[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"aplastic-anemia-idiopathic\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:aplastic-anemia-idiopathic":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,47,70],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100598994","phase-2-efficacy-and-safety-of-ucbt-with-tmi--based-conditioning-regimen-for-adults-with-refractoryrelapsed-aplastic-anemia-100598994",false,"NCT07078721","Efficacy and Safety of UCBT With TMI -Based Conditioning Regimen for Adults With Refractory\u002FRelapsed Aplastic Anemia","Efficacy and Safety Study of Umbilical Cord Blood Transplantation (UCBT) With Total Marrow Irradiation (TMI)-Based Conditioning Regimen for Adults With Refractory\u002FRelapsed Aplastic Anemia (AA).","TMI","Inclusion Criteria:\n\n* Subjects suitable for enrollment in this study must meet all of the following criteria:\n\n  1. Age ≥18 years and \\\u003C50 years, regardless of gender.\n  2. Diagnosis of AA according to the guidelines of the British Society for Haematology. The bone marrow shows hypocellularity, without infiltration of abnormal cells or marrow fibrosis. The peripheral blood must meet at least two of the following three criteria: absolute neutrophil count (ANC) \\\u003C1.5×10⁹\u002FL; platelet count \\\u003C50×10⁹\u002FL; hemoglobin \\\u003C100 g\u002FL. Based on the severity of the disease, AA is classified into severe aplastic anemia (SAA) and non-severe aplastic anemia (NSAA).\n\n     The diagnostic criteria for severe aplastic anemia (SAA) are as follows:\n\n     Peripheral blood criteria\n\n     At least two of the following three criteria must be met:\n\n     Absolute neutrophil count (ANC) \\\u003C0.5×10⁹\u002FL; Reticulocyte count \\\u003C20×10⁹\u002FL; Platelet count \\\u003C20×10⁹\u002FL; Bone marrow criteria The degree of bone marrow cellularity is \\\u003C25% of normal; if ≥25% but \\\u003C50% of normal, then the proportion of residual hematopoietic cells should be \\\u003C30%.\n\n     Bone marrow aspiration results show hypocellular or severely hypocellular marrow, with a marked decrease in hematopoietic cells and an increase in non-hematopoietic cells such as lymphocytes and reticular cells.\n\n     Other：Other diseases causing pancytopenia, such as myelodysplastic syndrome (MDS), paroxysmal nocturnal hemoglobinuria (PNH), and congenital marrow failure syndromes, should be excluded.\n\n     If ANC \\\u003C0.2×10⁹\u002FL, the diagnosis is very severe aplastic anemia (VSAA); Those who do not meet the criteria for SAA are classified as non-severe aplastic anemia (NSAA);\n  3. Meet the criteria for refractory AA: ①SAA: no response to first-line ATG + CSA treatment for more than 6 months; ②NSAA: ①persistent transfusion dependence of two or more blood cell lines, ②meet any of the following criteria: no response to immunosuppressive therapy with cyclosporine for more than 12 months, or no response to androgen and\u002For TPO receptor agonist therapy for more than 6 months, ④or progression to SAA; Meet the criteria for relapsed AA: initially effective to first-line immunosuppressive therapy, but pancytopenia recurs after reduction or discontinuation of immunosuppressive therapy, meeting the criteria for SAA.\n  4. Karnofsky score ≥60, ECOG score ≤2, HCT-CI index ≤2.\n  5. No HLA-identical sibling donor or no HLA-identical unrelated donor available.\n  6. Understanding of the study procedures and voluntary written informed consent.\n\nExclusion Criteria:\n\n* Subjects with any of the following conditions are not eligible for enrollment in this study:\n\n  1. Pancytopenia and hypoplastic marrow diseases caused by other reasons (including PNH, etc.).\n  2. Use of intermediate or high doses of cyclophosphamide (≥20mg\u002Fkg\u002Fd) for immunosuppressive therapy within 3 months before enrollment.\n  3. History of hematopoietic stem cell transplantation.\n  4. Known or suspected contraindications or allergies to fludarabine, melphalan, or other drugs.\n  5. Patients with uncontrolled bleeding and\u002For infection after standard treatment before screening.\n  6. Active viral hepatitis (hepatitis B, hepatitis C, etc.), HIV infection, or syphilis at baseline or screening.\n  7. Creatinine clearance rate \\\u003C60 ml\u002Fmin before treatment, or serum creatinine \\>140 μmol\u002FL.\n  8. Pregnant or breastfeeding women.\n  9. Participation in another clinical trial within 3 months before enrollment.\n  10. Any other condition that the investigator deems may prevent the subject from completing the study or pose significant risks to the subject.","ALL","18 Years","50 Years",{"count":21,"type":22},11,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","Refractory\u002Frelapsed aplastic anemia (AA) in adults remains a clinical challenge that is frequently encountered and urgently needs to be resolved. Salvage treatment should prioritize hematopoietic stem cell transplantation (HSCT). Unrelated cord blood is an ideal source of hematopoietic stem cells due to its easy availability, low immunogenicity, and low incidence of chronic graft-versus-host disease (cGVHD) after transplantation.The optimization of the conditioning regimen for UCBT is a crucial factor in determining patient outcomes.\n\nThis is a Phase II clinical study. A total of 11 adult patients with refractory\u002Frelapsed AA will be treated with a UCBT regimen based on a TMI-based conditioning regimen. Patients who meet the inclusion\u002Fexclusion criteria will sign an informed consent form before undergoing cord blood transplantation. The efficacy (12-month EFS) and safety of the regimen will be assessed 12 months after transplantation.",[28],"Aplastic Anemia Idiopathic",[30,31,32,33],"aplastic anemia","refractory\u002Frelapsed","Total Marrow Irradiation","Umbilical Cord Blood Transplantation","NOT_YET_RECRUITING","2025-07-12",{"date":37,"type":38},"2025-07-22","ACTUAL",{"date":40,"type":22},"2025-07-20",{"date":42,"type":22},"2028-06",{"name":44,"class":45},"Institute of Hematology & Blood Diseases Hospital, China","OTHER",1,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":51,"acronym":52,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":23,"phases":56,"briefSummary":58,"conditions":59,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":61,"lastUpdatePostDateStruct":62,"startDateStruct":64,"completionDateStruct":66,"leadSponsor":68,"locationsCount":46},"100562761","phase-1-rescuing-bone-marrow-function-in-patients-with-aplastic-anaemia-and-bone-marrow-failure-post-allogeneic-transplantation-2-100562761","NCT06607367","REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2","RESELECT2","Inclusion Criteria:\n\nPatient must meet ALL of the following:\n\n1. Poor Graft function OR Relapsed\u002Frefractory AA\n\n   1. Poor Graft Function defined as follows:≥95% donor chimerism at last reading OR ≥95% CD3 negative chimerism; ≥2 Lineage cytopenias defined as:\n\n      Thrombocytopenia:\n      * 30x109 \u002FL from D40-D60 OR\n      * 50 x10 9\u002FL from D60 onwards; Neutropenia requiring filgrastim support at any time post D40; Hb less than 80g\u002FL;\n   2. Relapsed \u002FRefractory AA defined as: Relapse after stem cell transplant OR relapsed post\u002Frefractory to 1st line immunosuppression without an unrelated donor identified.\n2. Age ≥18\n3. ECOG performance status 0-1\n4. Life expectancy greater than 6 months\n5. Patient's written informed consent\n\nExclusion Criteria:\n\n1. Active Grade 3-4 acute GVHD\n2. Relapsed or progressive disease on screening bone marrow biopsy or most recent PET imaging.\n3. Active second malignancy currently requiring treatment\n4. Human Immuno-deficiency Virus (HIV) infection.\n5. Any coexisting medical or psychological condition that would preclude participation in the required study procedures.\n6. Female patients who are both lactating and breast-feeding or have a positive serum pregnancy test during the screening period or a positive pregnancy test on Day 1 before first dose of study drug",{"count":55,"type":22},20,[57,25],"PHASE1","Allogeneic stem cell transplantation involves the transplanting of donor blood stem cells into a recipient, this is performed mainly for the treatment of blood cancers. The bone marrow is the organ that produces all blood cells and allogeneic stem cell transplantation results in the replacement of abnormal recipient bone marrow with donor blood cells as well as the production of donor immune cells from the donor bone marrow. The production of donor immune cells will hopefully lead to an immune response directed at any persisting cancerous cells leading to their eradication. As such, one of the key measures of success of a transplant is establishment of donor engraftment.\n\nEngraftment is considered successful when the patient has normal blood cell counts on routine laboratory testing as well as confirmation that the blood cells are being produced by donor bone marrow cells. Confirming donor blood cell production is done by a process called chimerism. Poor graft function (PGF) is a complication of allogeneic stem cell transplantation related to engraftment, manifested by low blood counts despite complete donor chimerism. This has significant consequences for the patient leaving them susceptible to infection because of low white blood cells and bleeding because of low platelets (the cell components that are important for blood clotting). There is currently no established treatment for this condition and patients with this condition who do not recover have a poor survival.\n\nAplastic anaemia (AA) is a rare autoimmune condition that results in a patient's own immune system attacking important components of their bone marrow resulting in low blood counts. The current treatments for AA include suppressing the immune system or a bone marrow transplant, however long term survival for patients who do not respond to these treatments or relapse is poor and more effective treatments are required.\n\nThere is emerging evidence that demonstrates that the components of the immune system are dysfunctional and result in excessive immune activation resulting in suppression of the bone marrow characteristic of PGF. Similar features of immune dysfunction has been demonstrated in AA. Ruxolitinib is a drug that may be able to reduce this excessive immune activation. Eltrombopag is a drug that has been shown to stimulate the production of blood cells. The aim of this study is to evaluate whether the combination of ruxolitinib and eltrombopag is safe and effective in the treatment of PGF and AA.",[60,28],"Poor Graft Function","2024-09-18",{"date":63,"type":38},"2024-09-23",{"date":65,"type":22},"2024-10-30",{"date":67,"type":22},"2026-10-30",{"name":69,"class":45},"Melbourne Health",{"id":71,"slug":72,"hasResults":11,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":77,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":78,"targetDuration":4,"studyType":23,"phases":79,"briefSummary":81,"conditions":82,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":4},"100554045","phase-3-outcomes-in-bone-marrow-aplasia-100554045","NCT06493981","Outcomes in Bone Marrow Aplasia.","Long Term Outcomes of Eltrombopag in Patients With Bone Marrow Aplasia, Assiut University Hospital Insight.","Inclusion Criteria:\n\n\\- Age \\> 18. Newely diagnosed bone marrow aplasia Eastern Cooperative Oncology Group (ECOG) performance status of 0-2. Patients started CSA plus Eltrombopag therapy Normal cardiac, hepatic \\& renal functions\n\nExclusion Criteria:\n\nHypersensitivity or contraindications to eltrombopag. Cardiovascular, pulmonary, hepatic, or renal diseases. History of malignancy. Pregnant, breastfeeding. Inherited bone marrow aplasia. Secondry bone marrow aplasia Previous thromboembolic events. Previous malignancies either solid or hematologic.\n\n\\-",true,{"count":5,"type":22},[80],"PHASE3","Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and fatty infiltration of the bone marrow space. Underlying mechanisms include immune-mediated attack, telomere defects, and inherent HSC compartment insufficiency. These events may occur individually or in concert, mostly involving effector T cells Historical treatment has included the use of high-dose chemotherapy and allogeneic stem cell transplantation as well as lymphotoxic immunosuppressive therapy (IST) Thrombopoietin (TPO) regulates platelet production, maturation, and release through binding of c-mpl on megakaryocytes.",[28],"2024-07-02",{"date":85,"type":38},"2024-07-10",{"date":87,"type":22},"2024-07",{"date":89,"type":22},"2030-07",{"name":91,"class":45},"Assiut University"]