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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04252066
• Other study ID #: AT1001-037
• Status: Recruiting
• Start date: April 17, 2020
• Est. completion date: February 2030

Study information

• Verified date: February 2024
• Source: Amicus Therapeutics
• Contact: Pregnancy Registry Call Center
• Phone: 888-239-0758
• Email: galafoldpregnancy[@]ubc.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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. All pregnant women with Fabry disease are eligible to enroll, an unexposed cohort potentially can be used for comparisons.

Cases will be reported voluntarily to the Pregnancy Coordinating Center (PCC) from any country by Healthcare Providers (HCPs), by patients and secondary contacts. The PCC will follow patients throughout their pregnancies and/or breastfeeding and infant through 1 year of age.

There will be 2 cohorts enrolled in the study. 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 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.

This is an observational study, it will enroll patients and collect data as described in this protocol for a minimum of 10 years.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: February 2030
• Est. primary completion date: August 2029
• Accepts healthy volunteers: No
• Gender: Female
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
United States	Amicus Therapeutics, Inc. Pregnancy Registry	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of major birth defects		Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Secondary	Incidence of spontaneous abortion		: up to 20 weeks
Secondary	Number of elective or induced abortion		Through the pregnancy, an average of 40 weeks
Secondary	Number of fetal death or stillbirth		Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week
Secondary	Number of live birth		at the delivery, an average of 40 weeks of pregnancy
Secondary	Number of neonatal death		up to 28 days of neonatal life
Secondary	Number of minor birth defects		Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Secondary	Number of ectopic or molar pregnancy	an ectopic or molar pregnancy occurs outside of the uterus.	Through the pregnancy, an average of 40 weeks
Secondary	Neurodevelopmental problems		Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Secondary	Adverse fetal outcomes other than birth defects		Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Secondary	Number of obstetric and delivery complications		At the delivery, an average of 40 weeks of pregnancy
Secondary	Occurrence of Jaundice cases in Infants		Up to 1 year
Secondary	Number of hospitalizations in infants		Up to 1 year
Secondary	Mortality in Infants		Up to 1 year
Secondary	Head circumference in Infants (cm)		Up to 1 year
Secondary	Weight in Infants (kilograms)		Up to 1 year
Secondary	Length in Infants (cm)		Up to 1 year
Secondary	Occurrence of milk allergic reaction in breastfed or formula supplemented infants		Up to 1 year
Secondary	Occurrence of allergic reaction in patients who are breastfeeding		Up to 1 year
Secondary	Frequency of adverse events effecting lactation		Up to 1 year
Secondary	Incidence of all serious adverse events		Through the pregnancy, an average of 40 weeks and up to 12 months of infant age