Ignite Creation Date:
2025-12-24 @ 1:14 PM
Ignite Modification Date:
2025-12-28 @ 7:53 PM
Study NCT ID:
NCT00413595
Status:
COMPLETED
Last Update Posted:
2021-04-12
First Post:
2006-12-19
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Belgium', 'Denmark', 'Finland', 'Ireland', 'Italy', 'Spain', 'United Kingdom']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D020521', 'term': 'Stroke'}], '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'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'EDTA-blood and urine sample for central laboratory analysis of agalsidase antibodies and Gb3 for safety issues.\n\nThere will be a proteomic analysis in blood to check whether there will be the possibility to characterize a biomarker for Fabry disease.'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 100}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2007-07', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-04', 'completionDateStruct': {'date': '2019-12-01', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-04-08', 'studyFirstSubmitDate': '2006-12-19', 'studyFirstSubmitQcDate': '2006-12-19', 'lastUpdatePostDateStruct': {'date': '2021-04-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2006-12-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2019-12-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Determination of the relapse rate of acute cerebrovascular events with clinical relevance in patients with different prophylactic approaches', 'timeFrame': '54 months study duration'}], 'secondaryOutcomes': [{'measure': 'Quality of Life measured with the SF-36', 'timeFrame': '54 months study duration'}, {'measure': 'Number of acute CVEs without clinical significance but with obvious signs in MRI diagnosis', 'timeFrame': '54 months study duration'}, {'measure': 'Beck Depression Inventory II (BDI II)', 'timeFrame': '54 months study duration'}, {'measure': 'Brief Pain Inventory (BPI)', 'timeFrame': '54 months study duration'}, {'measure': 'Rostocker Kopfschmerzfragen-Komplex (RoKoKo) (only in Austria and Germany)', 'timeFrame': '54 months study period'}, {'measure': 'Habi test (only in Austrian and German centers)', 'timeFrame': '54 months study duration'}, {'measure': 'Trail Making Test (TMT)', 'timeFrame': '54 months study duration'}, {'measure': 'Functional neurological deficits measured by the Mini Mental State Examination (MMSE)', 'timeFrame': '54 months study duration'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Cerebrovascular Accident', 'Cerebrovascular Accident, Acute', 'Fabry Disease', "Fabry's Disease", 'Anderson-Fabry Disease', 'CVA (Cerebrovascular Accident)', 'Stroke, acute', 'Cerebral Stroke'], 'conditions': ['Fabry Disease', 'Cerebrovascular Accident']}, 'referencesModule': {'references': [{'pmid': '16298216', 'type': 'BACKGROUND', 'citation': 'Rolfs A, Bottcher T, Zschiesche M, Morris P, Winchester B, Bauer P, Walter U, Mix E, Lohr M, Harzer K, Strauss U, Pahnke J, Grossmann A, Benecke R. Prevalence of Fabry disease in patients with cryptogenic stroke: a prospective study. Lancet. 2005 Nov 19;366(9499):1794-6. doi: 10.1016/S0140-6736(05)67635-0.'}]}, 'descriptionModule': {'briefSummary': 'New studies indicate that in about 1 - 2 percent of the younger stroke patients the cause could have been an undiagnosed genetic disease, the so called Fabry disease. In this case certain fat molecules are not digested and broken down by the body - but remain in the cells. These fat molecules build up to dangerous levels, which start to damage the body, because they accumulate e.g. in the walls of the blood vessels. This accumulation in the blood vessels of the whole body may cause life-threatening malfunctions in the brain, inducing a stroke.\n\nThe purpose of this study is to investigate the stroke rehabilitation of Fabry patients during different therapeutic standard approaches for stroke and for Fabry disease (if any). During this study, stroke patients with Fabry disease will be monitored in greater detail to determine whether the differences in treatment are significant for patient recovery and on what they depend.', 'detailedDescription': 'In a group of young stroke patients with diagnosed Fabry disease the stroke rehabilitation will be investigated during different prophylactic therapeutic approaches. In this study the investigator will not be given any instructions on stroke and Fabry therapy.\n\nAll patients with any etiology of stroke and a diagnosed Fabry disease submitted to the stroke unit of the participating centres which commit to work with the EUSI (European Stroke Initiative) recommendations for stroke management and diagnosis will be included into the study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT'], 'maximumAge': '55 Years', 'minimumAge': '18 Years', 'samplingMethod': 'PROBABILITY_SAMPLE', 'studyPopulation': 'Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack and genetic diagnosis (a-galactosidase defect) of Fabry disease.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Adult patients (18 - 55 years of age) with an acute cerebrovascular event (CVE) of any etiology defined as patients having an ischemic stroke or transient ischemic attack\n* Genetic diagnosis (a-galactosidase defect)of Fabry disease\n* Written informed consent from patient\n\nExclusion Criteria:\n\n* No proven Fabry disease\n* Participating in an other clinical trial with any investigational new drug or medical device\n* Contraindication to any of the diagnostic procedures like e.g. MRI investigation\n* Patient has been pretreated with Enzyme Replacement Therapy at the date of informed consent of sifap2'}, 'identificationModule': {'nctId': 'NCT00413595', 'acronym': 'sifap2', 'briefTitle': 'Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation', 'organization': {'class': 'INDUSTRY', 'fullName': 'CENTOGENE GmbH Rostock'}, 'officialTitle': 'Stroke in Young Fabry Patients (sifap2): Characterization of the Stroke Rehabilitation in Young Patients With Fabry Disease: An Epidemiological, International, Multicenter Prognosis Study', 'orgStudyIdInfo': {'id': 'II PV04/2006'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Observation', 'description': 'Adult patients (18 - 55 years of age) with an acute cerebrovascular event of any etiology and the genetic diagnosis (a-galactosidase defect) of Fabry disease', 'interventionNames': ['Other: No intervention']}], 'interventions': [{'name': 'No intervention', 'type': 'OTHER', 'description': 'Observational, epidemiological, prognosis study; no drug tested; only laboratory analysis and diagnostic interventions done.', 'armGroupLabels': ['Observation']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'A-8036', 'city': 'Graz', 'country': 'Austria', 'facility': 'Universitätsklinikum für Neurologie', 'geoPoint': {'lat': 47.06733, 'lon': 15.44197}}, {'zip': '10000', 'city': 'Zagreb', 'country': 'Croatia', 'facility': 'Department of Neurology, University Hospital Sestre Milosrdnice', 'geoPoint': {'lat': 45.81444, 'lon': 15.97798}}, {'zip': 'F-69003', 'city': 'Lyon', 'country': 'France', 'facility': "Hopital Neurologique de Lyon, Service d'urgences Neurovasculaires", 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '0179', 'city': 'Tbilisi', 'country': 'Georgia', 'facility': 'Department of Neurology, S. Khechinashvili University clinic of Tbilisi state medical university', 'geoPoint': {'lat': 41.69143, 'lon': 44.83412}}, {'zip': '95445', 'city': 'Bayreuth', 'country': 'Germany', 'facility': 'Department of Neurology, Klinikum Hohe Warte', 'geoPoint': {'lat': 49.94782, 'lon': 11.57893}}, {'zip': 'D-12200', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite Campus Benjamin Franklin, Dept. of Neurology', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '29223', 'city': 'Celle', 'country': 'Germany', 'facility': 'Department of Neurology, Allgemeines Krankenhaus Celle', 'geoPoint': {'lat': 52.62264, 'lon': 10.08047}}, {'zip': '09131', 'city': 'Chemnitz', 'country': 'Germany', 'facility': 'Department of Neurology, Klinikum Chemnitz gGmbH', 'geoPoint': {'lat': 50.8357, 'lon': 12.92922}}, {'zip': '01307', 'city': 'Dresden', 'country': 'Germany', 'facility': 'Department of Neurology, Universitaetsklinikum Carl Gustav Carus', 'geoPoint': {'lat': 51.05089, 'lon': 13.73832}}, {'zip': 'D-40225', 'city': 'Düsseldorf', 'country': 'Germany', 'facility': 'Heinrich-Heine-University Duesseldorf, Dept. of Neurology', 'geoPoint': {'lat': 51.22172, 'lon': 6.77616}}, {'zip': 'D-35385', 'city': 'Giessen', 'country': 'Germany', 'facility': 'University of Giessen-Marburg Dept. of Neurology', 'geoPoint': {'lat': 50.58727, 'lon': 8.67554}}, {'zip': '20246', 'city': 'Hamburg', 'country': 'Germany', 'facility': 'Department of Neurology, Universitaetsklinikum Hamburg-Eppendorf', 'geoPoint': {'lat': 53.55073, 'lon': 9.99302}}, {'zip': '07740', 'city': 'Jena', 'country': 'Germany', 'facility': 'Department of Neurology, Universitaetsklinikum Jena', 'geoPoint': {'lat': 50.92878, 'lon': 11.5899}}, {'zip': '04103', 'city': 'Leipzig', 'country': 'Germany', 'facility': 'Department of Neurology, Universitaetsklinikum Leipzig', 'geoPoint': {'lat': 51.33962, 'lon': 12.37129}}, {'zip': '99974', 'city': 'Mühlhausen', 'country': 'Germany', 'facility': 'Dept. of Neurology, Ökumenisches Hainich Klinikum gGmbH', 'geoPoint': {'lat': 51.20896, 'lon': 10.45275}}, {'zip': 'D-81377', 'city': 'München', 'country': 'Germany', 'facility': 'Ludwig-Maximilians-University of Munich, Klinikum München-Großhadern, Dept. of Neurology', 'geoPoint': {'lat': 51.60698, 'lon': 13.31243}}, {'zip': '72076', 'city': 'Tübingen', 'country': 'Germany', 'facility': 'Department of Neurology, University Tuebingen', 'geoPoint': {'lat': 48.52266, 'lon': 9.05222}}, {'zip': 'D-89081', 'city': 'Ulm', 'country': 'Germany', 'facility': 'University of Ulm, Department of Neurology', 'geoPoint': {'lat': 48.39841, 'lon': 9.99155}}, {'zip': '02-957', 'city': 'Warsaw', 'country': 'Poland', 'facility': 'Institute of Psychiatry and Neurology, Dept. of Neurology', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '1150-199', 'city': 'Lisbon', 'country': 'Portugal', 'facility': 'Centro Hospitalar de Lisboa Central, Servico de Neurologia', 'geoPoint': {'lat': 38.72509, 'lon': -9.1498}}], 'overallOfficials': [{'name': 'Arndt Rolfs, Prof., MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'CENTOGENE GmbH Rostock', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Shire Human Genetic Therapies, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}