Ignite Creation Date:
2025-12-25 @ 1:38 AM
Ignite Modification Date:
2026-03-13 @ 1:28 PM
Study NCT ID:
NCT02582294
Status:
TERMINATED
Last Update Posted:
2019-02-15
First Post:
2015-10-15
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Fabry's Disease and Pregnancy (PREFAB)
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': 'D011795', 'term': 'Surveys and Questionnaires'}], 'ancestors': [{'id': 'D003625', 'term': 'Data Collection'}, {'id': 'D004812', 'term': 'Epidemiologic Methods'}, {'id': 'D008919', 'term': 'Investigative Techniques'}, {'id': 'D017531', 'term': 'Health Care Evaluation Mechanisms'}, {'id': 'D011787', 'term': 'Quality of Health Care'}, {'id': 'D017530', 'term': 'Health Care Quality, Access, and Evaluation'}, {'id': 'D011634', 'term': 'Public Health'}, {'id': 'D004778', 'term': 'Environment and Public Health'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}, 'patientRegistry': False}, 'statusModule': {'whyStopped': 'Difficulty in patient recruitment', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-02', 'completionDateStruct': {'date': '2018-06-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-02-14', 'studyFirstSubmitDate': '2015-10-15', 'studyFirstSubmitQcDate': '2015-10-19', 'lastUpdatePostDateStruct': {'date': '2019-02-15', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-10-21', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-06-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': "Proportion of Fabry's disease patients having had an epidural block in childbirth.", 'timeFrame': "The duration of the interview for this study with women with Fabry disease's will be one hour"}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ["Fabry's Disease", 'Pregnancy']}, 'descriptionModule': {'briefSummary': "Fabry's disease is a progressive systemic disease X-linked which combines neurological (Fabry's pain crises), dermatologic (angiokeratomas), renal (renal failure), cardiovascular (hypertrophic cardiomyopathy, valvular disease, conduction disorder, coronary heart disease) and cerebral vascular (stroke) symptoms .\n\nIt is a glycosphingolipid metabolism disorder due to deficient or absent activity of the alpha-galactosidase A, causing accumulation of globotriaosylceramide in the lysosomes.\n\nThe incidence is estimated being 1/40 000. Some patients suffering from Fabry's disease today are of childbearing age and their multidisciplinary care (by neurologists, obstetricians and anesthetists) raises several questions.\n\nAbout the anesthetic, the question of epidural block is debated in patients with neurological diseases and recommendations are not unequivocal.\n\nIndeed one of the problems of the management in those conditions is the potential worsening of the disease because of the anesthetic procedure. In addition, the possibility of an antiplatelet and / or an anticoagulant treatment in these patients may also contre-indicate an epidural block.\n\nThe rate of epidural block achieved in patients with Fabry's disease is not currently known. Moreover, only sparse data on pregnancy outcomes in these patients are reported."}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['ADULT'], 'maximumAge': '45 Years', 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': "Fabry's disease patients having had at least one childbirth", 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Fabry's disease patients, aged 18 years or more, having had at least one childbirth\n\nExclusion Criteria:\n\n* Patient under a legal protection procedure\n* Patient denying to participate to the study\n* Lack of affiliation to a social security system"}, 'identificationModule': {'nctId': 'NCT02582294', 'acronym': 'PREFAB', 'briefTitle': "Fabry's Disease and Pregnancy (PREFAB)", 'organization': {'class': 'OTHER', 'fullName': 'Groupe Hospitalier Diaconesses Croix Saint-Simon'}, 'officialTitle': "Description of Obstetrical Care, Mainly Anesthesic, in Patients With Fabry's Disease", 'orgStudyIdInfo': {'id': 'OLE_2015_4'}}, 'armsInterventionsModule': {'interventions': [{'name': 'Questionnaire', 'type': 'OTHER'}]}, 'contactsLocationsModule': {'locations': [{'zip': '75020', 'city': 'Paris', 'state': 'Île-de-France Region', 'country': 'France', 'facility': 'Groupe hospitalier Diaconesses Croix Saint Simon', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Groupe Hospitalier Diaconesses Croix Saint-Simon', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}