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

11. Juni 2026 aktualisiert von: AstraZeneca

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:

  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.

Studienübersicht

Status

Noch keine Rekrutierung

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

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.

Studientyp

Beobachtungs

Einschreibung (Geschätzt)

320

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Nein

Probenahmeverfahren

Nicht-Wahrscheinlichkeitsprobe

Studienpopulation

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.

Beschreibung

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.

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

Kohorten und Interventionen

Gruppe / Kohorte
Intervention / Behandlung
Eplontersen users
D8450R00003 population of new and prior eplontersen users (including prior liver transplant and severe hepatic impairment subpopulations)
Sonstiges: None ( observational study )
Not applicable this is observational study no intervention is planned.

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Demographic and clinical characteristics of eplontersen users, including the prevalence of prior liver transplant and severe hepatic impairment
Zeitfenster: Quarter 2 2026- Quarter 1 2032
  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
Zeitfenster: Quarter 2 2026- Quarter 1 2032

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

Sekundäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Eplontersen treatment use
Zeitfenster: Quarter 2 2026- Quarter 1 2032
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".
Quarter 2 2026- Quarter 1 2032
Long-term safety in patients who initiate eplontersen treatment by subgroups
Zeitfenster: Quarter 2 2026- Quarter 1 2032
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.
Quarter 2 2026- Quarter 1 2032

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

AstraZeneca

Mitarbeiter

ICON Clinical Research

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Geschätzt)

1. September 2026

Primärer Abschluss (Geschätzt)

29. Januar 2032

Studienabschluss (Geschätzt)

29. Januar 2032

Studienanmeldedaten

Zuerst eingereicht

19. Mai 2026

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

11. Juni 2026

Zuerst gepostet (Tatsächlich)

17. Juni 2026

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

17. Juni 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

11. Juni 2026

Zuletzt verifiziert

1. Juni 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Schlüsselwörter

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • D8450R00022

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Ja

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Produkt, das in den USA hergestellt und aus den USA exportiert wird

Nein

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