The Eplontersen Pregnancy and Lactation Outcomes Study
ConditionTransthyretin Amyloidosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAstraZeneca
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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
Trial 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
Trial design
Design model
Case-only
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
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.
Time frame
November 2025 - December 2035Secondary outcomes
1
composite outcome of pregnancy and infant adverse outcomes ( except major congenital malformation
pregnancy complications, spontaneous abortion, stillbirth, induced abortion, preterm birth, small for gestational age, postnatal growth deficiencies, infant development delay, neonatal mortality, infant mortality, as well as any other infant adverse events
Time frame
November 2025-December 2035Sponsors and contacts
Click on the lead sponsor to view all of their trials.
AstraZeneca
Lead sponsor
ICON plc
Collaborator
Source ClinicalTrials.gov