Fabry Disease
Conditions
Keywords
migalastat, Fabry Disease, Women with Fabry Disease
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.
Interventions
DRUGmigalastat
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.
Sponsors
Amicus Therapeutics
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
FEMALE
Healthy volunteers
No
Inclusion criteria
Female patients meeting the following criteria will be eligible for study enrollment: 1. Patients with Fabry disease who are pregnant and/or breastfeeding, whether or not they are exposed to migalastat 2. Able and willing to provide informed consent or assent, if applicable. 3. Able and willing to provide HCP contact information.
Exclusion criteria
None
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of major birth defects | Through the pregnancy, an average of 40 weeks and up to 12 months of infant age |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| Number of ectopic or molar pregnancy | Through the pregnancy, an average of 40 weeks | an ectopic or molar pregnancy occurs outside of the uterus. |
| Neurodevelopmental problems | Through the pregnancy, an average of 40 weeks and up to 12 months of infant age | — |
| Adverse fetal outcomes other than birth defects | 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 | — |
| Incidence of spontaneous abortion | : up to 20 weeks | — |
| Number of hospitalizations in infants | Up to 1 year | — |
| Mortality in Infants | Up to 1 year | — |
| Head circumference in Infants (cm) | Up to 1 year | — |
| Weight in Infants (kilograms) | Up to 1 year | — |
| Length in Infants (cm) | Up to 1 year | — |
| 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 | — |
| 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 | — |
| Occurrence of Jaundice cases in Infants | Up to 1 year | — |
Countries
United States
Contacts
Primary ContactPregnancy Registry Call Center
Outcome results
None listed