[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100496538":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":28,"locations":34,"responsibleParty":50,"collaborators":20,"id":52,"slug":53,"hasResults":54,"nctId":55,"briefTitle":56,"officialTitle":56,"acronym":20,"eligibilityCriteria":57,"healthyVolunteers":54,"sex":58,"minAge":59,"maxAge":60,"enrollmentInfo":61,"targetDuration":20,"studyType":64,"phases":65,"briefSummary":67,"conditions":68,"keywords":20,"overallStatus":36,"whyStopped":20,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":79},{"fullName":5,"class":6},"Shenzhen Hemogen","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Experimental","EXPERIMENTAL","Ten transfusion-dependent β-thalassaemia subjects aged 8-16 years will be reinfused with β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q.",[13],"Biological: β-globin restored autologous hematopoietic stem cells",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"BIOLOGICAL","β-globin restored autologous hematopoietic stem cells","β-globin-restored autologous hematopoietic stem cells modified with LentiHBBT87Q",[9],null,[22,25],{"name":23,"affiliation":5,"role":24},"Chao Liu, PHD","PRINCIPAL_INVESTIGATOR",{"name":26,"affiliation":27,"role":24},"Sixi Liu, Professor","Shenzhen Children's Hospital",[29],{"name":30,"role":31,"phone":32,"phoneExt":20,"email":33},"Haigang Sun","CONTACT","13823168465","sunhaigang@genomics.cn",[35],{"facility":27,"status":36,"city":37,"state":38,"zip":39,"country":40,"countryCode":41,"cosmosGeoPoint":42,"geoPoint":47,"contacts":48},"RECRUITING","Shenzhen","Guangdong","518083","China","CN",{"type":43,"coordinates":44},"Point",[45,46],114.0683,22.54554,{"lat":46,"lon":45},[49],{"name":30,"role":31,"phone":32,"phoneExt":20,"email":33},{"type":51,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100496538","early-phase-1-safety-and-efficacy-evaluation-of--globin-restored-autologous-hematopoietic-stem-cells-in--thalassemia-major-patients-100496538",false,"NCT05745532","Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients","Inclusion Criteria:\n\n* 8-16 years old. Subject and\u002For subject's legal guardian fully understand and voluntarily sign informed consent;\n* Clinically diagnosed as transfusion-dependent β-thalassemia major;\n* With sufficient RBC infusion, subjects must maintain hemoglobin ≥9g\u002FdL, serum ferritin threshold ≤ 3000 ng\u002FmL and the liver iron overload mild or absent for at least 3 months before mobilization of hematopoietic stem cell;\n* Follow the arrangements for treatment and regular medical checks within two years post-transplantation\n\nExclusion Criteria:\n\n* The physical condition does not meet the requirements for hematopoietic stem cell mobilization and transplantation myeloablation;\n* Received gene therapy and allogeneic HSCT in the past.\n* Have an available HLA matched donor.\n* Enrolling in another clinical trial.\n* Other unsuitable conditions identified by doctors.","ALL","8 Years","16 Years",{"count":62,"type":63},10,"ESTIMATED","INTERVENTIONAL",[66],"EARLY_PHASE1","This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients",[69],"β-thalassemia","2024-11-26",{"date":72,"type":73},"2024-11-29","ACTUAL",{"date":75,"type":73},"2020-12-01",{"date":77,"type":63},"2025-12-30",{"name":5,"class":6},1]