[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100312778":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":24,"locations":29,"responsibleParty":48,"collaborators":19,"id":51,"slug":52,"hasResults":53,"nctId":54,"briefTitle":55,"officialTitle":56,"acronym":19,"eligibilityCriteria":57,"healthyVolunteers":53,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":19,"studyType":64,"phases":65,"briefSummary":67,"conditions":68,"keywords":70,"overallStatus":32,"whyStopped":19,"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},"Gene-modified autologous stem cells","EXPERIMENTAL","Autologous hematopoeitic stem cells and mesenchymal stem cells transduced with lentiviral vector carrying the FANCA gene ex vivo",[13],"Genetic: Gene-modified autologous stem cells",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"GENETIC","Infusion for 5x10\\^6\\~1x10\\^7 per kilogram of body weight of gene-modified cells; or more infusions depending on the circumstances",[9],null,[21],{"name":22,"affiliation":5,"role":23},"Lung-Ji Chang, Ph.D","PRINCIPAL_INVESTIGATOR",[25],{"name":22,"role":26,"phone":27,"phoneExt":19,"email":28},"CONTACT","86-13671121909","c@szgimi.org",[30],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":44},"Shenzhen Geno-immune Medical Institute","RECRUITING","Shenzhen","Guangdong","518000","China","CN",{"type":39,"coordinates":40},"Point",[41,42],114.0683,22.54554,{"lat":42,"lon":41},[45],{"name":46,"role":26,"phone":47,"phoneExt":19,"email":28},"Lung-Ji Chang, PhD","+86 0755-86573763",{"type":23,"investigatorFullName":49,"investigatorTitle":50,"investigatorAffiliation":5,"oldNameTitle":19,"oldOrganization":19},"Lung-Ji Chang","President","100312778","fanca-gene-transfer-for-fanconi-anemia-using-a-high-safety-high-efficiency-self-inactivating-lentiviral-vector-100312778",false,"NCT03351868","FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector","Gene Transfer for Fanconi Anemia Using a Self-inactivating Lentiviral Vector","Inclusion Criteria:\n\n1. Diagnosis of Fanconi anemia FANCA type based on DNA sequencing and sensitivity test for chromosomal cleavage by mitomycin C or butylene oxide.\n2. No cytogenetic abnormalities and the proportion of myelodysplastic abnormalities does not exceed 5% within 3 months prior to stem cell collection.\n3. Age: ≥ 4 years.\n4. Karnofsky: ≥ 70%.\n5. ANC ≥ 5×10\\^8\u002FL; PLT ≥ 2×10\\^10\u002FL.\n6. Hemoglobin ≥ 8g\u002FdL.\n7. Proper renal and hepatic functions (ULN denotes \"upper limit of normal range\") with\n\n   * serum creatinine ≤ 1.5×ULN;\n   * serum bilirubin ≤ 3×ULN;\n   * AST\u002FALT ≤ 5×ULN.\n8. Pulmonary function is normal; DLCO \\> 50%.\n9. Written, informed consent obtained prior to any study-specific procedures.\n\nExclusion Criteria:\n\n1. Diagnosis of active malignant disease or myelodysplastic syndrome.\n2. Diagnosis of myeloid leukemia.\n3. Pregnant or lactating females.\n4. Existence of an available HLA-identical related donor.\n5. Subject infected with HBV (HBsAg positive), HIV (HIV antibody positive), HTLV (HTLV antibody positive), Treponema pallidum antibody positive or TB culture positive.\n6. Patients, in the opinion of investigators, may not be eligible or not able to comply with the study.","ALL","2 Years","20 Years",{"count":62,"type":63},10,"ESTIMATED","INTERVENTIONAL",[66],"NA","This is a Phase I\u002FII clinical trial of gene therapy for treating Fanconi anemia using a self-inactivating lentiviral vector to functionally correct the defective gene. The objectives are to evaluate the safety and efficacy of the gene transfer clinical protocol.",[69],"Fanconi Anemia",[71,72,73,74],"Fanconi anemia","Lentiviral vector","FANCA","Gene","2026-06-18",{"date":77,"type":78},"2026-06-23","ACTUAL",{"date":80,"type":78},"2026-06-01",{"date":82,"type":63},"2030-12-31",{"name":5,"class":6},1]