Eplontersen exposure during pregnancy and breastfeeding: outcomes
The Eplontersen Pregnancy and Lactation Outcomes Study (EPPRO): A Descriptive Safety Study of Pregnant and Lactating Individuals and Their Offspring Exposed to Eplontersen
This project collects reports to see if exposure to eplontersen during pregnancy or breastfeeding affects mothers, fetuses, or infants with transthyretin amyloidosis.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10 (estimated) |
| Sex | All |
| Sponsor | AstraZeneca Industry-sponsored |
| Locations | 1 site (Frankfurt) |
| Trial ID | NCT07205666 on ClinicalTrials.gov |
What this trial studies
This descriptive pregnancy safety study compiles secondary data on people exposed to eplontersen during pregnancy or lactation using AstraZeneca's PV‑Argus database and a PRegnancy outcomes Intensive Monitoring (PRIM) pharmacovigilance approach. Eligible cases include reports from clinical trials, spontaneous post‑marketing submissions, observational studies, patient programs, published literature, and healthcare provider communications. Data are captured via a prespecified follow‑up schedule and questionnaires, and multiple attempts will be made to obtain missing information from reporters. The analysis will summarize maternal, fetal, neonatal, and infant outcomes associated with eplontersen exposure.
Who should consider this trial
Good fit: People diagnosed with transthyretin amyloidosis who were exposed to eplontersen during pregnancy or lactation (or infants up to 12 months linked to such exposures) are the intended cases for inclusion.
Not a fit: Individuals who were not exposed to eplontersen during pregnancy or breastfeeding, or whose reports lack minimum or verifiable data, will not benefit from enrollment in this dataset.
Why it matters
Potential benefit: If successful, this work could improve knowledge about the safety of eplontersen in pregnancy and breastfeeding and help patients and clinicians make more informed treatment decisions.
How similar studies have performed: There are no clinical studies of eplontersen in pregnancy to date, but the PRIM/pharmacovigilance approach follows established post‑marketing pregnancy surveillance methods used for other drugs.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Frankfurt
- Research Site — Frankfurt, Germany (Recruiting)
Study contacts
- Principal investigator: Myriam Alexander, PHD — AstraZeneca
- Study coordinator: AstraZeneca Clinical Study Information Center
- Email: information.center@astrazeneca.com
- Phone: 1-877-240-9479
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.