[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100341589":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":21,"locations":31,"responsibleParty":49,"collaborators":20,"id":51,"slug":52,"hasResults":53,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":20,"eligibilityCriteria":57,"healthyVolunteers":53,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":20,"studyType":64,"phases":65,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":34,"whyStopped":20,"lastUpdateSubmitDate":75,"lastUpdatePostDateStruct":76,"startDateStruct":79,"completionDateStruct":81,"leadSponsor":83,"locationsCount":84},{"fullName":5,"class":6},"Shenzhen Geno-Immune Medical Institute","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Lentivirus-mediated delivery of ABCD1 to the CNS and the body","EXPERIMENTAL","Intrathecal and intravenous injections with lentiviral TYF-ABCD1 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 multiplicity of infection\u002Fml which carry normal ABCD1 gene",[9],null,[22,27],{"name":23,"role":24,"phone":25,"phoneExt":20,"email":26},"Lung-Ji Chang, Ph.D","CONTACT","86-13671121909","c@szgimi.org",{"name":28,"role":24,"phone":29,"phoneExt":20,"email":30},"Rui Zhang, MSc","86-13488887093","13488887093@163.com",[32],{"facility":33,"status":34,"city":35,"state":36,"zip":37,"country":38,"countryCode":39,"cosmosGeoPoint":40,"geoPoint":45,"contacts":46},"Shenzhen Geno-immune Medical Institute","RECRUITING","Shenzhen","Guangdong","518000","China","CN",{"type":41,"coordinates":42},"Point",[43,44],114.0683,22.54554,{"lat":44,"lon":43},[47],{"name":48,"role":24,"phone":25,"phoneExt":20,"email":26},"Lung-Ji Chang, PhD",{"type":50,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100341589","it-and-iv-lentiviral-gene-therapy-for-x-ald-100341589",false,"NCT03727555","IT and IV Lentiviral Gene Therapy for X-ALD","Intrathecal and Intravenous Lentiviral Gene Therapy for X-linked Adrenoleukodystrophy (X-ALD)","Inclusion Criteria:\n\n1. X-ALD patients ≥ 1 year of age\n2. ALD diagnosis of the brain: evaluation of the VLCFA value in plasma\n3. Central imaging of the MRI to examine the damage on the CNS.\n4. Neurological function score (NFS) ≥ 1\n5. Parent \u002F guardian \u002F patient signing informed consent\n6. 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. Stablized condition after statins, Lorenzo's oil, or diet to reduce VLCFA levels\n3. Patients who are experiencing severe viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency\n4. Cannot perform an MRI\n5. Infection or dermatosis at pre-injection site","ALL","1 Year","60 Years",{"count":62,"type":63},30,"ESTIMATED","INTERVENTIONAL",[66],"NA","This is a Phase I\u002FII clinical trial of gene therapy for treating X-linked adrenoleukodystrophy using a high-safety, high-efficiency, self-inactivating lentiviral vector (LV) TYF-ABCD1 to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the intrathecal and intravenous lentiviral gene transfer clinical protocol.",[69],"X-linked Adrenoleukodystrophy",[71,72,73,74],"X-linked adrenoleukodystrophy","Lentiviral vector","Intrathecal injection","Intravenous injection","2025-09-08",{"date":77,"type":78},"2025-09-09","ACTUAL",{"date":80,"type":78},"2025-08-31",{"date":82,"type":63},"2028-12-31",{"name":5,"class":6},1]