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

Part of paid clinical trials in Los Angeles, California.

Sponsor
AstraZeneca
Study ID
NCT07654855
Status
Recruiting
Apply to this trial

Conditions

  • Transthyretin Amyloidosis
  • Transthyretin Amyloidosis With Cardiomyopathy
  • Transthyretin Amyloidosis With Polyneuropathy

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • None ( observational study ) — OTHER
    Not applicable this is observational study no intervention is planned.

Study Details

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.

Key Dates

First listed
Jun 17, 2026
Start date
Sep 1, 2026
Status verified
Sep 2026
Primary completion
Jan 29, 2032
Completion
Jan 29, 2032

Study Design

Enrollment
320 participants (estimated)

Arms

  • Arm: Eplontersen users
    D8450R00003 population of new and prior eplontersen users (including prior liver transplant and severe hepatic impairment subpopulations)

Primary Outcome Measure

Demographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment [ Time Frame: Quarter 2 2026- Quarter 1 2032 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Research SiteLos AngelesCalifornia90095-

Related Studies