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Search / Trial NCT04252066

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

Launched by AMICUS THERAPEUTICS · Jan 30, 2020

Trial Information

Current as of July 24, 2025

Recruiting

Keywords

Migalastat Fabry Disease Women With Fabry Disease

ClinConnect Summary

This clinical trial is exploring the experiences of women with Fabry disease who are pregnant or breastfeeding, along with their infants. Specifically, the study looks at how these women and their babies are affected by a treatment called migalastat, which some of them may be using. The goal is to gather valuable information on pregnancy and breastfeeding outcomes in this unique group of women.

To participate in this study, women must be diagnosed with Fabry disease and currently be pregnant or breastfeeding, regardless of whether they are taking migalastat. There are no specific exclusions, meaning most women with these conditions can join. Participants will be asked to share their experiences and health information during the study. This research is important as it aims to better understand the effects of Fabry disease and its treatment on both mothers and their infants, helping to improve care for future patients.

Gender

FEMALE

Eligibility 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

About Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company dedicated to developing innovative therapies for rare and orphan diseases, particularly those caused by genetic mutations. With a focus on lysosomal storage disorders, Amicus leverages advanced protein engineering and gene therapy approaches to create treatment options that address unmet medical needs. Committed to patient-centric research, the company collaborates with healthcare professionals and patient advocacy groups to ensure that its clinical trials are designed to deliver meaningful outcomes. Through its robust pipeline and dedication to scientific excellence, Amicus Therapeutics aims to enhance the quality of life for individuals affected by these complex conditions.

Locations

Philadelphia, Pennsylvania, United States

Patients applied

0 patients applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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