Ignite Creation Date:
2025-12-24 @ 4:40 PM
Ignite Modification Date:
2025-12-28 @ 2:14 PM
Study NCT ID:
NCT04252066
Status:
RECRUITING
Last Update Posted:
2025-07-08
First Post:
2019-12-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C090092', 'term': 'migalastat'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2025-03-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2030-02', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-07-02', 'studyFirstSubmitDate': '2019-12-18', 'studyFirstSubmitQcDate': '2020-01-30', 'lastUpdatePostDateStruct': {'date': '2025-07-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-02-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of major birth defects', 'timeFrame': 'Through the pregnancy, an average of 40 weeks and up to 12 months of infant age'}], 'secondaryOutcomes': [{'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', 'description': 'an ectopic or molar pregnancy occurs outside of the uterus.'}, {'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'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['migalastat', 'Fabry Disease', 'Women with Fabry Disease'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': '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.\n\nCases 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.\n\nThere 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.\n\nThis is an observational study, it will enroll patients and collect data as described in this protocol for a minimum of 10 years.'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'genderBased': True, 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Pregnant and/or breastfeeding patients with Fabry disease', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nFemale patients meeting the following criteria will be eligible for study enrollment:\n\n1. Patients with Fabry disease who are pregnant and/or breastfeeding, whether or not they are exposed to migalastat\n2. Able and willing to provide informed consent or assent, if applicable.\n3. Able and willing to provide HCP contact information.\n\nExclusion Criteria:\n\nNone'}, 'identificationModule': {'nctId': 'NCT04252066', 'briefTitle': 'A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amicus Therapeutics'}, 'officialTitle': 'A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding', 'orgStudyIdInfo': {'id': 'AT1001-037'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Cohort 1', 'description': '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.', 'interventionNames': ['Drug: migalastat']}, {'label': 'Cohort 2', 'description': 'Cohort 2 will be pregnant and/or breastfeeding patients who have Fabry Disease, who were not exposed to migalastat during pregnancy and/or breastfeeding.'}], 'interventions': [{'name': 'migalastat', 'type': 'DRUG', 'description': '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.', 'armGroupLabels': ['Cohort 1']}]}, 'contactsLocationsModule': {'locations': [{'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'facility': 'Amicus Therapeutics, Inc. Pregnancy Registry', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}], 'centralContacts': [{'name': 'Pregnancy Registry Call Center', 'role': 'CONTACT', 'email': 'galafoldpregnancy@ubc.com', 'phone': '888-239-0758'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amicus Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}