[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100341445":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":25,"locations":30,"responsibleParty":47,"collaborators":20,"id":49,"slug":50,"hasResults":51,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":20,"eligibilityCriteria":55,"healthyVolunteers":51,"sex":56,"minAge":57,"maxAge":58,"enrollmentInfo":59,"targetDuration":20,"studyType":62,"phases":63,"briefSummary":65,"conditions":66,"keywords":68,"overallStatus":33,"whyStopped":20,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":77,"completionDateStruct":79,"leadSponsor":81,"locationsCount":82},{"fullName":5,"class":6},"Shenzhen Geno-Immune Medical Institute","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Lentivirus-mediated delivery of ARSA to the CNS and the body","EXPERIMENTAL","Intrathecal and intravenous injections with lentiviral TYF-ARSA vector carrying the functional gene",[13],"Genetic: Intrathecal and intravenous LV gene therapy",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","Intrathecal and intravenous LV gene therapy","Direct IT and IV LV gene therapy to deliver high levels of LVs at 1-2×10\\^9 transduction units\u002Fml which carry a normal ARSA gene",[9],null,[22],{"name":23,"affiliation":5,"role":24},"Lung-Ji Chang, Ph.D","PRINCIPAL_INVESTIGATOR",[26],{"name":23,"role":27,"phone":28,"phoneExt":20,"email":29},"CONTACT","+86 0755-86573763","c@szgimi.org",[31],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":45},"Lung-Ji Chang","RECRUITING","Shenzhen","Guangdong","518000","China","CN",{"type":40,"coordinates":41},"Point",[42,43],114.0683,22.54554,{"lat":43,"lon":42},[46],{"name":23,"role":27,"phone":28,"phoneExt":20,"email":29},{"type":48,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100341445","direct-lentiviral-injection-gene-therapy-for-mld-100341445",false,"NCT03725670","Direct Lentiviral Injection Gene Therapy for MLD","Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)","Inclusion Criteria:\n\n1. MLD patient age \\>= 1 month\n2. ARSA gene sequence analysis to confirm MLD mutations\n3. Scoring system for brain MR Imaging confirmed MLD\n4. Parent \u002F guardian \u002F patient signing informed consent\n5. Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form\n\nExclusion Criteria:\n\n1. HIV positive patients\n2. Patients who are experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency\n3. Cannot perform an MRI\n4. Infection or dermatosis at pre-injection site\n5. Any condition that may increase the subjects' risk or interfere with the results of the trial. In addition to MLD, there are other neurological disorders.","ALL","1 Month","50 Years",{"count":60,"type":61},10,"ESTIMATED","INTERVENTIONAL",[64],"NA","This is a Phase I\u002FII clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.",[67],"Metachromatic Leukodystrophy (MLD)",[69,70,71,72],"Metachromatic leukodystrophy (MLD)","lentiviral vector","Intrathecal injection","Intravenous injection","2026-06-18",{"date":75,"type":76},"2026-06-23","ACTUAL",{"date":78,"type":76},"2025-05-31",{"date":80,"type":61},"2030-12-31",{"name":5,"class":6},1]