersen initiation (overall by reason for liver transplant), 2. Prevalence of severe hepatic impairment prior to eplontersen initiation These will be summarised as prevalence proportion and 95% CI. Secondary outcomes: Demographic characteristics (e.g. age, sex), general health factors (e.g. BMI), indication related characteristics (e.g. ATTR diagnoses dates and duration since first diagnosis, type, severity), prior and concomitant medications (as part of standard care of ATTR at eplontersen initiation), pre-existing and concurrent relevant morbidities will be described using descriptive statistics of counts, proportions and/or distribution characteristics (mean (SD), median (range)).
• Long term safety with patients who initiated eplonersen treatment — Quarter 2 2026- Quarter 1 2032
  Onset of new clinical events, abnormal laboratory values, serious adverse events will be analysed as follows: a) Counts and frequency of first events and total number of events, incidence rate and incidence risk of events per time periods (e.g., 0-6, 7-end of follow-up available at time of reporting). b) Cohort event monitoring analysis including a. Incidence densities per 6 months (in the first year) or 12-months (after one year) intervals will be estimated. b. Differences in incidence densities per 6 or 12months intervals c. Nelson-Aalen estimator of cumulative hazard rate function over time. c) For SAEs, qualitative case reports of clinical course and patients' characteristics and comorbidities will be provided. These outcomes are all relative to primary objective 2 of study and are all relative to the same outcome measure.

Trial sites (6)