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

Description

This is a global, decentralized, single-arm, prospective and retrospective observational registry designed to evaluate pregnancy and infant outcomes in women with Fabry disease who have been exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The registry aims to assess maternal and infant safety, pregnancy outcomes, and the occurrence of congenital malformations and other neonatal conditions.

The registry will enroll patients over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data collection will be facilitated through a secure, centralized web-based platform, where patients and physicians can enter information using electronic case report forms (eCRFs).

Enrollment & Data Collection:

Patients can be enrolled at any time, either during pregnancy or after delivery. Depending on the timing of enrollment, data will be collected retrospectively and/or prospectively.

Pregnancy and clinical outcomes will be monitored from enrollment until the infant reaches 12 months of age.

Collected data includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health parameters.

Reported congenital malformations will be classified according to established criteria (e.g., MACDP, EUROCAT) and adjudicated by an independent Scientific Advisory Committee.

The registry is observational and does not alter clinical care, physician treatment decisions, or patient management.