Идёт набор NCT04252066

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

— Наблюдательное Fabry Disease

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: migalastat.
Кому может быть актуально: Состояния в реестре: Fabry Disease. Базовые параметры: Без ограничений · Женщины.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: США
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Обзор

Подробное описание

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.

Вмешательства

Препарат 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.

Первичные конечные точки

Number of major birth defects [Срок оценки: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]

Вторичные конечные точки (12)

Incidence of spontaneous abortion [Срок оценки: : up to 20 weeks]
Number of elective or induced abortion [Срок оценки: Through the pregnancy, an average of 40 weeks]
Number of fetal death or stillbirth [Срок оценки: Greater than 20 weeks of pregnancy and through the pregnancy, average of 40 week]
Number of live birth [Срок оценки: at the delivery, an average of 40 weeks of pregnancy]
Number of neonatal death [Срок оценки: up to 28 days of neonatal life]
Number of minor birth defects [Срок оценки: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
Number of ectopic or molar pregnancy [Срок оценки: Through the pregnancy, an average of 40 weeks]
Neurodevelopmental problems [Срок оценки: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
Adverse fetal outcomes other than birth defects [Срок оценки: Through the pregnancy, an average of 40 weeks and up to 12 months of infant age]
Number of obstetric and delivery complications [Срок оценки: At the delivery, an average of 40 weeks of pregnancy]
Occurrence of Jaundice cases in Infants [Срок оценки: Up to 1 year]
Number of hospitalizations in infants [Срок оценки: Up to 1 year]

Критерии участия

Критерии включения

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.