A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Study Overview
=================
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.

Official Title
-----------------
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Conditions
-----------------
Fabry Disease

Intervention / Treatment
-----------------
* Drug: migalastat


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: 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. Exclusion Criteria: None
Sexes Eligible for Study
-----------------
Female

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Cohort 1<br>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<br>* 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.<br>|
| Cohort 2<br>Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding. | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of major birth defects |  | Through the pregnancy, an average of 40 weeks and up to 12 months of infant age | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Number of ectopic or molar pregnancy | an ectopic or molar pregnancy occurs outside of the uterus. | Through the pregnancy, an average of 40 weeks | 
| 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 | 
| Occurrence of Jaundice cases in Infants |  | Up to 1 year | 
| 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 | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
migalastat, Fabry Disease, Women with Fabry Disease