Study Observing Pregnancy and Breastfeeding Outcomes in People Exposed to Eplontersen for Transthyretin Amyloidosis

What this Trial Is About

Researchers are observing pregnancy and breastfeeding outcomes in people exposed to eplontersen, a treatment for transthyretin amyloidosis. This study aims to describe the occurrence of pregnancy and maternal complications, effects on the developing fetus and newborn, and infant adverse events associated with eplontersen exposure during pregnancy or lactation. The study addresses a gap since no clinical studies currently exist for eplontersen use in pregnant individuals, helping to fulfill health authorities post-marketing safety requirements. The study collects secondary data from multiple sources, including reports submitted to AstraZenecas global safety database and cases from clinical trials, post-marketing reports, observational studies, literature, and healthcare providers. Data on exposed pregnancies and lactation cases are gathered using a predefined follow-up schedule and questionnaires, with repeated attempts to obtain missing information. The study is descriptive, expecting to capture about five exposed pregnancies over the planned period from November 2025 to December 2035. Participants provide data through adverse event reports linked to eplontersen exposure during pregnancy or lactation and infant events within the first year of life. Researchers summarize demographics, medical histories, and disease characteristics using descriptive statistics. Outcomes such as major congenital malformations and other pregnancy or infant adverse events will be reported as proportions with confidence intervals when possible. If fewer than ten cases are available, reporting will focus on detailed case narratives. The study monitors participants long-term until December 2035.

The Eplontersen Pregnancy and Lactation Outcomes Study