A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
NCT ID: NCT04252066
Last Updated: 2025-07-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
20 participants
OBSERVATIONAL
2025-03-18
2030-02-28
Brief Summary
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Detailed Description
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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.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Cohort 1
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.
migalastat
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.
Cohort 2
Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.
No interventions assigned to this group
Interventions
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migalastat
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.
Eligibility Criteria
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Inclusion Criteria
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
FEMALE
No
Sponsors
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Amicus Therapeutics
INDUSTRY
Responsible Party
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Locations
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Amicus Therapeutics, Inc. Pregnancy Registry
Philadelphia, Pennsylvania, United States
Countries
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Central Contacts
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Other Identifiers
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AT1001-037
Identifier Type: -
Identifier Source: org_study_id
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