[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"aplastic-anemias\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:aplastic-anemias":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,47,76],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":17,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":19,"targetDuration":22,"studyType":23,"phases":4,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100610405","the-hope-biobank-resource-bmt-ctn-2402-hope-100610405",false,"NCT07227155","The HOPE Biobank Resource (BMT CTN 2402 HOPE)","Hematopoietic Cell Transplant and Gene Therapy for Non-Malignant Blood Disorders Biobank Resource (BMT CTN 2402)","HOPE Resource","HCT\u002FGT Inclusion:\n\n1. Patients with a diagnosis of Aplastic Anemia (AA), hemoglobinopathies or bone marrow failure from other causes except for malignant diseases will be eligible for enrollment on this protocol:\n\n   1. AA will be defined as having peripheral blood cytopenias with a hypocellular bone marrow for age and a clinical diagnosis of aplastic anemia as determined by their treating physicians.\n   2. Hemoglobinopathies include sickle cell disease or thalassemia. Patients receiving potentially curative therapy with HCT or GT for hemoglobinopathies will be eligible for this study.\n   3. Individuals with bone marrow failure due to clinical or molecularly diagnosed inherited bone marrow failure, inborn errors of immunity or other cause will be included.\n2. Patients must receive an HCT or GT for management of their underlying disease. Allogeneic transplants including all conditioning regimens, donors, and GVHD prophylaxis regimens are eligible. This study does not define how the transplant or transplant-supportive care will be performed.\n3. Patients or their legal guardian must consent to participate in the CIBMTR \"Protocol for a Research Database for Hematopoietic Cell Transplantation and Marrow Toxic Injuries\" (NCT 1166009) to allow linkage with the longitudinal clinical data collected by CIBMTR.\n4. All ages minorities, sexes and genders are eligible for the study, but participants must weigh at least 10 kilograms (kg) at the time of study enrollment given the volume and number of blood draws required.\n5. All participants or parent\u002Flegal guardian must sign an informed consent for this study. If there are questions regarding a patient's eligibility for the study, contact the Protocol Team for review and discussion by emailing bmtctn2402@emmes.com.\n\nHCT\u002FGT Exclusion\n\n1. Patients with aplastic anemia or hemoglobinopathies who are not pursuing allogeneic HCT or GT.\n2. Active malignancy.\n3. Hematologic malignancy or therapy for a prior hematologic malignancy in the previous five 5 years.\n4. Weight ≤ 10.0 kg at time of study enrollment.\n5. Prior autologous or allogeneic transplant.\n\nRelated Donor Inclusion:\n\n1\\. All related donors for eligible recipients undergoing allogeneic HCT for AA, hemoglobinopathies, or bone marrow failure as defined in the recipient eligibility criteria above are eligible. Note: HCT recipient participants will remain eligible if the related donor declines to participate in the study.\n\nRelated Donor Exclusion:\n\n1\\. Donor weight ≤ 10.0 kg at time of study enrollment",true,"ALL",{"count":20,"type":21},375,"ESTIMATED","5 Years","OBSERVATIONAL","A prospective, multicenter study that will establish a repository of biospecimens and clinical data from patients undergoing hematopoietic stem cell transplant (HCT) or gene therapy (GT) for treatment of non-malignant blood diseases.",[26,27,28],"Aplastic Anemias","Hemoglobinopathies","Bone Marrow Failure",[30,31,32,33],"Biorepository","Biospecimens","Allogenic Transplant","Gene Therapy","RECRUITING","2026-06-12",{"date":37,"type":38},"2026-06-16","ACTUAL",{"date":40,"type":38},"2026-05-15",{"date":42,"type":21},"2031-03-30",{"name":44,"class":45},"Medical College of Wisconsin","OTHER",6,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":18,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":59,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":75},"100566082","umbilical-cord-blood-supported-haplo-hsct-for-aplastic-anemia-treatment-study-100566082","NCT06650553","Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study","Clinical Study on Umbilical Cord Blood-Supported Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Aplastic Anemia","Inclusion Criteria:\n\n* A patient age of 6-75 years\n* Patients diagnosed with Severe Aplastic Anemia (SAA) , Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) or Paroxysmal nocturnal hemoglobinuria (PNH) acquired clonal-aplastic anemia(AA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT\n* Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study\n\nExclusion Criteria:\n\n* Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)\n* Patients with any conditions not suitable for the trial (investigators' decision)","6 Years","75 Years",{"count":57,"type":21},110,"INTERVENTIONAL",[60],"NA","Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74\u002F100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.",[26],[26,64,65],"Stem Cell Transplantation","Umbilical Cord Blood","2025-08-10",{"date":68,"type":38},"2025-08-14",{"date":70,"type":38},"2024-06-01",{"date":72,"type":21},"2027-07",{"name":74,"class":45},"Shanxi Bethune Hospital",1,{"id":77,"slug":78,"hasResults":11,"nctId":79,"briefTitle":80,"officialTitle":80,"acronym":4,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":18,"minAge":4,"maxAge":4,"enrollmentInfo":82,"targetDuration":4,"studyType":58,"phases":84,"briefSummary":86,"conditions":87,"keywords":88,"overallStatus":92,"whyStopped":4,"lastUpdateSubmitDate":93,"lastUpdatePostDateStruct":94,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":4},"100593050","phase-3-study-on-the-short-term-efficacy-and-safety-of-recombinant-human-thrombopoietin-combined-with-immunosuppressant-sequential-eltrombopag-ethanolamine-dry-suspension-in-the-treatment-of-saatd-nsaa-100593050","NCT07001397","Study on the Short-term Efficacy and Safety of Recombinant Human Thrombopoietin Combined With Immunosuppressant Sequential Eltrombopag Ethanolamine Dry Suspension in the Treatment of SAA\u002FTD-NSAA","Inclusion Criteria:\n\n1. Age ≥18 years old, gender not limited;\n2. Patients diagnosed with aplastic anemia (AA) through blood routine, bone marrow puncture, bone marrow biopsy and excluded examinations, and determined as SAA or TD-NSAA according to the Camitta criteria;\n3. Those who have not received treatment with immunosuppressants (such as cyclosporine, tacrolimus, ATG, etc.);\n4. The patient has no HLA-matched sibling donor or is not suitable for first-line allogeneic hematopoietic stem cell transplantation due to reasons such as age, complications, or the patient's wishes.\n5. Before treatment, the total bilirubin (TBIL) of the patient was less than 1.5 times the upper limit of normal value (ULN), alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were less than 3 times the ULN, and serum creatinine was less than 1.5 times the ULN.\n6. ECOG score ≤2 points;\n7. Voluntarily join this study, sign the informed consent form, have good compliance and be willing to cooperate with regular follow-up for therapeutic effect evaluation and side effect monitoring;\n\nExclusion Criteria:\n\n1. Pregnant or lactating patients;\n2. Have a history of any arterial or venous thrombosis (including stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within half a year before enrollment and have at least two of the following risk factors: Hormone replacement therapy, oral contraceptives (including estrogen), smoking, diabetes, hypercholesterolemia, drug-controlled hypertension, hereditary coagulation disorders;\n3. Patients with severe cardiovascular diseases (such as NYHA Cardiac Function Score grade III-IV) within half a year before enrollment, known arrhythmias that increase the risk of thromboembolism such as atrial fibrillation, after coronary stent implantation, angioplasty, and after coronary artery bypass grafting;\n4. Combined with malignant tumors that seriously affect survival;\n5. Within 2 weeks after the start of the study until the end of the study, continuous drug treatment (including but not limited to aspirin, clopidogrel and\u002For non-steroidal anti-inflammatory drugs NSAIDs) or anticoagulant treatment that affects platelet function for more than 3 days;\n6. Currently, there is a severe or difficult-to-control infection (CTC AE level 2 infection);\n7. Bone marrow biopsy within 4 weeks before the administration of the first dose of the study indicates the presence of primary diseases other than SAA that can cause thrombocytopenia (such as primary myelodysplastic syndrome (MDS), congenital bone marrow failure diseases (such as Fanconi anemia, congenital keratosis, etc.) Or accompanied by obvious paroxysmal nocturnal hemoglobinuria (PNH) clones (\\> 50%) and AA with clinical and biochemical indicators of hemolysis; And the researcher judged that the subject was not suitable to participate in this study.\n8. All laboratory or clinical evidence of HIV infection, previous clinical history of hepatitis C, previous hepatitis B infection, or active hepatitis at the time of screening. Laboratory tests during the screening period indicated hepatitis C infection or hepatitis B infection. (Defined as a positive HBsAg test. Additionally, if the HBsAg test is negative but HBcAb is positive, regardless of the status of HBsAb, HBV DNA testing is required. If it is positive, the subject should be excluded.)\n9. Chronic liver disease-related AA (hepatitis disease-related AA, liver cirrhosis, etc.);\n10. Had received treatment with TPO or TPO receptor agonists (TPO-RAS, including but not limited to eltrombopag, romisetine, avatrombopag, heltrombopag, rutrombopag, etc.) before screening;\n11. Suffer from severe autoimmune diseases or immunodeficiency diseases;\n12. Suffer from serious mental disorders;\n13. Patients who the researchers consider unsuitable to participate in this trial, such as those with any other medical, social or psychological factors that may affect safety or compliance with the research procedures.",{"count":83,"type":21},70,[85],"PHASE3","The study included adult patients ≥18 years old with SAA and TD-NSAA who had no HLA-matched sibling donors or were not suitable for first-line allogeneic hematopoietic stem cell transplantation due to age, complications, patient wishes, etc. After admission, they were treated with standard IST combined with rhTPO for at least 28 days. After discharge, the sequential treatment of EPAG-PFOS (with an initial dose of 150mg\u002Fd) was continued for 2 months (8 weeks), and the follow-up was conducted until the 6th month (24 weeks). It was compared with the historical cohort previously treated with IST combined with EPAG to explore the short-term efficacy and safety of this treatment regimen.",[26],[89,90,91],"Aplastic anemia","rhTPO","EPAG-pfos","NOT_YET_RECRUITING","2025-05-23",{"date":95,"type":38},"2025-06-03",{"date":97,"type":21},"2025-06-23",{"date":99,"type":21},"2027-03-23",{"name":101,"class":45},"Peking Union Medical College Hospital"]