[{"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:":40},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":4,"hasResults":10,"nctId":11,"briefTitle":12,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":10,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":20,"phases":21,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":5},"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":19},"ESTIMATED","INTERVENTIONAL",[22],"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",[25,26,27],"Developmental Delay Disorder","Intellectual Disability","Rare Diseases","RECRUITING","2025-03-05",{"date":31,"type":32},"2025-03-06","ACTUAL",{"date":34,"type":32},"2025-02-19",{"date":36,"type":19},"2025-09",{"name":38,"class":39},"Yongguo Yu","OTHER",""]