[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"myeloid-malignancies\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:myeloid-malignancies":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,42,61,93,121,140,164],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":41},"100545566","phase-1-phase-iii-study-of-engineered-t-cell-receptor-modified-nk-cells-targeting-prame-in-conjunction-with-lymphodepleting-chemotherapy-for-the-management-of-relapserefractory-myeloid-malignancies-100545566",false,"NCT06383572","Phase I\u002FII Study of Engineered T Cell Receptor-Modified NK Cells Targeting PRAME in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapse\u002FRefractory Myeloid Malignancies","Inclusion Criteria:\n\n1. 18- 80 years of age. English and non-English speaking patients are eligible.\n2. Patients with one of the following hematological malignances: AML, MDS\u002FCMML. Patients must meet disease specific eligibility criteria (see below)\n3. Patients at least 7 days from last cytotoxic chemotherapy at the time of starting lymphodepleting chemotherapy, except for Hydroxyurea which is allowed for peripheral blood count control in AML patients until the day prior to administration of lymphodepleting chemotherapy. Patients may continue tyrosine kinase inhibitors or other targeted therapies until up to three days prior to administration of lymphodepleting chemotherapy.\n4. Localized radiotherapy to one or more disease sites is allowed prior the infusion provided that there are additional disease sites that are not irradiated to assess response.\n5. Karnofsky Performance Scale \\> 50%.\n6. Adequate organ function: as described in 7-10\n7. Renal: Serum creatinine \\\u003C\u002F= 2.0 mg\u002FdL and estimated Glomerular Filtration Rate (eGFR using the CKD-EPI equation).GFR = 141 x \\[min(Scr\u002Fk), 1)a x max (Scr\u002Fk), 1) -1.209\\] x Age-0.993 x 1.018 \\[if female\\] x \\[ 1.157 if Black\\] (a is 0.329 for females and 0.411 for males; min indicates minimum of Scr\u002Fk or 1, and max indicates maximum of Scr\u002Fk or 1)\n8. Hepatic: ALT\u002FAST \\\u003C\u002F= 2.5 x ULN or \\\u003C\u002F= 5 x ULN if documented liver metastases, Total bilirubin \\\u003C\u002F= 1.5 mg\u002FdL, except in subjects with Gilbert's Syndrome in whom total bilirubin must be \\\u003C\u002F= 3.0 mg\u002FdL. No history of liver cirrhosis. No ascites.\n9. Cardiac: Cardiac ejection fraction \\>\u002F= 40%, no clinically significant pericardial effusion as determined by an ECHO\u002FMUGA, and no uncontrolled arrhythmias or symptomatic cardiac disease.\n10. Pulmonary: No clinically significant pleural effusion (per PI discretion), baseline oxygen saturation \\> 92% on room air and adequate pulmonary function with FEV1, FVC and DLCO (corrected for Hgb) \\>50%.\n11. Able to provide written informed consent.\n12. All participants who are able to have children must practice effective birth control while on study and up to 3 months post completion of study therapy. Acceptable forms of birth control for female patients include: hormonal birth control, intrauterine device, diaphragm with spermicide, condom with spermicide, or abstinence, for the length of the study. If the participant is a female and becomes pregnant or suspects pregnancy, she must immediately notify her doctor. If the participant becomes pregnant during this study, she will be taken off this study.\n\n    a. Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject\u002FPartner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physicaian immediately.\n13. Men treated or enrolled on this protocol must also agree to use adequate contraception, such as subject post vasectomy, partner with implantable or oninjectable contraceptives, and condoms plus spermicide, prior to the study, for the duration of study participation, and 4 months after completion of study agent administration. Men who are able to have children must use effective birth control while on the study. If the male participant fathers a child or suspects that he has fathered a child while on the study, he must immediately notify his doctor.\n14. Signed consent to long-term follow-up protocol PA17-0483 to fulfill the institutional responsibilities to various regulatory agencies.\n15. Life Expectancy \\>=3 mo, by PI assessment\n16. Patients are HLA-A\\*02:01 positive on HLA typing.\n17. Patients must have one of the following diseases:\n\nAcute myeloid leukemia (AML): one of the following:\n\n1\\. Patients with active (\\>5% of blasts or positive MRD at a level of \\>0.1% measured by multiparameter flow cytometry) relapsed or refractory AML. Who have received at least two lines of therapy. One or more of the lines of therapy must include hypomethylating agents and venetoclax. Patients who have mutations for which there are FDA approved targeted therapies (i.e. FLT3) must also have received at least one of such agent. Patients with diagnosis of acute promyelocytic leukemia are not eligible. Patients who relapse after allogeneic stem cell transplantation are eligible irrespective of the number and type of therapy received prior transplant.\n\n1. Relapsed AML is defined as patients who had a first complete remission (CR) before developing recurrent disease.\n2. Refractory AML is defined as patients that have not achieved a CR after 2 cycles of standard induction chemotherapy.\n\nOR determining the eligibility of the patient. For example, patients who received hypomethylating agents and venetoclax for the treatment of the MDS are not required to receive again hypomethylating agents and venetoclax to treat the secondary AML to be eligible. Similarly, patients who received two lines of therapy for the management of the MDS or MPD are not required to receive an additional two lines of therapy for the management of the AML to be eligible. Patients who relapse after allogeneic stem cell transplantation are eligible irrespective of the number and type of therapy received prior transplant.\n\nMyelodysplastic syndromes (MDS)\u002FChronic myelomonocytic leukemia (CMML):one of the following:\n\n1. Patients with high risk or intermediate risk MDS\u002FCMML who have received at least two lines of therapy and have positive MRD at a level of \\>0.1% measured by multiparameter flow cytometry or are not in morphological remission.\n\n   OR\n2. Patients with relapse high risk or intermediate risk MDS\u002FCMML after at least 2 lines of therapy. Relapse is defined as positive MRD at a level of \\>0.1% measured by multiparameter flow cytometry or loss of morphological remission.\n\n   OR\n3. CMML patients with refractory disease defined as failure to achieve complete remission after 4 therapy cycles of hypomethylating agent therapy or relapse or progression after any number of cycles of therapy.\n4. Patients with MDS\u002FCMML who relapse after allogeneic stem cell transplantation are eligible irrespective of the number and type of therapy received prior to transplant.\n\nExclusion Criteria:\n\n1. Positive beta HCG in female of child-bearing potential defined as not postmenopausal for 24 months or no previous surgical sterilization or lactating females.\n2. Presence of clinically significant Grade 3 or greater toxicity from the previous treatment, as determined by PI.\n3. Presence of uncontrolled fungal, bacterial, viral, or other infection not responding to appropriate therapy.\n4. Known active hepatitis B or C.\n5. Known HIV with detectable viral load\n6. Presence of active neurological disorder(s).\n7. Active autoimmune disease within 12 months of enrollment\n8. Active cerebral or meningeal involvement by the malignancy\n9. Active (defined as requiring therapy) acute or chronic GVHD\n10. Any other malignancy known to be active, except for treated cervical intra-epithelial neoplasia and non-melanoma skin cancer.\n11. Presence of any other serious medical condition that may endanger the patient at investigator discretion.\n12. Major surgery \\\u003C4 weeks prior to first dose of the preparatory chemotherapy\n13. Allogeneic SCT or DLI \\\u003C12 weeks prior to first dose of preparatory chemotherapy\n14. Concomitant use of other investigational agents.\n15. Concomitant use of other anti-cancer agents.\n16. Patients receiving systemic steroid therapy at time of enrollment (physiological substitutive doses deemed per PI assessment are allowed),or have received antithymocyte globulin or lymphocyte immune globulin within 14 days of enrollment or alemtuzumab within 28 days of enrollment.\n17. Patients receiving immunosuppressive therapy such as steroids and calcineurin inhibitors.\n\nCriteria to Receive Lymphodepletion Chemotherapy:\n\nWithin 72 hours prior to the start of Lymphodepletion chemotherapy, subjects must meet the eligibility criteria outlined below:\n\n1. No uncontrolled active systemic infection. Subjects must be fever free for at least 48 hours without antipyretics unless the patient is felt to have tumor fever.\n2. Absence of clinically significant Grade 3 or greater toxicity from the previous treatment, as determined by the Principal Investigator.\n3. Patient should not be receiving systemic steroids above physiologic dosing (excluding dexamethasone 25mg\u002Fm\\^2 PO as noted in the treatment plan from Day -10 through Day -6.) or calcineurin inhibitors.\n4. Patients should be at least 7 days from last cytotoxic chemotherapy at the time of starting lymphodepleting chemotherapy, except for Hydroxyurea which is allowed for peripheral blood count control in AML patients until the day prior to administration of lymphodepleting chemotherapy.\n5. Patients may continue tyrosine kinase inhibitors or other targeted therapies until up to three days prior to administration of lymphodepleting chemotherapy\n\nCriteria Prior to NK Cell Infusion:\n\nOn Day 0, prior to the NK Cell infusion, subjects must meet the eligibility criteria for infusion outlined\n\n1. No active and uncontrolled systemic infection. Subjects must be fever free for 48 hours without antipyretic unless patient is felt to have tumor fever.\n2. No clinical signs of cardiac failure.\n3. Oxygen saturation levels (SPO2) \\>92%\n4. No other ongoing medical condition that in the opinion of the investigator can affect patient's safety.\n5. No rapidly progressing AML\u002FMDS.\n6. Patient must be able to be off of all steroid treatment within 24hrs of cell infusion.","ALL","18 Years","80 Years",{"count":19,"type":20},44,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","To find a recommended dose of PRAME-TCR-NK cells that can be given to patients with AML or MDS.",[27,28],"Lymphodepleting Chemotherapy","Myeloid Malignancies","RECRUITING","2026-06-10",{"date":32,"type":33},"2026-06-12","ACTUAL",{"date":35,"type":33},"2024-06-26",{"date":37,"type":20},"2029-04-01",{"name":39,"class":40},"M.D. Anderson Cancer Center","OTHER",1,{"id":43,"slug":44,"hasResults":11,"nctId":45,"briefTitle":46,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":48,"targetDuration":4,"studyType":21,"phases":50,"briefSummary":51,"conditions":52,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":53,"lastUpdatePostDateStruct":54,"startDateStruct":56,"completionDateStruct":58,"leadSponsor":60,"locationsCount":41},"100587612","phase-1-a-phase-iii-study-of-car70-engineered-il15-transduced-cord-blood-derived-nk-cells-with-tgf-beta-receptor-2-tgfbr2-knock-out-in-conjunction-with-lymphodepleting-chemotherapy-for-the-management-of-relapsedrefractory-myeloid-malignancies-100587612","NCT06930651","A Phase I\u002FII Study of CAR.70-Engineered IL15-Transduced Cord Blood-Derived NK Cells With TGF-beta Receptor 2 (TGFBR2) Knock Out in Conjunction With Lymphodepleting Chemotherapy for the Management of Relapsed\u002FRefractory Myeloid Malignancies","Inclusion Criteria:\n\n1. Diagnosis: Age 18-80 years with diagnosis of:\n\n   1. Relapsed or refractory AML or \"treated secondary AML\"\n\n      * Patients with a mutation that is targetable with an FDA-approved targeted therapy should have received at least one on these agents. . \"Treated secondary AML \"includes patients with prior diagnosis of a myeloid neoplasm (e.g. MDS) who received hypomethylating agents for this disease and subsequently progressed to AML. These patients must have received all of the following: a hypomethylating agent + venetoclax and intensive chemotherapy (if a suitable candidate for intensive therapy). These patients may be enrolled at the time of AML diagnosis if they have already received all of the treatments above for their antecedent myeloid neoplasm.\n   2. MDS that is intermediate, high-risk or very-high risk by the Revised International Prognostic Scoring System (R-IPSS)\n\n      * Bone marrow blasts must be \\>5%.\n      * The disease must have either 1.) not have responded to at least 4 cycles of a hypomethylating agent or 2.) progressed or relapsed, regardless of the number of cycles received\n   3. CMML-1 or CMML-2\n\n      * Bone marrow blasts must be \\>5%.\n      * The disease must have either 1.) not have responded to at least 4 cycles of a hypomethylating agent or 2.) progressed or relapsed, regardless of the number of cycles received\n2. CD70 expression \\>10% measured by immunohistochemistry or multiparameter flow cytometry\n3. Performance status \\\u003C\u002F=2 (ECOG Scale)\n4. Adequate liver, cardiac, renal and pulmonary function as defined by the following criteria:\n\n   1. Total serum bilirubin \\\u003C\u002F=2 x upper limit of normal (ULN), unless due to Gilbert's syndrome, hemolysis or the underlying leukemia approved by the PI\n   2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\\u003C\u002F=3 x ULN, unless due to the underlying leukemia approved by the PI\n   3. Serum creatinine \\\u003C\u002F=2x ULN or creatinine clearance \\>\u002F=30 mL\u002Fmin\n   4. Left ventricular ejection fraction \\>\u002F=40% by echocardiogram or MUGA\n   5. Oxygen saturation \\>\u002F=93% on room air\n5. Ability to understand and the willingness to sign a written informed consent document\n6. Willingness to sign informed consent to long-term follow-up on protocol PA17-0483 to fulfill institutional responsibilities to regulatory agencies\n7. Willingness to use adequate contraception prior to study entry, for the duration of study participation, and for 3 months after completion of study participation. For women of childbearing potential, adequate methods of contraception include: complete abstinence,, hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), intrauterine device (IUD), tubal Ligation or hysterectomy, subject\u002Fpartner post vasectomy, implantable or injectable contraceptives, and condoms plus spermicide.\n\nExclusion Criteria:\n\n1. Active grade III-V cardiac failure as defined by the New York Heart Association Criteria\n2. Active serious infection not controlled by oral or intravenous antibiotics (e.g. persistent fever or lack of improvement despite antimicrobial treatment).\n3. Active central nervous system leukemia\n4. Known human immunodeficiency virus (HIV) seropositive, unless well-controlled on stable doses of anti-retroviral therapy.\n5. Known hepatitis B surface antigen seropositive or known or suspected active hepatitis C infection Note: Patients who have isolated positive hepatitis B core antibody (ie, in the setting of negative hepatitis B surface antigen and negative hepatitis B surface antibody) must have an undetectable hepatitis B viral load. Patients who have positive hepatitis C antibody may be included if they have an undetectable hepatitis C viral load.\n6. Patients with a prior or concurrent malignancy whose natural history or treatment is not anticipated to interfere with the safety or efficacy assessment of the investigational regimen may be included only after discussion with the PI\n7. Use of calcineurin inhibitors (e.g. tacrolimus) within the past 2 weeks\n8. Treatment with any investigational antileukemic agents or chemotherapy agents in the last 7 days before lymphodepletion, unless full recovery from side effects has occurred or patient has rapidly progressive disease judged to be life-threatening by the investigator.\n\n   i. Prior recent treatment with corticosteroids, hydroxyurea, and\u002For cytarabine (up to 2 g\u002Fm2 given for cytoreduction within the preceding 7 days) is permitted up until 1 day prior to lymphodepletion.\n\n   • Patients may continue on non-investigational targeted therapies up until 3 days prior to lymphodepletion.\n9. Pregnant or breastfeeding women will not be eligible",{"count":49,"type":20},42,[23,24],"The goal of this clinical research study is to find the recommended safe dose of TGFBR2 KO CAR27\u002FIL-15 NK cells that can be given to patients with relapsed\u002Frefractory disease. The safety and effectiveness of this treatment will also be studied.",[28],"2026-05-05",{"date":55,"type":33},"2026-05-06",{"date":57,"type":33},"2025-09-03",{"date":59,"type":20},"2030-05-01",{"name":39,"class":40},{"id":62,"slug":63,"hasResults":11,"nctId":64,"briefTitle":65,"officialTitle":65,"acronym":66,"eligibilityCriteria":67,"healthyVolunteers":11,"sex":15,"minAge":68,"maxAge":69,"enrollmentInfo":70,"targetDuration":4,"studyType":21,"phases":72,"briefSummary":73,"conditions":74,"keywords":81,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":83,"lastUpdatePostDateStruct":84,"startDateStruct":86,"completionDateStruct":88,"leadSponsor":90,"locationsCount":92},"100500462","phase-2-a-pilot-study-to-evaluate-the-feasibility-of-post-hematopoietic-stem-cell-transplant-prophylaxis-with-decitabine-combined-with-filgrastim-for-children-and-young-adults-with-aml-mds-and-related-myeloid-malignancies-100500462","NCT05796570","A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies","MORE","Inclusion Criteria:\n\n* Disease Criteria: Participants must have a histologically confirmed diagnosis of one of the following hematologic malignancies for eligibility, as defined by the criteria below:\n\n  * AML (relapsed, de-novo or secondary) based on WHO classification\n  * MDS (relapsed, de-novo or secondary) based on WHO classification\n  * Treatment myeloid neoplasm (tMDS\u002FAML; relapsed disease included)\n  * Myeloid Sarcoma\n  * Acute Undifferentiated Leukemia (MPAL and acute leukemia of ambiguous lineage\u002FNOS not eligible)\n* Note: MDS, AML, MDS\u002FAML, or tMDS\u002FAML as defined above may be idiopathic\u002Fde novo or derived from a germline predisposition to myeloid malignancy. For patients with an underlying germline disorder, those conditions that are not associated with increased risk for toxicity to treatment, including patients with known germline ANKRD26, DDX41, ELANE and other congenital neutropenia disorders, ETV6, GATA-2, Li-Fraumeni, RUNX1, SAMD9\u002FSAMD9L, or Shwachman-Diamond Syndrome, will be analyzed within the general treatment cohort (Cohort A, see Table 1) along with patients with idiopathic disease (Cohort B, see Table 2).\n\nMDS, AML, MDS\u002FAML, or tMDS\u002FAML derived from the following germline disorders will be enrolled in a separate cohort (B) and adverse events monitored closely for higher rates compared to cohort A:\n\n* Dyskeratosis Congenita or associated telomeropathies as defined by telomere length \\\u003C1st percentile on 3 out of 4 lymphocyte subsets and\u002For corresponding pathogenic genetic mutation.\n* Fanconi Anemia as defined by positive chromosomal breakage test to DEB\u002FMMC and\u002For corresponding pathogenic genetic mutation.\n* Nijmegen Breakage Syndrome as defined by positive chromosomal breakage test to DEB\u002FMMC and\u002For corresponding pathogenic genetic mutation\n* ERCC6L2 by genomic testing.\n\nTable 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)\n\n-iBMF with Standard risk for Treatment Related toxicities:\n\n* germline mutations in ANKRD26\n* germline mutations in DDX41\n* ELANE and other Congenital Neutropenia Disorders\n* germline mutations in ETV6\n* germline mutations in GATA-2\n* Li-Fraumeni\n* germline mutations in RUNX1\n* SAMD9\u002FSAMD9L\n* Shwachman-Diamond Syndrome\n* Familial MDS with thrombocytopenia\n* Diamond-Blackfan Anemia\n\nTable 2: Overview Inherited Bone Marrow Failure syndromes (iBMF)\n\n* iBMF with Increased Risk for Treatment Related Toxicities:\n\n  * Fanconi Anemia\n  * Dyskeratosis Congenita and associated Telomere Disorders\n  * Nijmegen Breakage Syndrome\n  * ERCC6L2\n* Patients must be receiving an allogeneic hematopoietic stem cell transplant. All donor types and graft sources are permitted. All conditioning regimens are permitted. All GVHD prophylaxis regimens are permitted.\n* Timing of Enrollment: Registration can occur from day - 40 to day - 1 prior to stem cell infusion.\n* Disease Status: Study enrollment will occur pre HCT. Any disease status is acceptable at the time of enrollment; however, patients must be in a MRD negative remission (as defined by multidimensional flow cytometry (MDF) post HCT prior to protocol treatment start). Post HCT\u002F pretreatment disease status will be performed by Hematologics.\n* No limitations on prior therapy.\n* Age ≥1 year and ≤ 39 year of age.\n* ECOG performance status ≤2 (Lansky, Karnofsky ≥60%).\n* Participants must have adequate organ function to be eligible for allogenic HCT as per institutional standard.\n* Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial. Anti- retroviral therapy must not have a non-acceptable drug interaction with protocol treatment.\n* For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. Suppressive therapy must not have a non-acceptable drug interaction with protocol treatment.\n* Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. Ongoing antiviral therapy must not have a non-acceptable drug interaction with protocol treatment.\n* Participants with a malignancy in remission are eligible for this trial.\n* Participants with known history or current symptoms of cardiac disease should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better.\n* The effects of filgrastim on the developing human fetus are unknown. For this reason and because decitabine is known to be teratogenic, 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 she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of decitabine administration.\n* Ability to understand and the willingness to sign a written informed consent document.\n\nExclusion Criteria:\n\n* Participants who have not recovered from adverse events due to prior anti-cancer therapy (i.e., have residual toxicities \\> Grade 2) except for bone marrow suppression.\n* Participants should not be enrolled on another study that prohibits initiation of maintenance therapy.\n* History of allergic reactions attributed to compounds of similar chemical or biologic composition to decitabine or filgrastim.\n* Participants with uncontrolled intercurrent illness.\n* Participant who are not able to present for clinic visits for at least 7 months after study treatment initiation.\n* Participant with FLT3\u002FITD mutations are excluded as maintenance therapy with tyrosine kinase therapy should be considered in this context. However, if a participant has a co-occurring NUP28 mutation, they will be considered eligible.\n* Participants with a concurrent active malignancy are not eligible for this trial.","1 Year","39 Years",{"count":71,"type":20},37,[24],"The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT.\n\nThe names of the study drugs involved in this study are:\n\n* Decitabine (a nucleoside metabolic inhibitor)\n* Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)",[75,76,28,77,78,79,80],"Acute Myeloid Leukemia","Myelodysplastic Syndromes","MDS","Inherited Bone Marrow Failure Syndrome","Myeloid Neoplasm","Aml",[75,76,28,77,78,79,82],"AML","2026-03-17",{"date":85,"type":33},"2026-03-19",{"date":87,"type":33},"2023-04-19",{"date":89,"type":20},"2029-09-01",{"name":91,"class":40},"Franziska Wachter",2,{"id":94,"slug":95,"hasResults":11,"nctId":96,"briefTitle":97,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":15,"minAge":99,"maxAge":100,"enrollmentInfo":101,"targetDuration":4,"studyType":21,"phases":103,"briefSummary":105,"conditions":106,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":112,"lastUpdatePostDateStruct":113,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":120},"100623426","phase-3-fludarabine-plus-melphalan-versus-addition-of-venetoclax-to-fludarabinemelphalan-conditioning-regimen-for-allogeneic-hematopoietic-stem-cell-transplantation-in-amlmds-patients-aged--50-years-a-multicenter-randomized-phase-3-trial-100623426","NCT07396480","Fludarabine Plus Melphalan Versus Addition of Venetoclax to Fludarabine\u002FMelphalan Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation in AML\u002FMDS Patients Aged > 50 Years: a Multicenter, Randomized, Phase 3 Trial","Inclusion Criteria:\n\n1. Aged \\> 50 years;\n2. Confirmed as acute myeloid leukemia (AML) in remission prior to transplantation, myelodysplastic syndrome (MDS; IPSS: Intermediate-2, high; or IPSS-R: Intermediate, high, very high; or IPSS-M: moderate-high, high, and very high), or myelodysplastic syndrome\u002Fmyeloproliferative neoplasm (MDS\u002FMPN) by morphology, immunology, cytogenetics and molecular biology (MICM) typing;\n3. Having an eligible donor and scheduled to undergo allogeneic hematopoietic stem cell transplantation (Allo-HCT) from a related or unrelated donor;\n4. Karnofsky Performance Score ≥ 70;\n5. Eastern Cooperative Oncology Group (ECOG) Performance Status \\\u003C 3;\n6. Expected survival time \\> 12 weeks;\n7. Voluntarily signing the informed consent form and being able to understand and comply with all study requirements.\n\nExclusion Criteria:\n\n1. Complicated with severe cardiac insufficiency with a left ventricular ejection fraction (EF) \\\u003C 60%; or complicated with severe arrhythmia, and assessed by the investigator as unable to tolerate the intensive conditioning regimen;\n2. Complicated with severe pulmonary insufficiency (obstructive and\u002For restrictive ventilatory disorder), and assessed by the investigator as unable to tolerate the intensive conditioning regimen;\n3. Complicated with severe liver function impairment, with liver function indicators (alanine aminotransferase \\[ALT\\], total bilirubin \\[TBIL\\]) exceeding 3 times the upper limit of normal (ULN); and assessed by the investigator as unable to tolerate the intensive conditioning regimen;\n4. Complicated with severe renal insufficiency, with serum creatinine (Cr) exceeding 2 times the upper limit of normal (ULN); or with a 24-hour creatinine clearance rate (Ccr) \\\u003C 50 ml\u002Fmin, and assessed by the investigator as unable to tolerate the intensive conditioning regimen;\n5. Suffering from severe active infection prior to transplantation, and assessed by the investigator as unable to tolerate the intensive conditioning regimen;\n6. Having a history of allergic reactions or severe adverse reactions to the drugs involved in the conditioning regimen, and assessed by the investigator as ineligible for enrollment;\n7. Other reasons for ineligibility assessed by the investigator.","50 Years","75 Years",{"count":102,"type":20},186,[104],"PHASE3","Allogeneic Hematopoietic Cell Transplantation (Allo-HCT) serves as a curative treatment modality for the vast majority of patients with hematological malignancies. Historically, due to the relatively high treatment-related mortality rate associated with Allo-HCT, this therapy was primarily administered to younger patients. However, the median age at onset of most hematological malignancies falls within the elderly population. For instance, the median ages at onset of Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) are 68 and 77 years, respectively. In recent years, with the advancement of transplantation techniques and the application of Reduced-intensity Conditioning (RIC) regimens, a growing number of elderly patients have undergone Allo-HCT. Data from the Center for International Blood and Marrow Transplant Research (CIBMTR) indicate that in 2017, 31% of patients who received Allo-HCT were aged over 60 years, and 6% were over 70 years old. Over the past decade, the number of elderly patients undergoing Allo-HCT has increased significantly.\n\nGiven that most elderly patients cannot tolerate conventional myeloablative conditioning regimens, RIC regimens based on Fludarabine (Flu) combined with Busulfan (Bu), or Fludarabine (Flu) combined with Melphalan (Mel) are currently widely used in elderly patients undergoing Allo-HCT. Nevertheless, the post-transplant relapse rate remains as high as 30%-55%, and the long-term GVHD-free and Relapse-free Survival (GRFS) rate fluctuates between 21% and 59%, suggesting that the efficacy of transplantation needs to be further improved. Further comparison of the commonly used RIC regimens in elderly patients-namely Flu+Bu (2-day), Flu+Bu (4-day) and Flu+Mel-has demonstrated that the Flu+Mel regimen yields superior transplantation outcomes over the Flu+Bu regimens.\n\nAt present, the optimal RIC regimen for elderly patients with hematological malignancies has not yet been clearly defined. The selection of transplantation conditioning regimens for elderly patients should strike a balance between reducing non-relapse mortality and decreasing post-transplant relapse. Over the past 20 years, an increasing number of targeted drugs acting on specific cellular signaling pathways, anti-apoptotic proteins, epigenetic regulators, and monoclonal antibodies have been introduced into clinical practice, thereby revolutionizing the treatment landscape of hematological malignancies. These novel targeted therapies not only bring hope of achieving remission to patients with hematological tumors resistant to traditional chemotherapy, but also the combined application of novel drugs and Allo-HCT is bound to fundamentally transform the overall technical system of hematopoietic stem cell transplantation. Venetoclax is a potent and selective oral inhibitor targeting the BH3 domain of the anti-apoptotic protein Bcl-2. In 2018, the FDA approved Venetoclax as a first-line induction chemotherapy agent for elderly AML patient's ineligible for intensive chemotherapy, with a complete remission rate of up to 67% and favorable tolerability¹¹. Preclinical studies using Allo-HCT animal models have confirmed that the addition of a Bcl-2 inhibitor to RIC regimens can promote donor cell engraftment, reduce the incidence of GVHD, without impairing the graft-versus-leukemia (GVL) effect¹². In recent years, clinical trials have reported the efficacy and safety of the conditioning regimen combining Venetoclax with Flu+Bu in patients with myeloid malignancies undergoing Allo-HCT. Our research center has demonstrated the favorable safety profile and promising long-term survival outcomes of the Venetoclax plus Flu+Mel conditioning regimen in a phase II clinical trial involving patients aged over 50 years with AML\u002FMDS undergoing Allo-HCT (2024 EBMT Poster B093; 2025 EBMT Poster B126). However, the long-term superiority of this novel regimen over the conventional Flu+Mel conditioning regimen remains to be clarified.\n\nTherefore, based on the existing findings from clinical studies and Allo-HCT animal model research, we hypothesize that incorporating Venetoclax into the Fludarabine+Melphalan conditioning regimen for elderly patients undergoing Allo-HCT is expected to improve long-term post-transplant survival and further enhance the transplantation efficacy in this patient population.",[107,28,108,109,110,111],"Venentoclax","Conditioning","ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION","Acute Myeloid Leucemia","Myeldysplastic Syndrome (MDS)","2026-03-16",{"date":83,"type":33},{"date":115,"type":20},"2026-03-01",{"date":117,"type":20},"2030-06-30",{"name":119,"class":40},"First Affiliated Hospital of Zhejiang University",18,{"id":122,"slug":123,"hasResults":11,"nctId":124,"briefTitle":125,"officialTitle":125,"acronym":4,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":100,"enrollmentInfo":127,"targetDuration":4,"studyType":21,"phases":129,"briefSummary":130,"conditions":131,"keywords":4,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":133,"startDateStruct":135,"completionDateStruct":137,"leadSponsor":139,"locationsCount":41},"100567468","phase-2-a-phase-ii-open-label-study-of-olutasidenib-post-transplant-maintenance-therapy-for-patients-with-idh1-mutated-myeloid-malignancies-100567468","NCT06668584","A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies","Inclusion Criteria:\n\n1. Diagnosis of AML, MDS, MPN, or CMML according to World Health Organization (WHO) classification that underwent first or second alloSCT regardless of stem cell source, donor type\u002Fmatch, conditioning regimen, or GVHD prophylaxis and is at least 30 days post stem cell transplant until day 120.\n2. Evdence of an IDH1 mutation presence by next generation sequencing panel at original diagnosis.\n3. Evidence of engraftment by Absolute neutrophil count (ANC) \\>\u002F= 1.0x 109\u002FL without daily use of myeloid growth factor (G-CSF) for at least 7 days; and Platelet \\>\u002F= 30 x 109\u002FL with out platelet transfusion within 1 week.\n4. Patient in morphologic remission with Day +30 bone marrow \\\u003C5% blast. Will include MRD positive or negative.\n5. Age 18 to 75 years old.\n6. ECOG performance status of 0, 1, 2. Or KPS above 70.\n7. Creatinine clearance greater or equal to 40cc\u002Fmin as defined by the Cockcroft-Gault Equation.\n\n   Males (mL\u002Fmin): (140-age) \\*IBW (kg) \u002F 72\\*(serum creatinine(mg\u002Fdl)) Females (mL\u002Fmin): 0.85\\*(140-age) \\*IBW (kg) \u002F 72\\*(serum creatinine(mg\u002Fdl)).\n8. Serum bilirubin \\\u003C\u002F= 1.5 x upper limit of normal (ULN) except in subjects with Gilbert's Syndrome in whom total bilirubin must be \\\u003C\u002F= 3.0 mg\u002Fdl. Aspartate transaminase (AST) or alanine transaminase (ALT) \\\u003C\u002F= 2.5 x ULN. Alkaline phoshatase \\\u003C\u002F=2.5 x ULN.\n9. No active bleeding.\n10. No clinical evidence of life-threatening infection.\n11. Capable of understanding the investigational nature, potential risks, and benefits of the study, and able to provide valid informed consent.\n12. Negative serum or urine pregancy test for wome with reproductive potential at screening.\n13. Female participants of non-childbearing potential must meet at least one of the following criteria:\n\n    1. Postmenopausal (no menses in greater than or equal to 12 consecutive months).\n    2. History of hysterectomy or bilateral salpingo-oophorectomey.\n    3. Ovarian failure (follicle-stimulating hormone and Estradiolin menopausal range, who have received Whole Pelvic Radiation Therapy).\n    4. History of bilateral tubal ligation or another surgical sterilization procedure\n14. Subjects who are of childbearing potential, sexually active, and at risk of pregnancy must agree to use a highly effective method of contraception for the duration of the active treatment and at least 3 months post-completion of the study therapy. See Appendix B.\n\nApproved methods of birth control are as follows: Hormonal contraception (i.e., birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject\u002FPartner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. See Appendix B.\n\nMen treated or enrolled in this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of Olutasidenib administration.\n\nExclusion Criteria:\n\n1. Use of any of the following after transplantation and prior to starting study therapy. Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous, or oral 5-azacitidine or FL T3 inhibitors for maintenance)\n2. Overall grade II-IV aGVHD. However, upon complete resolution of aGVHD-related symptoms with grade 1 or 0 overall clinical grade would be appropriate to enroll at that time. Patients may be eligible for enrollment if they are on prednisone 0.5 mg\u002Fkg daily dose or lower, tacrolimus, sirolimus, and\u002For ruxolitinib.\n3. cGVHD, moderate or servere by NIH criteria.\n4. Active uncontrolled systemic fungal, bacterial, or viral infection. However, patients receiving anti-micobial agents including antibiotics, antiviral and antifungal therapies are allowed if the infection is controlled, and the patient is hemodynamically stable.\n5. Known active viral infection with Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV).\n\n   * Patients with known active hepatitis B virus (HBV) infection will be excluded because of potential effects on immune function and\u002For drug interactions. However, if a patient has HBV history with an undetectable HBV load by polymerase chain reaction (PCR), no liver-related complications, and is on definitive HBV therapy that is not contraindicated in this study, then they would be eligible for study.\n   * Patients with known active hepatitis C virus (HCV) infection will be excluded because of potential effects on immune function and\u002For drug interactions. However, if a patient with a history of HCV infection has received definitive therapy (and is now HCV viral load negative), or if a patient has a reactive HCV antibody test but has an undetectable viral load by PCR, then they would be eligible.\n   * Patients with known active HIV infection will be excluded out of concern for the drug-drug interaction with venetoclax and highly active antiretroviral therapy (HART)\n6. QT prolongation of corrected QTcF interval (QTc) \\> 480 milliseconds\n7. Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures.\n8. Patients with cognitive impairments or psychiatric disorders that can interfere with safety or with obtaining informed consent of compliance with study procedures.",{"count":128,"type":20},31,[24],"The goal of this clinical research study is to learn about the safety and tolerability of giving olutasidenib to patients with IDH1-mutated myeloid malignancies as maintenance therapy after they receive a stem cell transplant.",[28],"2026-03-02",{"date":134,"type":33},"2026-03-04",{"date":136,"type":33},"2024-12-31",{"date":138,"type":20},"2029-12-31",{"name":39,"class":40},{"id":141,"slug":142,"hasResults":11,"nctId":143,"briefTitle":144,"officialTitle":145,"acronym":4,"eligibilityCriteria":146,"healthyVolunteers":11,"sex":15,"minAge":147,"maxAge":100,"enrollmentInfo":148,"targetDuration":4,"studyType":21,"phases":150,"briefSummary":151,"conditions":152,"keywords":4,"overallStatus":155,"whyStopped":4,"lastUpdateSubmitDate":156,"lastUpdatePostDateStruct":157,"startDateStruct":159,"completionDateStruct":161,"leadSponsor":162,"locationsCount":4},"100596972","phase-2-vendacbu2flu4-vs-bu2flu5-conditioning-regimen-for-elderly-myeloid-malignancies-undergoing-allo-hsct-100596972","NCT07052422","VEN+DAC+Bu2Flu4 vs Bu2Flu5 Conditioning Regimen for Elderly Myeloid Malignancies Undergoing Allo-HSCT","Venetoclax+Decitabine+Busulfan+Fludarabine (VEN+DAC+Bu2Flu4) vs Busulfan+Fludarabine Conditioning Regimen (Bu2Flu5 ) for Older Patients With Myeloid Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT)","Inclusion Criteria:\n\n* 60-75 years\n* Acute myeloid leukaemia in first complete remission or myelodysplastic syndrome\n* Willing to undergo the first allo-HSCT\n* Eastern Cooperative Oncology Group performance status of 0-2\n\nExclusion Criteria:\n\n* Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)\n* Patients with any conditions not suitable for the trial (investigators' decision)","60 Years",{"count":149,"type":20},160,[24,104],"The purpose of this study is to compare the efficacy and safety of venetoclax+decitabine+busulfan+fludarabine (VEN+DAC+Bu2Flu4) regimen with busulfan+fludarabine (Bu2Flu5) regimen in older patients with myeloid malignancies undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT).",[153,28,108,154],"Older Patients","Hematopoietic Stem Cell Transplantation (HSCT)","NOT_YET_RECRUITING","2025-07-01",{"date":158,"type":33},"2025-07-04",{"date":160,"type":20},"2025-07-15",{"date":138,"type":20},{"name":163,"class":40},"Nanfang Hospital, Southern Medical University",{"id":165,"slug":166,"hasResults":11,"nctId":167,"briefTitle":168,"officialTitle":168,"acronym":4,"eligibilityCriteria":169,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":170,"targetDuration":4,"studyType":21,"phases":172,"briefSummary":173,"conditions":174,"keywords":4,"overallStatus":155,"whyStopped":4,"lastUpdateSubmitDate":176,"lastUpdatePostDateStruct":177,"startDateStruct":179,"completionDateStruct":180,"leadSponsor":182,"locationsCount":41},"100586571","phase-1-exploratory-clinical-study-on-the-safety-and-efficacy-of-car-t-cell-therapy-in-the-treatment-of-relapsedrefractory-myeloid-malignancies-100586571","NCT06917105","Exploratory Clinical Study on the Safety and Efficacy of CAR-T Cell Therapy in the Treatment of Relapsed\u002FRefractory Myeloid Malignancies","Inclusion Criteria:\n\n* Informed Consent: Willing and able to provide written informed consent, with commitment to comply with scheduled visits, study treatment, laboratory tests, and other trial procedures.\n\nAge: ≥18 years, regardless of gender.\n\nDiagnosis: Pathologically confirmed myeloid malignancy (including but not limited to AML or MDS) meeting relapsed\u002Frefractory criteria:\n\nRelapsed Disease: Reappearance of leukemic cells in peripheral blood, bone marrow blasts \\>5%, or extramedullary relapse after achieving CR\u002FCRi with ≥2 lines of salvage therapy.\n\nRefractory Disease: Failure to achieve CR\u002FCRi after ≥2 cycles of standard intensive chemotherapy.\n\nAntigen Expression: Tumor cell positivity for CD33, CD123, and\u002For CLL-1 confirmed by immunohistochemistry (IHC) or flow cytometry.\n\nLife Expectancy: ≥3 months from the date of informed consent signing. Hematologic Criteria: Hemoglobin ≥70 g\u002FL (transfusion permitted).\n\nOrgan Function:\n\nRenal: Serum creatinine ≤1.5×ULN. Cardiac: Left ventricular ejection fraction (LVEF) ≥50%. Pulmonary: Oxygen saturation \\>90% on room air. Hepatic: Total bilirubin ≤1.5×ULN; ALT\u002FAST ≤2.5×ULN. Performance Status: Eastern Cooperative Oncology Group (ECOG) performance status score 0-2.\n\nExclusion Criteria:\n\n* Cardiac Dysfunction: Severe cardiac insufficiency with left ventricular ejection fraction (LVEF) \\\u003C50%.\n\nPulmonary Disease: History of severe pulmonary dysfunction (e.g., chronic respiratory failure, interstitial lung disease, or pulmonary hypertension requiring oxygen therapy).\n\nConcurrent Malignancy: Active\u002Fprogressive malignancy other than myeloid neoplasms (exceptions: adequately treated non-melanoma skin cancer or carcinoma in situ).\n\nUncontrolled Infection: Active severe infection requiring systemic antimicrobial therapy (antibacterial, antiviral, or antifungal) without clinical resolution.\n\nImmune Disorders:\n\nSevere autoimmune disease requiring immunosuppressive therapy within 6 months. Primary immunodeficiency disorders (e.g., common variable immunodeficiency, severe combined immunodeficiency).\n\nViral Infections:\n\nActive hepatitis B (HBV-DNA ≥2000 IU\u002FmL) or hepatitis C (HCV-RNA positive). HIV infection, AIDS, or untreated syphilis (confirmed by serological testing). Hypersensitivity: History of severe allergic reaction (Grade ≥3) to biological products, including antibiotics.\n\nTransplant Complications: Allogeneic hematopoietic stem cell transplant recipients with:\n\nAcute graft-versus-host disease (GvHD) ≥ Grade II within 3 months. Ongoing immunosuppressive therapy for GvHD within 4 weeks.\n\nGeneral Exclusion:\n\nAny physical, psychiatric, or laboratory abnormality that may:\n\nSignificantly increase study risk (e.g., uncontrolled diabetes, NYHA Class III\u002FIV heart failure).\n\nCompromise protocol compliance or data interpretation. Investigator-determined unsuitability for trial participation.",{"count":171,"type":20},45,[23,24],"This is an open-label, single-arm, exploratory clinical trial utilizing a \"3+3\" dose escalation followed by dose expansion to evaluate the safety, maximum tolerated dose (MTD), pharmacokinetics (PK), and preliminary efficacy of CD33\u002FCD123\u002FCLL-1 CAR-T cell therapy in patients with relapsed\u002Frefractory myeloid malignancies.\n\nPart A: Dose Escalation Phase. Follows a \"3+3\" dose escalation design with four predefined dose cohorts: 0.2×10⁶, 0.5×10⁶, 1×10⁶, and 2×10⁶ CAR-positive cells\u002Fkg.Anticipated enrollment: 12-24 subjects.Primary objectives: Assess safety, tolerability, and determine MTD.Dose-limiting toxicity (DLT) observation period: 28 days post-infusion.\n\nPart B: Dose Expansion Phase.Enrolls 21 additional subjects to receive CAR-T cell infusion at the recommended Phase 2 dose (RP2D) established in Part A.Primary objective: Further evaluate therapeutic efficacy.\n\nOverall Study Objectives:Safety profile of CD33\u002FCD123\u002FCLL-1 CAR-T therapy.Efficacy endpoints (e.g., response rates, survival outcomes).Pharmacokinetic characterization of CAR-T cells (expansion\u002Fpersistence).",[28,175],"CAR-T","2025-04-01",{"date":178,"type":33},"2025-04-08",{"date":176,"type":20},{"date":181,"type":20},"2029-04-30",{"name":183,"class":40},"Tongji Hospital"]