Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Launched by CHIESI FARMACEUTICI S.P.A. · Apr 22, 2025

Keywords

ClinConnect Summary

The Maternal and Postnatal Outcomes Study (MOS) is a clinical trial aimed at understanding how the medication Elfabrio® (also known as pegunigalsidase alfa) affects women with Fabry disease during pregnancy and breastfeeding. This study focuses on the health of the mothers and their infants, looking specifically at pregnancy outcomes and any potential birth defects or health issues in newborns. The trial will last for ten years and will collect information from women who have taken the medication within 30 days before becoming pregnant or during their pregnancy and breastfeeding period.

To be eligible for the study, women must have Fabry disease and have received at least one dose of Elfabrio® during pregnancy or while breastfeeding. Participants will share their experiences through a secure online system, and their information will be verified by their doctors. It’s important to note that this study is observational, meaning it will not change how participants receive their medical care. This trial is not currently recruiting participants, but it offers a valuable opportunity for women with Fabry disease to contribute to important research on their health and the health of their babies.

Gender

FEMALE

Eligibility criteria

• Inclusion Criteria:
• • Female patients with Fabry disease who have been exposed to at least 1 dose of pegunigalsidase alfa at any time during pregnancy (defined as having received pegunigalsidase alfa within 30 days prior to the DOC and/or during pregnancy) and/or during lactation, and their infants.
• • o DOC, defined as 20/7 gestational weeks, will be calculated from last menstrual period \[LMP\] or ultrasound
• • Patient or parent/legally authorized representative must be able to understand and provide consent through an Institutional Review Board / Independent Ethics Committee (IRB/IEC) approved Informed Consent Form.
• Exclusion Criteria:
• • None