[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"cln1-disease\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:cln1-disease":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":39,"overallStatus":73,"whyStopped":4,"lastUpdateSubmitDate":74,"lastUpdatePostDateStruct":75,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":5},"100409561","natural-history-and-longitudinal-clinical-assessments-in-ncl--batten-disease-the-international-dem-child-database-100409561",false,"NCT04613089","Natural History and Longitudinal Clinical Assessments in NCL \u002F Batten Disease, the International DEM-CHILD Database","Natural History and Long Term Clinical Assessments of All Forms of Neuronal Ceroid Lipofuscinoses - Capturing Key Symptoms and Disease Progression as Part of the Independent, International NCL DEM-CHILD Patient Database","Inclusion Criteria:\n\n\\- Patients with a confirmed molecular diagnosis of a form of NCL Disease\n\nAdditional inclusion criteria for Group\u002FCohort: \"CLN2 Disease - ERT (Brineura) Treated\":\n\n* Documented diagnosis of TPP1 deficiency\n* Previous or current treatment with intracerebroventricular ERT with cerliponase alpha\n* Patients that are currently participating in post-marketing studies will be allowed to participate.\n\nExclusion Criteria:\n\n\\- Patients with no confirmed molecular diagnosis of a form of NCL Disease","ALL",{"count":18,"type":19},500,"ESTIMATED","30 Years","OBSERVATIONAL","This is an observational study that aims at assessing the natural history of NCL diseases as part of the international DEM-CHILD Database.\n\n1. Patient data are collected from medical records, patient questionnaires and routine follow up clinical examinations with focus on assessing progression in key areas of disease such as motor, language, cognition, seizures, vision, and behavior.\n2. A local biorepository of samples from genetically defined NCL patients will be established as well as a virtual biorepository within the DEM-CHILD DB to be able to easily localize international availability of patient samples.",[24,25,26,27,28,29,30,31,32,33,34,35,36,37,38],"Neuronal Ceroid Lipofuscinosis","Batten Disease","CLN1 Disease","CLN2 Disease","CLN3 Disease","CLN4 Disease","CLN5 Disease","CLN6 Disease","CLN7 Disease","CLN8 Disease","CLN10 Disease","CLN11 Disease","CLN12 Disease","CLN13 Disease","CLN14 Disease",[40,41,42,43,44,45,46,47,48,49,50,51,52,53,54,55,56,57,58,59,60,61,62,63,64,65,66,67,68,69,70,71,72],"INCL","LINCL","VLINCL","JNCL","ANCL","NCL","CLN","Batten","Childhood Dementia","Lysosomal Storage Diseases","Neurodegenerative Diseases","Neurodegenerative Disorders","Metabolic Disorders","PME","EPMR","SCAR7","SGSH","PPT1","Haltia-Santavuori Disease","TPP1","Jansky-Bielschowsky Disease","Spielmeyer-Vogt-Sjögren-Batten Disease","DNAJC5","Parry Disease","Kufs Disease Type A","MFSD8","CTSD","GRN","ATP13A2","Kufor-Rakeb Syndrome","CTSF","Kufs Disease Type B","KCTD7","RECRUITING","2021-10-22",{"date":76,"type":77},"2021-10-29","ACTUAL",{"date":79,"type":77},"2020-04-08",{"date":81,"type":19},"2050-04-08",{"name":83,"class":84},"Universitätsklinikum Hamburg-Eppendorf","OTHER"]