A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
- Registration Number
- NCT04252066
- Lead Sponsor
- Amicus Therapeutics
- Brief Summary
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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Female
- Target Recruitment
- 20
Female patients meeting the following criteria will be eligible for study enrollment:
- Patients with Fabry disease who are pregnant and/or breastfeeding, whether or not they are exposed to migalastat
- Able and willing to provide informed consent or assent, if applicable.
- Able and willing to provide HCP contact information.
None
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Cohort 1 migalastat 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.
- Primary Outcome Measures
Name Time Method Number of major birth defects Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
- Secondary Outcome Measures
Name Time Method Incidence of spontaneous abortion : up to 20 weeks Number of elective or induced abortion Through the pregnancy, an average of 40 weeks Number of fetal death or stillbirth Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week Number of live birth at the delivery, an average of 40 weeks of pregnancy Number of neonatal death up to 28 days of neonatal life Number of minor birth defects Through the pregnancy, an average of 40 weeks and up to 12 months of infant age Weight in Infants (kilograms) Up to 1 year Length in Infants (cm) Up to 1 year Frequency of adverse events effecting lactation Up to 1 year Incidence of all serious adverse events Through the pregnancy, an average of 40 weeks and up to 12 months of infant age Neurodevelopmental problems Through the pregnancy, an average of 40 weeks and up to 12 months of infant age Number of obstetric and delivery complications At the delivery, an average of 40 weeks of pregnancy Occurrence of milk allergic reaction in breastfed or formula supplemented infants Up to 1 year Occurrence of allergic reaction in patients who are breastfeeding Up to 1 year Number of ectopic or molar pregnancy Through the pregnancy, an average of 40 weeks an ectopic or molar pregnancy occurs outside of the uterus.
Adverse fetal outcomes other than birth defects Through the pregnancy, an average of 40 weeks and up to 12 months of infant age Mortality in Infants Up to 1 year Head circumference in Infants (cm) Up to 1 year Occurrence of Jaundice cases in Infants Up to 1 year Number of hospitalizations in infants Up to 1 year
Trial Locations
- Locations (1)
Amicus Therapeutics, Inc. Pregnancy Registry
🇺🇸Philadelphia, Pennsylvania, United States