[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"neuronal-ceroid-lipofuscinosis-cln6\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:neuronal-ceroid-lipofuscinosis-cln6":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,49],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":32,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100640347","phase-1-gene-therapy-trial-for-cln6-batten-disease-100640347",false,"NCT07582484","Gene Therapy Trial for CLN6 Batten Disease","Phase 1\u002F2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9","Inclusion Criteria:\n\n* Diagnosis of CLN6\n* At least 4 months old\n\nExclusion Criteria:\n\n* Presence of another inherited neurologic disease\n* Prior stem cell transplantation\n* Prior gene transfer, gene editing, or viral vector therapy","ALL","4 Months",{"count":19,"type":20},12,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms.\n\nParticipants will:\n\nReceive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study",[27,28,29,30,31],"CLN6","Batten Disease","Batten's Disease","Neuronal Ceroid Lipofuscinosis CLN6","Neuronal Ceroid Lipofuscinosis",[27,33,34,35,31],"CLN6 Batten","Batten","Batten's","NOT_YET_RECRUITING","2026-05-12",{"date":39,"type":40},"2026-05-14","ACTUAL",{"date":42,"type":20},"2026-08",{"date":44,"type":20},"2028-08",{"name":46,"class":47},"The Charlotte and Gwenyth Gray Foundation","OTHER",1,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":55,"targetDuration":57,"studyType":58,"phases":4,"briefSummary":59,"conditions":60,"keywords":68,"overallStatus":71,"whyStopped":4,"lastUpdateSubmitDate":72,"lastUpdatePostDateStruct":73,"startDateStruct":75,"completionDateStruct":77,"leadSponsor":79,"locationsCount":48},"100199549","clinical-and-neuropsychological-investigations-in-batten-disease-100199549","NCT01873924","Clinical and Neuropsychological Investigations in Batten Disease","Inclusion Criteria:\n\n* child or adult with any form of Batten disease\n* parent or legal guardian of a child or adult with any form of Batten disease\n\nExclusion Criteria:\n\n\\- parent or guardian unable or unwilling to provide permission for the affected individual",{"count":56,"type":20},500,"10 Years","OBSERVATIONAL","This study aims to assess the natural history of Batten disease (Neuronal Ceroid Lipofuscinosis) by obtaining information about the motor, behavioral, and functional capabilities of individuals with Batten disease. This study will also refine and validate the Unified Batten Disease Rating Scale (UBDRS) as a clinical rating instrument for Batten disease.",[31,61,62,63,64,30,65,66,67,28],"Neuronal Ceroid Lipofuscinosis CLN1","Neuronal Ceroid Lipofuscinosis CLN2","Neuronal Ceroid Lipofuscinosis CLN3","Neuronal Ceroid Lipofuscinosis CLN5","Neuronal Ceroid Lipofuscinosis CLN7","Neuronal Ceroid Lipofuscinosis CLN8","Neuronal Ceroid Lipofuscinosis CLN10",[69,70,31],"Batten disease","NCL","RECRUITING","2025-09-05",{"date":74,"type":40},"2025-09-12",{"date":76,"type":4},"2004-08",{"date":78,"type":20},"2035-08",{"name":80,"class":47},"University of Rochester"]