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

NCT07654855 | Not Yet Recruiting FDA Drug

• 1 other identifier: D8450R00022
• Study Type: observational
• Target: 320
• Locations: 0 countries
• Sites: N/A

Brief 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. 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. 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.

Conditions

Transthyretin Amyloidosis; Transthyretin Amyloidosis With Cardiomyopathy; Transthyretin Amyloidosis With Polyneuropathy

Keywords

ATTR, Transthyretin amyloidosis

Outcome Measures

Primary Outcomes (2)

• Demographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment

  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)).

  Quarter 2 2026- Quarter 1 2032

• Long term safety with patients who initiated eplonersen treatment

  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.

  Quarter 2 2026- Quarter 1 2032

Secondary Outcomes (2)

• Eplontersen treatment use

  Quarter 2 2026- Quarter 1 2032

• Long-term safety in patients who initiate eplontersen treatment by subgroups

  Quarter 2 2026- Quarter 1 2032

Study Arms (1)

Eplontersen users

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

Other: None ( observational study )

Interventions

None ( observational study ) OTHER

Not applicable this is observational study no intervention is planned.

Eplontersen users

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The D8450R00003 study cohort includes individuals with confirmed diagnosis of ATTR, aged ≥18 years at the time of providing the informed consent. The sTTRing study population will be a subset of the D8450R00003 cohort who meet the additional criteria below.

You may qualify if:

• D8450R00003 participants who consented to have their data used for future related research studies.
• 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

You may not qualify if:

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

Sponsors & Collaborators

• AstraZeneca: lead
• ICON Clinical Research: collaborator

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related

Interventions

Observation

Intervention Hierarchy (Ancestors)

Methods; Investigative Techniques

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2026
• First Posted: June 17, 2026
• Study Start (Estimated): September 1, 2026
• Primary Completion (Estimated): January 29, 2032
• Study Completion (Estimated): January 29, 2032
• Last Updated: June 17, 2026

Record last verified: 2026-06