[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"aplastic-anemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:aplastic-anemia":30},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,28,0,25,[9,44,70,103,127,148,178,210,240,273,297,321,354,390,417,451,477,508,540,561,585,606,631,654,674],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":4},"100054233","phase-2-dexamethasone-intravenous-injection-of-human-immunoglobulin-and-increased-infusion-of-mononuclear-cells-to-reduce-donor-specific-antibodies-in-haploid-hematopoietic-stem-cell-transplantation-a-prospective-multicenter-study-100054233",false,"NCT07698080","Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploid Hematopoietic Stem Cell Transplantation: a Prospective, Multicenter Study","Inclusion Criteria:\n\n1. Diagnosis of benign or malignant hematological diseases (including leukemia, lymphoma, thalassemia, aplastic anemia, myelodysplastic syndromes, etc.) confirmed by NCCN guidelines, and determined by the investigator to require allogeneic hematopoietic stem cell transplantation.\n2. Eastern Cooperative Oncology Group (ECOG) performance status ≤ 3.\n3. Donor-specific antibody (DSA) mean fluorescence intensity (MFI) \\> 500.\n4. Age between 18 and 65 years (age limits are also captured separately in the eligibility module).\n5. Body weight between 40 kg and 100 kg.\n6. No other suitable donor available (i.e., no DSA-negative related donor or unrelated donor).\n\nExclusion Criteria:\n\n1. Patients unsuitable for transplantation or without willingness to undergo transplantation, or diagnosed with non-hematological diseases.\n2. Estimated life expectancy \\\u003C 1 month.\n3. Known allergy to any drug or intervention used in the study regimen.\n4. Pregnancy, lactation, active severe infection, or severe major organ dysfunction.\n5. Severe psychiatric or neurological disorders that may affect the ability to provide informed consent and\u002For to report adverse events or comply with observation.\n6. Refusal or inability to sign the informed consent form.","ALL","18 Years","65 Years",{"count":20,"type":21},60,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower harmful antibodies called donor-specific antibodies (DSA) in patients who need a stem cell transplant from a half-matched (haploidentical) family donor.\n\nIn these transplants, DSA are antibodies made by the patient's own body that attack the donor's stem cells. If DSA levels are high, the transplant is more likely to fail - the donor cells may not \"take\" (engraft). Currently, there is no single, simple, and reliable way to reduce DSA, and many existing methods have drawbacks.\n\nBased on our earlier experience in 11 patients, this three-part approach seemed to work well. All patients successfully engrafted, and DSA levels dropped quickly. Now we want to confirm these results in a larger, prospective, multicenter study.\n\nWe plan to enroll 60 patients aged 18-65 with blood cancers or other blood disorders who need a haploidentical transplant, have DSA levels above 500 MFI (a measure of antibody strength), and have no other suitable donor available. Participants will receive:\n\n* Dexamethasone (25 mg\u002Fm²) for 4 days before transplant,\n* IVIG (1 g\u002Fkg) one day before transplant,\n* Extra mononuclear cells on transplant day - the extra amount depends on how high their DSA level is (low, medium, or high).\n\nThe main goal is to see how many patients have primary graft failure (when the donor cells never engraft). We will also measure how long it takes for blood counts to recover, rates of graft-versus-host disease, survival, and side effects. All participants will be followed for 1 year.\n\nThis study will help us find out whether this combination is a safe, simple, and effective way to improve transplant success for patients with DSA who have no other donor options.",[27,28,29,30,31],"Leukemia","Lymphoma","Thalassemia","Aplastic Anemia","Myelodysplastic Syndromes","NOT_YET_RECRUITING","2026-07-07",{"date":35,"type":36},"2026-07-13","ACTUAL",{"date":38,"type":21},"2026-07-03",{"date":40,"type":21},"2028-07-03",{"name":42,"class":43},"Hematology department of the 920th hospital","OTHER",{"id":45,"slug":46,"hasResults":12,"nctId":47,"briefTitle":48,"officialTitle":49,"acronym":4,"eligibilityCriteria":50,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":51,"targetDuration":4,"studyType":22,"phases":53,"briefSummary":54,"conditions":55,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":60,"lastUpdatePostDateStruct":61,"startDateStruct":63,"completionDateStruct":65,"leadSponsor":67,"locationsCount":69},"100180554","phase-2-methylprednisolone-horse-anti-thymocyte-globulin-cyclosporine-filgrastim-andor-pegfilgrastim-or-pegfilgrastim-biosimilar-in-treating-patients-with-aplastic-anemia-or-low-or-intermediate-risk-myelodysplastic-syndrome-100180554","NCT01624805","Methylprednisolone, Horse Anti-Thymocyte Globulin, Cyclosporine, Filgrastim, and\u002For Pegfilgrastim or Pegfilgrastim Biosimilar in Treating Patients With Aplastic Anemia or Low or Intermediate-Risk Myelodysplastic Syndrome","Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients With Aplastic Anemia (AA), or Low\u002FInt-1 Risk Myelodysplastic Syndrome (MDS)","Inclusion Criteria:\n\n1. Patients with the diagnosis of MDS (Low, Int-1 by IPSS, or hypocellular) who are either previously treated or untreated are eligible for this trial.\n2. Patients with the diagnosis of aplastic anemia who are either previously treated or untreated are eligible if they are not currently candidates for an allogeneic stem cell transplant.\n3. Patients ages 18 years and older are eligible\n4. Patients must have been off of cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks prior to entering this study, and have recovered from the toxic effects of that therapy to grade 1 or less.\n5. Adequate organ function as defined below:\n\n   * liver function (bilirubin \\\u003C 2mg\u002FdL, AST \\\u003C3 x ULN)\n   * kidney function (creatinine \\\u003C 2.5 x ULN ).\n6. ECOG performance status of ≤ 2.\n7. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.\n8. A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.\n9. Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol.\n10. Patients should have an indication for therapy for their disease such as transfusion dependence or morbidity associated with their cytopenia(s) such as bleeding, severe fatigue, or frequent\u002Fmultiple infections (eg. neutropenia).\n\nExclusion Criteria:\n\n1. Pregnant women are excluded from this study. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated on this study.\n2. Known HIV infection.\n3. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n4. Patient with documented hypersensitivity to any of the component medications.",{"count":52,"type":21},140,[24],"This phase II trial studies methylprednisolone, horse anti-thymocyte globulin, cyclosporine, filgrastim, and\u002For pegfilgrastim or pegfilgrastim biosimilar in treating patients with aplastic anemia or low or intermediate-risk myelodysplastic syndrome. Horse anti-thymocyte globulin is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in aplastic anemia and in some cases of myelodysplastic syndromes, killing these cells may help treat the disease. Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in aplastic anemia and myelodysplastic syndromes. Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count. Giving methylprednisolone and horse anti-thymocyte globulin together with cyclosporine, filgrastim, and\u002For pegfilgrastim may be an effective treatment for patients with aplastic anemia or myelodysplastic syndrome.",[30,56,57,58],"de Novo Myelodysplastic Syndrome","Myelodysplastic Syndrome","Previously Treated Myelodysplastic Syndrome","RECRUITING","2026-06-18",{"date":62,"type":36},"2026-06-22",{"date":64,"type":36},"2012-06-25",{"date":66,"type":21},"2029-06-30",{"name":68,"class":43},"M.D. Anderson Cancer Center",1,{"id":71,"slug":72,"hasResults":12,"nctId":73,"briefTitle":74,"officialTitle":75,"acronym":4,"eligibilityCriteria":76,"healthyVolunteers":12,"sex":16,"minAge":77,"maxAge":4,"enrollmentInfo":78,"targetDuration":4,"studyType":22,"phases":79,"briefSummary":81,"conditions":82,"keywords":88,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":94,"lastUpdatePostDateStruct":95,"startDateStruct":97,"completionDateStruct":99,"leadSponsor":101,"locationsCount":69},"100569096","lets-get-real-family-health-communication-tool-in-pediatric-stem-cell-transplant-and-cellular-therapy-100569096","NCT06689800","Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy","Let's Get REAL: A Pilot Trial of a Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy","Inclusion Criteria Youth:\n\n* Children or adolescents 8-17 years of age referred for SCTCT.\n* Diagnosis of malignant or nonmalignant disorder.\n* Referred for any type of SCTCT. Autologous and allogeneic stem cell and cellular therapies are eligible.\n* Planning to meet with a provider to discuss SCTCT.\n* Must have the ability to understand and willingness to consent to participate after reviewing an IRB approved informed assent document.\n* Must speak English and be cognitively able to participate.\n\nInclusion Criteria Parents:\n\n* Parent or guardian of a child 8-17 years of age with any diagnosis referred for any type of SCTCT. Diagnoses may include malignant and nonmalignant disorders. Autologous and allogeneic stem cell and cellular therapies are eligible. Parent or guardian is defined as an adult who usually cares for the youth and has authority to make medical decisions for them.\n* Must have the ability to understand and willingness to consent to participate after reviewing an IRB approved informed consent document.\n* Must speak English and be cognitively able to participate.\n\nExclusion Criteria Youth:\n\n* Active medical problems severe enough to preclude study participation at the time of recruitment.\n\n  * Patients who are otherwise eligible, but whose primary transplant physician does not want them to participate in the study.\n* Lacks cognitive capacity to complete study activities, as determined by consenting professional.\n\nExclusion Criteria Parents:\n\n* Their youth referred for SCTCT does not assent to participate.","8 Years",{"count":20,"type":21},[80],"NA","The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention among 24 youth and their parents, stratified by youth age (stratum 1, 8-12 years of age and stratum 2, 13-17 years of age).",[83,84,85,30,86,87],"Hematologic Malignancy","Solid Tumor","Sickle Cell Disease","Immune Deficiency","Metabolic Disorder",[89,90,91,92,93],"Pediatric","Family","Decision making","Health Communication","Intervention","2026-06-01",{"date":96,"type":36},"2026-06-04",{"date":98,"type":36},"2024-11-26",{"date":100,"type":21},"2026-11-30",{"name":102,"class":43},"Washington University School of Medicine",{"id":104,"slug":105,"hasResults":12,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":111,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":114,"conditions":115,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":118,"lastUpdatePostDateStruct":119,"startDateStruct":121,"completionDateStruct":123,"leadSponsor":125,"locationsCount":69},"100639998","brain-cognitive-network-abnormalities-in-anemia-patients-using-fnirs-100639998","NCT07578675","Brain Cognitive Network Abnormalities in Anemia Patients Using fNIRS","Construction of Abnormal Projection Regions of Cognitive Neural Networks in Patients With Anemia Using Functional Near-Infrared Spectroscopy (fNIRS)","AN-CNA","Inclusion Criteria:\n\n* Diagnosis of anemia\n* First hospitalization\n* Male or female, aged 18-65 years\n* Willing and able to comply with the requirements for this study and written informed consent\n\nExclusion Criteria:\n\n* History of psychiatric illness\n* History of severe brain disease\n* Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study",{"count":112,"type":21},323,"OBSERVATIONAL","Anemia is a global public health concern and is closely linked to dysfunction of brain cognitive neural networks, a key mechanism underlying cognitive impairment. Such deficits-including declines in memory, learning, processing speed, and executive function-reduce daily living abilities and increase risks of falls and depression.\n\nFunctional near-infrared spectroscopy (fNIRS) enables real-time monitoring of oxygenated and deoxygenated hemoglobin fluctuations, reflecting brain network function. This study aims to identify affected neural network regions in anemic patients and visualize connectivity changes using heatmaps and arc-based mapping. The findings will support early detection of cognitive impairment and guide precise clinical interventions, ultimately informing individualized treatment strategies to enhance therapeutic outcomes and quality of life.",[30,31,116,117],"Autoimmune Hemolytic Anemia","Functional Near-Infrared Spectroscopy","2026-05-05",{"date":120,"type":36},"2026-05-11",{"date":122,"type":36},"2025-10-11",{"date":124,"type":21},"2028-12-31",{"name":126,"class":43},"Institute of Hematology & Blood Diseases Hospital, China",{"id":128,"slug":129,"hasResults":12,"nctId":130,"briefTitle":131,"officialTitle":132,"acronym":4,"eligibilityCriteria":133,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":134,"targetDuration":4,"studyType":22,"phases":136,"briefSummary":137,"conditions":138,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":139,"lastUpdatePostDateStruct":140,"startDateStruct":142,"completionDateStruct":144,"leadSponsor":146,"locationsCount":4},"100636930","phase-2-romiplostim-n01-in-chemoradiotherapy-induced-aplastic-anemia-100636930","NCT07572084","Romiplostim N01 in Chemoradiotherapy-induced Aplastic Anemia","Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced Aplastic Anemia: a Single-center, Prospective, Open-label Study","Inclusion Criteria:\n\n1. Age ≥ 18 years old;\n2. Diagnosed with relapsed\u002Frefractory chemoradiotherapy-induced AA. Relapsed\u002F refractory was defined as patients who had no response to at least 3 months of cyclosporine or thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag;\n3. At least one of the following conditions must be met: hemoglobin \\\u003C 90 g\u002F L, platelet count \\\u003C 30×109\u002FL, neutrophil count \\\u003C 1.0×109\u002FL;\n4. With baseline liver and kidney functions \\\u003C2 upper limit of normal (ULN);\n5. Without active infection that cannot be controlled by standard treatment;\n6. Signed the informed consent;\n7. ECOG score ≤ 2;\n\nExclusion Criteria:\n\n1. Had other primary or secondary bone marrow failure (BMF) diseases, such as Fanconi anemia, congenital keratinization disorder, etc.;\n2. With evidence of clonal hematological bone marrow diseases (MDS, AML) in cytogenetics;\n3. Paroxysmal nocturnal hemoglobinuria (PNH) clone ≥ 50%;\n4. Received hematopoietic stem cell transplantation before enrollment;\n5. Received ATG treatment before enrollment;\n6. Severe bleeding or infection that cannot be controlled by standard treatment;\n7. Allergic or intolerant to romiplostim N01;\n8. Active infections of HIV, HCV, or HBV, liver cirrhosis, or portal hypertension\n9. History of malignant tumors within 5 years;\n10. History of arterial or venous thrombosis;\n11. Pregnant or lactating patients;\n12. Participated in other clinical trials within 3 months.",{"count":135,"type":21},43,[24],"This study aimed to explore the efficacy and safety of romiplostim N01 in the treatment of chemoradiotherapy-induced aplastic anemia (AA)",[30],"2026-05-02",{"date":141,"type":36},"2026-05-07",{"date":143,"type":21},"2026-04",{"date":145,"type":21},"2029-12",{"name":147,"class":43},"Peking Union Medical College Hospital",{"id":149,"slug":150,"hasResults":12,"nctId":151,"briefTitle":152,"officialTitle":153,"acronym":154,"eligibilityCriteria":155,"healthyVolunteers":12,"sex":16,"minAge":156,"maxAge":157,"enrollmentInfo":158,"targetDuration":4,"studyType":22,"phases":160,"briefSummary":161,"conditions":162,"keywords":163,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":168,"lastUpdatePostDateStruct":169,"startDateStruct":171,"completionDateStruct":173,"leadSponsor":175,"locationsCount":177},"100565770","phase-2-indication-of-hsct-in-patients-with-refractoryrelapse-aa-after-first-line-standard-immunosuppressive-therapy-aged-more-than-40-years-100565770","NCT06646497","Indication of HSCT in Patients With Refractory\u002FRelapse AA After First-line Standard Immunosuppressive Therapy Aged More Than 40 Years","Evaluation of an Optimized Allogeneic Hematopoietic Stem Cell Transplantation Protocol With Post-transplant Cyclophosphamide in Patients Aged 40 to 60 Years Old With Acquired Aplastic Anemia Refractory or in Relapse After Immunosuppression","APARR","Inclusion Criteria:\n\n* Aged from 40 to 60 years old\n* Suffering from acquired refractory severe idiopathic aplastic anemia after at least 6 months treatment with anti-thymocyte globulin, cyclosporine with Eltrombopag or in relapse\n* Allograft validated in the National Multidisciplinary expertise meetings of the French reference centre for aplastic anemia\n* With an available geno-identical donor or 10\u002F10 matched donor or haploidentical donor\n* With the absence of donor specific antibody detected in the patient with a MFI \\\u003C 1500 (antibodies to the distinct haplotype between donor and recipient)\n* Usual criteria for HSCT:\n\n  * ECOG ≤ 2\n  * No severe and uncontrolled infection\n  * Cardiac function compatible with high dose of cyclophosphamide\n  * With an adequate organ function ASAT and ALAT ≤ 3N, conjugated bilirubin ≤ 2N (or total bilirubin ≤ 2N if not available), clearance creatinine ≥ 50ml \u002F min\n* With health insurance coverage\n* Women of childbearing potential and men must use contraceptive methods during their participation to the research and for 12 months and 6 months after the last dose of cyclophosphamide, respectively.\n* Having signed a written informed consent\n\nNB: The authorized contraceptive methods are: For women of childbearing age and in absence of permanent sterilization:\n\n* oral, intravaginal or transdermal combined hormonal contraception,\n* oral, injectable or transdermal progestogen-only hormonal contraception,\n* intrauterine hormonal-releasing system (IUS),\n* sexual abstinence (need to be evaluated in relation to the duration of clinical trial and the preferred and usual lifestyle of the participants).\n\nFor men in absence of permanent sterilization: sexual abstinence, condoms.\n\nIndividuals must meet all of the inclusion criteria as verified at the screening \u002F inclusion visit to be eligible to participate at the study.\n\nExclusion Criteria:\n\nPatients:\n\n* With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype).\n* With seropositivity for HIV or HTLV-1-2 or active hepatitis B or C and associated hepatic cytolysis\n* Cancer in the last 5 years (except basal cell carcinoma of the skin or \"in situ\" carcinoma of the cervix)\n* Pregnant (βHCG positive) or breast-feeding\n* Yellow fever vaccine and all others live virus vaccines within 2 months before transplantation and during the research\n* With uncontrolled coronary insufficiency, recent myocardial infarction \\\u003C 6-month, current manifestations of heart failure according to NYHA (II or more), ventricular ejection fraction \\\u003C50%\n* With renal failure with creatinine clearance \\\u003C50ml \u002Fmin\n* Any contraindication mentioned in the SmPC and the Investigator's brochure of all medicinal products planned to be used in the trial including conditioning regimen, GVHD prophylaxis, prevention of EBV reactivation, infection prophylaxis\n* Known allergy or intolerance to all medicinal products and\u002For excipients planned to be used in the trial including conditioning regimen, GVHD prophylaxis, prevention of EBV reactivation, infection prophylaxis, according to Investigator's brochure and SmPC.\n* Who have any debilitating medical or psychiatric illness, which precludes understanding the inform consent as well as optimal treatment and follow-up\n* Under legal protection (tutorship or curatorship)\n* Under state medical aid\n* Participation to another interventional trial on a medicinal product or cell therapy\n\nIndividuals meeting any of the exclusion criteria as verified at the screening \u002F inclusion visit will be ineligible to participate at the study.","40 Years","60 Years",{"count":159,"type":21},52,[24],"Outcomes for adult patients with Severe Aplastic Anemia (SAA) aged more than 40 years who are refractory or in relapse after first-line IST remain poor. Hematopoietic stem cell transplantation (HSCT) is the unic valid therapeutic option but results have always been disappointing in patients aged 40 years or older. The first cause of death after HSCT in those refractory\u002Frelapse SAA patients is still graft versus host disease (GvHD). Recently, new strategies to prevent GvHD, including T-cell replete grafts with administration of post-transplantation cyclophosphamide (PTCy), have revolutionized the field, notably in haplo-identical donor setting. Using marrow as source of stem cells and a PTCy strategy not only in haplo-identical donor setting but also in case of an available matched sibling or unrelated donor might prevent drastically GvHD and eventually be practice changing. Evaluating this new strategy is the main objectives of \"APARR\".",[30],[164,165,166,167],"Refractory\u002Frelapse idiopathic anemia","Idiopathic aplastic anemia","Hematopoietic stem cell transplantation","Post-transplantation cyclophosphamide","2026-04-27",{"date":170,"type":36},"2026-04-28",{"date":172,"type":36},"2025-01-24",{"date":174,"type":21},"2030-01-24",{"name":176,"class":43},"Assistance Publique - Hôpitaux de Paris",26,{"id":179,"slug":180,"hasResults":12,"nctId":181,"briefTitle":182,"officialTitle":182,"acronym":4,"eligibilityCriteria":183,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":184,"targetDuration":4,"studyType":22,"phases":186,"briefSummary":187,"conditions":188,"keywords":189,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":200,"lastUpdatePostDateStruct":201,"startDateStruct":203,"completionDateStruct":205,"leadSponsor":207,"locationsCount":4},"100631317","phase-2-ck0801-for-treatment-of-aplastic-anemia-100631317","NCT07499102","CK0801 for Treatment of Aplastic Anemia","Inclusion Criteria:\n\n* Age 18 years or older\n* Diagnosis of aplastic anemia\n* Transfusion dependent after at least one prior line of treatment, or intolerance to standard-of-care treatment\n* Female subjects of childbearing potential must have a negative urine or serum pregnancy test\n* Agrees to comply with all protocol-required procedures, including study-related assessments, visits, and long-term follow-up\n* Willing and able to provide written informed consent\n\nExclusion Criteria:\n\n* Known HIV seropositivity\n* Uncontrolled infection not responding to appropriate antimicrobial agents after 7 days of therapy; the Protocol PI is the final arbiter of eligibility\n* Uncontrolled intercurrent illness that, in the opinion of the investigator, would place the subject at greater risk of severe toxicity and\u002For impair the activity of CK0801\n* Pregnant or breastfeeding\n* Unable to provide consent or, in the opinion of the investigator, unlikely to fully comply with protocol requirements",{"count":185,"type":21},12,[24],"This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care treatment. CK0801 is an allogeneic cord blood-derived regulatory T-cell therapy administered intravenously. The study is designed to assess safety and clinical activity, including hematologic response, transfusion independence, duration of response, survival outcomes, and patient-reported outcomes. Exploratory assessments include immune reconstitution, biomarkers, pharmacokinetics, immunogenicity, and donor-specific antibodies. The primary endpoint is overall response at Day 180.",[30],[30,190,191,192,193,194,195,196,197,198,199],"Bone Marrow Failure","CK0801","Regulatory T Cells","Treg Cell Therapy","Cord Blood-Derived Tregs","Allogeneic Cell Therapy","Transfusion Dependent","Immune Dysregulation","Open-Label Phase 2 Study","Cord Blood Unit","2026-03-30",{"date":202,"type":36},"2026-04-03",{"date":204,"type":21},"2026-08-03",{"date":206,"type":21},"2029-08-03",{"name":208,"class":209},"Cellenkos, Inc.","INDUSTRY",{"id":211,"slug":212,"hasResults":12,"nctId":213,"briefTitle":214,"officialTitle":215,"acronym":4,"eligibilityCriteria":216,"healthyVolunteers":12,"sex":16,"minAge":217,"maxAge":218,"enrollmentInfo":219,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":221,"conditions":222,"keywords":223,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":230,"lastUpdatePostDateStruct":231,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":239},"100538164","revolade-tablets-specified-drug-use-survey-100538164","NCT06287268","Revolade Tablets Specified Drug-use Survey","Revolade Tablets Specified Drug-use Survey (Pediatric Aplastic Anemia Naive to Treatment With Anti-thymocyte Immunoglobulin, CETB115G1401)","Inclusion Criteria:\n\n* Patients whose legally acceptable representative has given written consent for cooperation in this survey prior to enrollment in this survey\n* Patients aged ≥ 6 years and \\\u003C 18 years at the start of treatment with eltrombopag\n* Pediatric patients with AA who receive eltrombopag for the first time in combination with ATG after the approval of additional dosage and administration for \"ATG-naïve pediatric patients with AA\"\n\nExclusion Criteria:\n\n* Patients who have received ATG without concomitant use of eltrombopag\n* Patients with congenital AA\n* Patients with suspected or confirmed diagnosis of myelodysplastic syndrome (MDS) at the start of treatment with eltrombopag\n* Patients who have received any drug products containing the same ingredient as eltrombopag (including investigational products)","6 Years","17 Years",{"count":220,"type":21},10,"This is a multicenter, single-arm, non-interventional study (NIS) to confirm the safety and efficacy of eltrombopag in Anti-Thymocyte Globulin (ATG) treatment naive pediatric patients with aplastic anemia (AA).",[30],[224,225,226,227,228,229],"NIS","Pediatric Aplastic Anemia","AA","Revolade Tablets","eltrombopag","Drug-use Survey","2026-02-18",{"date":232,"type":36},"2026-02-19",{"date":234,"type":36},"2024-07-17",{"date":236,"type":21},"2028-10-31",{"name":238,"class":209},"Novartis Pharmaceuticals",17,{"id":241,"slug":242,"hasResults":12,"nctId":243,"briefTitle":244,"officialTitle":245,"acronym":246,"eligibilityCriteria":247,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":248,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":250,"conditions":251,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":265,"lastUpdatePostDateStruct":266,"startDateStruct":268,"completionDateStruct":270,"leadSponsor":272,"locationsCount":69},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":249,"type":21},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[252,253,254,255,256,57,257,28,27,30,258,259,260,261,262,263,264],"Multiple Myeloma","Acute Myeloid Leukemia","Hemophilia","Hemophilia A","Hemophilia B","MDS","Bleeding Disorder","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","Paroxysmal Nocturnal Hemoglobinuria, PNH","2026-02-09",{"date":267,"type":36},"2026-02-12",{"date":269,"type":36},"2020-12-01",{"date":271,"type":21},"2030-12-01",{"name":126,"class":43},{"id":274,"slug":275,"hasResults":12,"nctId":276,"briefTitle":277,"officialTitle":278,"acronym":4,"eligibilityCriteria":279,"healthyVolunteers":12,"sex":16,"minAge":280,"maxAge":281,"enrollmentInfo":282,"targetDuration":4,"studyType":22,"phases":284,"briefSummary":285,"conditions":286,"keywords":287,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":288,"lastUpdatePostDateStruct":289,"startDateStruct":291,"completionDateStruct":293,"leadSponsor":295,"locationsCount":69},"100593039","phase-2-ui-romi-02-romiplostim-added-to-standard-of-care-for-treatment-naive-and-relapsed-or-refractory-severe-aplastic-anemia-100593039","NCT07001254","UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia","Phase II Open-label Clinical Trial Evaluating Efficacy of Romiplostim Added to Standard of Care for Children and Young Adults With Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia","INCLUSION CRITERIA\n\nIn order to be eligible to participate in this study, an individual must meet all of the following criteria:\n\n* Age ≥2 years to ≤21 years\n* Child should be receiving ongoing care with pediatric hematology\u002Foncology provider.\n* Confirmed Diagnosis of SAA and other related conditions based on following criteria.\n\nDiagnosis of severe Aplastic anemia (newly diagnosed or refractory based on history of prior treatments) is established if bone marrow cellularity \\\u003C25% to 30% and at least two of the following criteria are met: (a) absolute neutrophil count \\\u003C0.5 × 10\\^9\n\n\u002FL, (b) platelet count \\\u003C20 × 10\\^9\u002FL, and (c) hemoglobin \\\u003C8 g\u002FdL. In the event bone marrow cellularity is \\>30% but patient presents with severe pancytopenia and its complications; the diagnosis of SAA will be considered at the discretion of PI. For relapsed or refractory AA, minor variations in hematological parameters will be acceptable, e.g., platelet count of \\\u003C50 x 10\\^9\u002FL or hemoglobin of ≤9 g\u002FdL.\n\nOR Diagnosis of refractory aplastic anemia will include a confirmed diagnosis of SAA and the clinical assessment by the treating physician that the patient has not responded to the frontline IST by 6 months.\n\nOR Diagnosis of relapsed aplastic anemia will be determined by previous diagnosis of SAA and the prior history of successful hematologic response to IST with subsequent loss of response and\u002For requirement of supportive therapy\\*.\n\n\\*Adequate organ function within 7 days of enrollment defined as: Creatinine: ≤2.0 mg\u002FdL Hepatic function: Elevation of liver enzymes is acceptable for patients with hepatitis-induced SAA if patient does not have history of chronic liver problem such as liver cirrhosis. If necessary, liver biopsy will be performed.\n\n* Females of childbearing potential agree to use effective contraception during the study period and for 4 months after completion of therapy.\n* Patient with available allogenic stem cell donor chooses to defer HSCT option at least for 12-weeks.\n* Parent, guardian, or patient (if \\>18 years of old) must be able to provide written and voluntary informed consent and assent (if \\>12 years of age).\n\nEXCLUSION CRITERIA\n\nAn individual who meets any of the following criteria will be excluded from participation in this study:\n\n* Age \\\u003C 2 years or \\>21 years \\*Availability of suitable HLA-matched related or HLA-matched unrelated (9\u002F10 or 10\u002F10) allogenic stem cell donor and the participant fulfills the requirement for HSCT and opts to undergo allogenic HSCT.\\*\\*\n* Patients with Symptomatic Paroxysmal Nocturnal Hemoglobinuria (PNH) and\u002For PNH clones \\>50% of granulocytes or RBC at time of enrollment.\n* Preexisting condition with predisposition for thrombosis such as protein C, S, antithrombin deficiency, homozygous factor V Leiden or prothrombin 20210 polymorphism, history of idiopathic thromboembolism with patient or first degree relative.\n* Diagnosis of bone marrow failure syndrome with cancer predisposition, including chromosomal fragility disorders (Fanconi anemia, Bloom syndrome, Ataxia Telangiectasia) and other conditions with known association towards cancer predisposition.#\n* Presence of complex karyotype or monosomy 7 or 5q- or other cytogenetic abnormality with known predisposition to cancer.\n* Diagnosis of MDS.\n* Patients taking concurrent therapy with eltrombopag, avatrombopag, and lusutrombopag\\*\\*\\*.\n* Patients taking concurrent therapy with androgens\\*\\*\\*.\n* Females who are nursing or pregnant (positive serum or urine β-human chorionic gonadotropin \\[β-hCG\\] pregnancy test) at screening or pre-dose on Day 1.\n* Current alcohol or drug abuse.\n* Treatment with an investigational drug within 30 days or 5 half-lives (whichever is longer) preceding the first dose of study medication.\n* Active and uncontrolled infections (e.g., sepsis, hepatitis B, hepatitis C).\n* Chronic liver disease, i.e., fibrosis or cirrhosis.\n* Patients infected with Human Immunodeficiency Virus (HIV).\n* Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to romiplostim that contraindicates the patient's participation.\n* Known history of sensitivity or allergy to the active substance, to any of the excipients, or to any E. coli-derived product.\n* Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely.\n* Current pregnancy or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during this study.\n* Inability to understand investigational nature of the study or give informed consent.\n\n  \\# Clarification about exclusion criteria #5: Low cell counts could lead to failure to perform specific assays (e.g., cytogenetics, chromosomal fragility testing) for inherited bone marrow failure (BMF) syndrome. Furthermore, genetic testing for BMF could take weeks to get the results. Therefore, the intervention will be commenced based on diagnosis of SAA with peripheral cytopenia and bone marrow hypocellularity as per Camitta's criteria. If the participant is diagnosed with inherited BMF syndrome after the commencement of therapy requiring HSCT or other alternative therapy, the participant will be withdrawn from the study. The decision to withdraw the participant will be discussed with the adjudication committee.\n* All cases with relapsed and refractory aplastic anemia will be reviewed by the central adjudication committee to determine whether a patient qualifies for the inclusion criteria.\n\n  * Prior to participation in the study, patients will be offered available therapeutic options and concurrent competing studies evaluating alternative therapies for SAA. Matched sibling donor transplant is the preferred curative option and will be offered to all patients. If patient has a HLA-matched unrelated donor (9\u002F10 or 10\u002F10), and if there is an option to participate in a research study evaluating a cure with matched-unrelated donor transplant, the patient will offered those options prior to recruiting in the study. In US, TransIT (NCT05600426): \"A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia\" is currently recruiting newly diagnosed children with SAA for matched unrelated donor transplant who are not eligible for matched sibling donor transplant. However, the study requires availability of two matched unrelated donors (9\u002F10 or 10\u002F10). If the patient chooses to undergo matched-unrelated donor transplantation either with participation with the research study or outside the research study, the patient will not be recruited in the study.\n\n    * Prior history of use of another TPO-mimetic (eltrombopag, avatrombopag and lusutrombopag) and androgen for more than 30 days ago will not be a contraindication. Dysplastic changes with diagnosis of SAA are acceptable as long as hematopathologist is not concerned about alternative diagnosis of MDS or refractory cytopenia of childhood. Patients who have discontinued androgen therapy for more than 2- weeks will be considered for study participation.","2 Years","21 Years",{"count":283,"type":21},15,[24],"This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed\u002Frefractory, in inducing trilineage hematopoiesis in children and young adults.",[30],[30],"2025-12-17",{"date":290,"type":36},"2025-12-19",{"date":292,"type":21},"2026-08-01",{"date":294,"type":21},"2031-12-31",{"name":296,"class":43},"Anjali Sharathkumar",{"id":298,"slug":299,"hasResults":12,"nctId":300,"briefTitle":301,"officialTitle":302,"acronym":303,"eligibilityCriteria":304,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":305,"targetDuration":4,"studyType":22,"phases":307,"briefSummary":309,"conditions":310,"keywords":313,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":315,"lastUpdatePostDateStruct":316,"startDateStruct":317,"completionDateStruct":319,"leadSponsor":320,"locationsCount":69},"100615025","phase-1-the-safety-and-efficacy-of-daratumumab-in-patients-with-refractory-aplastic-anemia-100615025","NCT07287228","The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia","An Ib\u002FII Phase Clinical Study Evaluating the Safety and Preliminary Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia","DARA-AA-001","Inclusion Criteria:\n\n1. Clearly diagnosed with primary acquired aplastic anemia according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition).\n2. Refractory aplastic anemia meeting one of the following criteria:\n\n   2.1 Patients previously diagnosed with severe aplastic anemia (SAA) # or transfusion-dependent non-severe aplastic anemia (TD-NSAA) \\&: have received antithymocyte\u002Fantilymphocyte globulin (ATG\u002FALG) combined with standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist\\* therapy for at least 4 months, but showed no response or relapsed; 2.2 Patients previously diagnosed with non-transfusion-dependent non-severe aplastic anemia (NTD-NSAA): have received standard-dose cyclosporine for at least 6 months, and standard-dose thrombopoietin (TPO) receptor agonist\\* therapy for at least 4 months, but showed no response or relapsed.\n3. Hemoglobin \\\u003C90 g\u002FL or platelet count \\\u003C30×10⁹\u002FL.\n4. Not suitable for or unwilling to undergo hematopoietic stem cell transplantation; no other better treatment options available.\n5. Age ≥18 years, no sex limitation.\n6. Eastern Cooperative Oncology Group (ECOG) performance status score ≤2 (see Appendix 2).\n7. Voluntarily signed the informed consent form, understands the nature, purpose, and procedures of the trial, and is willing to comply with the trial requirements.\n\nExclusion Criteria:\n\n1. Patients with congenital bone marrow failure syndromes.\n2. Patients with bone marrow reticulin staining ≥ Grade 2.\n3. Patients with paroxysmal nocturnal hemoglobinuria (PNH) clone ≥50% or with active hemolysis.\n4. Patients with clonal chromosomal abnormalities characteristic of myelodysplastic syndrome (MDS) (except +8, 20q-, and -Y).\n5. Presence of active bacterial, viral, or fungal infection within 2 weeks prior to the first administration of the investigational drug (excluding common cold and onychomycosis), or any other severe infection. Any anti-infective therapy for infections must be completed at least 2 weeks before the first dose. History of HIV infection or HIV antibody positive during screening; positive treponema pallidum antibody during screening; active pulmonary tuberculosis (evidenced by chest imaging or other relevant examinations within 3 months prior to first administration or during screening indicating active TB infection); active hepatitis during screening (positive hepatitis B surface antigen \\[HBsAg\\], or positive hepatitis B core antibody \\[HBcAb\\] with HBV DNA ≥30 IU\u002FmL, or positive hepatitis C antibody \\[HCV Ab\\] with positive HCV RNA).\n6. Presence of active bleeding in the gastrointestinal tract, respiratory tract, central nervous system, or other sites.\n7. History of significant clinical diseases that, in the investigator's judgment, would pose a safety risk for the subject's participation or affect the evaluation of efficacy or safety if the disease\u002Fcondition worsens during the study. Examples include:\n\n   7.1 Cardiovascular disease: acute myocardial infarction or unstable angina within the past year, severe arrhythmias (such as frequent multifocal PVCs, ventricular tachycardia, ventricular fibrillation, etc.), congestive heart failure, arterial or venous thrombosis, NYHA Class III-IV cardiac function.\n\n   7.2 History of psychiatric disorders or severe cerebrovascular disease or cognitive sequelae.\n8. Use of B-cell or plasma-cell targeting agents within 3 months prior to the first administration of the investigational drug or expected use during the study period.\n9. Receipt of antithymocyte globulin or antilymphocyte globulin within 6 months prior to the first administration of the investigational drug.\n10. Treatment with tacrolimus, sirolimus, cyclophosphamide, anti-CD52 monoclonal antibodies, or similar agents within 4 weeks or 5 half-lives (whichever is shorter) prior to the first administration of the investigational drug.\n11. Planned participation in another clinical trial or prior exposure to another investigational product within less than 4 weeks or 5 half-lives (whichever is shorter) before the first dose.\n12. Vaccination with live attenuated vaccines within 4 weeks prior to the first administration of the investigational drug or planned during the study period, or COVID-19 vaccination within 7 days prior to first administration.\n13. Prior exposure to CD38-targeted therapy.\n14. Pregnant or breastfeeding women, or women planning to become pregnant or breastfeed during the study; female subjects of childbearing potential and male subjects with partners of childbearing potential who are unwilling to use highly effective contraception (see Appendix 3 for specific methods) throughout the study (from signing the ICF to 6 months after the last dose of study drug), or who plan to donate eggs or sperm during the study.\n15. Any other conditions deemed unsuitable for participation in this study by the investigator.",{"count":306,"type":21},37,[308,24],"PHASE1","This study consists of two phases, phase Ib and phase II. The primary objective of phase Ib is to evaluate the safety and tolerability of multiple doses of daratumumab in patients with refractory aplastic anemia. The primary objective of phase II is to preliminarily assess the efficacy of multiple doses of daratumumab in patients with refractory aplastic anemia. Secondary objectives include evaluation of other efficacy endpoints, safety (phase II), and pharmacodynamic characteristics of multiple doses of daratumumab in these patients. The exploratory objective is to assess biological functional changes in peripheral blood and bone marrow before and after daratumumab treatment.",[30,311,312],"Refractory","Relapse",[314],"Aplastic anemia, Daratumumab","2025-12-16",{"date":288,"type":36},{"date":318,"type":21},"2025-12-10",{"date":124,"type":21},{"name":126,"class":43},{"id":322,"slug":323,"hasResults":12,"nctId":324,"briefTitle":325,"officialTitle":326,"acronym":4,"eligibilityCriteria":327,"healthyVolunteers":328,"sex":16,"minAge":329,"maxAge":330,"enrollmentInfo":331,"targetDuration":4,"studyType":22,"phases":333,"briefSummary":334,"conditions":335,"keywords":338,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":346,"lastUpdatePostDateStruct":347,"startDateStruct":348,"completionDateStruct":350,"leadSponsor":352,"locationsCount":353},"100549190","phase-2-a-study-of-emapalumab-for-pediatric-aplastic-anemia-100549190","NCT06430788","A Study of Emapalumab for Pediatric Aplastic Anemia","Phase 2a\u002F2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia","Inclusion Criteria:\n\n* Patients undergoing workup for suspected newly diagnosed sAA:\n\n  * Patients with severe cytopenias and a hypocellular marrow concerning for sAA\n  * Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:\n\nMarrow Cellularity: \\\u003C25%, or 25-50% with \\\u003C30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \\\u003C500 x 10\\^9\u002FL Platelets: \\\u003C20 x 10\\^9\u002FL Absolute Reticulocyte Count: \\\u003C60 x 10\\^9\u002FL\n\n* Patients that do not have evidence of leukemia or MDS\n* Patients \\\u003C 25 years of age at time of diagnosis\n* Able to tolerate emapalumab and IST (with standard institutional organ function criteria)\n\nExclusion Criteria:\n\n* Uncontrolled infection at presentation.\n* Patients who have undergone previous treatment for sAA.\n* Patients with known inherited bone marrow failure\n* Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial\n* Patients with leukemia or MDS\n* Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.",true,"0 Years","25 Years",{"count":332,"type":21},35,[24],"The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options.\n\nFunding Source- FDA OOPD",[30,336,337],"Cytopenia","Hypocellular Marrow",[339,340,341,342,343,344,345],"pediatric aplastic anemia","aplastic anemia","cytopenia","hypocellular marrow","Emapalumab","Memorial Sloan Kettering Cancer Center","23-278","2025-12-11",{"date":288,"type":36},{"date":349,"type":36},"2024-05-21",{"date":351,"type":21},"2029-05-21",{"name":344,"class":43},6,{"id":355,"slug":356,"hasResults":12,"nctId":357,"briefTitle":358,"officialTitle":359,"acronym":360,"eligibilityCriteria":361,"healthyVolunteers":12,"sex":16,"minAge":362,"maxAge":363,"enrollmentInfo":364,"targetDuration":4,"studyType":22,"phases":366,"briefSummary":367,"conditions":368,"keywords":372,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":380,"lastUpdatePostDateStruct":381,"startDateStruct":383,"completionDateStruct":385,"leadSponsor":387,"locationsCount":389},"100454196","phase-2-exercise-training-and-nr-supplementation-trial-to-improve-fitness-in-aya-hct-survivors-100454196","NCT05194397","Exercise Training and NR Supplementation Trial to Improve Fitness in AYA HCT Survivors","Intensive Tailored Exercise Training With NAD+ Precursor Supplementation to Improve Muscle Mass and Fitness in Adolescent and Young Adult Hematopoietic Cell Transplant Survivors","IAMFIT","Inclusion Criteria:\n\n1. Males and females, ages 10-30 years at enrollment\n2. Able to understand and speak English\n3. Diagnosis of leukemia (myeloid, lymphoid), aplastic anemia, bone marrow failure syndrome, or myelodysplastic syndrome requiring allogeneic HCT\n4. 6-48 months from allogeneic HCT\n5. Females must have a negative urine\u002Fserum pregnancy test and must use an acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study.\n6. Subject's informed consent or parental\u002Fguardian permission (informed consent) and if appropriate, child assent\n7. Minimum weight of 24 kg\n\nExclusion Criteria:\n\n1. Known sensitivity to NR\n2. Concurrent use of any medications, including statins, likely to increase risk of NR toxicity\n3. Active malignancy, investigational agent(s) within 4 wks, or supraphysiological glucocorticoids at the baseline visit\n4. Currently meeting public health exercise guidelines\n5. Use of NAD+ precursors (supra-physiologic) within 4 weeks\n6. Hemoglobin \\\u003C 10 g\u002FdL\n7. Platelets \\\u003C 50K\n8. Diabetes Mellitus requiring insulin or insulin secretagogue\n9. HbA1C ≥ 8% or fasting glucose \\> 125 mg\u002FdL; However if the participant is diabetic, both fasting glucose level and HbA1C must meet the criteria to be deemed eligible.\n10. Kidney disease (eGFR \\\u003C 60 ml\u002Fmin\u002F1.73 m2)\n11. Liver disease Alanine aminotransferase (ALT)\u002FAspartate aminotransferase (AST) (ALT\u002FAST \\> 3 x ULN)\n12. Limitations in physical function preventing exercise testing\u002Ftraining\n13. Unstable angina or history of acute myocardial infarction (\\\u003C5 days of planned study procedures)\n14. Recurrent syncope\n15. Symptomatic severe aortic stenosis\n16. Uncontrolled arrhythmia causing symptoms\n17. Pulmonary embolus \\\u003C3 months of study procedures\n18. Thrombosis of lower extremities\n19. Symptomatic moderate or severe persistent asthma based on Forced Expiratory Volume (FEV) from pre-HCT pulmonary function testing\n20. Room air desaturation at rest ≤85%\n21. Females: Pregnant or planning pregnancy\n22. Non-cardiopulmonary disorders that may affect exercise performance or be aggravated by exercise (e.g. infection, renal failure, thyrotoxicosis, \\>moderate Graft versus host disease (GVHD) resulting in physical or functional impairment)\n23. Parents\u002Fguardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures.\n24. For MRI procedure: Contraindications to MRI. Individuals will be screened to ensure they can safely complete the MRI. \\*participants who cannot complete the MRI will not be excluded from participation in the remainder of the study procedures if they meet those inclusion and exclusion criteria.\n25. Current weight precludes safe completion of study procedures","10 Years","30 Years",{"count":365,"type":21},80,[24],"This will be a randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on skeletal muscle quality and VO2max in AYA HCT survivors.\n\nThe primary outcome is the change in muscle strength (isometric knee extension) from baseline to 16 weeks. Key secondary outcomes are the change in muscle strength (ankle plantarflexion) from baseline to 16 weeks, the change in grip strength from baseline to 16 weeks, the change in lower extremity muscle mass from baseline to 16 weeks, the change in muscle OXPHOS capacity from baseline to 16 weeks, and the change in aerobic capacity (VO2 max) from baseline to 16 weeks.",[369,370,30,31,371],"Lymphoblastic Leukemia","Cancer Survivors","Myeloid Leukemia",[373,374,375,376,377,378,370,379],"Sarcopenia","NAD+","Exercise Intervention","OXPHOS Capacity","VO2 Max","Adolescent young adults","Hematopoietic Stem Cell Transplantation (HCT)","2025-12-02",{"date":382,"type":36},"2025-12-04",{"date":384,"type":36},"2023-02-23",{"date":386,"type":21},"2026-06-30",{"name":388,"class":43},"Children's Hospital of Philadelphia",3,{"id":391,"slug":392,"hasResults":12,"nctId":393,"briefTitle":394,"officialTitle":395,"acronym":4,"eligibilityCriteria":396,"healthyVolunteers":12,"sex":397,"minAge":398,"maxAge":399,"enrollmentInfo":400,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":402,"conditions":403,"keywords":406,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":409,"lastUpdatePostDateStruct":410,"startDateStruct":412,"completionDateStruct":413,"leadSponsor":415,"locationsCount":69},"100600767","a-prediction-model-in-pregnant-women-with-aplastic-anemia-100600767","NCT07101770","A Prediction Model in Pregnant Women With Aplastic Anemia","Development and Validation of a Prediction Model for Adverse Pregnancy Outcomes in Women With Aplastic Anemia","Inclusion Criteria:\n\n1. pregnant women who had aplastic anemia before pregnancy\n2. pregnant women with newly diagnosed aplastic anemia during pregnancy\n\nExclusion Criteria:\n\n1. age less than 20 years\n2. without coagulation series results\n3. termination of pregnancy before 20 weeks of gestation\n4. multiple pregnancy","FEMALE","20 Years","50 Years",{"count":401,"type":21},300,"Aplastic anemia (AA) is a rare haematologic disorder characterized by bone marrow failure and pancytopenia. Its occurrence during pregnancy is exceedingly rare but poses significant risks, including maternal hemorrhage, infections, and perinatal complications such as preterm birth and fetal growth restriction. Consequently, pregnancy with AA is highly perilous for both mothers and infants. Early management is critical to ensure the health of both the mother and the baby. However, there are currently no predictive tools available to assess adverse outcomes in pregnant women with AA. Our center plans to conduct a multicenter, combined retrospective and prospective cohort study.",[30,404,405],"Pregnancy","Prediction Model",[340,407,408],"pregnancy","prediction model","2025-11-27",{"date":411,"type":36},"2025-12-01",{"date":409,"type":21},{"date":414,"type":21},"2030-12-31",{"name":416,"class":43},"Peking University People's Hospital",{"id":418,"slug":419,"hasResults":12,"nctId":420,"briefTitle":421,"officialTitle":422,"acronym":4,"eligibilityCriteria":423,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":424,"targetDuration":4,"studyType":22,"phases":426,"briefSummary":427,"conditions":428,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":442,"lastUpdatePostDateStruct":443,"startDateStruct":445,"completionDateStruct":447,"leadSponsor":449,"locationsCount":69},"100441709","phase-2-two-step-haplo-with-radiation-conditioning-100441709","NCT05031897","Two Step Haplo With Radiation Conditioning","A 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning","Inclusion Criteria:\n\n* Radiation-based cohort diagnoses:\n\n  * Acute myeloid leukemia\n  * Acute lymphoid leukemia in remission\n  * Myelodysplasia (MDS)\n  * Chronic lymphocytic leukemia (CLL) with no or minimal lymph node involvement\n  * Multiple myeloma\n  * Chronic myeloid leukemia\n  * Myelofibrosis\n  * Myeloid malignancy not otherwise specified\n  * Chronic myelomonocytic leukemia\n  * Essential thrombocytopenia or polycythemia vera\n  * T cell leukemia\n  * T cell lymphoma without significant lymph node disease burden\n  * Any hematological malignancy or dyscrasia not cited above in which HSCT is potentially curable\n  * Any patient who has a hematological disease that would normally be treated on a myeloablative study, but is prevented from doing so by factors in their past medical history. Examples are patients with previous treatment with radiation therapy precluding total-body irradiation (TBI), or a past history of myeloablative therapy, precluding a 2nd myeloablative regimen.\n  * Patients must have a donor who is one-haplotype mismatched (number of mismatches in either direction not considered)\n* Chemotherapy-based cohort diagnoses:\n\n  * Hodgkin or non-Hodgkin lymphoma\n  * Small lymphocytic lymphoma\u002FCLL\n  * Any other diagnosis in which chemotherapy is thought to be superior to radiotherapy for treatment of the disease\n  * Hematological malignancy in patients who cannot receive \\> 2 Gy radiation\n  * Aplastic anemia and other non-malignant hematologic dyscrasias\n  * Patients must have a donor who is one-haplotype mismatched (number of mismatches in either direction not considered)\n* Human leukocyte antigen (HLA) identical cohort diagnoses:\n\n  \\* Patients in this group will be treated in parallel to the radiation-based cohort or the chemotherapy-based group based on what category their diagnosis falls into. However, these patients will have HLA identical related donors (one-antigen cross-over event included).\n* Left ventricular ejection fraction of \\>= 50%\n* Diffusion lung capacity of oxygen \\>= 50% and forced expiratory volume at 1 second \\>= 50% of predicted corrected for hemoglobin\n* Serum bilirubin =\\\u003C 1.8\n* Aspartate aminotransferase or alanine aminotransferase =\\\u003C 2.5 x upper limit of normal\n* Creatinine clearance of \\>= 60 mL\u002Fmin\n* Patients must have adequate Karnofsky performance status (KPS) and Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) scores:\n\n  * Patients \\\u003C age 60 years must have a KPS of \\>= 60% and an HCT-CI score of 5 or less\n  * Patients aged 60 to 65 years must have a KPS of \\>= 60% and an HCT-CI score of 4 or less\n  * Patients aged 66 to 69 years must have a KPS of 90% and an HCT-CI score of 3 or less\n  * Patients aged 70 years or more must have a KPS of 90% and an HCT-CI score of 2 or less\n  * (Patients with greater than the allowable HCT-CI points for age can be enrolled for trial with approval of the principal investigator (PI) and at least 1 co-investigator (CI) not on the primary care team of the patient). This is an adjustment to account for healthy patients who meet the spirit of this protocol but have histories that result in higher than guideline HCT-CI points. An example is a patient with a solid tumor malignancy in their remote history (adds 3 points to HCT-CI total) where the treatment for the malignancy occurred years to decades before and there has been complete recovery of toxicities\n* Patients must be willing to use contraception if they have childbearing potential\n* Patient or patient's guardian is able to give informed consent\n* Patients should have a life expectancy of \\>= 6 months for reasons other than their underlying hematologic\u002Foncologic disorder\n* Patients with evidence of another malignancy, exclusive of a skin cancer that requires only local treatment, should not be enrolled on this protocol\n* Patients should not be:\n\n  * Human immunodeficiency virus positive\n  * Have active involvement of the central nervous system with malignancy. This can be documented by a normal neurological exam, magnetic resonance imaging (MRI) of the head, and\u002For a negative cerebral spinal fluid analysis\n* Pregnant or breastfeeding",{"count":425,"type":21},63,[24],"This phase II clinical trial evaluates whether a modified modality of conditioning reduces treatment-related mortality (TRM) in patients who undergo a hematopoietic stem cell transplant (HSCT) for a hematological malignancy. HSCT is a curative therapy for many hematopoietic malignancies, however this regimen results in higher rates of TRM than other forms of treatment. In recent years, less intense conditioning regimens with radiation and chemotherapy prior to HSCT have been developed. Radiation therapy uses high energy sources to kill cancer cells and shrink tumors while chemotherapy drugs like fludarabine and cyclophosphamide work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. This study evaluates whether a two-step approach with lower-intensity regimens of these treatments prior to HSCT reduces the rate of TRM.",[429,253,430,30,431,432,433,434,435,436,252,57,437,438,439,440,441],"Acute Lymphoblastic Leukemia","Adult T-Cell Leukemia\u002FLymphoma","Chronic Lymphocytic Leukemia","Chronic Myeloid Leukemia, BCR-ABL1 Positive","Chronic Myelomonocytic Leukemia","Essential Thrombocythemia","Hematopoietic and Lymphatic System Neoplasm","Hodgkin Lymphoma","Myelofibrosis","Myeloid Neoplasm","Non-Hodgkin Lymphoma","Polycythemia Vera","Small Lymphocytic Lymphoma","2025-10-28",{"date":444,"type":36},"2025-10-30",{"date":446,"type":36},"2021-10-25",{"date":448,"type":21},"2032-04",{"name":450,"class":43},"Thomas Jefferson University",{"id":452,"slug":453,"hasResults":12,"nctId":454,"briefTitle":455,"officialTitle":455,"acronym":4,"eligibilityCriteria":456,"healthyVolunteers":12,"sex":16,"minAge":457,"maxAge":458,"enrollmentInfo":459,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":461,"conditions":462,"keywords":4,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":468,"lastUpdatePostDateStruct":469,"startDateStruct":471,"completionDateStruct":473,"leadSponsor":475,"locationsCount":69},"100287065","umbilical-cord-blood-transplantation-from-unrelated-donors-100287065","NCT03016806","Umbilical Cord Blood Transplantation From Unrelated Donors","Inclusion Criteria:\n\n* Appropriate diagnosis: Patients must have a disease or syndrome amenable to therapy with hematopoietic stem cell transplantation. Diagnoses include, but are not limited to:\n* Congenital and Other Non-malignant Disorders:\n* Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)\n* Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)\n* Metabolic disorders (e.g. Hurler's Syndrome)\n* Severe aplastic anemia\n* High-Risk Leukemia:\n* Acute Myelogenous Leukemia\n* Refractory to standard induction therapy (more than 1 cycle required to achieve remission)\n\n  * Recurrent (in CR ≥ 2)\n  * Treatment-related AML or MDS\n  * Evolved from myelodysplastic syndrome\n  * Presence of FLT3 abnormalities\n  * FAB M6 or M7\n  * Adverse cytogenetics\n  * Myelodysplastic Syndrome\n  * Acute Lymphoblastic Leukemia including T lymphoblastic leukemia:\n  * Refractory to standard induction therapy (time to CR \\>4 weeks)\n  * Recurrent (in CR ≥ 2)\n  * WBC count \\>30,000\u002FmcL at diagnosis\n  * Age \\>30 at diagnosis\n* Adverse cytogenetics, such as t(9:22), t(1:19), t(4:11), and other MLL rearrangements.\n* Chronic Myelogenous Leukemia in accelerated phase or blast crisis\n* Biphenotypic or undifferentiated leukemia\n* Burkitt's leukemia or lymphoma\n* Lymphoma:\n* Large cell, Mantle cell, Hodgkin lymphoma refractory or recurrent, chemo-sensitive, and ineligible for an autologous stem cell transplant or previously treated with autologous SCT\n* Marginal zone or follicular lymphoma that is progressive after at least two prior therapies\n* Multiple Myeloma, recurrent following high-dose therapy and autologous SCT or ineligible for an autologous HSCT\n* Solid tumors, with efficacy of allogeneic HSCT demonstrated for the specific disease and disease status\n* Adequate organ function:\n* Cardiac - LVEF \\>45%, or shortening fraction \\>25%, Absence of congestive heart failure or conduction disturbances with high risk for sudden death\n* Pulmonary - DLCO (corrected for hemoglobin), FEV1 and FVC ≥ 50% predicted;\n* Renal - serum Cr \\\u003C 1.5 times the upper limit of normal for age or GFR ≥ 50 ml\u002Fmin\u002F1.73m2\n* Hepatic - total bilirubin level \\\u003C 2 times the upper limit of normal (except for patients with Gilbert's syndrome or hemolysis); if the primary disease process is causal, this criterion will be reconsidered. ALT, AST, and Alkaline phosphatase ≤ 5 times upper limit of normal.\n* Performance Status Karnofsky or Lansky score ≥ 70%.\n* Informed Consent must be obtained prior to initiating conditioning therapy.\n* Receipt of viable cord blood product(s), single or dual, must be confirmed with the stem cell processing laboratory prior to initiating conditioning therapy.\n\nExclusion Criteria:\n\n* Availability of 10\u002F10 or 9\u002F10 HLA-matched related or unrelated donor within a reasonable timeframe dictated by the clinical urgency of the transplant\n* Autologous HSCT \\\u003C 6 months prior to proposed UCB transplant\n* Pregnant or breast feeding\n* Current uncontrolled infection\n* Evidence of HIV infection or positive HIV serology","2 Months","75 Years",{"count":460,"type":21},30,"This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated donor fails. Another purpose will be to document the rate of disease-free survival and the rate of relapse (a return of your disease or syndrome) as well as the incidence and severity of graft versus host disease (GvHD) following cord blood stem cell transplantation. GvHD is a complication of stem cell transplants in which white blood cells from the transplanted tissue (graft) attack the transplant recipient's body (host).",[463,464,465,466,30,31,467,28,252,84],"Acute Leukemia","Immune Deficiency Disorder","Congenital Hematological Disorder","Metabolism Disorder","Chronic Leukemia","2025-10-08",{"date":470,"type":36},"2025-10-10",{"date":472,"type":4},"2015-06",{"date":474,"type":21},"2027-06",{"name":476,"class":43},"University of Rochester",{"id":478,"slug":479,"hasResults":12,"nctId":480,"briefTitle":481,"officialTitle":482,"acronym":483,"eligibilityCriteria":484,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":485,"targetDuration":487,"studyType":113,"phases":4,"briefSummary":488,"conditions":489,"keywords":497,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":499,"lastUpdatePostDateStruct":500,"startDateStruct":502,"completionDateStruct":504,"leadSponsor":506,"locationsCount":69},"100608743","high-altitude-hematology-observation-stem-cell-transplantation-halo-sct-100608743","NCT07205523","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)","High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT): A Prospective Real-World Cohort Study in the Qinghai-Tibet Plateau","HALO-SCT","Inclusion Criteria:\n\n1. Patients diagnosed with hematologic diseases who are admitted to the HSCT center of Qinghai University Affiliated Hospital on or after September 1, 2023.\n2. Planned or actual hematopoietic stem cell transplantation (HSCT).\n3. Provision of signed informed consent.\n\nExclusion Criteria:\n\n1. Inability to provide long-term follow-up data due to severe comorbidities or logistical reasons.\n2. Substance abuse compromising adherence.\n3. Any condition judged by investigators to jeopardize safety or compliance.",{"count":486,"type":21},1000,"100 Years","The High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT) study is the first prospective real-world cohort of hematologic diseases and transplantation in the Qinghai-Tibet Plateau. Patients undergoing hematopoietic stem cell transplantation (HSCT) at Qinghai University Affiliated Hospital, together with their donors, are systematically enrolled. The registry collects demographic, diagnostic, treatment, prognosis, and medical expense information, as well as biospecimens for future analyses. Historical data are incorporated, and prospective data collection is ongoing with long-term follow-up planned. The registry is designed as a sustainable research infrastructure to provide comprehensive data on disease incidence, treatment patterns, outcomes, and resource utilization in a high-altitude setting.",[490,491,492,493,254,57,257,28,27,30,494,259,252,495,496],"Hematopoietic Stem Cell Transplantation (HSCT)","Acute Myeloid Leukemia (AML)","Leukemias, Acute Myeloid","Myeloid Leukemias, Acute","Bleeding Disorders","Myeloma, Multiple","Immune Reconstitution",[498],"High-altitude Bone marrow transplantation Allogeneic HSCT Autologous HSCT Immune reconstitution Graft-versus-host disease Relapse Survival Quality of life","2025-09-25",{"date":501,"type":36},"2025-10-03",{"date":503,"type":36},"2023-09-01",{"date":505,"type":21},"2100-12-31",{"name":507,"class":43},"Yigeng Cao,MD,PhD",{"id":509,"slug":510,"hasResults":12,"nctId":511,"briefTitle":512,"officialTitle":513,"acronym":4,"eligibilityCriteria":514,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":515,"targetDuration":4,"studyType":22,"phases":517,"briefSummary":519,"conditions":520,"keywords":526,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":499,"lastUpdatePostDateStruct":532,"startDateStruct":534,"completionDateStruct":536,"leadSponsor":538,"locationsCount":69},"100546708","early-phase-1-darzalex-faspro-daratumumab-and-hyaluronidase-fihj-before-standard-desensitization-and-allogeneic-peripheral-blood-stem-cell-transplantation-in-adult-patients-at-high-risk-for-primary-graft-failure-secondary-to-donor-specific-antibodies-100546708","NCT06398457","Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies","A Pilot Study of Darzalex Faspro (Daratumumab and Hyaluronidase-fihj) Before Standard Desensitization and Allogeneic Peripheral Blood Stem Cell Transplantation in Adult Patients at High-risk for Primary Graft Failure Secondary to Donor Specific Antibodies","Inclusion Criteria:\n\n1. Participates must meet all other institutional criteria for the planned reduced intensity conditioning allogeneic peripheral blood stem cell transplant (RIC alloHSCT) as defined in Johns Hopkins BMT Policy; all potential non-cord blood donor sources are included: matched related, haploidentical, matched unrelated, mismatched unrelated.\n2. Participants must be ≥18 years of age.\n3. Participants must have adequate organ function for undergoing RIC allogeneic peripheral blood stem cell transplant, and for undergoing a clinical trial.\n\n   a. Hematologic. i. White blood cell (WBC). ANC ≥ 500\u002Fmm3 (growth factor support allowed). ii. Hemoglobin. No specific cut-off. (PRBC transfusion allowed). iii. Platelets. Platelets ≥ 10,000\u002Fmm3 (platelet transfusion allowed). b. Liver. Bilirubin ≤ 3.0 mg\u002FdL (unless due to Gilbert's syndrome or hemolysis), and Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) \\\u003C 5x Upper limit of normal (ULN) c. Renal. Serum creatinine ≤ 2.0 mg\u002FdL. d. Cardiac. Left ventricular ejection fraction ≥ 35%. e. Pulmonary. FEV1 ≥ 50%.\n4. Subjects are eligible if there are high levels of Donor Specific Antibody levels based on protocol specific scoring system regardless of prior attempts at standard desensitization.\n5. Participants must have a no other readily available suitable alternative donor.\n6. All potential Participants must be pre-approved by BMT faculty consensus.\n7. Participants must have adequate willingness to participate in a clinical trial.\n\nExclusion Criteria:\n\n1. Previous exposure to Daratumumab-SC or other anti-CD38 therapy\n\n   1. Exposure to Daratumumab-SC or other anti-CD38 therapies (unless a re-treatment study)\n   2. Exposure to an investigational drug (including investigational vaccine) or invasive investigational medical device for any indication within 4 weeks or 5 pharmacokinetic half-lives, whichever is longer.\n   3. Focal radiation therapy within 14 days prior to beginning of planned RIC allogeneic peripheral blood stem cell transplant regimen with the exception of palliative radiotherapy for symptomatic management but not on measurable extramedullary plasmacytoma\n2. Chronic obstructive pulmonary disease (COPD) with a forced expiratory volume in 1 second (FEV1) \\\u003C 50% of predicted normal. Note that FEV1 testing is required for participants suspected of having COPD and participants must be excluded if FEV1 is \\\u003C 50% of predicted normal.\n3. Moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any classification. Note that participants who currently have controlled intermittent asthma or controlled mild persistent asthma are allowed to participate.\n4. Known hypersensitivity or intolerance to boron or mannitol, sorbitol, corticosteroids, monoclonal antibodies or human proteins, or the excipients\n5. Diagnosis of multiple myeloma or Amyloid light-chain (AL) amyloidosis\n6. A planned myeloablative alloBMT or the planned use of bone marrow or cord blood as a stem cell source\n7. History of HIV infection at any time in past.\n8. Seropositive for hepatitis B (HBV) (defined by a positive test for hepatitis B surface antigen \\[HBsAg\\] positive, or antibodies to hepatitis B surface and\u002For core antigens \\[antiHBs or antiHBc, respectively\\] with hepatitis B virus \\[HBV\\]- DNA quantitation positive). Patients who are positive for antiHBs and\u002For antiHBc must have a negative polymerase chain reaction (PCR) for HBV-DNA quantitation result during screening. Patients with serologic findings suggestive of HBV vaccination (antiHBs positivity as the only serologic marker) AND a known history of prior HBV vaccination do not need to be tested for HBV DNA by PCR. Those who are PCR positive will be excluded.\n9. Seropositive for hepatitis C (except in the setting of a sustained virologic response (SVR), defined as aviremia at least 12 weeks after completion of antiviral therapy)\n10. Clinically significant cardiac disease, including:\n\n    1. Myocardial infarction within 6 months before RIC alloHSCT or unstable or uncontrolled disease\u002Fcondition related to or affection cardiac function (e.g., unstable angina, congestive heart failure, New York Heart Association Class III-IV)\n    2. Uncontrolled cardiac arrhythmia",{"count":516,"type":21},8,[518],"EARLY_PHASE1","This research is being done to investigate the safety and effectiveness of Darzalex Faspro (daratumumab and hyaluronidase-fihj) (a monoclonal antibody that targets plasma cells that make antibodies) and whether it can lower donor specific antibodies (DSA) levels to low enough levels to permit patients to proceed with allogeneic peripheral blood transplant (alloBMT). Those being asked to participate have high DSA levels that puts those being asked to participate at high risk of rejecting the available donor's blood stem cells and making those being asked to participate ineligible to receive a stem cell transplant.",[83,521,491,522,523,252,30,28,524,436,525,437],"Bone Marrow Transplant Rejection","Myelodysplastic Syndromes (MDS)","Acute Lymphoblastic Leukemia (ALL), Adult","Non Hodgkin Lymphoma","Chronic Myeloid Leukemia",[527,528,529,530,531],"DSA","donor specific antibodies","desensitization","BMT","allogeneic stem cell transplant",{"date":533,"type":36},"2025-09-26",{"date":535,"type":36},"2024-09-19",{"date":537,"type":21},"2027-03",{"name":539,"class":43},"Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins",{"id":541,"slug":542,"hasResults":12,"nctId":543,"briefTitle":544,"officialTitle":545,"acronym":4,"eligibilityCriteria":546,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":156,"enrollmentInfo":547,"targetDuration":4,"studyType":113,"phases":4,"briefSummary":549,"conditions":550,"keywords":552,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":553,"lastUpdatePostDateStruct":554,"startDateStruct":556,"completionDateStruct":558,"leadSponsor":560,"locationsCount":69},"100588095","reproductive-and-mental-health-of-patients-with-aplastic-anemia-100588095","NCT06936930","Reproductive and Mental Health of Patients With Aplastic Anemia","A Study on Reproductive and Psychological Health in Aplastic Anemia Patients","Inclusion Criteria:\n\n* Diagnosis of AA\n* Male or female, aged 18-40 years\n* ECOG performance status ≤2\n* Willing and able to comply with the requirements for this study and written informed consent\n\nExclusion Criteria:\n\n* Diagnosis of inherited bone marrow failure syndromes\n* Combined with other hematological diseases\n* Chemotherapy-induced bone marrow failure\n* Pregnant or breastfeeding women\n* Subjects with a history of mental illness or severe psychological disorders\n* Subjects with a history of reproductive system cancer (eg, penile cancer, testicular cancer, prostate cancer, ovarian cancer, cervical cancer, vaginal cancer, etc.)\n* Subjects that the investigator believes have other reasons that make them unsuitable for inclusion in this study",{"count":548,"type":21},600,"Aplastic anemia (AA) is a hematological disease characterized by bone marrow failure, leading to varying degrees of anemia, leukopenia, and thrombocytopenia. With the advancement of immunosuppressive therapy and hematopoietic stem cell transplantation, the survival of AA patients has significantly improved. However, these treatment approaches may result in reproductive system impairment.\n\nReproductive health has been a major concern among reproductive-age AA patients. In female patients, it often manifests as irregular menstruation, amenorrhea, and infertility; while in male patients, it may present as reduced sperm count and low sperm motility. The reproductive impairment observed in AA patients may be attributed to various factors, including the disease's underlying pathophysiology, side effects of used medications such as androgens and the toxicity conditioning agents during transplantation.\n\nNotably, immunosuppressive agents (such as cyclosporine and antithymocyte globulin) and chemotherapeutic drugs (such as cyclophosphamide) can suppress hematopoiesis and directly damage the gonads, thereby impairing reproductive function. Furthermore, long-term use of these medications may disrupt the endocrine system, affecting the secretion of sex hormones and overall reproductive capacity. Additionally, chronic anemia in AA patients can lead to compensatory physiological changes in other body systems, which may also indirectly affect reproductive health.",[30,551],"Reproductive Health",[30],"2025-07-31",{"date":555,"type":36},"2025-08-05",{"date":557,"type":36},"2025-03-03",{"date":559,"type":21},"2027-12-31",{"name":126,"class":43},{"id":562,"slug":563,"hasResults":12,"nctId":564,"briefTitle":565,"officialTitle":566,"acronym":4,"eligibilityCriteria":567,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":281,"enrollmentInfo":568,"targetDuration":4,"studyType":22,"phases":570,"briefSummary":571,"conditions":572,"keywords":574,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":576,"lastUpdatePostDateStruct":577,"startDateStruct":579,"completionDateStruct":581,"leadSponsor":583,"locationsCount":69},"100405389","phase-2-haploidentical-hct-for-severe-aplastic-anemia-100405389","NCT04558736","Haploidentical HCT for Severe Aplastic Anemia","Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia","Inclusion Criteria for Transplant Recipient\n\n1. Age less than or equal to 21 years at time of enrollment.\n2. Confirmed diagnosis of SAA or a single lineage cytopenia\n\n   (a) SAA or single lineage cytopenia will be defined as follows:\n   * i. Bone marrow cellularity \\\u003C 25% or hypocellular marrow for age, AND\n   * ii. One or more of the following (in peripheral blood): (i) Neutrophils \\\u003C 0.5 x10\\^9\u002FL (ii) Platelets \\\u003C 20 x10\\^9\u002FL, or platelet transfusion dependence (iii) Hemoglobin \\\u003C8g\u002FdL, or red blood cell transfusion dependence\n3. Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10\u002F10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.\n4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial improvement of cytopenias after first-line IST but then a later return to fulfillment of SAA disease criteria when IST is decreased or ceased). IST could have included ATG based regimens, calcineurin inhibitors and\u002For other higher dose therapy directed at the treatment of primary SAA. Patients with very severe aplastic anemia who are likely not to benefit from IST do not need to have failed a trial of IST and can proceed directly to HCT if they meet the rest of the criteria.\n5. Has a suitable single haplotype matched (≥ 3 of 6) family member donor.\n6. Patient and\u002For legal guardian must sign informed consent for HCT.\n7. Adequate organ function defined as:\n\n   1. Left ventricular ejection fraction \\> 40% or shortening fraction ≥ 25%.\n   2. Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml\u002F min\u002F1.73m2.\n   3. Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry\n   4. ≥ 92% on room air if patient is unable to perform pulmonary function testing.\n   5. Karnofsky or Lansky (age-dependent) performance score ≥ 50.\n   6. Bilirubin ≤ 3 times the upper limit of normal for age.\n   7. Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.\n8. Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered\n\nExclusion Criteria for Transplant Recipient:\n\n1. Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.\n2. Known clinical or genetic diagnosis of dyskeratosis congenita\n3. Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).\n4. Diagnosis of myelodysplastic syndrome (MDS).\n5. Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement- dependent cytotoxicity or flow cytometric testing or the presence of anti- donor HLA antibody to the high expression loci HLA-A, B, C, DRB1, or DPB1 with mean fluorescence intensity (MFI) \\> 1000 by solid phase immunoassay).\n6. Prior allogeneic hematopoietic cell transplant.\n7. Prior solid organ transplant.\n8. Known life-threatening reaction (i.e., anaphylaxis) to ATG that would prohibit use for the patient.\n9. Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as progression or no clinical improvement on appropriate medical treatment.\n10. Female patients who are pregnant (per institutional practice) or breast- feeding.\n11. Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent \\> 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years previously will not be allowed unless approved by the PI.\n12. Alemtuzumab or ATG within 2 weeks of enrollment.\n\nInclusion Criteria for Haploidentical Donor\n\n1. At least single haplotype matched (≥ 3 of 6) family member.\n2. At least 18 years of age.\n3. HIV negative.\n4. Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).\n5. Not breast feeding.\n6. Related donors must be ruled out for telomere disease by appropriate clinical and diagnostic measures (for example, clinical evaluation, telomere length testing, genetic testing, and\u002For bone marrow examination).\n7. The HAPLO donor and\u002For legal guardian must be able to sign informed consent documents.\n8. The potential HAPLO donor must be willing and able to donate PBSCs.",{"count":569,"type":21},21,[24],"This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT).\n\nPrimary Objectives\n\n* To estimate the rate of engraftment at 30 days after TCR αβ+ T-cell-depleted graft infusion in patients receiving a single dose of post graft infusion cyclophosphamide.\n* To estimate the overall survival and event free survival at 1-year post transplantation.\n\nSecondary Objectives\n\n* To calculate the incidence of acute and chronic GVHD after HCT.\n* To calculate the rate of secondary graft rejection at 1-year post transplantation\n* To calculate the cumulative incidence of viral reactivation (CMV, EBV and adenovirus).\n* To describe the immune reconstitution after TCR αβ+ T-cell-depleted graft infusion at 1 month, 3 months, 6 months, 9 months, and 1 year.\n\nExploratory Objectives\n\n* To longitudinally assess the phenotype and epigenetic profile of T-cells in SAA patients receiving HCT for SAA.\n* To assess the phenotype and epigenetic profile of T-cells in DLI administered to SAA patients post HCT.\n* To longitudinally assess CD8 T cell differentiation status in SAA patients using an epigenetic atlas of human CD8 T cell differentiation.\n* To examine the effector functions and proliferative capacity of CD8 T cells isolated from SAA patients before and after DLI.\n* Quantify donor derived Treg cells at different time points in patients received HCT.\n* Determine Treg activation status at different stages after HCT.\n* Are specific features of the DLI product associated with particular immune repertoire profiles post-transplant?\n* How does the diversity and functional profile of the DLI product alter the response to pathogens in the recipient?\n* Do baseline features of the recipient's innate and adaptive immune cells correlate with post-transplant immune repertoires and response profiles?",[30,573],"Bone Marrow Failure Syndrome",[575],"Haploidentical Transplant","2025-07-03",{"date":578,"type":36},"2025-07-04",{"date":580,"type":36},"2021-01-21",{"date":582,"type":21},"2030-07-01",{"name":584,"class":43},"St. Jude Children's Research Hospital",{"id":586,"slug":587,"hasResults":12,"nctId":588,"briefTitle":589,"officialTitle":590,"acronym":4,"eligibilityCriteria":591,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":592,"targetDuration":4,"studyType":22,"phases":594,"briefSummary":595,"conditions":596,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":597,"lastUpdatePostDateStruct":598,"startDateStruct":600,"completionDateStruct":602,"leadSponsor":604,"locationsCount":69},"100576940","phase-1-study-of-cm313sc-injection-in-subjects-with-relapsedrefractory-aplastic-anemia-100576940","NCT06791824","Study of CM313(SC) Injection in Subjects With Relapsed\u002FRefractory Aplastic Anemia","A Phase IB\u002FII Clinical Study to Assess the Safety, Tolerability and Preliminary Efficacy of CM313(SC) Injection in Subjects With Relapsed\u002FRefractory Aplastic Anemia","Inclusion Criteria:\n\n1. Diagnosis of idiopathic aplastic anemia.\n2. Patients must meet the requirements of relapsed\u002Frefractory aplastic anemia defined in the protocol.\n3. Male or female, age≥18 years.\n4. The Eastern Cooperative Oncology Group (ECOG) physical status score ≤2.\n5. Willing and able to comply with the requirements for this study and written informed consent.\n\nExclusion Criteria:\n\n1. The inherited bone marrow failure syndromes.\n2. Previously treated with the anti-CD38 monoclonal antibody.\n3. Have an allergy to humanized monoclonal antibody or any part of CM313.\n4. Pregnant or breastfeeding females, or females planning to become pregnant during the study.\n5. Any condition considered to be ineligible for the study by the investigator.",{"count":593,"type":21},125,[308,24],"This is a randomized, open-label, phase IB\u002FII clinical study to evaluate the safety, tolerability and preliminary efficacy of CM313(SC) injection in patients with relapsed\u002Frefractory aplastic anemia",[30],"2025-02-14",{"date":599,"type":36},"2025-02-18",{"date":601,"type":21},"2025-03",{"date":603,"type":21},"2027-12",{"name":605,"class":209},"Keymed Biosciences Co.Ltd",{"id":607,"slug":608,"hasResults":12,"nctId":609,"briefTitle":610,"officialTitle":611,"acronym":4,"eligibilityCriteria":612,"healthyVolunteers":12,"sex":16,"minAge":613,"maxAge":4,"enrollmentInfo":614,"targetDuration":4,"studyType":22,"phases":616,"briefSummary":617,"conditions":618,"keywords":620,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":623,"lastUpdatePostDateStruct":624,"startDateStruct":626,"completionDateStruct":628,"leadSponsor":630,"locationsCount":69},"100563262","phase-2-standard-immunosuppressive-therapy-combined-with-romiplostim-n01-as-first-line-treatment-for-severe-aplastic-anemia-100563262","NCT06613880","Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia","A Prospective, Single Center, Single-arm Study on the Efficacy and Safety of Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia","Inclusion Criteria:\n\n* Age ≥ 12 years\n* Diagnosis of severe or very severe aplastic anemia\n* Patients are unconditionally or unacceptable undergoing allogeneic hematopoietic stem cell transplantation\n* ECOG performance status ≤2\n* Willing and able to comply with the requirements for this study and written informed consent.\n\nExclusion Criteria:\n\n* Received \\> 4 weeks of TPO-RA drug before treatment\n* Received \\> 4 weeks of immunosuppressive therapy before treatment\n* History of thromboembolic disease\n* Intolerance to Romiplostim N01 or cyclosporine\n* Allergy to ALG\n* Presence of uncontrolled active infection\n* Presence of uncontrolled hypertension (≥140\u002F90mmHg), diabetes mellitus (fasting blood glucose≥ 7.0 mmol\u002FL or random blood glucose ≥ 11.1 mmol\u002FL)\n* Abnormal liver and kidney function: Aspartate Transaminase (AST) or Alanine Transaminase (ALT) \\> 3 ULN, creatinine ≥ 2.5 ULN\n* History of chemoradiotherapy for malignant solid tumors\n* History of other systemic serious illnesses\n* Females who are pregnant\u002Flactating or need pregnancy\n* Patients considered to be ineligible for the study by the investigator for reasons other than the above","12 Years",{"count":615,"type":21},48,[24],"This prospective, single center, single-arm study aims to evaluate the efficacy and safety of anti-human T lymphocyte porcine immunoglobulin (p-ALG) in combination with cyclosporine and Romiplostim in treating severe aplastic anemia (SAA). It will include 48 patients with newly diagnosed SAA.",[30,619],"TPO Receptor Agonists",[621,622],"Severe aplastic anemia","Romiplostim N01","2024-11-29",{"date":625,"type":36},"2024-12-04",{"date":627,"type":36},"2024-10-24",{"date":629,"type":21},"2026-09-30",{"name":126,"class":43},{"id":632,"slug":633,"hasResults":12,"nctId":634,"briefTitle":635,"officialTitle":636,"acronym":4,"eligibilityCriteria":637,"healthyVolunteers":12,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":638,"targetDuration":4,"studyType":22,"phases":639,"briefSummary":640,"conditions":641,"keywords":642,"overallStatus":59,"whyStopped":4,"lastUpdateSubmitDate":645,"lastUpdatePostDateStruct":646,"startDateStruct":648,"completionDateStruct":650,"leadSponsor":652,"locationsCount":69},"100564757","early-phase-1-cd7-car-t-bridging-to-allohsct-for-severe-aplastic-anemia-100564757","NCT06633328","CD7 CAR-T Bridging to alloHSCT for Severe Aplastic Anemia","Clinical Study on the Safety and Efficacy of Donor Derived CD7 CAR-T Cell Bridging Allogeneic Hematopoietic Stem Cell Transplantation for the for Patients With Severe Aplastic Anemia","Inclusion Criteria:\n\n* Chinese expert consensus on the diagnosis and treatment of aplastic anemia（2017）, Diagnosis of severe aplastic anemia ，1. The degree of bone marrow cell proliferation \\\u003C 25%, or 25%-50% but residual hematopoietic cells \\\u003C 30%；2. With pancytopenia (at least two of the following peripheral blood parameters) : (1) absolute neutrophil \\\u003C0.5×10\\^9\u002FL; (2) Platelet count\\\u003C 20×10\\^9\u002FL; (3) The absolute value of reticulocytes \\\u003C20×109\u002FL;\n* Suitable donors (relatives) with allogeneic HSCT indications and at least haploid allogeneic transplantation;\n* Patients who are not suitable or unwilling to undergo traditional allogeneic hematopoietic stem cell transplantation;\n* creatinine clearance \\> 60ml\u002Fmin; without liver invasion, serum total bilirubin ≤ 1.5 times the upper limit of normal, and serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) were both ≤ 3 times the upper limit of the normal range. If there is liver invasion, serum erythrambirubin ≤ 3 times the upper limit of normal, and serum ALT and AST are both ≤ 5 times the upper limit of the normal range;\n* Echocardiogram shows left ventricular ejection fraction (LVEF) ≥ 50%;\n* No active infection in the lungs, blood oxygen saturation in indoor air is ≥ 92%;\n* Estimated survival time ≥ 3 months;\n* ECOG performance status 0 to 1;\n* Females and males of childbearing potential must agree to use adequate contraception prior to study entry, during study participation, and for 6 months after infusion (the safety of this therapy for unborn children is not known and has unknown risks);\n* Those who voluntarily participated in this trial and provided informed consent;\n\nExclusion Criteria:\n\n* Allergy to pre-treatment measures；\n* Those with acute graft versus host disease (GvHD) or moderate to severe chronic GvHD within 4 weeks before screening; Those who have received systemic drug therapy for GvHD within 4 weeks before the reinfusion；\n* History of epilepsy or other central nervous system disorders；\n* Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythmia in the past;\n* Less than 100 days after allogeneic hematopoietic stem cell transplantation；\n* Patients with HIV infection，Active infection of hepatitis B virus or hepatitis C virus，Uncured active infection；\n* The proiferation rate is less than 5 times response to CD3\u002FCD28 co-stimulation signal;\n* Received anti-cancer chemotherapy or other drug treatment within 2 weeks prior to screening;\n* Any condition that, in the opinion of the investigator, may increase the risk to the subject or interfere with the results of the study.",{"count":460,"type":21},[518],"This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with severe aplastic anemia",[30],[643,644],"allo-HSCT","CD7 CAR-T","2024-10-08",{"date":647,"type":36},"2024-10-09",{"date":649,"type":21},"2024-10-20",{"date":651,"type":21},"2027-10-20",{"name":653,"class":43},"Zhejiang University",{"id":655,"slug":656,"hasResults":12,"nctId":657,"briefTitle":658,"officialTitle":659,"acronym":4,"eligibilityCriteria":660,"healthyVolunteers":328,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":661,"targetDuration":4,"studyType":22,"phases":663,"briefSummary":665,"conditions":666,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":667,"lastUpdatePostDateStruct":668,"startDateStruct":670,"completionDateStruct":671,"leadSponsor":673,"locationsCount":69},"100557251","phase-4-a-study-of-romiplostim-for-the-treatment-of-refractory-transfusion-dependent-nsaa-100557251","NCT06535685","A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA","A Single-centre, Prospective, Open-label, Single-arm Study of Romiplostim for the Treatment of Refractory Transfusion-dependent Non-severe Aplastic Anaemia (NSAA)","Inclusion Criteria:\n\n1. Age ≥18 years, male or female.\n2. Diagnosis consistent with refractory transfusion dependence NSAA defines refractory as patients who have failed to respond to at least 6 months of prior first-line treatment with adequate doses of cyclosporine (3-5 mg\u002Fkg) and who have been treated with an adequate dose of at least one povidone for 3 months. Definition of transfusion dependence: at least 1 component transfusion on average every 8 weeks and duration of transfusion dependence ≥ 4 months.\n3. Satisfy at least one of the following conditions at the time of enrolment: haemoglobin \\\u003C90 g\u002FL, platelets \\\u003C30×10\\^9\u002FL, neutrophils \\\u003C1.0×10\\^9\u002FL.\n4. Agree to sign the consent form.\n5. An Eastern Cooperative Oncology Group (ECOG) score of 0-2.\n\nExclusion Criteria:\n\n1. Other causes of whole blood cytopenia, such as myelodysplastic syndromes (MDS).\n2. Presence of cytogenetic evidence of clonal haematological bone marrow disorders (MDS, AML).\n3. PNH clones ≥50%.\n4. Haematopoietic stem cell transplantation (HSCT) prior to enrolment.\n5. Prior treatment with ATG.\n6. Infection or haemorrhage uncontrolled by standard therapy.\n7. Allergy to roprostin.\n8. Active HIV, HCV or HBV infection or cirrhosis or portal hypertension.\n9. Any concomitant malignancy, localised basal cell carcinoma of the skin within 5 years.\n10. Liver and renal function at baseline that is more than two times normal.\n11. Active infection.\n12. Past history of thromboembolic events, heart attack or stroke (including antiphospholipid antibody syndrome) and current use of anticoagulants.\n13. Pregnant or lactating (breastfeeding) women.\n14. Participation in another clinical trial within 3 months.",{"count":662,"type":21},40,[664],"PHASE4","Efficacy and safety of Romiplostim in the treatment of refractory transfusion-dependent NSAA.",[30],"2024-07-30",{"date":669,"type":36},"2024-08-02",{"date":669,"type":21},{"date":672,"type":21},"2025-12-31",{"name":147,"class":43},{"id":675,"slug":676,"hasResults":12,"nctId":677,"briefTitle":678,"officialTitle":679,"acronym":4,"eligibilityCriteria":680,"healthyVolunteers":12,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":681,"targetDuration":4,"studyType":22,"phases":683,"briefSummary":684,"conditions":685,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":687,"lastUpdatePostDateStruct":688,"startDateStruct":690,"completionDateStruct":691,"leadSponsor":693,"locationsCount":69},"100556503","phase-4-efficacy-and-safety-of-rhtpo-in-combination-with-cyclosporine-versus-cyclosporine-alone-in-the-treatment-of-td-nsaa-100556503","NCT06525948","Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of TD-NSAA","Efficacy and Safety of rhTPO in Combination With Cyclosporine Versus Cyclosporine Alone in the Treatment of Transfusion-dependent Non-severe Aplastic Anaemia (TD-NSAA)","Inclusion Criteria:\n\n1\\. aged ≥ 18 years, male and female; 2. patients with a clear diagnosis of NSAA who are dependent on transfusion therapy:\n\n1. Meet the Camitta NSAA criteria;\n2. accompanied by at least one of the following abnormalities: (1) dependence on component blood transfusion therapy, at least one component blood transfusion every 8 weeks on average, and the duration of transfusion dependence ≥ 4 months, the indication of component blood transfusion: HGB ≤ 60g \u002F L; (2) PLT ≤ 10 × 10 \\^ 9 \u002F L, or PLT ≤ 30 × 10 \\^ 9 \u002F L with a significant tendency to bleed; (3) neutrophils ≤ 0.5 × 10 \\^ 9 \u002F L.\n3. Excluding other haematological and non-haematological diseases that cause pancytopenia; 3. ECOG PS score 0-2, expected survival ≥ 3 months with follow-up; 4. functional levels of major organs must meet the following requirements: 1) Total bilirubin (TBIL) ≤ 1.5 times the upper limit of normal (ULN); 2) alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 2.5 x ULN; 3) blood creatinine (Cr) ≤ 1.5 x ULN; 5. has not been treated with platelet receptor agonist (TPO-RA) analogues and other immunosuppressant analogues; 6. the subject is not suitable or willing to receive haematopoietic stem cell transplantation therapy; 7. no history of serious heart, lung, liver, kidney and other important organs and endocrine system diseases; 8. Voluntarily enroll in the study, sign the informed consent, have good compliance and willing to cooperate with the follow-up.\n\nExclusion Criteria:\n\n1. have used other clinical investigational drugs within 4 weeks;\n2. a history of primary myelodysplastic syndromes (MDS), primary paroxysmal sleep haemoglobinuria (PNH) and leukaemia, as well as congenital bone marrow failure syndromes (IBMFS), such as Fanconi's anaemia (FA) and congenital dyskeratosis (DC)\n3. history of cirrhosis or history of portal hypertension;\n4. congestive heart failure, arrhythmia, peripheral arteriovenous thrombosis requiring medication within 1 year prior to enrolment, or myocardial infarction or cerebral infarction within 3 months prior to enrolment;\n5. HIV infection;\n6. severe autoimmune disease or immunodeficiency disease;\n7. suffering from malignant tumour\n8. severe mental disorders;\n9. a known history of allergy to the drug components of this regimen;\n10. in the opinion of the investigator, it is not appropriate to participate in this trial, e.g., any other medical, social or psychological factors that may affect safety or compliance with the study procedures.\n\nCompliance with study procedures.",{"count":682,"type":21},54,[664],"Investigating the efficacy and safety of rhTPO in combination with cyclosporine versus cyclosporine alone for the treatment of TD-NSAA",[30,686],"Transfusion-dependent Anemia","2024-07-26",{"date":689,"type":36},"2024-07-29",{"date":667,"type":21},{"date":692,"type":21},"2025-12-30",{"name":147,"class":43}]