[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100551858":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":29,"locations":35,"responsibleParty":79,"collaborators":20,"id":81,"slug":82,"hasResults":83,"nctId":84,"briefTitle":85,"officialTitle":86,"acronym":20,"eligibilityCriteria":87,"healthyVolunteers":83,"sex":88,"minAge":89,"maxAge":90,"enrollmentInfo":91,"targetDuration":20,"studyType":94,"phases":95,"briefSummary":97,"conditions":98,"keywords":20,"overallStatus":38,"whyStopped":20,"lastUpdateSubmitDate":100,"lastUpdatePostDateStruct":101,"startDateStruct":104,"completionDateStruct":106,"leadSponsor":108,"locationsCount":109},{"fullName":5,"class":6},"Shanghai BDgene Co., Ltd.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"BD211 Single-Dose group","EXPERIMENTAL","Route of Administrate: infusion intravenously. Dosage form: injection solution. Dose: ≥ 5×10\\^6 cells \u002Fkg. Frequency of administration: One dosing intravenously. Intervention: Single dose of BD211",[13],"Genetic: BD211",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"GENETIC","BD211","Genetically modified CD34+ autologous stem cells were transfused intravenously with single dosing.",[9],null,[22,26],{"name":23,"affiliation":24,"role":25},"Sujiang Zhang, M.D.","Shanghai Ruijin Hospital, Shanghai Jiaotong University","PRINCIPAL_INVESTIGATOR",{"name":27,"affiliation":28,"role":25},"Jianpei Fang, M.D.","Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University",[30],{"name":31,"role":32,"phone":33,"phoneExt":20,"email":34},"fujun Li","CONTACT","+86-19121311061","fujun.li@bdgene.cn",[36,52,65],{"facility":37,"status":38,"city":39,"state":40,"zip":41,"country":42,"countryCode":43,"cosmosGeoPoint":44,"geoPoint":49,"contacts":50},"Sun Yat-sen Memorial Hospital","RECRUITING","Guangzhou","Guandong","510120","China","CN",{"type":45,"coordinates":46},"Point",[47,48],113.25,23.11667,{"lat":48,"lon":47},[51],{"name":27,"role":32,"phone":20,"phoneExt":20,"email":20},{"facility":53,"status":38,"city":54,"state":55,"zip":56,"country":42,"countryCode":43,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"The First Affiliated Hospital of Guangxi Medical University","Nanning","Guangxi","530021",{"type":45,"coordinates":58},[59,60],108.31667,22.81667,{"lat":60,"lon":59},[63],{"name":64,"role":32,"phone":20,"phoneExt":20,"email":20},"Yongrong Lai, M.D.",{"facility":24,"status":38,"city":66,"state":67,"zip":68,"country":42,"countryCode":43,"cosmosGeoPoint":69,"geoPoint":73,"contacts":74},"Shanghai","Shanghai City","200025",{"type":45,"coordinates":70},[71,72],121.45806,31.22222,{"lat":72,"lon":71},[75],{"name":76,"role":32,"phone":77,"phoneExt":20,"email":78},"Sujiang zhang, M.D.","+86-17717285030","zbruce.zhang@hotmail.com",{"type":80,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","100551858","phase-1-a-phase-1-study-of-gene-modified-autologous-hematopoietic-stem-cell-bd211-treating--thalassemia-major-100551858",false,"NCT06465550","A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major","A Phase 1 Clinical Trail of the Safety and Efficacy of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Intravenous Infusion for the Treatment of Transfusion-dependent β-thalassaemia Patients","Inclusion Criteria:\n\n1. Participants aged 3 years (inclusive) to 18 years (exclusive), with no gender restrictions.\n2. Parents\u002Flegal guardians have fully understood and voluntarily signed a written informed consent form; and it is recommended that children aged 8 and above be involved in the decision to participate in this clinical trial and obtain a written consent form.\n3. Transfusion-dependent β-thalassemia patients. \"Transfusion-dependent\" is defined as: requiring at least 100 mL\u002Fkg of packed red blood cells annually; the genotype can be β0\u002Fβ0, β0\u002Fβ+, or β+\u002Fβ+, diagnosed through hemoglobin studies.\n4. Eligible for allogeneic hematopoietic stem cell transplantation, but without a donor or those refusing to undergo allogeneic hematopoietic stem cell transplantation.\n5. Have undergone symptomatic treatment for at least the past 2 years and have retained medical records including transfusion history.\n6. Stable condition and maintained an appropriate iron chelation regimen.\n7. Good status of organ function.\n8. Good compliance from the individual and parents\u002Flegal guardians, willing to adhere to visit schedules, trial plans, laboratory tests, and other trial procedures as stipulated in this protocol.\n9. Willing to participate in long-term follow-up research.\n\nExclusion Criteria:\n\n1. Has a fully HLA-matched hematopoietic stem cell donor and is willing to receive a fully HLA-matched hematopoietic stem cell transplant. Enrollment is otherwise only advised after review by the safety review committee.\n2. Positive for antibodies against Human Immunodeficiency Virus 1\u002F2 (HIV-1\u002FHIV-2), Treponema pallidum (TP) specific antibodies, Human T-lymphotropic Virus 1 or 2 (HTLV-1\u002FHTLV-2) antibodies, and Vesicular Stomatitis Virus G (VSV-G).\n3. Positive for Hepatitis B Virus (HBV) HbsAg or HBV-DNA; Hepatitis C Virus (HCV) HCAb positive; positive nucleic acid test for Epstein-Barr Virus (EBV) or Cytomegalovirus (CMV).\n4. Severe active bacterial, viral, fungal, malarial, or parasitic infections.\n5. Has had, or currently has, a malignant, myeloproliferative, or immunodeficiency disorder.\n6. Direct relatives with known or suspected hereditary cancer syndromes (including but not limited to breast cancer, colorectal cancer, ovarian cancer, prostate cancer, and pancreatic cancer).\n7. Autoimmune diseases that could result in transfusion difficulties.\n8. Major organ diseases or abnormal lab tests, including:\n\n   1. Liver cirrhosis, fibrosis, or active hepatitis, and\u002For abnormal liver function tests (Serum total bilirubin (TBIL) ≥ 1.5x Upper Limit of Normal (ULN); Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) ≥ 2.5x ULN; Alkaline phosphatase ≥ 2.5x ULN).\n   2. Heart disease, or Left Ventricular Ejection Fraction (LVEF) \\\u003C 60%.\n   3. Kidney diseases, or serum creatinine ≥ 1.5ULN, creatinine clearance rate \\\u003C 30% of the normal level (measured or calculated by the Cockcroft-Gault equation).\n   4. Endocrine disorders, such as insulin-dependent diabetes, hyperthyroidism, or hypothyroidism.\n   5. Severe iron overload, serum ferritin ≥ 5000 ng\u002FmL.\n   6. Cardiac T2\\* \\\u003C 20 ms, and\u002For liver iron content (LIC) ≥ 15mg\u002Fg liver weight by MRI.\n   7. Significant pulmonary hypertension diagnosed clinically according to guidelines, requiring clinical medical intervention.\n9. Uncorrected bleeding disorders.\n10. Severe psychiatric disorders.\n11. Peripheral blood white cell (WBC) count \\\u003C 3x10\\^9\u002FL or platelets count \\\u003C 120x10\\^9\u002FL.\n12. Received hydroxyurea treatment within the last 3 months before stem cell collection.\n13. Used erythropoiesis-stimulating agents within the 3 months prior to HSC collection.\n14. History of allogeneic transplantation.\n15. Previously received any type of gene and\u002For cell therapy.\n16. Participating in another clinical trial and is within a 30-day screening period.\n17. Has contraindications to anesthesia.\n18. Has contraindications to hematopoietic stem cell collection.\n19. Allergic to the investigational drug or its excipients.\n20. Any other conditions determined by the investigator as unsuitable for participation in this clinical trial.","ALL","3 Years","35 Years",{"count":92,"type":93},9,"ESTIMATED","INTERVENTIONAL",[96],"PHASE1","This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.",[99],"β-thalassemia","2024-06-19",{"date":102,"type":103},"2024-06-24","ACTUAL",{"date":105,"type":103},"2024-01-05",{"date":107,"type":93},"2026-12",{"name":5,"class":6},3]