Observational Study of Fabry Disease in Pregnancy and Breastfeeding

This study is looking at how pregnancy and breastfeeding affect women with Fabry disease and their babies. It's also observing the effects of a medication called migalastat during this time. You can join if you are a pregnant or breastfeeding woman with Fabry disease, whether or not you are taking migalastat. The main goal is to see how many major birth defects occur in babies. This is an observational study, meaning researchers will collect information without giving you a specific treatment. The study is ongoing and plans to enroll 20 participants.

Study design
This is a global observational study with two groups of participants, aiming to enroll 20 women. One group will have taken migalastat during pregnancy or breastfeeding, and the other will not have.
What's involved
Healthcare providers, patients, or secondary contacts will voluntarily report cases to a coordinating center. The coordinating center will then follow you throughout your pregnancy and/or breastfeeding period, and your infant will be followed until 1 year of age.
Compensation
Not stated in the trial record.
Follow-up
Your baby will be followed for up to 12 months of age after birth.

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NCT04252066

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

Recruiting
Not specifiedAll AgesObservational
Amicus Therapeutics
~20 participants
Updated 2025-07-08 on ClinicalTrials.gov
What's tested:migalastat

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of major birth defects
Measured over Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Fabry Disease
1 sites across 1 states
Pennsylvania1

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  • Number of major birth defectsThrough the pregnancy, an average of 40 weeks and up to 12 months of infant age