[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100311346":3},{"organization":4,"armGroups":7,"interventions":8,"overallOfficials":7,"centralContacts":16,"locations":22,"responsibleParty":38,"collaborators":7,"id":42,"slug":7,"hasResults":43,"nctId":44,"briefTitle":45,"officialTitle":45,"acronym":7,"eligibilityCriteria":46,"healthyVolunteers":43,"sex":47,"minAge":7,"maxAge":7,"enrollmentInfo":48,"targetDuration":7,"studyType":51,"phases":7,"briefSummary":52,"conditions":53,"keywords":81,"overallStatus":25,"whyStopped":7,"lastUpdateSubmitDate":96,"lastUpdatePostDateStruct":97,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":105},{"fullName":5,"class":6},"University of Pittsburgh","OTHER",null,[9,12],{"type":6,"name":10,"description":11,"armGroupLabels":7,"otherNames":7},"Palliative Care","Collecting information about the natural progression of these diseases",{"type":13,"name":14,"description":15,"armGroupLabels":7,"otherNames":7},"BIOLOGICAL","Hematopoetic Stem Cell Transplantation","Following patients who have received HSCT as part of their clinical care.",[17],{"name":18,"role":19,"phone":20,"phoneExt":7,"email":21},"Deepa Rajan, MD","CONTACT","412-692-8388","rajands@upmc.edu",[23],{"facility":24,"status":25,"city":26,"state":27,"zip":28,"country":29,"cosmosGeoPoint":30,"geoPoint":35,"contacts":36},"UPMC Children's Hospital of Pittsburgh","RECRUITING","Pittsburgh","Pennsylvania","15224","United States",{"type":31,"coordinates":32},"Point",[33,34],-79.99589,40.44062,{"lat":34,"lon":33},[37],{"name":18,"role":19,"phone":20,"phoneExt":7,"email":21},{"type":39,"investigatorFullName":40,"investigatorTitle":41,"investigatorAffiliation":5,"oldNameTitle":7,"oldOrganization":7},"PRINCIPAL_INVESTIGATOR","Deepa Soundara Rajan","Associate Professor","100311346",false,"NCT03333200","Longitudinal Study of Neurodegenerative Disorders","Inclusion Criteria:\n\n* Any patient with a genetic neurodegenerative disorder\n\nExclusion Criteria:\n\n* none","ALL",{"count":49,"type":50},1500,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.",[54,55,56,57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,80],"MLD","Krabbe Disease","ALD","MPS I","MPS II","MPS III","Vanishing White Matter Disease","GM3 Gangliosidosis","PKAN","Tay-Sachs Disease","NP Deficiency","Osteopetrosis","Alpha-Mannosidosis","Sandhoff Disease","Niemann-Pick Diseases","MPS IV","Gaucher Disease","GAN","GM1 Gangliosidoses","Morquio Disease","S-Adenosylhomocysteine Hydrolase Deficiency","Batten Disease","Pelizaeus-Merzbacher Disease","Leukodystrophy","Lysosomal Storage Diseases","Purine Nucleoside Phosphorylase Deficiency","Multiple Sulfatase Deficiency Disease",[82,83,84,85,86,87,88,89,90,91,92,93,94,95],"Pediatric","Rare","Neurodegenerative","Genetic","Neurodevelopment","Brain","MRI","Biorepository","NDRD","Longitudinal","Cognitive","Motor","Language","Adaptive behavior","2026-02-04",{"date":98,"type":99},"2026-02-09","ACTUAL",{"date":101,"type":99},"2012-01-11",{"date":103,"type":50},"2035-01",{"name":5,"class":6},1]