An observational study in Fabry Disease, Pregnancy and Pregnancy Complications, sponsored by Chiesi Farmaceutici S.p.A.. Recruiting at 5 sites in 5 countries. Open to female participants. Per ClinicalTrials.gov, last updated 2026-03-19.
Sponsored by Chiesi Farmaceutici S.p.A. · Observational
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:
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.
This is a global, decentralized, single-arm, prospective and retrospective observational registry designed to evaluate pregnancy and infant outcomes in women with Fabry disease who have been exposed to pegunigalsidase alfa within 30 days prior to conception and/or during pregnancy and lactation. The registry aims to assess maternal and infant safety, pregnancy outcomes, and the occurrence of congenital malformations and other neonatal conditions.
The registry will enroll patients over a 10-year period. Eligible patients may be enrolled by their physician or may self-enroll, where permitted by local regulations. Data collection will be facilitated through a secure, centralized web-based platform, where patients and physicians can enter information using electronic case report forms (eCRFs).
Enrollment \& Data Collection:
Patients can be enrolled at any time, either during pregnancy or after delivery. Depending on the timing of enrollment, data will be collected retrospectively and/or prospectively.
Pregnancy and clinical outcomes will be monitored from enrollment until the infant reaches 12 months of age.
Collected data includes maternal health, pregnancy complications, delivery outcomes, congenital malformations, and infant health parameters.
Reported congenital malformations will be classified according to established criteria (e.g., MACDP, EUROCAT) and adjudicated by an independent Scientific Advisory Committee.
The registry is observational and does not alter clinical care, physician treatment decisions, or patient management.
242 studies on the registry are indexed under Fabry Disease; 54 are open to participants now.
This study's planned enrollment of 10 is below the median of 100 across 123 observational studies indexed under Fabry Disease.
Browse Fabry Disease studies →Chiesi Farmaceutici S.p.A. is the lead sponsor of 182 studies on the registry; 22 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 11 (65%) have results posted.
Counted across the registry records on this site, refreshed daily.
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.
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
Exclusion Criteria:
Other: Not applicable- observational study
Not applicable - observational study
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
Number of neonates/infants with MCMs
Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of ectopic or molar pregnancies
Time frame: through the pregnancy, an average of 40 weeks
Number of women with obstetric and delivery complications
Time frame: at the delivery, an average of 40 weeks of pregnancy
Number of women with complications of preeclampsia or eclampsia
Time frame: through the pregnancy, an average of 40 weeks
Number of women with complications of preterm prelabour rupture of membrane
Time frame: at delivery, prior to 37 weeks of gestation
Number of neonates/infants with minor congenital malformations
Time frame: through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of infants with developmental deficiency
Time frame: up to 12 months of infant age
Number of hospitalisations in infants
Time frame: up to 12 months of infant age
Mortality in infants, including neonatal death and infant death
Time frame: up to 12 months of infant age
Head circumference in infants (cm)
Time frame: up to 12 months of infant age
Weight in infants (kilograms)
Time frame: up to 12 months of infant age
Length in infants (cm)
Time frame: up to 12 months of infant age
Number of infants born as SGA
Time frame: up to 12 months of infant age
Number of infants with postnatal growth deficiency or FTT
Time frame: up to 12 months of infant age
Duration of breastfeeding, number of exclusively breastfeeding women and number of breastfeeding women supplemented with formula
Time frame: up to 12 months of infant age
Number of adverse events in infants exposed to pegunigalsidase alfa during breastfeeding
Time frame: up to 12 months of infant age
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Chiesi Farmaceutici S.p.A.