[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"aplastic-anaemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:aplastic-anaemia":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,44,68,90,115],{"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":27,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":32,"lastUpdatePostDateStruct":33,"startDateStruct":36,"completionDateStruct":38,"leadSponsor":40,"locationsCount":43},"100631331","a-multicenter-randomized-open-label-study-of-haplo-cord-hsct-for-the-treatment-of-aplastic-anemia-100631331",false,"NCT07499284","A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia","A Multicenter, Randomized, Open-Label Study to Analyze the Efficacy of Unrelated Cord Blood Combined With Haploidentical Stem Cell Transplantation in Patients With Aplastic Anemia","Inclusion Criteria: (1)Aged ≥14 years. (2) Patients diagnosed with aplastic anemia meeting the diagnostic criteria of the Chinese Guidelines for the Diagnosis and Management of Aplastic Anemia (2024 Edition). (3) Patients who were eligible for allogeneic bone marrow or peripheral blood hematopoietic stem cell transplantation in accordance with the Clinical Application and Management Specifications for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition). (4) The patient understands the study protocol and voluntarily signs the informed consent form. (5) Life expectancy of ≥3 months; (6) Pre-transplant assessment meets the following criteria: Karnofsky Performance Status (KPS) score≥70, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score≤2, and Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score≤2.\n\nExclusion Criteria: (1)Patients with aplastic anemia secondary to malignancy treatment or those with concurrent active malignancy. (2) Women who are pregnant or lactating. (3) Patients with psychiatric or psychological disorders that preclude adequate compliance with the treatment protocol. (4) Patients with positive serologic testing for any of the four major infectious diseases (hepatitis B, hepatitis C, syphilis, HIV). (5) Patients with systemic infection or localized severe infection requiring active antimicrobial therapy. (6) Patients with significant dysfunction of major organs (e.g., heart, lung, liver, kidney). (7) Patients with a known allergy or hypersensitivity to any drug or component used in this study. (8) Patients who are currently participating in or plan to participate in any other clinical trial. (9) Any other condition deemed by the investigator to render the patient unsuitable for study participation.","ALL","14 Years",{"count":19,"type":20},224,"ESTIMATED","INTERVENTIONAL",[23],"NA","Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell\u002Fprogenitor cell deficiencies and immunity disorders.\n\nCurrently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte\u002Flymphocyte globulin (ATG\u002FALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy.\n\nUmbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation.\n\nCurrently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P \\\u003C 0.0001).\n\nBased on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV\u002FEBV reactivation rate, donor chimerism dynamics, and immune reconstitution.",[26],"Aplastic Anaemia",[28,29,30],"Haplo-Cord HCT","Haplo HCT","Aplastic Anemia","RECRUITING","2026-03-24",{"date":34,"type":35},"2026-03-30","ACTUAL",{"date":37,"type":35},"2025-10-01",{"date":39,"type":20},"2028-09-30",{"name":41,"class":42},"The First Affiliated Hospital of Soochow University","OTHER",4,{"id":45,"slug":46,"hasResults":11,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":53,"phases":4,"briefSummary":54,"conditions":55,"keywords":56,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":58,"lastUpdatePostDateStruct":59,"startDateStruct":61,"completionDateStruct":63,"leadSponsor":65,"locationsCount":67},"100628398","adverse-effects-of-atgalg-therapy-in-aplastic-anemia-100628398","NCT07461116","Adverse Effects of ATG\u002FALG Therapy in Aplastic Anemia","A Study on the Dynamics and Grading Criteria Construction of Adverse Reactions to ATG\u002FALG Therapy in Aplastic Anemia","Inclusion Criteria:\n\n* Meet the diagnostic criteria for aplastic anemia (Camitta criteria);\n* Receiving ATG\u002FALG immunosuppressive therapy for the first time;\n\nExclusion Criteria:\n\n* Previously received hematopoietic stem cell transplantation;\n* Aplastic anemia patients receiving ATG\u002FALG as part of transplant conditioning regimen;",{"count":52,"type":20},200,"OBSERVATIONAL","This prospective, single-center, observational cohort study aims to systematically observe and describe the clinical characteristics of adverse reactions in patients with aplastic anemia undergoing ATG (Anti-Thymocyte Globulin) \u002FALG (Anti-Lymphocyte Globulin) treatment, providing a data foundation for the development of relevant management strategies. This study plans to enroll 200 aplastic anemia patients undergoing ATG\u002FALG treatment",[26],[26,57],"ATG\u002FALG","2026-03-04",{"date":60,"type":35},"2026-03-10",{"date":62,"type":35},"2026-02-23",{"date":64,"type":20},"2027-12-31",{"name":66,"class":42},"Institute of Hematology & Blood Diseases Hospital, China",1,{"id":69,"slug":70,"hasResults":11,"nctId":71,"briefTitle":72,"officialTitle":73,"acronym":4,"eligibilityCriteria":74,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":75,"enrollmentInfo":76,"targetDuration":4,"studyType":21,"phases":78,"briefSummary":79,"conditions":80,"keywords":4,"overallStatus":81,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":85,"completionDateStruct":87,"leadSponsor":89,"locationsCount":4},"100618961","hid-hsct-versus-ist-as-first-line-treatment-for-saa-100618961","NCT07338422","HID-HSCT Versus IST as First-line Treatment for SAA","Nonrandomized Controlled Study of HLA-Haploidentical Hematopoietic Stem Cell Transplantation Versus Immunosuppressive Therapy as First-Line Treatment for Severe Aplastic Anemia","Inclusion Criteria:\n\n* (1) Confirmed diagnosis of severe aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, and hepatitis-associated severe aplastic anemia.\n\n  (2) Age 14-70 years. (3) No HLA-matched related donor available. (4) HIV negative, HBV negative, HCV negative. (5) No absolute contraindications to transplantation or immunosuppressive therapy.\n\n  (6) Signed informed consent form must be obtained before study procedures begin; for subjects aged 18 years or older, informed consent shall be signed by the patient themselves or direct family members. Considering the patient's medical condition, if the patient's own signature is medically inadvisable, the informed consent shall be signed by a legal guardian or the patient's direct family member.\n\nExclusion Criteria:\n\n1. Inherited bone marrow failure syndromes (IBMFS), including Fanconi anemia, dyskeratosis congenita, Shwachman-Diamond syndrome (SDS), etc.;\n2. Clonal cytogenetic abnormalities or bone marrow examination suggesting pre-MDS or MDS;\n3. Known severe allergy to ATG;\n4. Previous allogeneic or autologous hematopoietic stem cell transplantation;\n5. Previous solid organ transplantation;\n6. Uncontrolled infection at enrollment, or requiring mechanical ventilation or hemodynamic instability;\n7. Active HIV replication at enrollment, detectable HCV antibody positivity and HCV-RNA positivity within 90 days prior to enrollment, or HBsAg positivity; known seropositivity for HIV or active hepatitis C virus;\n8. History of malignant tumors (except resected basal cell carcinoma or treated cervical carcinoma in situ);\n9. Psychiatric disorders or other conditions that prevent compliance with study treatment and monitoring requirements;\n10. Inability or unwillingness to sign the consent form;\n11. Other special circumstances deemed ineligible by the investigator.","70 Years",{"count":77,"type":20},116,[23],"This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.",[26],"NOT_YET_RECRUITING","2026-01-05",{"date":84,"type":35},"2026-01-13",{"date":86,"type":20},"2026-01-14",{"date":88,"type":20},"2030-09-14",{"name":66,"class":42},{"id":91,"slug":92,"hasResults":11,"nctId":93,"briefTitle":94,"officialTitle":94,"acronym":4,"eligibilityCriteria":95,"healthyVolunteers":11,"sex":16,"minAge":96,"maxAge":4,"enrollmentInfo":97,"targetDuration":4,"studyType":21,"phases":99,"briefSummary":101,"conditions":102,"keywords":103,"overallStatus":31,"whyStopped":4,"lastUpdateSubmitDate":106,"lastUpdatePostDateStruct":107,"startDateStruct":109,"completionDateStruct":111,"leadSponsor":113,"locationsCount":67},"100577727","phase-2-efficacy-and-safety-of-sirolimus-with-or-without-cyclosporin-a-in-chinese-patients-with-aplastic-anemia-refractoryintolerant-to-cyclosporin-a-100577727","NCT06802055","Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory\u002FIntolerant to Cyclosporin A","Inclusion Criteria:\n\n1. Age ≥ 18 years;\n2. Diagnosed with acquired aplastic anemia (AA), excluding congenital AA;\n3. At least one of the following criteria met at enrollment: hemoglobin \\\u003C 100 g\u002FL, platelets \\\u003C 50 × 10⁹\u002FL, or neutrophils \\\u003C 1.0 × 10⁹\u002FL;\n4. At enrollment, meeting at least one of the following conditions:\n\n   ① Cyclosporine A (CsA) ineffective: (CsA) used for at least 3 months without achieving partial response (PR), or disease relapse occurred;\n\n   ② CsA intolerant: Unsuitable for standard dose CsA treatment due to adverse events or underlying conditions.\n5. No active infections;\n6. Not pregnant or breastfeeding;\n7. Willing to sign the consent form;\n8. Eastern Cooperative Oncology Group (ECOG) performance status score of 0-2.\n\nExclusion Criteria:\n\n1. Pancytopenia caused by other reasons, such as myelodysplastic syndrome (MDS);\n2. Evidence of clonal hematopoietic system bone marrow diseases (e.g., MDS or acute myeloid leukemia, AML);\n3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;\n4. History of hematopoietic stem cell transplantation (HSCT) before enrollment;\n5. Previous use of sirolimus or allergy to sirolimus;\n6. Severe adverse events to CsA in the past, making it unsuitable for reuse;\n7. Uncontrolled infection or bleeding with standard treatment;\n8. Active infections with HIV, HCV, or HBV, liver cirrhosis, portal hypertension;\n9. Any concurrent malignancy within the past 5 years, except for localized basal cell carcinoma of the skin;\n10. History of thromboembolic events, myocardial infarction, or stroke (including antiphospholipid antibody syndrome), or current use of anticoagulants;\n11. Pregnant or breastfeeding women;\n12. Participation in other clinical trials within the past 3 months.","18 Years",{"count":98,"type":20},40,[100],"PHASE2","This is a single center, randomized, open-label, phase II study to compare the efficacy of sirolimus combined with cyclosporin A (CsA) to sirolimus alone in Chinese subjects with aplastic anemia refractory\u002Fintolerant to CsA. The safety would also be evaluated. Patients would be randomized to receive sirolimus alone or sirolimus combined with CsA at a 1:3 ratio. Treatment with sirolimus will be started at 1-3 mg once daily orally, with a target trough blood concentration of 4-12 ng\u002Fml. CsA will be given at 25-150 mg orally every 12 hours, with the dose adjusted based on renal function and trough concentration. For patients with normal renal function, the target trough concentration is approximately 150 ng\u002Fml. For patients with impaired renal function, the cyclosporine A dose is reduced to 25-50 mg every 12 hours, aiming for recovery or stabilization of renal function. The hematological response rate and safety will be recorded and compared at 3 and 6 months after starting the study treatment (Week 13 and 25).",[26],[104,105],"Sirolimus","Relapsed\u002Frefractory aplastic anemia","2025-05-29",{"date":108,"type":35},"2025-06-04",{"date":110,"type":35},"2025-01-25",{"date":112,"type":20},"2027-01",{"name":114,"class":42},"Peking Union Medical College Hospital",{"id":116,"slug":117,"hasResults":11,"nctId":118,"briefTitle":119,"officialTitle":120,"acronym":4,"eligibilityCriteria":121,"healthyVolunteers":11,"sex":16,"minAge":122,"maxAge":4,"enrollmentInfo":123,"targetDuration":4,"studyType":21,"phases":125,"briefSummary":126,"conditions":127,"keywords":131,"overallStatus":81,"whyStopped":4,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":136,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":141,"locationsCount":4},"100575229","phase-2-reduced-dose-conditioning-regimen-containing-tbi-in-hsct-treating-elderly-patients-with-aplastic-anemia-100575229","NCT06769568","Reduced-dose Conditioning Regimen Containing TBI in HSCT Treating Elderly Patients With Aplastic Anemia","The Safety and Efficacy of Reduced-dose Conditioning Regimen Containing TBI in Hematopoietic Stem Cell Transplantation Treating Elderly Patients With Aplastic Anemia","Inclusion Criteria:\n\n* ( 1 ) Diagnosis of aplastic anemia, including very severe aplastic anemia, severe aplastic anemia, chronic aplastic anemia and hepatitis-associated aplastic anemia.\n\n( 2 ) Age older than or equal to 50 years old. ( 3 ) Informed consent can be signed by themselves. ( 4 ) HIV negative, HBV, HCV negative. ( 5 ) Informed consent must be signed before the start of the study procedure. Informed consent must be signed by the patient or immediate family members who are 18 years old or older. Considering the patient 's condition, if the patient 's signature is not conducive to the treatment of the disease, the informed consent is signed by the legal guardian or the patient 's immediate family.\n\nExclusion Criteria:\n\n* ( 1 ) Patients who underwent more than one time of transplantation before enrollment ; ( 2 ) Uncontrolled infection, mechanical ventilation or hemodynamic instability at the time of enrollment ; ( 3 ) Diagnosis of clinically significant severe liver dysfunction ( defined as Child-Pugh C grade ) within 5 days before enrollment ; or within 5 days before enrollment, AST or ALT was 5 times higher than the upper limit of normal, or serum total bilirubin was 2 times higher than the upper limit of normal ; ( 4 ) End-stage renal insufficiency was diagnosed within 5 days before enrollment, and creatinine clearance rate was less than 10ML \u002F min.\n\n( 5 ) Simultaneous diagnosis of moderate hepatic insufficiency and moderate renal insufficiency ( moderate hepatic insufficiency was defined as Child-Pugh B grade ; moderate renal insufficiency was defined as creatinine clearance rate less than 50ML \u002F min ).\n\n( 6 ) In addition to localized basal cell or squamous cell skin cancer or treatment conditions ( such as lymphoma ), there are active solid tumor malignant tumors.\n\n( 7 ) There was active HIV replication before enrollment. HCV antibody positive, HCV-RNA positive, or HBsAg positive could be detected within 90 days before enrollment. The serological response to HIV or active hepatitis C virus is known to be positive.\n\n( 8 ) Having a mental illness or other condition that does not cooperate with the requirements of research, treatment and monitoring.\n\n( 9 ) unable or unwilling to sign the consent form. ( 10 ) Patients with other special conditions who were assessed as unqualified by the researchers.","50 Years",{"count":124,"type":20},20,[100],"The TBI-containing reduced-dose conditioning regimen was used to treat elderly patients with aplastic anemia who received hematopoietic stem cell transplantation. The overall survival rate, GVHD-free survival rate, all-cause mortality,et al were studied. The modified conditioning regimen included TBI 2Gy, -7d, busulfan 3.2mg \u002F kg-6d ; fludarabine 30mg \u002F m2 \u002F d-5 \\~ -1d ; cyclophosphamide 25-30mg \u002F kg \u002F d-5 \\~ -2d ; ATG ( rabbit ) 2 mg \u002F kg \u002F d-5 \\~ -1d.",[26,128,129,130],"HSCT","TBI","Old Age",[132,128,133,134],"aplastic anemia","old age","conditioning regimen","2025-01-07",{"date":137,"type":35},"2025-01-10",{"date":139,"type":20},"2025-01-31",{"date":39,"type":20},{"name":66,"class":42}]