[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Yongguo Yu\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":41},{"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":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":5},"100582235","early-phase-1-clinical-trial-of-the-dual-vector-base-editor-for-the-treatment-of-the-chd3-r1025w-mutation-100582235",false,"NCT06860672","Clinical Trial of the Dual Vector Base Editor for the Treatment of the CHD3-R1025W Mutation","Safety, Tolerability and Preliminary Efficacy Study of a Single Intrathecal Injection of the Dual Vector AAV-CHD3-R1025W Base Editor for the Treatment of Developmental Disorders Caused by the R1025W Mutation in the CHD3 Gene","Inclusion Criteria:\n\n* Clinical diagnosis of Snijders Blok-Campeau syndrome\n* Heterozygous mutation of c.3073C\\>T, p.(Arg1025Trp) in the CHD3 gene\n* Normal liver, heart and immune function\n* Normal coagulation and platelet counts\n\nExclusion Criteria:\n\n* Brain tumor or intracranial space-occupying lesion\n* Contraindications to administration of lumbar puncture or sheath injection administration\n* Persistent status epilepticus or recurrent epileptic control instability\n* Presence of unstable systemic disease including active bacterial, fungal or HIV, hepatitis A, hepatitis B infection\n* Serum anti-AAV neutralizing antibody titer \\>1:50 (ELISA immunoassay)\n* Treatment with immunological agents other than protocol-specified prophylaxis within 3 months\n* Prior gene therapy\n* Participation in another clinical trial, or treatment with another investigational product within 30 days or 5 half-lives\n* Known allergy to any investigational product","ALL","2 Years","10 Years",{"count":5,"type":20},"ESTIMATED","INTERVENTIONAL",[23],"EARLY_PHASE1","To evaluate the safety, tolerability and preliminary efficacy study of a single intrathecal injection of the dual vector AAV-CHD3-R1025W base editor for the treatment of developmental disorders caused by the R1025W mutation in the CHD3 gene",[26,27,28],"Developmental Delay Disorder","Intellectual Disability","Rare Diseases","RECRUITING","2025-03-05",{"date":32,"type":33},"2025-03-06","ACTUAL",{"date":35,"type":33},"2025-02-19",{"date":37,"type":20},"2025-09",{"name":39,"class":40},"Yongguo Yu","OTHER",""]