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.
Study Type
OBSERVATIONAL
Enrollment
20
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.
Amicus Therapeutics, Inc. Pregnancy Registry
Philadelphia, Pennsylvania, United States
RECRUITINGNumber 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
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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