An observational study in Fabry Disease, sponsored by Amicus Therapeutics. Recruiting at 1 site in United States. Open to female participants. Per ClinicalTrials.gov, last updated 2026-10-28.
Sponsored by Amicus Therapeutics (part of BioMarin Pharmaceutical) · Observational
This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat.
This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat. All pregnant women with Fabry disease are eligible to enroll, an unexposed cohort potentially can be used for comparisons.
Cases will be reported voluntarily to the Pregnancy Coordinating Center (PCC) from any country by Healthcare Providers (HCPs), by patients and secondary contacts. The PCC will follow patients throughout their pregnancies and/or breastfeeding and infant through 1 year of age.
There will be 2 cohorts enrolled in the study. Cohort 1 will be pregnant and/or breastfeeding patients who have Fabry Disease, and have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding. Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.
This is an observational study, it will enroll patients and collect data as described in this protocol for a minimum of 10 years.
244 studies on the registry are indexed under Fabry Disease; 55 are open to participants now.
This study's planned enrollment of 20 is below the median of 100 across 122 observational studies indexed under Fabry Disease.
Browse Fabry Disease studies →Amicus Therapeutics is the lead sponsor of 42 studies on the registry; 8 are open to participants now.
First submitted before Amicus Therapeutics became part of BioMarin Pharmaceutical.
Counted across the registry records on this site, refreshed daily.
Pregnant and/or breastfeeding patients with Fabry disease
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Female patients meeting the following criteria will be eligible for study enrollment:
Exclusion Criteria:
None
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cohort 1 will be pregnant and/or breastfeeding patients who have Fabry Disease, and have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding.
Drug: migalastat
Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.
This is an observational study. Patients as described in Cohort 1 should have been exposed to at least 1 dose of migalastat during pregnancy and/or breastfeeding.
Number of major birth defects
Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Incidence of spontaneous abortion
Time frame: : up to 20 weeks
Number of elective or induced abortion
Time frame: Through the pregnancy, an average of 40 weeks
Number of fetal death or stillbirth
Time frame: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week
Number of live birth
Time frame: at the delivery, an average of 40 weeks of pregnancy
Number of neonatal death
Time frame: up to 28 days of neonatal life
Number of minor birth defects
Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of ectopic or molar pregnancy
an ectopic or molar pregnancy occurs outside of the uterus.
Time frame: Through the pregnancy, an average of 40 weeks
Neurodevelopmental problems
Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Adverse fetal outcomes other than birth defects
Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Number of obstetric and delivery complications
Time frame: At the delivery, an average of 40 weeks of pregnancy
Occurrence of Jaundice cases in Infants
Time frame: Up to 1 year
Number of hospitalizations in infants
Time frame: Up to 1 year
Mortality in Infants
Time frame: Up to 1 year
Head circumference in Infants (cm)
Time frame: Up to 1 year
Weight in Infants (kilograms)
Time frame: Up to 1 year
Length in Infants (cm)
Time frame: Up to 1 year
Occurrence of milk allergic reaction in breastfed or formula supplemented infants
Time frame: Up to 1 year
Occurrence of allergic reaction in patients who are breastfeeding
Time frame: Up to 1 year
Frequency of adverse events effecting lactation
Time frame: Up to 1 year
Incidence of all serious adverse events
Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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