The Eplontersen Pregnancy and Lactation Outcomes Study

NCT07205666 | Recruiting

• 1 other identifier: D8451R00002
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Transthyretin Amyloidosis

Keywords

Eplontersen; pregnancy; Wainua; Wainzua

Outcome Measures

Primary Outcomes (1)

• Major congenital malformations

  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.

  November 2025 - December 2035

Secondary Outcomes (1)

• composite outcome of pregnancy and infant adverse outcomes ( except major congenital malformation

  November 2025-December 2035

Eligibility Criteria

• Gender Eligibility Details: Pregnant and/or lactating individuals
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include all PV reports of pregnant and/or lactating individuals diagnosed with an indication approved for use of eplontersen who administered at least one dose of eplontersen during pregnancy or lactation

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AstraZeneca: lead
• ICON plc: collaborator

Study Sites (1)

Research Site

Frankfurt, Germany

RECRUITING

Related Publications (1)

• Geissbuhler Y, Rezaallah B, Moore A. An alternative to product-specific pregnancy registries? PRIM; PRegnancy outcomes Intensive Monitoring. Reprod Toxicol. 2020 Jun;94:13-21. doi: 10.1016/j.reprotox.2020.03.004. Epub 2020 Mar 10.

  PMID: 32169556 RESULT

MeSH Terms

Conditions

Amyloidosis, Hereditary, Transthyretin-Related

Study Officials

• Myriam Alexander, PHD

  AstraZeneca

  PRINCIPAL INVESTIGATOR

Central Study Contacts

AstraZeneca Clinical Study Information Center

CONTACT

1-877-240-9479; information.center@astrazeneca.com

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 17, 2025
• First Posted: October 3, 2025
• Study Start: November 30, 2025
• Primary Completion (Estimated): December 30, 2035
• Study Completion (Estimated): December 30, 2035
• Last Updated: May 1, 2026

Record last verified: 2026-04

Locations

DE (1)