A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of major birth defects
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 性别
- Female
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- 未提供
研究组 & 干预措施
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.
干预措施: migalastat (Drug)
结局指标
主要结局
Number of major birth defects
时间窗: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age
次要结局
- Incidence of spontaneous abortion(: up to 20 weeks)
- Number of elective or induced abortion(Through the pregnancy, an average of 40 weeks)
- Number of fetal death or stillbirth(Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week)
- Number of live birth(at the delivery, an average of 40 weeks of pregnancy)
- Number of neonatal death(up to 28 days of neonatal life)
- Number of minor birth defects(Through the pregnancy, an average of 40 weeks and up to 12 months of infant age)
- Weight in Infants (kilograms)(Up to 1 year)
- Length in Infants (cm)(Up to 1 year)
- Frequency of adverse events effecting lactation(Up to 1 year)
- Incidence of all serious adverse events(Through the pregnancy, an average of 40 weeks and up to 12 months of infant age)
- Neurodevelopmental problems(Through the pregnancy, an average of 40 weeks and up to 12 months of infant age)
- Number of obstetric and delivery complications(At the delivery, an average of 40 weeks of pregnancy)
- Occurrence of milk allergic reaction in breastfed or formula supplemented infants(Up to 1 year)
- Occurrence of allergic reaction in patients who are breastfeeding(Up to 1 year)
- Number of ectopic or molar pregnancy(Through the pregnancy, an average of 40 weeks)
- Adverse fetal outcomes other than birth defects(Through the pregnancy, an average of 40 weeks and up to 12 months of infant age)
- Mortality in Infants(Up to 1 year)
- Head circumference in Infants (cm)(Up to 1 year)
- Occurrence of Jaundice cases in Infants(Up to 1 year)
- Number of hospitalizations in infants(Up to 1 year)