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

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Amicus Therapeutics
Study ID
NCT04252066
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
FEMALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • migalastat — DRUG
    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.

Study Details

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.

Key Dates

Start date
Mar 18, 2025
Status verified
Jul 2025
Primary completion
Aug 31, 2029
Completion
Feb 28, 2030

Study Design

Enrollment
20 participants (estimated)

Arms

  • Arm: 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.
  • Arm: 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.

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

Locations (1)

FacilityCityStateZIPSite coordinators
Amicus Therapeutics, Inc. Pregnancy RegistryPhiladelphiaPennsylvania19104-

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Amicus Therapeutics, Inc. Pregnancy Registry· Philadelphia, PA

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