Pregnancy and postpartum outcomes after pegunigalsidase alfa exposure in women with Fabry disease
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
This registry will collect information to see how pregnancies and babies do when women with Fabry disease received pegunigalsidase alfa shortly before conception, during pregnancy, or while breastfeeding.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 10 (estimated) |
| Sex | Female |
| Sponsor | Chiesi Farmaceutici S.p.A. Industry-sponsored |
| Locations | 5 sites (Washington D.C., District of Columbia and 4 other locations) |
| Trial ID | NCT06941025 on ClinicalTrials.gov |
What this trial studies
This global, decentralized registry will collect both retrospective and prospective data on pregnancies and infant health when mothers with Fabry disease were exposed to pegunigalsidase alfa within 30 days prior to conception or during pregnancy or lactation. Enrollment is planned over a 10-year period and participants may be entered by their clinician or may self-enroll where local regulations permit, with data entered through a secure web-based platform and electronic case report forms. Pregnancy outcomes, delivery details, reported congenital malformations (adjudicated by an independent Scientific Advisory Committee), and infant health up to 12 months of age will be documented. The registry is observational, does not dictate treatment, and collects real-world safety and outcome data without exclusion criteria beyond exposure and ability to consent.
Who should consider this trial
Good fit: Women with Fabry disease who received at least one dose of pegunigalsidase alfa within 30 days before conception or at any time during pregnancy or lactation, and their infants, who can provide consent or have a legal representative, are ideal candidates.
Not a fit: Patients who were not exposed to pegunigalsidase alfa, whose exposure fell outside the defined window, who cannot provide consent, or who cannot complete follow-up are unlikely to gain useful data from this registry.
Why it matters
Potential benefit: If successful, the registry could provide clearer safety information to help clinicians and pregnant women with Fabry disease make informed treatment decisions about pegunigalsidase alfa during pregnancy and breastfeeding.
How similar studies have performed: Pregnancy registries for enzyme replacement therapies in other lysosomal storage disorders have generated useful safety signals, but published data specifically on pegunigalsidase alfa in pregnancy are limited.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Washington D.C., District of Columbia and 4 other locations
- No physical study sites - Decentralized, web-based registry — Washington D.C., District of Columbia, United States (Recruiting)
- No physical study sites - Decentralized, web-based registry — Berlin, Germany (Recruiting)
- No physical study sites - Decentralized, web-based registry — Rome, Italy (Recruiting)
- No physical study sites - Decentralized, web-based registry — Madrid, Spain (Recruiting)
- No physical study sites - Decentralized, web-based registry — London, United Kingdom (Not_yet_recruiting)
Study contacts
- Study coordinator: Chiesi Clinical Trial
- Email: clinicaltrials_info@chiesi.com
- Phone: +3905212791
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.