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

Last updated: July 2, 2025
Sponsor: Amicus Therapeutics
Overall Status: Active - Recruiting

Phase

N/A

Condition

Fabry Disease

Kidney Disease

Treatment

migalastat

Clinical Study ID

NCT04252066
AT1001-037
  • Female

Study 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.

Eligibility Criteria

Inclusion

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 notthey 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

Exclusion Criteria:

None

Study Design

Total Participants: 20
Treatment Group(s): 1
Primary Treatment: migalastat
Phase:
Study Start date:
March 18, 2025
Estimated Completion Date:
February 28, 2030

Study 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.

Connect with a study center

  • Amicus Therapeutics, Inc. Pregnancy Registry

    Cranbury, New Jersey 08512
    United States

    Site Not Available

  • Amicus Therapeutics, Inc. Pregnancy Registry

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

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