Fabry Disease Clinical Trials

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Maternal and Postnatal Outcomes Study (MOS) A Worldwide Decentralized Observational Registry to Evaluate the Safety in Women With Fabry Disease and Their Infants Exposed to Elfabrio® (Pegunigalsidase Alfa-iwxj/Pegunigalsidase Alfa) During Pregnancy and/or Lactation

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Other
Study Type: Observational
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. 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.

Eligibility
Participation Requirements
Sex: Female
Healthy Volunteers: f
View:

• 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.

Locations
United States
Washington, D.c.
No physical study sites - Decentralized, web-based registry
RECRUITING
Washington D.c.
Other Locations
Germany
No physical study sites - Decentralized, web-based registry
RECRUITING
Berlin
Italy
No physical study sites - Decentralized, web-based registry
RECRUITING
Rome
Spain
No physical study sites - Decentralized, web-based registry
RECRUITING
Madrid
United Kingdom
No physical study sites - Decentralized, web-based registry
NOT_YET_RECRUITING
London
Contact Information
Primary
Chiesi Clinical Trial
clinicaltrials_info@chiesi.com
+3905212791
Time Frame
Start Date: 2025-05-23
Estimated Completion Date: 2034-12
Participants
Target number of participants: 10
Treatments
Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation
Related Therapeutic Areas
Sponsors
Collaborators: ICON plc
Leads: Chiesi Farmaceutici S.p.A.

This content was sourced from clinicaltrials.gov