[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Shenzhen Hemogen\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":107},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,41,59,76],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100505652","early-phase-1-the-safety-and-efficacy-evaluation-of-hgi-001-injection-in-patients-with-transfusion-dependent--thalassemia-100505652",false,"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.","ALL","18 Years","35 Years",{"count":20,"type":21},3,"ESTIMATED","INTERVENTIONAL",[24],"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",[27],"β-thalassemia","RECRUITING","2024-11-26",{"date":31,"type":32},"2024-11-29","ACTUAL",{"date":34,"type":32},"2022-05-27",{"date":36,"type":21},"2025-12-30",{"name":38,"class":39},"Shenzhen Hemogen","INDUSTRY",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":16,"minAge":47,"maxAge":18,"enrollmentInfo":48,"targetDuration":4,"studyType":22,"phases":49,"briefSummary":50,"conditions":51,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":53,"startDateStruct":54,"completionDateStruct":56,"leadSponsor":58,"locationsCount":40},"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":20,"type":21},[24],"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",[52],"α-thalassemia",{"date":31,"type":32},{"date":55,"type":32},"2022-10-08",{"date":57,"type":21},"2026-12-30",{"name":38,"class":39},{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":63,"acronym":4,"eligibilityCriteria":64,"healthyVolunteers":11,"sex":16,"minAge":65,"maxAge":66,"enrollmentInfo":67,"targetDuration":4,"studyType":22,"phases":69,"briefSummary":25,"conditions":70,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":71,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":75,"locationsCount":40},"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":68,"type":21},10,[24],[27],{"date":31,"type":32},{"date":73,"type":32},"2020-12-01",{"date":36,"type":21},{"name":38,"class":39},{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":4,"eligibilityCriteria":82,"healthyVolunteers":11,"sex":16,"minAge":83,"maxAge":18,"enrollmentInfo":84,"targetDuration":4,"studyType":22,"phases":86,"briefSummary":88,"conditions":89,"keywords":91,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":20},"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":85,"type":21},8,[87],"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.",[90],"Β-thalassemia",[27,92,93,94,95,96,97,98],"Thalassemia","Genetic Diseases, Inborn","Hemoglobinopathies","Hematologic Diseases","Anemia","Anemia, Hemolytic","Anemia, Hemolytic, Congenital","2024-10-22",{"date":101,"type":32},"2024-10-23",{"date":103,"type":32},"2024-06-12",{"date":105,"type":21},"2026-12-31",{"name":38,"class":39},""]