[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Bioray Laboratories\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":213},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,42,65,83,107,129,150,172,194],{"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":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100612022","universal-car-t-cell-therapy-for-mm-100612022",false,"NCT07248176","Universal CAR-T Cell Therapy for MM","A Clinical Study on the Safety and Efficacy of Allogeneic CAR T Cells Targeting BCMA in the Treatment of Adult r\u002Fr Multiple Myeloma","Inclusion Criteria:\n\n1. Expected survival of at least 3 months;\n2. Subjects should have measurable disease that meets the IMWG 2016 criteria;\n3. Previously received at least two lines of prior anti-myeloma therapy ；\n4. Relapse , failure to achieve at least a minimal response, or disease progression after the last treatment ；\n5. BCMA positive；\n6. ECOG score 0-1；\n7. No severe impairment or suppression of liver, kidney, coagulation, bone marrow, or lung function.\n\nExclusion Criteria:\n\n1. Pregnant or breastfeeding women;\n2. History of other malignant tumors;\n3. Active autoimmune diseases requiring immunotherapy;\n4. Previously received allogeneic stem cell transplantation;\n5. Previous use of CAR-T cells or other genetically modified T cell therapies;\n6. Previously received targeted BCMA therapy;\n7. Severe cardiovascular disease;\n8. Active infection;\n9. Positive virology test;\n10. Clinically significant central nervous system (CNS) diseases or pathological changes.","ALL","18 Years",{"count":19,"type":20},6,"ESTIMATED","INTERVENTIONAL",[23],"NA","This trial aims to evaluate the safety and efficacy of BCMA-UCART in treating patients with r\u002Fr multiple myeloma.",[26,27,28],"Multiple Myeloma","Multiple Myeloma in Relapse","Multiple Myeloma, Refractory","RECRUITING","2025-11-28",{"date":32,"type":33},"2025-12-01","ACTUAL",{"date":35,"type":33},"2025-04-10",{"date":37,"type":20},"2028-04-18",{"name":39,"class":40},"Bioray Laboratories","INDUSTRY",1,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":47,"acronym":4,"eligibilityCriteria":48,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":49,"targetDuration":4,"studyType":21,"phases":51,"briefSummary":53,"conditions":54,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":56,"lastUpdatePostDateStruct":57,"startDateStruct":59,"completionDateStruct":61,"leadSponsor":63,"locationsCount":64},"100496217","phase-1-the-safety-and-efficacy-of-brl-201-in-the-treatment-of-rr-b-lymphocyte-non-hodgkin-lymphoma-100496217","NCT05741359","The Safety and Efficacy of BRL-201 in the Treatment of r\u002Fr B Lymphocyte Non-Hodgkin Lymphoma","A Phase I\u002FII Clinical Study of the Safety and Efficacy of CD19-targeted Non-viral PD1 Site-specific Integrated CAR-T Cell Injection (BRL-201) in the Treatment of Relapsed or Refractory B Lymphocyte Non-Hodgkin Lymphoma","Inclusion Criteria:\n\n1. Willing to participate in this clinical study and sign an informed consent form;\n2. Age ≥ 18 years old;\n3. Estimated survival time ≥ 3 months;\n4. Presence of at least one measurable lesion as assessed according to Lugano Classification 2014 for response assessment in lymphomas (i.e., the cross-sectional images obtained by CT show that the long diameter of lymph node lesions is \\> 15 mm or the long diameter of extranodal lesions is \\> 10 mm, and FDG-PET scan results are positive). Lesions, for which radiotherapy was provided, can be regarded as measurable lesions only if there is an unequivocal progression after radiotherapy;\n5. Histopathologically confirmed aggressive B-NHL; positive expression of CD19 in tumors detected by immunohistochemistry or flow cytometry; pathological types of B-NHL (according to WHO Lymphoma Classification 2016);\n6. Relapsed or refractory diseases;\n7. Subjects who must receive adequate prior therapy;\n8. Absence of invasion of central nervous system (CNS) lymphoma by cranial magnetic resonance imaging (MRI);\n9. Hematological parameters meeting the requirements;\n10. Blood biochemistry meeting the requirements;\n11. LVEF ≥ 55%;\n12. No severe pulmonary disorders;\n13. Toxic reactions induced by prior anti-lymphoma therapy must be stable and resolved to grade ≤ 1;\n14. Eastern Cooperative Oncology Group (ECOG) performance status score of 0-1;\n15. Patients with physical conditions for apheresis of peripheral blood; 16 . Willing to abide by the rules formulated in the study protocol.\n\nExclusion Criteria:\n\n1. Pregnant or lactating women;\n2. Subjects who previously received allogeneic cell therapies, including allogeneic stem cell transplant;\n3. Subjects who previously received anti-CD19 targeted therapy, except those who receive BRL-201 and are eligible to receive reinfusion in this study;\n4. Prior treatment with any CAR-T cell product or other genetically modified T cell therapies;\n5. History of Richter's transformation of chronic lymphocytic leukemia (CLL);\n6. Presence of uncontrollable fungal, bacterial, viral, or other infections requiring systemic therapy. Patients can be enrolled if the simple urinary tract infection or pharyngitis responds to treatment;\n7. Subjects with positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and peripheral blood HBV DNA titer higher than the upper limit of detection; hepatitis C virus (HCV) antibody positive and peripheral blood HCV RNA positive; human immunodeficiency virus (HIV) antibody positive; syphilis test positive;\n8. Severe mental disorders; history of CNS disorders (e.g., epileptic seizure, cerebrovascular ischemia\u002Fhemorrhage, dementia, cerebellar diseases, or any CNS-involved autoimmune disorders);\n9. Active autoimmune disorders requiring immunotherapy, including but not limited to end organ damages caused by autoimmune disorders (e.g., Crohn's disease, rheumatoid arthritis, and systemic lupus erythematosus) in the past 2 years, or requiring systemic application of immunosuppressive drugs or other drugs for systemic control of diseases;\n10. Primary immunodeficiency;\n11. History of other malignancies;\n12. Patients with severe cardiovascular disorders, including but not limited to those with lymphoma infiltration in the cardiac atrium or ventricles and those with a history of myocardial infarction, cardioangioplasty or stent implantation, unstable angina, or other clinically significant heart diseases within 12 months before enrollment;\n13. History of deep venous thrombosis or pulmonary embolism within 6 months before enrollment;\n14. Patients who are receiving oral anticoagulant therapy; prothrombin time (PT), activated partial thromboplastin time (APTT), or international normalized ratio (INR) \\> 1.5 × ULN without anticoagulant therapy;\n15. Presence of any indwelling tube or catheter (e.g., tube or catheter for percutaneous nephrostomy, indwelling catheter, or catheter in pleural cavity\u002Fperitoneal cavity\u002Fpericardium). Dedicated central venous access catheters (e.g., Port-a-Cath or Hickman catheter) are permitted;\n16. Lymphoma cells detected in cerebrospinal fluid, presence of brain metastases, history of CNS lymphoma, or history of lymphoma cells detected in cerebrospinal fluid or brain metastases;\n17. Conditions (e.g., intestinal obstruction or vascular compression) requiring emergency treatment due to tumor masses;\n18. History of severe immediate hypersensitivity to any drug to be used in this study;\n19. Vaccination of live vaccines, excluding corona virus disease 2019 (COVID-19) vaccines, within ≤ 6 weeks before the start of the pretreatment regimen;\n20. Any circumstances that possibly increase the risk of subjects or interfere with the study results as judged by the investigator.",{"count":50,"type":20},18,[52],"PHASE1","This is a multi-center, single-arm, open-label clinical study, and the sample size is set to 12-18 subjects.",[55],"Non-hodgkin Lymphoma,B Cell","2025-11-20",{"date":58,"type":33},"2025-11-25",{"date":60,"type":33},"2023-04-25",{"date":62,"type":20},"2027-01-15",{"name":39,"class":40},3,{"id":66,"slug":67,"hasResults":11,"nctId":68,"briefTitle":69,"officialTitle":70,"acronym":4,"eligibilityCriteria":71,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":72,"targetDuration":4,"studyType":21,"phases":73,"briefSummary":74,"conditions":75,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":76,"lastUpdatePostDateStruct":77,"startDateStruct":78,"completionDateStruct":80,"leadSponsor":82,"locationsCount":41},"100612021","universal-car-t-cell-therapy-for-nhl-100612021","NCT07248163","Universal CAR-T Cell Therapy for NHL","A Clinical Study of the Safety and Efficacy of Universal CAR-T Cells Targeting CD19 in the Treatment of r\u002Fr B Lymphocyte Non-Hodgkin Lymphoma","Inclusion Criteria:\n\n1. Willing to participate in this clinical study and sign an informed consent form;\n2. Age ≥ 18 years old;\n3. Estimated survival time ≥ 3 months;\n4. At least one measurable lesion;\n5. CD19 positively expressed;\n6. ECOG score 0-1；\n7. Hematology, coagulation and biochemistry parameters meeting the requirements;\n8. LVEF ≥ 55%;\n9. No severe pulmonary disorders;\n\nExclusion Criteria:\n\n1. Pregnant or lactating women;\n2. Subjects who previously received allogeneic cell therapies, including allogeneic stem cell transplant;\n3. Subjects who previously received anti-CD19 targeted therapy;\n4. Prior treatment with any CAR-T cell product or other genetically modified T cell therapies;\n5. History of Richter's transformation of chronic lymphocytic leukemia (CLL);\n6. Presence of uncontrollable fungal, bacterial, viral, or other infections requiring systemic therapy;\n7. Subjects with positive hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and peripheral blood HBV DNA titer higher than the upper limit of detection; hepatitis C virus (HCV) antibody positive and peripheral blood HCV RNA positive; human immunodeficiency virus (HIV) antibody positive; syphilis test positive;\n8. Severe mental disorders; history of CNS disorders (e.g., epileptic seizure, cerebrovascular ischemia\u002Fhemorrhage, dementia, cerebellar diseases, or any CNS-involved autoimmune disorders);\n9. Active autoimmune disorders requiring immunotherapy, including but not limited to end organ damages caused by autoimmune disorders (e.g., Crohn's disease, rheumatoid arthritis, and systemic lupus erythematosus) in the past 2 years, or requiring systemic application of immunosuppressive drugs or other drugs for systemic control of diseases;\n10. Primary immunodeficiency;\n11. History of other malignancies;\n12. Patients with severe cardiovascular disorders;\n13. Any circumstances that possibly increase the risk of subjects or interfere with the study results as judged by the investigator.",{"count":19,"type":20},[23],"This is a single-center, single-arm, open-label clinical study, and the sample size is set to 3-6 subjects.",[55],"2025-11-18",{"date":58,"type":33},{"date":79,"type":33},"2025-03-11",{"date":81,"type":20},"2028-03-11",{"name":39,"class":40},{"id":84,"slug":85,"hasResults":11,"nctId":86,"briefTitle":87,"officialTitle":88,"acronym":4,"eligibilityCriteria":89,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":90,"enrollmentInfo":91,"targetDuration":4,"studyType":21,"phases":93,"briefSummary":95,"conditions":96,"keywords":4,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":4},"100568446","early-phase-1-universal-car-t-cell-therapy-for-refractory-lupus-nephritis-100568446","NCT06681337","Universal CAR-T Cell Therapy for Refractory Lupus Nephritis","A Clinical Study of the Safety and Efficacy of Universal CAR-T Cells Targeting BCMA and CD19 for the Treatment of Refractory Lupus Nephritis","Inclusion Criteria:\n\n* Aged 18-65 years; both genders eligible.\n* Subjects diagnosed with lupus nephritis.\n* Previous treatment outcomes were unsatisfactory.\n* Diagnosis of active nephritis type III or IV with or without type V according to 2018 International Society of Nephrology and Society of Renal Pathology (ISN\u002FRPS) criteria.\n* NIH Activity Index \\> 2 and elevated chronicity index.\n* Urine protein: creatinine ratio (UPCR) ≥ 1.0 g\u002Fg, or 24-hour urine protein ≥ 1.0 g, with or without active urine sediment with red blood cell casts.\n* Receiving hormones with or without antimalarials.\n* SLEDAI-2K score ≥ 6.\n* Antinuclear antibody positive, and\u002For anti-ds-DNA antibody positive, and\u002For anti-Smith antibody positive.\n* Positive expression of CD19 on B cells in peripheral blood.\n* Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.\n* Provides written informed consent.\n\nExclusion Criteria:\n\n* History of solid organ transplantation.\n* Malignant tumor within the last two years.\n* Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.\n* Primary immunodeficiency (congenital or acquired).\n* Severe cardiac disease.\n* History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.\n* Allergic constitution or a history of severe allergies.\n* Pregnant or breastfeeding women.","65 Years",{"count":92,"type":20},10,[94],"EARLY_PHASE1","This investigator-initiated trial aims to assess the efficacy and safety of combination therapy using universal CAR-T cells targeting BCMA and CD19 in refractory lupus nephritis.",[97],"Lupus Nephritis","NOT_YET_RECRUITING","2024-11-07",{"date":101,"type":33},"2024-11-08",{"date":103,"type":20},"2024-11-25",{"date":105,"type":20},"2025-12",{"name":39,"class":40},{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":4,"eligibilityCriteria":113,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":90,"enrollmentInfo":114,"targetDuration":4,"studyType":21,"phases":115,"briefSummary":116,"conditions":117,"keywords":119,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":126,"leadSponsor":128,"locationsCount":4},"100564735","phase-1-the-safety-and-efficacy-of-universal-car-t-cells-targeting-bcma-in-the-treatment-of-refractory-nmosd-100564735","NCT06633042","The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory NMOSD","The Safety and Efficacy of Universal CAR-T Cells Targeting BCMA in the Treatment of Refractory AQP4 Antibody Positive Neuromyelitis Optica Spectrum Disease","Inclusion Criteria:\n\n* Aged 18-65 years ; both genders eligible.\n* Meets the criteria for Refractory NMOSD.\n* Anticipated survival of ≥ 12 weeks as judged by the researcher.\n* Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.\n* Provides written informed consent.\n\nExclusion Criteria:\n\n* History of solid organ transplantation.\n* Malignant tumor within the last two years.\n* Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.\n* Primary immunodeficiency (congenital or acquired).\n* Severe cardiac disease.\n* History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.\n* Allergic constitution or a history of severe allergies.\n* Pregnant or breastfeeding women.",{"count":50,"type":20},[52],"This is an open label, Multi-center，dose-escalation study in up to 18 participants with refractory NMOSD. This study aims to evaluate the safety and efficacy of universal CAR-T Cells targeting BCMA in the Treatment of refractory NMOSD.",[118],"Autoimmune Disease",[120,121,122],"Universal BCMA CART","AQP4 Antibody Positive","RefractoryNeuromyelitis Optica Spectrum Disease",{"date":124,"type":33},"2024-11-12",{"date":103,"type":20},{"date":127,"type":20},"2025-12-12",{"name":39,"class":40},{"id":130,"slug":131,"hasResults":11,"nctId":132,"briefTitle":133,"officialTitle":134,"acronym":4,"eligibilityCriteria":135,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":90,"enrollmentInfo":136,"targetDuration":4,"studyType":21,"phases":138,"briefSummary":139,"conditions":140,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":142,"lastUpdatePostDateStruct":143,"startDateStruct":145,"completionDateStruct":147,"leadSponsor":149,"locationsCount":41},"100515185","universal-car-t-cells-brl-301-in-refractory-systemic-lupus-erythematosus-100515185","NCT05988216","Universal CAR-T Cells (BRL-301) in Refractory Systemic Lupus Erythematosus","A Clinical Study on the Safety and Efficacy of BRL-301 (Allogeneic Chimeric Antigen Receptor T Cell Injection Targeting CD19 Gene) in the Treatment of Refractory Systemic Lupus Erythematosus","Inclusion Criteria:\n\n1. Age range from 18 to 65 years old (including threshold), regardless of gender;\n2. Subjects diagnosed with SLE according to the 2019 EULAR\u002FACR SLE classification criteria; ANA ≥ 1:80, or positive for anti dsDNA and\u002For anti Sm antibodies;\n3. The condition becomes active again after conventional treatment is ineffective or the disease relapses. Conventional treatment is defined as the use of two or more drugs, including corticosteroids (more than 1mg\u002Fkg\u002Fd) and any one or more of the following immunomodulatory drugs for over six months: antimalarial drugs, cyclophosphamide, azathioprine, mycophenolate mofetil, methotrexate, leflunomide, tacrolimus, cyclosporine, as well as biologics including rituximab, belimumab, etanercept, etc.\n4. At least one BILAG2004 Class A or two Class B score, or both;\n5. SELENA-SLEDAI score ≥ 8 points;\n6. The positive expression and expression rate of CD19 on peripheral blood B cells determined by flow cytometry;\n7. The functions of important organs meet the following requirements:\n\n   Bone marrow function needs to meet:\n   1. White blood cell count ≥ 3 × 109\u002FL;\n   2. Neutrophil count ≥ 1 × 109\u002FL (no Colony-stimulating factor treatment within 2 weeks before examination);\n   3. Platelets ≥ 50 × 109\u002FL;d. Hemoglobin ≥ 80g\u002FL\n\n   Liver function:\n   1. Alanine Aminotransferase (ALT) ≤ 3 × ULN;\n   2. Asparagus cochinchinensis transase (AST) ≤ 3 × ULN;\n   3. Total Bilirubin (TBIL) in serum ≤ 1.5 × ULN (excluding Gilbert syndrome, total bilirubin ≤ 3.0 × ULN); Renal function: Creatinine Clearance Rate (CrCl) ≥ 60 ml\u002Fminute (Cockcroft\u002FFault formula) ;\n\n   Coagulation function:\n   1. International Normalized Ratio (INR) ≤ 1.5 × ULN,\n   2. Prothrombin time (PT) ≤ 1.5 × ULN.\n\n   Cardiac function: good hemodynamic stability, left ventricular Ejection fraction (LVEF) ≥ 55%;\n8. Female patients of childbearing potential and male patients whose female sexual partners are of childbearing age should adopt medically recognized contraceptive measures or abstain from sex within at least 6 months after infusion of BRL-301; female patients of childbearing age should have a negative serum HCG test result within 7 days before study enrollment and be not breastfeeding;\n9. Willing to participate in this clinical study, sign an ICF, and complete follow-ups, with good compliance.\n\nExclusion Criteria:\n\n1. Have a serious history of Drug allergy or allergic constitution;\n2. Fungi, bacteria, viruses, or other infections that are uncontrollable or require intravenous medication treatment exist or are suspected;\n3. Active central nervous system disease caused by SLE or not (including epilepsy, psychosis, organic encephalopathy syndrome, cerebrovascular accident, encephalitis, central nervous system Vasculitis);\n4. Individuals with relatively serious heart diseases, such as angina pectoris, myocardial infarction, heart failure, and arrhythmia;\n5. Subjects with congenital immunoglobulin deficiency;\n6. Other malignant tumors (excluding non Melanoma skin cancer, cervical cancer in situ, bladder cancer cancer and breast cancer that have survived for more than 5 years without disease);\n7. Subjects with end-stage renal failure;\n8. Have received any of the following SLE treatments:\n\n   1. Corticosteroid (defined as prednisone or equivalent\\>20 mg\u002Fday) of therapeutic dose were used before enrollment or within 72 hours before BRL-301 infusion.\n   2. Use any other clinical study drugs for SLE within 4 weeks prior to enrollment. However, if the research treatment period is ineffective or the disease progresses, and at least 3 half-lives have passed before enrollment, enrollment is allowed.\n   3. Had received anti CD20 monoclonal antibody (such as Rituximab) within 4 weeks before screening, tetaximab within 6 weeks, or belizumab within 12 weeks.\n   4. Previous CAR-T cell or other genetically modified T Cell therapy.\n9. Subjects with positive hepatitis B B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb) and HBV DNA titer in peripheral blood higher than the upper limit of detection; Patients with positive hepatitis C virus (HCV) antibodies and positive peripheral blood HCV RNA; People who are positive for human immunodeficiency virus (HIV) antibodies; Those who have tested positive for syphilis;\n10. Having mental illness and severe cognitive impairment;\n11. Those who have participated in other clinical trials within the first 3 months of enrollment;\n12. Pregnant or intending to conceive women;\n13. Patients who are unsuitable for being included into this study as deemed by the investigator due to other reasons.",{"count":137,"type":20},12,[23],"This is an investigator initiated trial to assess the efficacy and safety of BRL-301 in the refractory systemic lupus erythematosus.",[141],"Systemic Lupus Erythematosus (SLE)","2024-09-25",{"date":144,"type":33},"2024-09-27",{"date":146,"type":33},"2023-08-14",{"date":148,"type":20},"2025-09",{"name":39,"class":40},{"id":151,"slug":152,"hasResults":11,"nctId":153,"briefTitle":154,"officialTitle":155,"acronym":4,"eligibilityCriteria":156,"healthyVolunteers":11,"sex":16,"minAge":157,"maxAge":158,"enrollmentInfo":159,"targetDuration":4,"studyType":21,"phases":160,"briefSummary":161,"conditions":162,"keywords":4,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":164,"lastUpdatePostDateStruct":165,"startDateStruct":167,"completionDateStruct":169,"leadSponsor":171,"locationsCount":4},"100538150","clinical-study-on-the-safety-and-efficacy-of-brl-101-in-the-treatment-of-sickle-cell-disease-100538150","NCT06287086","Clinical Study on the Safety and Efficacy of BRL-101 in the Treatment of Sickle Cell Disease","Clinical Study on the Safety and Efficacy of a Single Intravenous Dose of CRISPR\u002FCas9-Edited Autologous CD34+ Hematopoietic Stem\u002FProgenitor Cells (BRL-101) in the Treatment of Sickle Cell Disease","Inclusion Criteria:\n\nSubjects must meet all the following inclusion criteria to be eligible for enrolment into the study:\n\n1. Subject (or their legally authorized representative or guardian) will sign and date an informed consent form (ICF) and, where applicable, an assent form.\n2. Subjects 3 to 35 years of age, inclusive, on the date of informed consent.\n3. Clinically confirmed severe SCD, genotypes include: βS\u002FβS, βS\u002Fβ + or βS\u002Fβ0. Severe SCD is defined as having at least 2 VOC events per year during the 2 years prior to screening and requiring appropriate supportive care, including a pain management program, HU therapy (if indicated).\n4. Karnofsky performance status of ≥80% for subjects ≥16 years of age. Lansky performance status of ≥80% for subjects \\\u003C16 years of age (see Appendix 1 and 2).\n5. Eligible for autologous stem cell transplant as per investigator's judgment.\n6. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.\n7. Willing to participate in an additional long-term follow-up study after completion of this study (Study 2022-LTF-BRL-101).\n8. Subjects of childbearing potential must use effective contraception for at least 6 months after BRL-101 infusion during the study.\n\nExclusion Criteria\n\nSubjects meeting any of the following criteria are not eligible for enrolment in the study:\n\n1. Known contraindications, intolerance, or hypersensitivity to hematopoietic stem cell mobilizers, busulfan injection, or dimethyl sulfoxide (DMSO) or study drug-related components.\n2. Eligible for allogeneic hematopoietic stem cell transplantation and have found HLA-identical donors.\n3. Prior allo-HSCT, gene therapy or gene editing therapy.\n4. Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator.\n5. HbF level \\>15.0%, irrespective of concomitant treatment with HbF inducing treatments such as HU.\n6. Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment.\n7. More than 10 unplanned hospitalizations or emergency department visits related to SCD in the 1 year before screening and the investigator considered this to be a significant chronic pain rather than an acute pain crisis.\n8. A history of clinically significant transcranial Doppler (TCD) test abnormalities or test abnormalities in the opinion of the investigator.\n9. History of untreated Moyamoya disease or presence of Moyamoya disease at Screening that in the opinion of the investigator puts the subjects at the risk of bleeding.\n10. the subject has participated in other clinical studies and used drugs within 3 months before screening.\n11. White blood cell count \\\u003C 3 × 109\u002FL and\u002For platelet count \\\u003C 100 × 109\u002FL not due to hypersplenism as judged by the investigator.\n12. INR \\> 1.5×ULN, APTT \\> 1.5×ULN.\n13. Creatinine \\> 1.5 × ULN or endogenous creatinine clearance \\\u003C 60 ml\u002Fmin (calculated according to the Cockcroft-Gault formula, see Appendix 3).\n14. ALT or AST\\> 3×ULN, or direct bilirubin value \\> 2.5×ULN.\n15. Severe iron overload with serum ferritin ≥ 5000 ng\u002Fml, liver iron \\> 15 mg Fe\u002Fg dry weight (or liver MRIT2\\* \\\u003C 1.4 ms or \\> 588 Hz), or heart MRI-T2\\* \\\u003C 10 ms.\n16. LVEF \\\u003C 50%.\n17. DLco \\\u003C 50% predicted (corrected haemoglobin or\u002Fand alveolar volume) or forced vital capacity (FVC) (measured\u002Fpredicted) \\\u003C 60% (For children for whom DLco could not be determined), or abnormal blood gas analysis (for younger children with undetectable ventilatory function only).\n18. Hepatitis B virus surface antigen (HBsAg) positive or HBV-DNA positive; hepatitis C virus (HCV) antibody positive; human immunodeficiency virus (HIV) antibody positive; syphilis (TP) -specific antibody positive; Epstein-Barr virus EBV-DNA positive; cytomegalovirus CMV-DNA positive.\n19. History of a significant bleeding disorder.\n20. History or family history of malignancy or myeloproliferative disorder.\n21. Any prior or current cardiovascular system diseases, such as congestive heart failure, arrhythmia, myocardial disease, valvular heart disease or pulmonary hypertension; cirrhosis, liver fibrosis or active hepatitis; central nervous system diseases or mental illness.\n22. Presence of immune dysfunction or endocrine disorders, such as insulin-dependent diabetes mellitus, hyperthyroidism, or insufficiency.\n23. Pregnant or breastfeeding females.\n24. Any condition that, in the opinion of the investigator, would make participation in this clinical study inappropriate.","3 Years","35 Years",{"count":41,"type":20},[23],"This is a single center, non-randomized, open label, single-dose study in subjects with Sickle Cell Disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (BRL-101)",[163],"Sickle Cell Disease","2024-05-30",{"date":166,"type":33},"2024-06-03",{"date":168,"type":20},"2024-06-14",{"date":170,"type":20},"2026-06-10",{"name":39,"class":40},{"id":173,"slug":174,"hasResults":11,"nctId":175,"briefTitle":176,"officialTitle":177,"acronym":4,"eligibilityCriteria":178,"healthyVolunteers":11,"sex":16,"minAge":157,"maxAge":158,"enrollmentInfo":179,"targetDuration":4,"studyType":181,"phases":4,"briefSummary":182,"conditions":183,"keywords":4,"overallStatus":98,"whyStopped":4,"lastUpdateSubmitDate":185,"lastUpdatePostDateStruct":186,"startDateStruct":188,"completionDateStruct":190,"leadSponsor":192,"locationsCount":193},"100539037","long-term-follow-up-study-of-brl-101-for-tdt-100539037","NCT06298630","Long-term Follow-up Study of BRL-101 for TDT","Long-term Follow-up Study of BRL-101 in the Treatment of Transfusion-dependent β-thalassemia","Inclusion Criteria:\n\n1. Provision of written informed consent for this study by subjects, or as applicable, subject's parent(s)\u002Flegal guardian(s)\n2. Treated with BRL-101 for therapy of transfusion-dependent β-thalassemia.\n\nExclusion Criteria:\n\n\\- There are no exclusion criteria for this study",{"count":180,"type":20},45,"OBSERVATIONAL","Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.",[184],"Thalassemia, Beta","2024-03-21",{"date":187,"type":33},"2024-03-22",{"date":189,"type":20},"2024-12-08",{"date":191,"type":20},"2038-10-15",{"name":39,"class":40},7,{"id":195,"slug":196,"hasResults":11,"nctId":197,"briefTitle":198,"officialTitle":199,"acronym":4,"eligibilityCriteria":178,"healthyVolunteers":11,"sex":16,"minAge":200,"maxAge":158,"enrollmentInfo":201,"targetDuration":4,"studyType":181,"phases":4,"briefSummary":202,"conditions":203,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":204,"lastUpdatePostDateStruct":205,"startDateStruct":207,"completionDateStruct":209,"leadSponsor":211,"locationsCount":212},"100540260","long-term-follow-up-study-of-bhc001-for-tdt-100540260","NCT06314529","Long-term Follow-up Study of BHC001 for TDT","Long-term Follow-up Study of BHC001 in the Treatment of Transfusion-dependent β-thalassemia","5 Years",{"count":19,"type":20},"Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.",[184],"2024-03-11",{"date":206,"type":33},"2024-03-18",{"date":208,"type":33},"2022-12-14",{"date":210,"type":20},"2037-05-10",{"name":39,"class":40},2,""]