Clinical trial

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

NCT04252066

Name

AT1001-037

Description

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.

Trial arms

Trial start

2020-04-17

Estimated PCD

2029-08-01

Trial end

2030-02-01

Status

Recruiting

Treatment

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.

Arms:

Cohort 1

Size

Primary endpoint

Number of major birth defects

Through the pregnancy, an average of 40 weeks and up to 12 months of infant age

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

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 20, 'type': 'ESTIMATED'}}

Updated at

2024-02-08

1 organization

1 product

1 indication

Organization

Amicus Therapeutics

Product

migalastat

Indication

Fabry Disease