A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: migalastat.
- Who it may be relevant to
- Registry conditions: Fabry Disease. Basic parameters: No limits · Female.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Overview
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.
Detailed 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.
Interventions
- 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.
Primary outcome measures
- Number of major birth defects [Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
Secondary outcome measures (12)
- Incidence of spontaneous abortion [Time frame: : up to 20 weeks]
- Number of elective or induced abortion [Time frame: Through the pregnancy, an average of 40 weeks]
- Number of fetal death or stillbirth [Time frame: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week]
- Number of live birth [Time frame: at the delivery, an average of 40 weeks of pregnancy]
- Number of neonatal death [Time frame: up to 28 days of neonatal life]
- Number of minor birth defects [Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
- Number of ectopic or molar pregnancy [Time frame: Through the pregnancy, an average of 40 weeks]
- Neurodevelopmental problems [Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
- Adverse fetal outcomes other than birth defects [Time frame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
- Number of obstetric and delivery complications [Time frame: At the delivery, an average of 40 weeks of pregnancy]
- Occurrence of Jaundice cases in Infants [Time frame: Up to 1 year]
- Number of hospitalizations in infants [Time frame: Up to 1 year]
Eligibility criteria
Inclusion criteria
Female patients meeting the following criteria will be eligible for study enrollment:
- Patients with Fabry disease who are pregnant and/or breastfeeding, whether or not they are exposed to migalastat
- Able and willing to provide informed consent or assent, if applicable.
- Able and willing to provide HCP contact information.
Exclusion criteria
None
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Amicus Therapeutics, Inc. Pregnancy Registry — Philadelphia
Identifiers
NCT: NCT04252066 · AT1001-037