Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at below P25 for all trials

Timeline

104mo left

Started May 2025

Longer than P75 for all trials

Geographic Reach

5 countries

5 active sites

Status

recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

9.5 years study duration

Study Progress10%

May 2025Dec 2034

First Submitted

Initial submission to the registry

April 16, 2025

Completed

7 days until next milestone

First Posted

Study publicly available on registry

April 23, 2025

Completed

1 month until next milestone

Study Start

First participant enrolled

May 23, 2025

Completed

9.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2034

Expected

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2034

Last Updated

March 19, 2026

Status Verified

March 1, 2026

Enrollment Period

9.5 years

First QC Date

April 16, 2025

Last Update Submit

March 17, 2026

Conditions

Fabry Disease Pregnancy Pregnancy Complications

Outcome Measures

Primary Outcomes (3)

Pregnancy outcome: Number of live births
at the delivery, after an average of 40 weeks of pregnancy
Pregnancy outcome: Number of preterm birth
at delivery, prior to 37 weeks of gestation
Pregnancy outcome: Number of pregnancy losses (number of spontaneous abortions , number of pregnancy terminations, number of foetal deaths or stillbirths)
spontaneous abortions: up to 20 weeks of pregnancy; pregnancy terminations: through the pregnancy; number of foetal deaths or stillbirths: greater than 20 weeks of pregnancy and through the pregnancy, average of 40 weeks

Secondary Outcomes (16)

Number of neonates/infants with MCMs
through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of ectopic or molar pregnancies
through the pregnancy, an average of 40 weeks
Number of women with obstetric and delivery complications
at the delivery, an average of 40 weeks of pregnancy
Number of women with complications of preeclampsia or eclampsia
through the pregnancy, an average of 40 weeks
Number of women with complications of preterm prelabour rupture of membrane
at delivery, prior to 37 weeks of gestation
+11 more secondary outcomes

Study Arms (1)

Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation

Other: Not applicable- observational study

Interventions

Not applicable- observational studyOTHER

Not applicable - observational study

Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation

Eligibility Criteria

Sexfemale(Gender-based eligibility)

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

Pregnant and/or breastfeeding women with Fabry disease and their infants, after exposure to at least 1 dose of pegunigalsidase alfa during pregnancy and/or during lactation.

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

Contact the study team to confirm eligibility.