Clinical trial NCT04252066: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Official title: A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Fabry Disease
Keywords: migalastat
Fabry Disease
Women with Fabry Disease
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-02-05
Enrollment: 20
Sex: FEMALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pregnant and/or breastfeeding patients with Fabry disease

Brief 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.

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.

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

Primary outcomes:
measure: Number of major birth defects
timeFrame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Secondary outcomes:
measure: Incidence of spontaneous abortion
timeFrame: : up to 20 weeks
measure: Number of elective or induced abortion
timeFrame: Through the pregnancy, an average of 40 weeks
measure: Number of fetal death or stillbirth
timeFrame: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week
measure: Number of live birth
timeFrame: at the delivery, an average of 40 weeks of pregnancy
measure: Number of neonatal death
timeFrame: up to 28 days of neonatal life
measure: Number of minor birth defects
timeFrame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
measure: Number of ectopic or molar pregnancy
timeFrame: Through the pregnancy, an average of 40 weeks
measure: Neurodevelopmental problems
timeFrame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
measure: Adverse fetal outcomes other than birth defects
timeFrame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
measure: Number of obstetric and delivery complications
timeFrame: At the delivery, an average of 40 weeks of pregnancy
measure: Occurrence of Jaundice cases in Infants
timeFrame: Up to 1 year
measure: Number of hospitalizations in infants
timeFrame: Up to 1 year
measure: Mortality in Infants
timeFrame: Up to 1 year
measure: Head circumference in Infants (cm)
timeFrame: Up to 1 year
measure: Weight in Infants (kilograms)
timeFrame: Up to 1 year
measure: Length in Infants (cm)
timeFrame: Up to 1 year
measure: Occurrence of milk allergic reaction in breastfed or formula supplemented infants
timeFrame: Up to 1 year
measure: Occurrence of allergic reaction in patients who are breastfeeding
timeFrame: Up to 1 year
measure: Frequency of adverse events effecting lactation
timeFrame: Up to 1 year
measure: Incidence of all serious adverse events
timeFrame: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
Other outcomes:
Locations:
city: Philadelphia
country: United States
facility: Amicus Therapeutics, Inc. Pregnancy Registry
state: Pennsylvania
Study officials:
References: