[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myelodysplastic-neoplasm\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myelodysplastic-neoplasm":33},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,54,86,119],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":34,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100633910","phase-1-proton-based-total-marrow-irradiation-for-allogeneic-transplantation-in-high-risk-amlmds-100633910",false,"NCT07532824","Proton-Based Total Marrow Irradiation for Allogeneic Transplantation in High-Risk AML\u002FMDS","Proton Total Marrow Irradiation-Based Conditioning for Allogeneic Hematopoietic Stem Cell Transplantation in High-Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome","UHKT-PTC-TMI-1","Inclusion Criteria:\n\n1. Underlying diagnosis of acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS),\n\n   A) Acute Myeloid Leukemia (AML), meeting at least one of the following criteria:\n\n   i. Relapsed disease after a prior complete remission (CR) or\n\n   ii. Disease refractory to at least two cycles of intensive chemotherapy or\n\n   iii. High-risk AML in complete remission (CR), defined by at least one of the following:\n\n   iii a) Adverse molecular or cytogenetic risk according to ELN 2022 classification or\n\n   iii b) Presence of measurable\u002Fminimal residual disease (MRD).\n\n   B) Myelodysplastic Syndrome (MDS), meeting at least one of the following criteria:\n\n   i. Relapsed MDS with increased blasts (MDS-IB) or\n\n   ii. MDS-IB2 without reduction of bone marrow blasts below 10% after induction chemotherapy or after at least two cycles of azacitidine or\n\n   iii. IPSS-M score \\> 0.5 (high-risk or very high-risk disease).\n2. Eligibility confirmed by the institutionalal Transplant Indication Committee according to standard criteria.\n3. Age ≥ 18 years and ≤ 65 years\n4. Ability to understand and voluntarily sign written informed consent\n\nExclusion Criteria:\n\nSevere comorbidity, defined as the presence of one or more of the following conditions:\n\n1. Left ventricular ejection fraction (LVEF) \\\u003C 40%\n2. Creatinine clearance \\\u003C 0.5 mL\u002Fs\n3. Total bilirubin \\> 40 µmol\u002FL (unless attributable to Gilbert's syndrome or hemolysis) and alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 5 × upper limit of normal (ULN)\n4. Pulmonary function impairment defined as forced expiratory volume in 1 second (FEV1) and forced vital capacity (FVC) \\\u003C 50% of predicted value, or diffusing capacity of the lung for carbon monoxide (DLCO) \\\u003C 50% of predicted value after correction for anemia\n5. Karnofsky Performance Status \\\u003C 70%\n6. Active viral hepatitis or human immunodeficiency virus (HIV) infection\n7. Presence of liver cirrhosis\n8. Pregnancy","ALL","18 Years","65 Years",{"count":21,"type":22},16,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","This is an open-label, single-center, non-randomized phase I\u002FII pilot study evaluating proton-based Total Marrow Irradiation (TMI) as part of the conditioning regimen prior to allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult patients with high-risk or relapsed\u002Frefractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). These patients have an unfavorable prognosis with standard conditioning approaches.\n\nParticipants will receive a standard conditioning regimen consisting of either myeloablative or reduced-intensity chemotherapy, selected according to age and comorbidities, combined with proton TMI delivered at a total dose of 12 Gy in three fractions. Graft-versus-host disease (GvHD) prophylaxis will be administered according to institutional standards, preferentially using post-transplant cyclophosphamide. Patients will subsequently undergo standard allo-HSCT and will be followed for at least 24 months after transplantation.\n\nThe primary objective of the study is to assess the safety and tolerability of proton TMI added to standard conditioning, as measured by non-relapse mortality and treatment-related toxicity within the first 100 days after transplantation. Secondary objectives include evaluation of engraftment kinetics, incidence of relapse, overall and relapse-free survival, GvHD outcomes, and quality of life. Study outcomes will be analyzed descriptively and compared with a matched historical cohort.",[29,30,31,32,33],"Acute Myeloid Leukemia (AML)","Myelodysplastic Syndrome (MDS)\u002FAML","Proton Therapy","MDS and AML Prior to Allogeneic SCT","Myelodysplastic Neoplasm",[35,36,37,31,38,39,40],"High-Risk Hematologic Malignancy","Total Marrow Irradiation","TMI","Conditioning Regimen","Targeted Radiotherapy","HSCT","RECRUITING","2026-04-09",{"date":44,"type":45},"2026-04-16","ACTUAL",{"date":47,"type":45},"2025-11-21",{"date":49,"type":22},"2029-11",{"name":51,"class":52},"Institute of Hematology and Blood Transfusion, Czech Republic","OTHER",2,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":4,"eligibilityCriteria":60,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":61,"targetDuration":4,"studyType":23,"phases":63,"briefSummary":64,"conditions":65,"keywords":70,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":76,"lastUpdatePostDateStruct":77,"startDateStruct":79,"completionDateStruct":81,"leadSponsor":83,"locationsCount":85},"100575892","phase-2-oral-ato-for-tp53-mutated-myeloid-malignancies-100575892","NCT06778187","Oral-ATO for TP53-mutated Myeloid Malignancies","A Phase 2 Study of Oral Arsenic Trioxide (Arsenol ®)-Based Low-intensity Treatment for Previously Untreated or Relapsed\u002FRefractory TP53-mutated Myeloid Malignancies","Inclusion Criteria:\n\n1. Willing and able to provide informed consent\n2. Age ≥18 years\n3. Diagnosis of acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS) or chronic myelonocytic leukaemia (CMML) by World Health Organization (WHO) 2022 criteria (1, 3)\n4. Presence of TP53 mutation\n5. Previously untreated patients for Cohort A (Treatment-naïve), or Patients failing 1 or more lines of prior treatment for Cohort B (Relapsed and Refractory)\n6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2\n7. Women of childbearing potential and fertile men must agree to use an approved method of contraception from Screening until 30 days after the last dose of oral arsenic trioxide, ascorbic acid, venetoclax and azacitidine\u002Fdecitabine\u002Foral-decitabine-cedazuridine.\n\nExclusion Criteria:\n\nInclusion Criteria\n\n1. Willing and able to provide informed consent\n2. Age ≥18 years\n3. Diagnosis of acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS) or chronic myelonocytic leukaemia (CMML) by World Health Organization (WHO) 2022 criteria (1, 3)\n4. Presence of TP53 mutation\n5. Previously untreated patients for Cohort A (Treatment-naïve), or Patients failing 1 or more lines of prior treatment for Cohort B (Relapsed and Refractory)\n6. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2\n7. Women of childbearing potential and fertile men must agree to use an approved method of contraception from Screening until 30 days after the last dose of oral arsenic trioxide, ascorbic acid, venetoclax and azacitidine\u002Fdecitabine\u002Foral-decitabine-cedazuridine.\n\nExclusion Criteria\n\n1. Use of an investigational agent within 14 days of study treatment (or at least 7 half-lives of that agent, whichever is longer), prior to the first dose of oral arsenic trioxide\n2. Known hypersensitivity to arsenic trioxide, ascorbic acid, venetoclax or azacitidine\u002Fdecitabine\u002Foral-decitabine-cedazuridine or their excipients.\n3. Uncontrolled, active infection\n4. Major surgery within 4 weeks of starting the study drug, or not recovered from side effects of surgery\n5. Any other serious medical conditions that could compromise study participation, in the opinion of the investigator\n6. Known HIV infection or known, active hepatitis B or hepatitis C infection\n7. Concurrent second active and non-stable malignancy (patients with a concurrent second active but stable malignancy, i.e., non-melanoma skin cancers, are eligible)\n8. Known history of long QT syndrome (LQTS) or corrected QT interval by Fridericia formula (QTcF) ≥ 480 ms\n9. Evidence at the time of Screening of significant renal or hepatic insufficiency (unless due to hemolysis) as defined by any of the following local laboratory parameters:\n\n   1. Calculated glomerular filtration rate (GFR; using the Cockcroft-Gault equation or eGFR; using CKD-EPI) \\\u003C 40 mL\u002Fmin\n   2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥ 3 x the local upper limit of normal\n10. Pregnant or lactating females, or females planning to become pregnant at any time during the study\n11. Unwilling or unable to comply with the study protocol",{"count":62,"type":22},30,[26],"This is an open-label, phase 2 study of oral arsenic trioxide (Arsenol ®) in combination with ascorbic acid and investigator choice of low-intensity therapy in patients with previously untreated or relapse\u002Frefractory TP53-mutated acute myeloid leukemia (AML), myelodysplastic neoplasm (MDS), chronic myelomonocytic leukemia (CMML).",[66,33,67,68,69],"Acute Myeloid Leukemia","Chronic Myelomonocytic Leukemia","TP53 Gene Mutation","Arsenic Trioxide",[71,72,73,74,75],"Acute myeloid leukemia","Myelodysplastic neoplasm","Chronic myelomonocytic leukemia","TP53 mutation","Oral arsenic trioxide","2025-06-05",{"date":78,"type":45},"2025-06-10",{"date":80,"type":45},"2025-02-07",{"date":82,"type":22},"2028-02",{"name":84,"class":52},"The University of Hong Kong",1,{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":93,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":94,"targetDuration":4,"studyType":23,"phases":96,"briefSummary":97,"conditions":98,"keywords":102,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":85},"100419455","phase-2-repurposing-metformin-as-a-leukemia-preventive-drug-in-ccus-and-lr-mds-100419455","NCT04741945","Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS","STOP-LEUKEMIA: Repurposing Metformin As a Leukemia-preventive Drug in CCUS and LR-MDS","Patients are eligible to be included in WP1 if they meet all of the following criteria:\n\nInclusion criteria:\n\n* A diagnosis of:\n\n  * LR-MDS according to the revised international prognostic scoring system (IPSS-R), i.e., very low- or low-risk disease (IPSS-R score ≤3) in addition to a bone marrow blast percentage \\\u003C5 OR\n  * CCUS defined as the presence of somatic mutation(s) or cytogenetic abnormality not diagnostic of MDS or any other malignancy in the context of persistent cytopenia (\\>6 months) with other common causes of cytopenia ruled out in the setting of bone marrow morphology that is not diagnostic of MDS or any other malignancy, and hematolytic conditions have been ruled out. Peripheral blood cytopenia is defined as hemoglobin (hgb) \\\u003C11.3 g\u002FdL (7 mmol\u002FL) in women and hgb \\\u003C12.9 g\u002FdL (8 mmol\u002FL) in men, platelet count \\\u003C150 x 109\u002FL, or neutrophil count \\\u003C1.8 x 109\u002FL\n* Menopause, if being a female, defined as females \\>45 years of age who have experienced amenorrhea for minimum 12 months, without any other obvious pathological or physiological cause\n* ≥18 years of age\n* Written informed consent\n* Willingness to comply with mandatory aspects of the protocol\n* Ability to swallow pills\n\nExclusion criteria:\n\n* Any prior treatment with metformin\n* A diagnosis of diabetes mellitus\n* Therapeutic radiation, immunosuppressive therapy (with the exception of corticosteroids), or chemotherapy within the past year\n* Treatment with granulocyte colony-stimulating factor within the past 30 days\n* Prior therapy with hypomethylating agents (i.e., azacitidine, decitabine)\n* eGFR \\\u003C45 mL\u002Fmin\n* Performance status according to the Eastern Cooperative Oncology Group \\>2\n* Other active malignancy within the past five years\n* Uncontrolled comorbidity including impaired hepatic function (total serum bilirubin \\>1.5 × upper limit of the normal range (ULN), serum alanine transaminase \\>3 × ULN), chronic hepatitis with decompensated cirrhosis, disabling psychiatric disease, severe neurologic disease, uncontrolled metabolic disease, or severe cardiac disease (NYHA class 3-4)\n\nAn eGFR calculation performed up to one month prior to inclusion may be used to assess renal function. If such an assessment is not available, it is performed at screening.\n\nHealthy volunteers are eligible to be included in WP0 if they meet all of the following criteria:\n\nInclusion criteria:\n\n* Healthy individuals matched on age, sex, and BMI, if possible, to individual patient participants in WP1\n* Written informed consent\n* Willingness to comply with mandatory aspects of the protocol\n\nExclusion criteria:\n\n* Use of metformin within the past 3 years\n* A diagnosis of diabetes mellitus, rheumatological disorders, autoimmune diseases or other inflammatory disorders, celiac disease, inflammatory bowel disease, or other gastrointestinal disorders or symptoms\n* Treatment with immunosuppressive drugs (with the exception of corticosteroids) or chemotherapy within the past year or antibiotics within the past 6 months\n* Any contraindications to MRS",true,{"count":95,"type":22},40,[26],"This is a single-arm pilot study of the feasibility and safety of metformin in patients with clonal cytopenia of undetermined significance (CCUS) or lower-risk myelodysplastic neoplasms (LR-MDS).",[99,33,100,101],"Preleukemia","Cytopenia","Preleukemic Anemia",[103,33,104,105,106,107,108,109],"Metformin","Clonal Cytopenia of Undetermined Significance","Safety","Feasibility","Bone Marrow Adipose Tissue","Gut Microbiota","Intestinal Permeability","2024-11-19",{"date":112,"type":45},"2024-11-22",{"date":114,"type":45},"2021-12-13",{"date":116,"type":22},"2026-02",{"name":118,"class":52},"Kirsten Grønbæk",{"id":120,"slug":121,"hasResults":11,"nctId":122,"briefTitle":123,"officialTitle":124,"acronym":4,"eligibilityCriteria":125,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":126,"enrollmentInfo":127,"targetDuration":4,"studyType":129,"phases":4,"briefSummary":130,"conditions":131,"keywords":4,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":139,"leadSponsor":141,"locationsCount":85},"100524659","intensive-conditioning-withthibufluara-c-in-allo-hsct-for-myeloid-malignancies-with-extramedullary-involvement-100524659","NCT06111612","Intensive Conditioning withTHI\u002FBu\u002FFlu\u002FAra-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement","Intensive Conditioning Regimen With Thiotepa Combined With Busulfan, Fludarabine and Cytarabine for Allogeneic Hematopoietic Stem Cell Transplantation in the Treatment of Myeloid Malignancies With Extramedullary Involvement","Inclusion Criteria:\n\n1. Age between 18 and less than 55 years, regardless of gender.\n2. Criteria for myeloid tumors with extramedullary involvement:\n\n   1. AML (Acute Myeloid Leukemia) with at least one extramedullary lesion achieving hematological remission (CR1 or CR2) after induction therapy.\n   2. MDS (Myelodysplastic Syndrome) with at least one extramedullary lesion and bone marrow blast percentage ≥ 5% achieving hematological CR after treatment; CMML (Chronic Myelomonocytic Leukemia) with at least one extramedullary lesion (diagnosed according to WHO standards) achieving hematological CR after treatment.\n   3. Control and remission of extramedullary lesions, including those in the central nervous system, testes, skin, and other extramedullary tissues.\n   4. Granulocytic sarcoma with or without bone marrow involvement, and achieving remission after treatment.\n3. Patients must have a suitable hematopoietic stem cell donor:\n\n   1. Related donors must have at least 5\u002F10 matches for HLA-A, -B, -C, -DQB1, and - DRB1.\n   2. Unrelated donors must have at least 8\u002F10 matches for HLA-A, -B, -C, -DQB1, and\n\n      * DRB1.\n4. Hematopoietic cell transplantation comorbidity index (HCT-CI) score ≤ 2.\n5. ECOG (Eastern Cooperative Oncology Group) performance status: 0-2.\n6. Adequate liver, kidney, and cardiopulmonary function, meeting the following requirements:\n\n   1. Serum creatinine ≤ 1.5x ULN (the upper limit of normal).\n   2. Cardiac function: Ejection fraction ≥ 50%.\n   3. Baseline oxygen saturation \\> 92%.\n   4. Total bilirubin ≤ 1.5 x ULN; ALT and AST ≤ 2.0 x ULN.\n   5. Pulmonary function: DLCO (corrected for hemoglobin) ≥ 40% and FEV1 (Forced Expiratory Volume in 1 second) ≥ 50%.\n7. Patients must have the ability to understand and be willing to participate in this study and sign an informed consent form.\n\nExclusion Criteria:\n\n1. History of malignancies other than myeloid tumors within the 5 years prior to screening, except for adequately treated in situ cervical cancer, basal cell carcinoma, squamous cell carcinoma of the skin, and curatively treated localized prostate cancer or ductal carcinoma in situ.\n2. ECOG \\> 2.\n3. HCT-CI score ≥ 3.\n4. Any unstable systemic diseases, including but not limited to unstable angina, recent cerebrovascular accidents or transient ischemic attacks within the 3 months prior to screening, myocardial infarction within the 3 months prior to screening, congestive heart failure (New York Heart Association \\[NYHA\\] class ≥ III), severe arrhythmias requiring drug treatment after pacemaker implantation, significant liver, kidney, or metabolic diseases, and pulmonary arterial hypertension.\n5. Active, uncontrolled infections, including those associated with hemodynamic instability, new or worsening infection symptoms or signs, new infectious lesions on imaging, or persistent unexplained fever without signs or symptoms of infection.\n6. Conditions requiring treatment such as grade 2 or higher seizures, paralysis, aphasia, recent severe cerebral infarction, severe traumatic brain injury, dementia, Parkinson's disease, or schizophrenia.\n7. HIV-infected individuals.\n8. Active hepatitis B (HBV) or active hepatitis C (HCV) requiring antiviral therapy.\n\n   Patients at risk of HBV reactivation, are defined as those who are positive for hepatitis B surface antigen or core antibody without receiving antiviral therapy.\n9. Pregnant or breastfeeding women.\n10. Fertile males and females unwilling to use contraception during the treatment period and for 12 months after treatment.","55 Years",{"count":128,"type":22},50,"OBSERVATIONAL","This study is a multicenter, single-arm, prospective phase II clinical trial that evaluates the efficacy and safety of an intensive conditioning regimen with thiotepa combined with busulfan, fludarabine, and cytarabine for allogeneic hematopoietic stem cell transplantation in the treatment of myeloid malignancies with extramedullary involvement. The conditioning regimen includes thiotepa at a dose of 5mg\u002Fkg\u002Fd from d -9 to d -8 (2 days), fludarabine at 30mg\u002Fm2\u002Fd from d -7 to d -3 (5 days), cytarabine at 1-1.5g\u002Fm2\u002Fd from d -7 to d -3 (5 days), and busulfan at 3.2mg\u002Fkg\u002Fd from d -5 to d -3 (3 days). Conditioning begins on day -9, and donor hematopoietic stem cell infusion is performed on day 0. All patients will undergo bone marrow examination on day 14 and day 28 post-transplant, followed by bone marrow examinations every 30 days within the first year after transplantation, and every 60 days within the second year after transplantation. If disease relapse is suspected during the follow-up period, bone marrow or extramedullary relapse site examinations will be conducted at any time. The primary study endpoints are the 1-year and 2-year progression-free survival (PFS) rates post-transplant. Secondary study endpoints include the incidence of acute graft-versus-host disease (GVHD) within 180 days post-transplant, cumulative relapse rates at 1 year and 2 years post-transplant, 1-year and 2-year overall survival (OS), graft-versus-host disease-free, relapse-free survival (GRFS), non-relapse mortality (NRM), cumulative incidence of chronic GVHD, and the incidence of Cytomegalovirus （CMV）and Epstein-Barr virus（EBV）reactivation within 1 year.",[66,33,67,132,133],"Myeloid Sarcoma","Extramedullary Myeloid Tumor","2024-07-19",{"date":136,"type":45},"2024-07-22",{"date":138,"type":45},"2024-01-20",{"date":140,"type":22},"2027-01-01",{"name":142,"class":52},"Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine"]