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

Recruiting now

Conditions studied: Fabry Disease, Pregnancy, Pregnancy Complications

In brief

The goal of this observational registry is to evaluate the safety and outcomes of pregnancy and lactation in women with Fabry disease who are exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The main objectives are to: * Assess pregnancy outcomes, including maternal and infant health. * Evaluate the occurrence of congenital malformations and other neonatal outcomes. This is a global, decentralized, single-arm, prospective and retrospective registry planned to enroll participants over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data will be collected through a secure web-based platform, allowing patients and physicians to enter information via electronic case report forms (eCRFs). Pregnancy and clinical outcomes will be documented throughout pregnancy and up to 12 months post-birth. Data from self-enrolled patients will be confirmed by their primary care or attending physician. This registry is observational and does not impact clinical care or treatment decisions.

Key facts

Study ID

NCT06941025

Run by

Chiesi Farmaceutici S.p.A.

People needed

10

Starts

2025-05-23

Expected to finish

2034-12-01

Last updated by the study team

2026-03-19

Who can join

Age: any. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• None

Where it is running

• No physical study sites - Decentralized, web-based registry — Washington D.C., District of Columbia, United States (enrolling)
• No physical study sites - Decentralized, web-based registry — Berlin, Germany (enrolling)
• No physical study sites - Decentralized, web-based registry — Rome, Italy (enrolling)
• No physical study sites - Decentralized, web-based registry — Madrid, Spain (enrolling)
• No physical study sites - Decentralized, web-based registry — London, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.