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

Amicus Therapeutics

Status: Enrolling

Conditions: Fabry Disease

Treatments: Drug: migalastat

Study type: Observational

Funder types: Industry

Identifiers

NCT04252066

AT1001-037

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 estimated patients

Sex: Female

Volunteers: No Healthy Volunteers

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

Trial design

20 participants in 2 patient groups

Cohort 1

Description:

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.

Treatment:

Drug: migalastat

Cohort 2

Description:

Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.