[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100107860":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":17,"locations":22,"responsibleParty":43,"collaborators":45,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":62,"acronym":10,"eligibilityCriteria":63,"healthyVolunteers":60,"sex":64,"minAge":10,"maxAge":10,"enrollmentInfo":65,"targetDuration":10,"studyType":68,"phases":10,"briefSummary":69,"conditions":70,"keywords":76,"overallStatus":25,"whyStopped":10,"lastUpdateSubmitDate":101,"lastUpdatePostDateStruct":102,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":110},{"fullName":5,"class":6},"University of Minnesota","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":10},"Gangliosidosis Diseases Study Population",null,"This study observes one cohort: 42 infantile or juvenile Tay-Sachs disease, Sandhoff disease, or GM1 gangliosidosis affected subjects; and 10 late-onset gangliosidosis disease affected subjects.",[13],{"name":14,"affiliation":15,"role":16},"Jeanine R. Jarnes, PharmD","University of Minnesota - Fairview","PRINCIPAL_INVESTIGATOR",[18],{"name":14,"role":19,"phone":20,"phoneExt":10,"email":21},"CONTACT","612-626-5131","utzx0002@umn.edu",[23],{"facility":24,"status":25,"city":26,"state":27,"zip":28,"country":29,"countryCode":30,"cosmosGeoPoint":31,"geoPoint":36,"contacts":37},"University of Minnesota - Pediatric Genetics and Metabolism","RECRUITING","Minneapolis","Minnesota","55455","United States","US",{"type":32,"coordinates":33},"Point",[34,35],-93.26384,44.97997,{"lat":35,"lon":34},[38,39,40],{"name":14,"role":19,"phone":20,"phoneExt":10,"email":21},{"name":14,"role":16,"phone":10,"phoneExt":10,"email":10},{"name":41,"role":42,"phone":10,"phoneExt":10,"email":10},"Chester B. Whitley, MD, PhD","SUB_INVESTIGATOR",{"type":44,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[46,49,52,54,56],{"name":47,"class":48},"Rare Diseases Clinical Research Network","NETWORK",{"name":50,"class":51},"National Center for Advancing Translational Sciences (NCATS)","NIH",{"name":53,"class":51},"National Institute of Neurological Disorders and Stroke (NINDS)",{"name":55,"class":51},"National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)",{"name":57,"class":6},"Lysosomal Disease Network","100107860","a-natural-history-study-of-the-gangliosidoses-100107860",false,"NCT00668187","A Natural History Study of the Gangliosidoses","Inclusion Criteria:\n\n1. Subjects must have a documented gangliosidosis disease.\n2. Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.\n3. Late-onset gangliosidosis subjects must be able to tolerate a head MRI.\n\nExclusion Criteria:\n\n1\\. There are no exclusion criteria, beyond a desire not to participate.","ALL",{"count":66,"type":67},52,"ESTIMATED","OBSERVATIONAL","Hypothesis: To characterize and describe disease progression and heterogeneity of the gangliosidosis diseases.\n\nThis research study seeks to develop a quantitative method to delineate disease progression for the gangliosidosis diseases (Tay-Sachs disease, Sandhoff disease, and GM1 gangliosidosis) in order to better understand the natural history and heterogeneity of these diseases. Such a quantitative method will also be essential for evaluating any treatments that may become available in the future, such as gene therapy. The data from this study will be necessary to provide end-points for future therapies, guide medical decisions about treatment, provide objective measurement of treatment outcomes, and accurately inform parents regarding potential outcomes.",[71,72,73,74,75],"Tay-Sachs Disease","Sandhoff Disease","Late Onset Tay-Sachs Disease","GM1 Gangliosidosis","GM2 Gangliosidosis",[77,78,79,80,81,82,83,84,85,86,87,88,89,90,91,92,93,94,95,96,97,98,99,100],"Tay-Sachs disease","Sandhoff disease","Late Onset Tay-Sachs disease","LOTS","hexosaminidase A deficiency","hexosaminidase A and B deficiency","infantile Tay-Sachs disease","adult-onset Tay-Sachs disease","prospective","natural history","GM1 gangliosidosis","gangliosidoses","β-galactosidase","β-galactosidase deficiency","hexosaminidase","hexosaminidase deficiency","Tay-Sachs","Sandhoff","juvenile Tay-Sachs","juvenile Tay-Sachs disease","late onset Tay-Sachs","juvenile Sandhoff","juvenile Sandhoff disease","GM2 gangliosidosis","2026-03-02",{"date":103,"type":104},"2026-03-04","ACTUAL",{"date":106,"type":104},"2010-12",{"date":108,"type":67},"2027-03-01",{"name":5,"class":6},1]