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