Observational study of women with Fabry disease during pregnancy and breastfeeding
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
This study looks at how pregnancy and breastfeeding affect women with Fabry disease and their babies, comparing those who have taken a treatment called migalastat to those who haven't.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 20 (estimated) |
| Sex | Female |
| Sponsor | Amicus Therapeutics Industry-sponsored |
| Locations | 1 site (Philadelphia, Pennsylvania) |
| Trial ID | NCT04252066 on ClinicalTrials.gov |
What this trial studies
This global prospective observational study focuses on women with Fabry disease and their infants during pregnancy and breastfeeding. It aims to evaluate the outcomes of these experiences in women and infants who have been exposed to the treatment migalastat. The study will enroll two cohorts: one of women who have been treated with migalastat and another of those who have not. Data will be collected over a minimum of 10 years, with ongoing follow-up through the Pregnancy Coordinating Center.
Who should consider this trial
Good fit: Ideal candidates for this study are pregnant and/or breastfeeding women diagnosed with Fabry disease, regardless of their exposure to migalastat.
Not a fit: Patients who are not pregnant or breastfeeding, or those without a diagnosis of Fabry disease, may not receive any benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the safety and outcomes of migalastat exposure during pregnancy and breastfeeding for women with Fabry disease.
How similar studies have performed: While this study is observational and focuses on a specific population, similar studies have shown success in understanding treatment outcomes in pregnant women with chronic conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Philadelphia, Pennsylvania
- Amicus Therapeutics, Inc. Pregnancy Registry — Philadelphia, Pennsylvania, United States (Recruiting)
Study contacts
- Study coordinator: Pregnancy Registry Call Center
- Email: galafoldpregnancy@ubc.com
- Phone: 888-239-0758
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.