[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100582235":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":27,"locations":33,"responsibleParty":52,"collaborators":11,"id":56,"slug":57,"hasResults":58,"nctId":59,"briefTitle":60,"officialTitle":61,"acronym":11,"eligibilityCriteria":62,"healthyVolunteers":58,"sex":63,"minAge":64,"maxAge":65,"enrollmentInfo":66,"targetDuration":11,"studyType":69,"phases":70,"briefSummary":72,"conditions":73,"keywords":11,"overallStatus":36,"whyStopped":11,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":81,"completionDateStruct":83,"leadSponsor":85,"locationsCount":67},{"fullName":5,"class":6},"Xinhua Hospital, Shanghai Jiao Tong University School of Medicine","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Dual vector treatment group","EXPERIMENTAL",null,[13],"Genetic: Dual vector DNA base editor",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":11},"GENETIC","Dual vector DNA base editor","The base editor is delivered using a dual vector adeno-associated virus (AAV) system and introduced into the child via intrathecal injection to correct the mutated CHD3 gene. The vital signs of the child will be closely monitored during treatment to assess possible acute adverse effects. The child will be followed up regularly after treatment to monitor the success of gene editing and the neurodevelopmental improvement of the child. Possible long-term adverse events will be closely monitored to assess the safety of the treatment.",[9],[21,24],{"name":22,"affiliation":5,"role":23},"Yongguo Yu, Dr, MD, PhD","PRINCIPAL_INVESTIGATOR",{"name":25,"affiliation":26,"role":23},"Zilong Qiu, PhD","Shanghai Jiao Tong University School of Medicine Songjiang Research Institute",[28],{"name":29,"role":30,"phone":31,"phoneExt":11,"email":32},"Xiaomei Luo, Ms., Master","CONTACT","+86-25-25076466","luoxiaomei@shsmu.edu.cn",[34],{"facility":35,"status":36,"city":37,"state":38,"zip":39,"country":40,"countryCode":41,"cosmosGeoPoint":42,"geoPoint":47,"contacts":48},"Xinhua Hospital affiliated to Shanghai Jiao Tong University School of Medicine","RECRUITING","Shanghai","Shanghai Municipality","200092","China","CN",{"type":43,"coordinates":44},"Point",[45,46],121.45806,31.22222,{"lat":46,"lon":45},[49],{"name":50,"role":30,"phone":51,"phoneExt":11,"email":32},"Xiaomei Luo, Master","+86-21-25076466",{"type":53,"investigatorFullName":54,"investigatorTitle":55,"investigatorAffiliation":5,"oldNameTitle":11,"oldOrganization":11},"SPONSOR_INVESTIGATOR","Yongguo Yu","Chief physician","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":67,"type":68},1,"ESTIMATED","INTERVENTIONAL",[71],"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",[74,75,76],"Developmental Delay Disorder","Intellectual Disability","Rare Diseases","2025-03-05",{"date":79,"type":80},"2025-03-06","ACTUAL",{"date":82,"type":80},"2025-02-19",{"date":84,"type":68},"2025-09",{"name":54,"class":6}]