Featured Trial

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

Study Identifier:
CLI-06657AA1-06
CT.gov Identifier
NCT06941025
EudraCT Identifier:
N/A
Study Contact Information

Recruiting

Protocol Summary

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.

his 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.

Trial Details

Medical Condition
Fabry Disease
Pregnancy
Study Drug
N/A
Age
N/A
Phase
N/A
Type
Observational
Trial Dates
May 2025 - Dec 2034

Trial Participant Requirements

Age
N/A
Sex
Female
Healthy Volunteers
No

Key Inclusion and Exclusion Criteria

Participants Must:

  • Sex: Female

Requirements information
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

Study Locations

Location
Status
Location
No physical study sites - Decentralized, web-based registry
Washington D.C., District of Columbia, United States, 20001
Status
Recruiting
Location
No physical study sites - Decentralized, web-based registry
Berlin, Germany
Status
Recruiting
Location
No physical study sites - Decentralized, web-based registry
Rome, Italy
Status
Recruiting
Location
No physical study sites - Decentralized, web-based registry
Madrid, Spain
Status
Recruiting
Location
No physical study sites - Decentralized, web-based registry
London, United Kingdom
Status
Not yet recruiting