Recruiting

Eplontersen

Sponsor:

AstraZeneca

Code:

NCT07654855

Conditions

Transthyretin Amyloidosis

Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis With Polyneuropathy

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

None ( observational study )

Study Details

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. 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.

Conditions

Transthyretin Amyloidosis

Transthyretin Amyloidosis With Cardiomyopathy

Transthyretin Amyloidosis With Polyneuropathy

Study ID

NCT07654855

Start date

Sep 1, 2026

Status verified date

Sep, 2026

Completion date

Jan 29, 2032

Anticipated

Primary completion date

Jan 29, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

320 participants

Anticipated

Interventions and Outcome Measures

Arms

Eplontersen users

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

Interventions

None ( observational study )

Not applicable this is observational study no intervention is planned.

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 ]
  • Long term safety with patients who initiated eplonersen treatment [ Time Frame: Quarter 2 2026- Quarter 1 2032 ]

Central Contacts and Locations

Central contacts

AstraZeneca Clinical Study Information Center

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

Locations

Research Site

Recruiting

Los Angeles, California, United States, 90095

Research Site

Recruiting

Vancouver, British Columbia, Canada, V6Z 1Y6

More Information

Sponsor

AstraZeneca

Last update posted

Sep 14, 2026

Last verified

Sep, 2026

Keywords

  • ATTR, Transthyretin amyloidosis

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-15. This information was provided to ClinicalTrials.gov by AstraZeneca on 2026-09-14.