ClinicalTrials.gov record NCT07654855
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
Post-Authorisation Safety Study: A Cohort Event Monitoring Study to Characterise the 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)
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This retrospective observational cohort study uses data from an existing ATTR cohort (D8450R00003) to describe how eplontersen is used and to monitor long-term safety in people who start the drug. The study will report patient characteristics at treatment start and track new clinical events, abnormal laboratory values, and serious adverse events. It focuses particularly on people with prior liver transplant and on those with pre-existing severe hepatic impairment. Data collection is planned from Q3 2026 through Q1 2032 with annual interim reports.
What it is studying. The study aims to learn who starts eplontersen and how it is used in routine care, and to identify and describe long-term safety events (new clinical events, abnormal lab results, and serious adverse events), with separate looks at people with prior liver transplant and those with severe hepatic impairment.
What the researchers hope to learn. Describe demographic and clinical characteristics of people starting eplontersen, including prevalence of prior liver transplant and severe hepatic impairment. Describe long-term safety among new eplontersen users (timing and frequency of new clinical events, abnormal laboratory values, and serious adverse events). Describe eplontersen treatment use (duration and reasons for stopping), overall and in the liver-transplant and severe hepatic impairment subgroups.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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:
- 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).
- To describe long-term safety in patients who initiate eplontersen treatment, including onset of new clinical events, abnormal laboratory values and serious adverse events.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Other
- None ( observational study )
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- 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
Exclusion criteria
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
Study D8450R00003 enrolls adults (≥18 years) with a confirmed ATTR diagnosis at the time of informed consent. The sTTRing study will represent a subset of D8450R00003 participants who consent to secondary research use of their data and who initiated eplontersen treatment within 1 year prior to enrollment in D8450R00003. For comparative analyses, patients not exposed to eplontersen who initiated alternative ATTR therapies during the D8450R00003 observation period will also be included. sTTRing data collection will begin in Q3 2026 and conclude in Q1 2032. Interim analyses will be reported annually from Q3 2027 through Q3 2031, with a final report submitted in Q1 2033. The primary endpoints supporting the primary study objective are: (1) the prevalence of patients with a history of liver transplantation, categorized by reason for liver transplant, and (2) the prevalence of severe hepatic impairment. Safety outcomes will be summarized by incidence risk and event rates across prespecified time intervals (e.g., 0-6 months and from 7 months to the end of available follow-up) and over the full duration of follow-up. These summaries will be included in interim reports as part of both primary and secondary objectives. Cohort Event Monitoring will be used to identify safety events requiring further characterization. When more than 10 events are observed for a given outcome, cumulative hazard rates will be estimated. To optimize sample size, comparative analyses will be conducted, where feasible, at the final analysis and presented in the final report.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participation means allowing researchers to use clinical data already collected in the parent D8450R00003 study for retrospective analysis (no study treatment is assigned). The study team will analyze medical history, treatment start dates (including eplontersen or other ATTR therapies), lab results, transplant history, and recorded adverse events; cohort event monitoring methods will be used to identify safety events. There are no new interventions planned in this observational study; any additional contact or procedures are not specified in the registry record.
How the study is structured. A retrospective observational cohort study nested within the existing D8450R00003 cohort. Investigators will use cohort event monitoring of existing medical records to identify and describe safety events and treatment patterns; where possible, comparisons with patients who started other ATTR treatments will be made. Data collection is scheduled Q3 2026–Q1 2032, with annual interim analyses and a final report.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Canada, China, Germany, Spain, United Kingdom, United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on wild-type ATTR amyloidosis
This trial is also listed on this site under ATTRv amyloidosis.
Important dates
- Study start
- Sep 1, 2026
- Primary completion
- Jan 29, 2032 (estimated)
- Study completion
- Jan 29, 2032 (estimated)
- Target enrollment
- 320 participants (estimated)
- Last updated on registry
- Sep 14, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- AstraZeneca
- Collaborators
- ICON Clinical Research
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- AstraZeneca Clinical Study Information Center 📞 1-877-240-9479 ✉ information.center@astrazeneca.com
Participating sites (6)
- Research SiteRecruiting
Los Angeles, California, 90095 · United States
- Research SiteRecruiting
Vancouver, British Columbia, V6Z 1Y6 · Canada
- Research SiteRecruiting
Beijing · China
- Research SiteRecruiting
Hanover · Germany
- Research SiteRecruiting
Majadahonda · Spain
- Research SiteRecruiting
Birmingham · United Kingdom
Terms used on this page · explanations by MedicalUpdates.ai
- serious adverse events
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- enrolment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- interventional
- A study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- endpoints
- Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- Cohort
- A group of people who share a characteristic, such as a diagnosis, and are studied together. 'Prospective' means they are followed forward from enrolment; 'retrospective' means existing records are reviewed.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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