[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"-thalassemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:-thalassemia":24},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,40,65,83,100,130,152,171],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":25,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":28,"lastUpdatePostDateStruct":29,"startDateStruct":32,"completionDateStruct":34,"leadSponsor":36,"locationsCount":39},"100609545","a-real-world-study-to-evaluate-luspatercept-in-adults-with-transfusion-dependent-beta-thalassemia-in-the-middle-east-100609545",false,"NCT07215975","A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East","REal-World Application of Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East (RELATE): A Non-interventional Retrospective and Prospective Observational Study","Inclusion Criteria:\n\n* Male or female participants of any race aged at least 18 years at time of initiation of luspatercept treatment\n* Participants with documented diagnosis of transfusion-dependent β-thalassemia (TDT).\n* Participants who have been initiated on treatment with luspatercept as per the product's Summary of Product Characteristics (SmPC) no longer than 12 months prior to informed consent signature, and for whom therapy is ongoing.\n* Participants for whom the decision to prescribe luspatercept treatment is clearly separated from the physician's decision to include the participant in the current study.\n* Participants who have provided signed informed consent for participating in the study and for collecting and analyzing medical data pertinent to the objectives of this study\n\nExclusion Criteria:\n\n* Participants that meet any of the contraindications to the administration of luspatercept as outlined in the latest version of the locally approved SmPC.\n* Participants who are currently receiving or are planned to receive treatment with any investigational drug\u002Fdevice\u002Fintervention or who have received any investigational product within 1 month or 5 half-lives of the investigational agent (whichever is longer) prior to luspatercept therapy initiation.\n* Participants who are currently pregnant, breastfeeding, or planning a pregnancy during the study observation period.\n* Participants who have not provided signed informed consent for participating in the study and for collecting and analysing medical data pertinent to the objectives of this study.","ALL","18 Years",{"count":19,"type":20},200,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East",[24],"β-thalassemia",[26],"Transfusion-dependent β-thalassemia","RECRUITING","2026-06-01",{"date":30,"type":31},"2026-06-02","ACTUAL",{"date":33,"type":20},"2026-06-25",{"date":35,"type":20},"2031-04-17",{"name":37,"class":38},"Bristol-Myers Squibb","INDUSTRY",2,{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":47,"enrollmentInfo":48,"targetDuration":4,"studyType":50,"phases":51,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":55,"lastUpdatePostDateStruct":56,"startDateStruct":58,"completionDateStruct":60,"leadSponsor":62,"locationsCount":64},"100505652","early-phase-1-the-safety-and-efficacy-evaluation-of-hgi-001-injection-in-patients-with-transfusion-dependent--thalassemia-100505652","NCT05864170","the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia","the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia(Child)","Inclusion Criteria:\n\n1. Aged 18-35 years (inclusive), ICF can be provided by the patient and\u002For legal guardian;\n2. Definitively diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;\n3. Average transfusion volume \\> 100 mL\u002Fkg\u002Fyear or transfusion frequency \\> 8 times\u002Fyear within 2 years prior to enrollment, or has been definitively diagnosed with TDT;\n4. At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g\u002FdL;\n5. Ferritin load \\\u003C 3000 μg\u002FL, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months before screening (including prescription or receipt) can be provided;\n6. Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged by the investigator;\n7. Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-001 injection.\n\nExclusion Criteria:\n\n1. Patients with fully HLA-matched donors;\n2. Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;\n3. Have previously undergone splenectomy;\n4. Uncorrected bleeding disorder;\n5. Uncontrolled epilepsy and mental illness;\n6. Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;\n7. Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;\n8. Patients with pulmonary hypertension who have not been given effective intervention;\n9. Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment;\n10. Positive for anti-RBC antibodies in antibody screening;\n11. Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number \\> upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled). In certain clinical environments\u002Fregions, subjects who are positive for other tests can also be excluded from the trial, such as, human lymphocytic virus-1 (HTLV-1) or -2 (HTLV-2), tuberculosis, and toxoplasmosis.\n12. Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;\n13. Immediate family member with or suspected of having a familial cancer (including but not limited to hereditary breast and ovarian cancers, nonpolyposis colorectal cancer, and adenomatous polyposis);\n14. Severe bacterial, viral, fungal or parasitic infection;\n15. Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin \\> 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance \\\u003C 30% of normal;\n16. WBC \\\u003C 3 × 109\u002FL and\u002For PLT \\\u003C 100 × 109\u002FL;\n17. Has diabetes, abnormal thyroid functions or other endocrine disorder;\n18. Participated in other interventional clinical studies within 4 weeks before the trial;\n19. Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.","35 Years",{"count":49,"type":20},3,"INTERVENTIONAL",[52],"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",[24],"2024-11-26",{"date":57,"type":31},"2024-11-29",{"date":59,"type":31},"2022-05-27",{"date":61,"type":20},"2025-12-30",{"name":63,"class":38},"Shenzhen Hemogen",1,{"id":66,"slug":67,"hasResults":11,"nctId":68,"briefTitle":69,"officialTitle":69,"acronym":4,"eligibilityCriteria":70,"healthyVolunteers":11,"sex":16,"minAge":71,"maxAge":47,"enrollmentInfo":72,"targetDuration":4,"studyType":50,"phases":73,"briefSummary":74,"conditions":75,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":55,"lastUpdatePostDateStruct":77,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":64},"100504651","early-phase-1-the-safety-and-efficacy-evaluation-of-hgi-002-injection-in-patients-with-transfusion-dependent--thalassemia-100504651","NCT05851105","the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia","Inclusion Criteria:\n\n1. Aged 12-35 years (inclusive), ICF can be provided by the patient and\u002For legal guardian;\n2. Definitively α- thalassemia diagnosed with severe TDT without genotype restriction, and a valid test report can be provided;\n3. Average transfusion volume \\> 100 mL\u002Fkg\u002Fyear or transfusion frequency \\> 8 times\u002Fyear within 2 years prior to enrollment, or has been definitively diagnosed with TDT;\n4. At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g\u002FdL;\n5. Ferritin load \\\u003C 3000 μg\u002FL, cardiac and liver iron indicates moderate or lesser iron overload; records of iron chelation treatments within 3 months before screening (including prescription or receipt) can be provided;\n6. Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged by the investigator;\n7. Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-002 injection.\n\nExclusion Criteria:\n\n1. Patients with fully HLA-matched donors;\n2. Received allogeneic transplantation, which needs to be weighed and evaluated by an expert committee; received other gene therapies;\n3. Have previously undergone splenectomy;\n4. Uncorrected bleeding disorder;\n5. Uncontrolled epilepsy and mental illness;\n6. Received hydroxyurea, ruxolitinib, decitabine, or cytarabine within 3 months prior to enrollment;\n7. Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;\n8. Patients with pulmonary hypertension who have not been given effective intervention;\n9. Persistent toxicity (≥ CTCAE grade 2) induced by previous treatment;\n10. Positive for anti-RBC antibodies in antibody screening;\n11. Positive for hepatitis B surface antigen (HBsAg) and HBV DNA copy number \\> upper limit of normal (ULN) (HBV DNA test not required for patients negative for HBsAg), positive for hepatitis C virus (HCV) antibody, positive human immunodeficiency virus (HIV), or positive for Treponema pallidum antibody (TP-Ab) (subjects who are positive for the antibody due to vaccination can be enrolled). In certain clinical environments\u002Fregions, subjects who are positive for other tests can also be excluded from the trial, such as, human lymphocytic virus-1 (HTLV-1) or -2 (HTLV-2), tuberculosis, and toxoplasmosis.\n12. Has or has had malignant tumors or myeloproliferative disease or immunodeficiency disease;\n13. Immediate family member with or suspected of having a familial cancer (including but not limited to hereditary breast and ovarian cancers, nonpolyposis colorectal cancer, and adenomatous polyposis);\n14. Severe bacterial, viral, fungal or parasitic infection;\n15. Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin \\> 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance \\\u003C 30% of normal;\n16. WBC \\\u003C 3 × 109\u002FL and\u002For PLT \\\u003C 100 × 109\u002FL;\n17. Has diabetes, abnormal thyroid functions or other endocrine disorder;\n18. Participated in other interventional clinical studies within 4 weeks before the trial;\n19. Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.","12 Years",{"count":49,"type":20},[52],"This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients",[76],"α-thalassemia",{"date":57,"type":31},{"date":79,"type":31},"2022-10-08",{"date":81,"type":20},"2026-12-30",{"name":63,"class":38},{"id":84,"slug":85,"hasResults":11,"nctId":86,"briefTitle":87,"officialTitle":87,"acronym":4,"eligibilityCriteria":88,"healthyVolunteers":11,"sex":16,"minAge":89,"maxAge":90,"enrollmentInfo":91,"targetDuration":4,"studyType":50,"phases":93,"briefSummary":53,"conditions":94,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":55,"lastUpdatePostDateStruct":95,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":99,"locationsCount":64},"100496538","early-phase-1-safety-and-efficacy-evaluation-of--globin-restored-autologous-hematopoietic-stem-cells-in--thalassemia-major-patients-100496538","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.","8 Years","16 Years",{"count":92,"type":20},10,[52],[24],{"date":57,"type":31},{"date":97,"type":31},"2020-12-01",{"date":61,"type":20},{"name":63,"class":38},{"id":101,"slug":102,"hasResults":11,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":4,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":16,"minAge":107,"maxAge":47,"enrollmentInfo":108,"targetDuration":4,"studyType":50,"phases":109,"briefSummary":111,"conditions":112,"keywords":114,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":49},"100566475","phase-1-evaluating-safety-and-efficacy-of-lentiviral-transduced-cd34-hscs-in--thalassaemia-patients-100566475","NCT06655662","Evaluating Safety and Efficacy of Lentiviral-transduced CD34+ HSCs in Β-thalassaemia Patients.","An Open, Multi-center, Phase I Clinical Study on the Safety and Efficacy of HGI-001 Injection in Patients with Transfusion-Dependent Β-Thalassemia.","Inclusion Criteria:\n\n1. Aged 6-35 years (inclusive), ICF can be provided by the patient and\u002For legal guardian;\n2. Definitively diagnosed with severe TDT without genotype restriction (excluding patients with coexisting α-thalassemia), and a valid test report can be provided;\n3. Average transfusion volume \\> 100 mL\u002Fkg\u002Fyear or transfusion frequency \\> 8 times\u002Fyear within 2 years prior to enrollment；\n4. At least 3 months of full volume transfusion (verification of blood transfusion records can be provided) prior to screening, and Hb is maintained at ≥ 9.0 g\u002FdL;\n5. Serum ferritin level less than 5000μg\u002FL, with moderate or lower iron overload in the heart and liver as indicated by magnetic resonance imaging (MRI T2\\*), specifically liver MRI T2\\* greater than 1.4ms and cardiac MRI T2\\* greater than 10ms;\n6. Acceptable organ functions (including heart, liver, kidney, lung and coagulation functions), stable disease condition, and suitable for busulfan pre-treatment and hematopoietic stem cell (HSC) transplantation as judged by the investigator;\n7. Meets follow-up requirements, adheres to treatment arrangements, and is able to return to the hospital regularly to undergo various examinations within 2 years after reinfusion of HGI-001 injection.\n\nExclusion Criteria:\n\n1. Patients with fully HLA-matched donors;\n2. Having previously received gene therapy, gene editing therapy, or allogeneic hematopoietic stem cell transplantation;\n3. Uncorrected bleeding disorder;\n4. Uncontrolled epilepsy and mental illness;\n5. Within the past 3 months prior to enrollment, the use of Luspatercept, Hydroxyurea, Ruxolitinib, Thalidomide, Decitabine, or Ara-c has been administered；\n6. Psychoactive substance abuse, drug or alcohol abuse within 6 months prior to enrollment;\n7. Patients with pulmonary hypertension who have not been given effective intervention;\n8. Positive for anti-RBC antibodies in antibody screening;\n9. Hepatitis B surface antigen (HBsAg) is positive and the HBV DNA copy number is greater than the upper limit of the normal value of the detection unit (those who are negative do not need to test for HBV DNA copy number), antibodies to Hepatitis C virus (HCV) are positive, antibodies to Human Immunodeficiency Virus (HIV) are positive, or antibodies to Treponema pallidum (TP-Ab) are positive (subjects who are positive due to vaccination are eligible for enrollment). Additionally, the results of Hepatitis B Virus (HBV) DNA testing, Hepatitis C Virus (HCV) RNA testing, Cytomegalovirus DNA testing, and Epstein-Barr Virus (EBV) DNA testing are abnormal；\n10. Have or have had malignant tumors or myeloproliferative diseases or immunodeficiency disorders or autoimmune diseases;\n11. Have a first-degree relative with a history of or suspected hereditary cancer (including but not limited to hereditary breast and ovarian cancer, nonpolyposis colorectal cancer, and adenomatous polyposis);\n12. Severe bacterial, viral, fungal or parasitic infection;\n13. Other illnesses which render the subject unsuitable for participation (e.g., severe liver, kidney or heart disease); Definition of severe liver and kidney disease: a. Aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin \\> 3 × ULN; b. Liver magnetic resonance imaging (MRI) indicates significant cirrhosis; c. Liver biopsy indicates cirrhosis, severe fibrosis or active hepatitis (liver biopsy is only performed when liver MRI indicates active hepatitis and significant fibrosis without evidence for cirrhosis); d. Creatinine clearance \\\u003C60 mL\u002F(min·1.73m\\^2);\n14. WBC \\\u003C 3 × 10\\^9\u002FL and\u002For PLT \\\u003C 100 × 10\\^9\u002FL;\n15. Has diabetes, abnormal thyroid functions or other endocrine disorder;\n16. Participated in other interventional clinical studies within 4 weeks before the trial;\n17. Poor adherence or other conditions that renders the subject unsuitable for participation as judged by the investigator.","6 Years",{"count":5,"type":20},[110],"PHASE1","This is a single-arm, open label, multi-center, single-dose Phase 1 clinical trial in subjects with transfusion dependent β-thalassaemia. The study aims to evaluate the safety and efficacy of autologous lentiviral-transduced CD34+ human hematopoietic stem cells (hHSCs) using the gene therapy product HGI-001.",[113],"Β-thalassemia",[24,115,116,117,118,119,120,121],"Thalassemia","Genetic Diseases, Inborn","Hemoglobinopathies","Hematologic Diseases","Anemia","Anemia, Hemolytic","Anemia, Hemolytic, Congenital","2024-10-22",{"date":124,"type":31},"2024-10-23",{"date":126,"type":31},"2024-06-12",{"date":128,"type":20},"2026-12-31",{"name":63,"class":38},{"id":131,"slug":132,"hasResults":11,"nctId":133,"briefTitle":134,"officialTitle":135,"acronym":4,"eligibilityCriteria":136,"healthyVolunteers":11,"sex":16,"minAge":137,"maxAge":47,"enrollmentInfo":138,"targetDuration":4,"studyType":50,"phases":140,"briefSummary":141,"conditions":142,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":143,"lastUpdatePostDateStruct":144,"startDateStruct":146,"completionDateStruct":148,"leadSponsor":150,"locationsCount":49},"100551858","phase-1-a-phase-1-study-of-gene-modified-autologous-hematopoietic-stem-cell-bd211-treating--thalassemia-major-100551858","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.","3 Years",{"count":139,"type":20},9,[110],"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.",[24],"2024-06-19",{"date":145,"type":31},"2024-06-24",{"date":147,"type":31},"2024-01-05",{"date":149,"type":20},"2026-12",{"name":151,"class":38},"Shanghai BDgene Co., Ltd.",{"id":153,"slug":154,"hasResults":11,"nctId":155,"briefTitle":156,"officialTitle":157,"acronym":4,"eligibilityCriteria":158,"healthyVolunteers":11,"sex":16,"minAge":137,"maxAge":17,"enrollmentInfo":159,"targetDuration":4,"studyType":50,"phases":160,"briefSummary":141,"conditions":162,"keywords":4,"overallStatus":27,"whyStopped":4,"lastUpdateSubmitDate":163,"lastUpdatePostDateStruct":164,"startDateStruct":166,"completionDateStruct":168,"leadSponsor":170,"locationsCount":64},"100498705","safety-and-efficacy-of-gene-modified-autologous-hematopoietic-stem-cells-to-treat-transfusion-dependent-beta-thalassemia-100498705","NCT05773729","Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia","Safety and Efficacy of Lentiviral Vector Transduction of β-globin Genetically Modified Autologous CD34+ Hematopoietic Stem Cells in Patients With Transfusion-dependent β-thalassemia","Inclusion Criteria:\n\n1. Ages 3 to 18 years old, including:\n\n   The parents or legal guardians must be able to understand and provide ICFs. If available, it is strongly recommended that children aged ≥8 years in treatment decisions and obtain written ICFs and be clearly documented; Diagnosed as Transfusion Dependent β-thalassemia with any genotype (β0, β+, βE\u002Fβ0, βS\u002FS, βS\u002Fβ0, βS\u002Fβ+), confirmed the Hb analysis. No alfa chain genetic abnormalities. Subjects must stabilize and maintain an appropriate iron chelation regimen. Transfusion-dependent types are defined as requiring at least 100 mL\u002Fkg\u002F year of red blood cells (pRBCs).\n2. No eligiblity for allogeneic hematopoietic stem cell transplantation.\n3. The treatment of erythrocyte maturation agent luspatercept cannot be financially supported.\n4. The subjects' parents\u002Flegal guardians must be willing and able to follow the study procedures in the study protocol.\n5. Good organs' functions.\n6. Having complete medical records including a history of blood transfusions testified subject received treatment and followed up for at least two years prior to screening .\n\nExclusion Criteria:\n\n1. Availability of voluntary, fully HLA-matched hematopoietic cell donors, unless recommended for inclusion by the Monitoring Committee.\n2. HIV-1 and HIV-2 were positive, and \u002F or HTLV-1, HTLV-2 and VSV-G antibodies were positive.\n3. An active bacterial, viral, fungal or parasitic infection.\n4. Contraindicated for the extraction of bone marrow under anesthesia.\n5. Any malignancy, myeloproliferative, or immunodeficient disease and relevant medical history.\n6. Peripheral blood white blood cell (WBC) count \\\u003C 3×10\\^9\u002FL or platelet count \\\u003C 120×10\\^9\u002FL.\n7. A history of allo-transplantation.\n8. Erythropoietin was used within 3 months prior to HSC cell collection.\n9. Immediate family members with known or suspected familial cancer syndromes (including but not limited to breast, colorectal, ovarian, prostate, and pancreatic cancers).\n10. Subjects with a diagnosis of major mental illness may had a serious disability to participate in the study.\n11. Active recurrent malaria.\n12. Had autoimmune diseases that may make blood transfusions difficult.\n13. History of major organ injury including:\n\n    Liver disease, transaminase \\> 3 times the upper limit of normal. (If the liver biopsy does not reveal evidence of widespread bridging fibrosis, cirrhosis, or acute hepatitis, this indicator will not be used as a criterion for the exclusion); Widely bridging fibrosis, histopathological evidence of acute hepatitis or cirrhosis showed in liver biopsy Heart disease, left ventricular ejection fraction \\\u003C 25%; Kidney disease, creatinine clearance \\\u003C 30% normal level; Of severe iron overload, confirmed by the study doctor; An heart MRI detection of T2 \\* \\\u003C 10 ms; Significant pulmonary hypertension needing clinical medical intervention.\n14. There are bleeding diseases that have not been cured.\n15. The subject involved with another clinical study in a 30-day screening period.\n16. Allergic to the research drug and its excipients.\n17. Prior treatment with any type of gene and\u002For cell therapy.\n18. As assessed by the investigator, the subjects or their parents are unable to comply well with the study procedures per protocol.\n19. Hydroxyurea treatment within 3 months prior to hematopoietic stem cell collection.\n20. Had diseases that interfere with hematopoietic stem cells collections.\n21. Any other conditions being ineligible for HSC transplantation determined by the investigator.",{"count":92,"type":20},[161],"NA",[24],"2024-06-13",{"date":165,"type":31},"2024-06-14",{"date":167,"type":31},"2023-09-15",{"date":169,"type":20},"2026-10",{"name":151,"class":38},{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":4,"eligibilityCriteria":177,"healthyVolunteers":178,"sex":16,"minAge":179,"maxAge":180,"enrollmentInfo":181,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":183,"conditions":184,"keywords":4,"overallStatus":185,"whyStopped":4,"lastUpdateSubmitDate":186,"lastUpdatePostDateStruct":187,"startDateStruct":189,"completionDateStruct":191,"leadSponsor":193,"locationsCount":4},"100549941","eklf-gene-expression-in--thalassemia-100549941","NCT06440603","EKLF Gene Expression in β-thalassemia","Erythroid Krüppel Like Factor (EKLF) Gene Expression in β-thalassemia Patients","Inclusion Criteria:\n\n* patients with β-thalassemia (major and intermedia).\n* patients are of both sexes (male or female) at any age\n\nExclusion Criteria:\n\n* patients with any other types of hemolytic anaemia",true,"5 Years","80 Years",{"count":182,"type":20},150,"1. Studying the effect of expression pattern of EKLF gene in β-thalassemic patients.\n2. Detecting the correlation between the gene expression of EKLF and the clinical phenotype of β-thalassemic patients.",[24],"NOT_YET_RECRUITING","2024-05-31",{"date":188,"type":31},"2024-06-04",{"date":190,"type":20},"2024-07-01",{"date":192,"type":20},"2027-07-01",{"name":194,"class":195},"Rofaida Hassan Ahmed","OTHER"]