Transthyretin AmyloidosissTTRingNCT07654855
Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
An observational study of Transthyretin Amyloidosis and Transthyretin Amyloidosis With Cardiomyopathy, run by AstraZeneca at 6 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- What is being tested?
- None ( observational study )
- How many people
- 320 participants
- Where
- 6 sitesin 6 countries
- Study dates
- 1 Sep 2026to 29 Jan 2032
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
6 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
AstraZeneca Clinical Study Information Center contact
6 sites in 6 countries
United States1 site, 1 recruiting
- Recruiting
Research Site
Los Angeles, California, 90095
Canada1 site, 1 recruiting
- Recruiting
Research Site
Vancouver, British Columbia, V6Z 1Y6
About this study
Official title: 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)
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.
Read the detailed description
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.
- Conditions
- Transthyretin Amyloidosis, Transthyretin Amyloidosis With Cardiomyopathy, Transthyretin Amyloidosis With Polyneuropathy
- Being tested
- None ( observational study )
- Keywords
- ATTR, Transthyretin amyloidosis
- Medical subject headings
- Amyloidosis, Hereditary, Transthyretin-Related
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
None ( observational study )Other
Not applicable this is observational study no intervention is planned.
| Study group | Role | What they receive |
|---|---|---|
| Eplontersen users | D8450R00003 population of new and prior eplontersen users (including prior liver transplant and severe hepatic impairment subpopulations) Receives: Other: None ( observational study ) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Demographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment
- Prevalence of liver transplant prior to eplontersen initiation (overall by reason for liver transplant),
- 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)).
Measured: 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:
- 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.
Measured: Quarter 2 2026- Quarter 1 2032
Secondary outcomes (2)
Eplontersen treatment use
To describe eplontersen treatment use, including duration of treatment and reasons for discontinuation (overall, in patients with prior liver transplant, in patients with pre-existing severe hepatic impairment). These outcomes are all relative to secondary objective 1 of the study which is to describe "eplontersen treatment use".
Measured: Quarter 2 2026- Quarter 1 2032
Long-term safety in patients who initiate eplontersen treatment by subgroups
Same as primary objective 2, separately in i) patients with prior liver transplant and ii) patients with pre-existing severe hepatic impairment. These outcomes are relative to primary objective 2 of the study and to the same outcome measure, i.e. "long-term safety in patients who initiated eplontersen treatment". This is a long-term safety study without a prespecified adverse event of interest; therefore, all safety events are in scope, as defined in the protocol: all serious adverse events identified; other adverse events, including any new diagnosis reported as a co-morbidity, new sign or symptom, or new abnormal laboratory value within 115 days of last eplontersen dose. To support systematic and comprehensive listing of all AEs, clinical data in D8450R00003, in addition to serious adverse events, will be coded using MedDRA. Clinical data that cannot be mapped to MedDRA will also be considered.
Measured: Quarter 2 2026- Quarter 1 2032
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2026
- Study start1 Sep 2026
- Primary completion29 Jan 2032 (estimated)
- Study completion29 Jan 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Transthyretin AmyloidosisStudies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 320 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ICON Clinical Research
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07654855
- Sponsor's protocol ID
- D8450R00022
- Acronym
- sTTRing
- Status last verified
- Sep 2026
- First submitted
- 19 May 2026
- Last updated
- 14 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No