ClinicalTrials.gov record NCT07205666
The Eplontersen Pregnancy and Lactation Outcomes Study
The Eplontersen Pregnancy and Lactation Outcomes Study (EPPRO): A Descriptive Safety Study of Pregnant and Lactating Individuals and Their Offspring Exposed to Eplontersen
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a descriptive, observational safety study of people exposed to eplontersen during pregnancy or lactation and their infants. It uses existing safety reports and other sources to record pregnancy complications, fetal and neonatal outcomes, and infant events. If few cases are available, reporting may be limited to individual case summaries rather than formal statistical analysis.
What it is studying. The study aims to describe and summarize pregnancy, birth, neonatal, and infant outcomes seen after exposure to eplontersen during pregnancy or breastfeeding.
What the researchers hope to learn. To add to knowledge about maternal, fetal, neonatal, and infant outcomes after eplontersen exposure and to meet post-marketing safety reporting requirements. The study will summarize occurrences such as major congenital malformations and a composite of pregnancy and infant adverse outcomes.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Show the detailed description from the registry
Currently, there are no clinical studies of eplontersen use in pregnant individuals. Although it is expected that exposure to eplontersen during pregnancy or lactation is very rare, it is important to capture as many exposed cases as possible. This descriptive pregnancy safety study (DPSS) will add to the current body of knowledge regarding the safety of eplontersen exposure during pregnancy and lactation and maternal, fetal, and infant outcomes following exposure to eplontersen. The study is designed to fulfill health authorities' post-marketing requirements. This DPSS will analyze secondary data on cases of eplontersen-exposed pregnant and lactating individuals collected through a PRegnancy outcomes Intensive Monitoring (PRIM) enhanced pharmacovigilance (PV) approach utilizing AstraZeneca's PV Global Safety Database (PV-Argus). Data sources: All eplontersen-exposed cases reported to AstraZeneca PV-Argus as notifiable events related to pregnancy and lactation; and cases reported from clinical trials, spontaneous post-marketing reports, post-marketing observational studies, patient-oriented programs, published literature, and personal communication by healthcare providers will be eligible for inclusion in the analysis. Data on eplontersen-exposed cases will be captured and processed using a pre-specified follow-up schedule and questionnaires. In case of missing data, there will be several attempts to collect data using all contact methods supplied by the reporters at the time of initial report. Study size: This DPSS is descriptive in nature, and a formal sample size calculation is not performed. Feasibility calculations indicate that the DPSS may capture five exposed pregnancies over the study period. Data analysis: Analyses will be conducted in accordance with the study objectives, table/listing shells, and applicable guidelines. Demographics, medical and obstetric history, and disease characteristics will be summarized using descriptive statistics. The outcomes will be reported as a proportion and 95% confidence interval (if applicable) and calculated by dividing the number of cases of the outcome by the appropriate denominator for the particular outcome. If fewer than ten cases are reported, formal analyses will not be performed, and reporting will be limited to case narratives.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is an observational safety study that uses existing pharmacovigilance and published reports; it does not assign treatment. If a person or infant's case is included, the study team will extract medical, obstetric, and outcome information and may use pre-specified questionnaires. When information is missing, study staff may attempt follow-up contact using the reporter's contact details supplied at the time of the original report. The registry does not specify additional tests or invasive procedures as part of participation.
How the study is structured. A prospective, case-only observational study using secondary data from AstraZeneca's global safety database and other reports (clinical trials, published reports, spontaneous post-marketing reports, and communications). Reported cases will be followed using a pre-specified schedule and questionnaires when possible; missing information will be pursued using available reporter contact details. Analyses are descriptive; if fewer than ten exposed pregnancies are captured, results may be presented as individual case narratives.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The registry lists this study under Transthyretin Amyloidosis. Does it apply to my diagnosis?
- This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
- The listed sites are in Germany. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on ATTRv amyloidosis
This trial is also listed on this site under wild-type ATTR amyloidosis.
Important dates
- Study start
- Nov 30, 2025
- Primary completion
- Dec 30, 2035 (estimated)
- Study completion
- Dec 30, 2035 (estimated)
- Target enrollment
- 10 participants (estimated)
- Last updated on registry
- Aug 27, 2026
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- AstraZeneca
- Collaborators
- ICON plc
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- AstraZeneca Clinical Study Information Center 📞 1-877-240-9479 ✉ information.center@astrazeneca.com
Participating sites (1)
- Research SiteRecruiting
Frankfurt · Germany
Terms used on this page · explanations by MedicalUpdates.ai
- adverse event
- Any unwanted medical problem that happens during a study, whether or not it was caused by the treatment. A 'serious' adverse event is one that meets specific criteria, such as causing death, being life-threatening, requiring or prolonging hospital admission, causing significant disability, or another medically important outcome.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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