Recruiting

Observational Study

Sponsor:

Amicus Therapeutics

Code:

NCT04252066

Conditions

Fabry Disease

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

Interventions

migalastat

Study Details

Brief 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.

Conditions

Fabry Disease

Study ID

NCT04252066

Start date

Mar 18, 2025

Status verified date

Jul, 2025

Completion date

Feb, 2030

Anticipated

Primary completion date

Aug, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Female

Age: 0+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

20 participants

Anticipated

Interventions and Outcome Measures

Arms

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.

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.

Interventions

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.

Primary outcome measure

  • Number of major birth defects [ Time Frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age ]

Central Contacts and Locations

Central contacts

Pregnancy Registry Call Center

888-239-0758galafoldpregnancy@ubc.com

Locations

Amicus Therapeutics, Inc. Pregnancy Registry

Recruiting

Philadelphia, Pennsylvania, United States, 19104

More Information

Sponsor

Amicus Therapeutics

Last update posted

Jul 8, 2025

Last verified

Jul, 2025

Keywords

  • migalastat
  • Fabry Disease
  • Women with Fabry Disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Amicus Therapeutics on 2025-07-08.