A cohort event monitoring study to characterise 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)

Study identification

• EU PAS number:
  EUPAS1000000890
• Study ID:
  1000000890
• Official title and acronym:
  A cohort event monitoring study to characterise 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)
• DARWIN EU® study:
  No
• Study countries:
  Canada

  China

  Germany

  Spain

  United Kingdom

  United States
• Study description:
  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. The sTTRing study is a retrospective observational cohort study of patients initiating eplontersen in the real-world (and compared to patients initiating other transthyretin amyloidosis [ATTR] treatments, if feasible) which combines deidentified data from several existing data sources. Primarily sTTRing will be a secondary data use of the ‘non-interventional prospective, multi-country study collecting real-world data on the characteristics, treatment patterns, and outcomes of patients with amyloid transthyretin (ATTR) amyloidosis’ (clinicaltrials.gov identifier NCT06465810). The sTTRing dataset will extract deidentified data from patients enrolled in NCT06465810 and who initiated eplontersen, and if comparative analyses are performed, a balanced cohort of patients unexposed to eplontersen will also be identified. Additionally, tokenized data for NCT06465810 patients in the USA will be linked to claims, if feasible, and deidentified individual case safety report (ICSR) data from AstraZeneca’s PV database will also be used, if feasible to link those data to NCT06465810.The primary objectives of the study 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.
• Study status:
  Planned

Research institutions and networks

Institutions

AstraZeneca

ICON Commercialisation & Outcomes

Germany

Ireland

Study timelines

Date when funding contract was signed

Actual:10/04/2025

Study start date

Planned:15/11/2026

Data analysis start date

Planned:15/05/2027

Date of interim report, if expected

Planned:12/11/2027

Date of final study report

Planned:15/02/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

AstraZeneca

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  Non-EU RMP only

Other study registration identification numbers and links

Protocol number: D8450R00022

Link to Clinicaltrials.gov