Global Observational Study of Pregnant or Breastfeeding Women With Fabry Disease and Their Infants, Including Those Exposed to Migalastat

What this Trial Is About

Researchers are conducting a global prospective observational study involving women with Fabry disease who are pregnant or breastfeeding, along with their infants. The study aims to evaluate outcomes related to pregnancy and breastfeeding in women and infants who have been exposed to the drug migalastat. An unexposed group of women with Fabry disease may also be included for comparison. This study will collect data over a minimum of 10 years to gain comprehensive insights. The study includes two groups Cohort 1 consists of pregnant or breastfeeding women with Fabry disease who have taken at least one dose of migalastat during this time, while Cohort 2 includes similar women who have not been exposed to migalastat. Participants are reported voluntarily from any country by healthcare providers, patients, or secondary contacts. The Pregnancy Coordinating Center will follow the mothers through pregnancy and breastfeeding, and their infants up to one year old. Participants will have their pregnancies and infants monitored for various outcomes such as birth defects, miscarriage, fetal death, delivery complications, infant growth measurements, allergic reactions, hospitalizations, and mortality up to one year of age. Data collection includes monitoring major and minor birth defects, neurodevelopmental problems, jaundice, and serious adverse events in both mothers and infants. This long-term follow-up helps researchers understand the effects of migalastat exposure during pregnancy and breastfeeding.

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