The Eplontersen Pregnancy and Lactation Outcomes Study

NCT07205666 · tracked via the Priya Life Science Germany tracker

Sponsor

AstraZeneca

Phase

Observational

Started

2025-11-30

Last updated

2026-08-27

Condition(s) studied

Transthyretin Amyloidosis

Study summary

The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to eplontersen during pregnancy and/or lactation.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria: * all pregnancy and/or lactation cases with exposure to eplontersen and a diagnosis of an approved indication for treatment with eplontersen * all adverse event reports in infants in the first 12 months of age that are or can be linked to pregnancy or lactation reports in individuals previously diagnosed with an approved indication and exposed to eplontersen during pregnancy or lactaction Exclusion Criteria: * all case reports considered invalid (i.e. minimum data is not provided at first report nor follow-up), or where reporter indicates that they do not wish to be contacted to obtain follow-up information, or the reporter/patient cannot be identified

Primary outcome measure(s)

Major congenital malformations — November 2025 - December 2035
All major congenital malformations diagnosed in utero, at birth or during the the first year of life. Considering the small number of patients anticipated to be included in this study, it is possible that only case narratives will be developed. However, if at least ten exposed pregnancies are reported, the data will be summarized quantitatively, using relevant measures. Major congenital malformation will be reported as numbers and proportions. When presented, the 95% confidence intervals (CIs) for proportion of the outcome of interest will be constructed using the exact (Clopper-Pearson) method.

Trial sites (1)

Facility	City	Region	Status
Research Site	Frankfurt	Germany	Recruiting

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Other trials for the same condition

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis Recruiting

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

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07205666 on ClinicalTrials.gov ↗ ← All trials in Germany