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

ConditionsFabry Disease Pregnancy Pregnancy Complications

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexFemale

AgeNot listed

SponsorChiesi Farmaceutici S.p.A.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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.

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.

Disqualifiers

None

Trial 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.

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

Not applicable- observational study
Other intervention
Not applicable - observational study

Treatment groups

10 Participants

are divided into 1 treatment group

Group A: Women with Fabry disease who were exposed to pegunigalsidase alfa during pregnancy and/or lactation1 intervention

Trial outcomes

Primary outcomes

Pregnancy outcome: Number of live births

Time frame

at the delivery, after an average of 40 weeks of pregnancy

Pregnancy outcome: Number of preterm birth

Time frame

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)

Time frame

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