[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"aml\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:aml":27},{"pageToken":4,"total":5,"offset":6,"count":7,"results":8},null,54,0,25,[9,62,90,165,189,231,251,276,297,320,340,367,399,422,444,464,492,518,536,568,620,641,665,690,717],{"id":10,"slug":11,"hasResults":12,"nctId":13,"briefTitle":14,"officialTitle":15,"acronym":4,"eligibilityCriteria":16,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":50,"lastUpdatePostDateStruct":51,"startDateStruct":54,"completionDateStruct":56,"leadSponsor":58,"locationsCount":61},"100053848","phase-1-a-phase-13-study-of-t-cell-receptor-engineered-donor-t-cells-in-subjects-undergoing-allogeneic-peripheral-blood-stem-cell-transplantation-alloha-2-100053848",false,"NCT05473910","A Phase 1\u002F3 Study of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation (ALLOHA-2)","A Phase 1\u002F3 Study Evaluating the Efficacy and Safety of T-Cell Receptor Engineered Donor T Cells in Subjects Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation (ALLOHA-2)","Inclusion Criteria:\n\n* Male or female aged ≥ 18 years at the time of signing the informed consent.\n* Eastern Cooperative Oncology Group (ECOG)-PS ≤ 2 at the time of the screening visit.\n* Contraceptive use by male and female participants must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Male Participants:\n* A male participant must agree to use a highly effective contraceptive as detailed in Appendix 4 of this protocol during the intervention period and for at least 12 months after the last dose of study intervention and refrain from donating sperm during this period.\n* Female Participants:\n* A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:\n* Not a woman of childbearing potential (WOCBP) OR\n* A WOCBP who agrees to follow the contraceptive guidance during the intervention period and for at least 12 months after the last dose of study intervention.\n* Preparing to undergo allogeneic HCT for either of the following:\n* AML, MDS, ALL\n* Participants in the treatment arms must express HLA-A\\*0201. Participants in the control arm may express any HLA type.\n* Having the HA1+\u002F- or HA-1+\u002F+ (HA-1 positive) genotype to be eligible for TSC-100 treatment.\n* Having the HA2+\u002F- HA-2+\u002F+ (HA-2 positive) genotype to be eligible for TSC-101 treatment.\n* Having a haploidentical donor, MMUD, or MUD for HCT who is adequately HLA-matched by institutional standards and meets the donor inclusion criteria.\n\nConsidered to be clinically indicated for haploidentical donor, MMUD, or MUD transplantation at the discretion of the treating investigator.\n\nConsidered to be clinically indicated for RIC at the discretion of the treating investigator.\n\nConsidered to be clinically indicated for peripheral blood stem cell transplantation at the discretion of the treating investigator.\n\nOrgan function parameters for transplant eligibility are met per institutional standards.\n\nCapable of giving signed informed consent - which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nParticipants must provide consent for mandatory study procedures including bone marrow biopsy and blood sampling for research analyses in the ICF.\n\nParticipants must agree to participate in long-term follow-up for up to 15 years post initial product treatment if they are enrolled in the study and receive the investigational Tcell infusion.\n\nDonor Inclusion Criteria :\n\nMale or female aged ≥ 18 years at the time of signing the informed consent. Able to undergo peripheral blood stem cell (PBSC) collection and up to 2 rounds of leukapheresis (for TSC-100 or TSC101 manufacturing for treatment arms only, and f for stem cell collection for both treatment arms and the control arm).\n\nDonors matched to TSC-100 participants should be HA-1-\u002F- (negative) and\u002For negative for all HLA-A\\*02 alleles Donors matched to TSC-101 participants should be negative for all HLA-A\\*02 alleles Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nExclusion Criteria:\n\nMedical or psychological conditions that would make the participant an unsuitable candidate for cell therapy including another concurrent uncontrolled malignancy or active CNS disease.\n\nThe presence of organ toxicities will not necessarily exclude participants from enrolling on the protocol at the discretion of the PI; however, a delay in the infusion of HA1\u002FHA2 TCRT cells may be required at the discretion of the treating investigator Participants with levels of donor-specific HLA antibodies that are considered by the treating investigator to be high enough to warrant desensitization protocols and who have no alternate donors.\n\nParticipants who meet inclusion criteria for TSC-101 but who are also positive for HLAA\\*02:07.\n\nParticipants with evidence of clinically significant infection or uncontrolled viral r reactivation of cytomegalovirus (CMV), Epstein-Barr virus (EBV), Adenovirus, BK virus (BKV), or human herpesvirus 6 (HHV-6).\n\nParticipants with active cardiac disease, defined as:\n\nUncontrolled or symptomatic angina within the past 3 months. History of clinically significant arrhythmias (such as ventricular tachycardia, ventricular fibrillation, torsades de pointes). Atrial fibrillation with controlled ventricular response on treatment is not an exclusion.\n\nMyocardial infarction \\\u003C 3 months from study entry. Uncontrolled or symptomatic congestive heart failure. Prior allogeneic HCT. Participants who have a history of hypersensitivity to murine proteins. Enrollment on a concomitant trial with a novel investigational agent. Use of anti-thymocyte globulin, alemtuzumab, or other in vivo T-cell depleting agents from Day -14 through end of study.\n\nDonor Exclusion Criteria :\n\nDonors for TSC-100 positive for any HLA-A\\*02 allele would be excluded unless they are HA-1 negative. If donors with any HLA-A\\*02 allele are considered for patients eligible for TSC-100, the donor would undergo HA-1 testing to ensure that the donor is HA-1 negative (40% probability).\n\nDonors for TSC-101 positive for any HLA-A\\*02 allele are excluded regardless of HA- 2 status.\n\nDonors who test positive for any of the following: HIV-1, HIV-2, human T-lymphotropic virus (HTLV)-1, HTLV-2 seropositive or with active hepatitis B or hepatitis C virus infection, syphilis, West Nile virus through central lab testing. Donors who screen positive for risk of CreutzfeldtJakob disease or Zika virus infection using donor history questionnaires will also be excluded. Donors with evidence of past CMV or EBV infections will be allowed.\n\nRelated donor residing outside of the United States of America (USA). If the donor screening, testing and leukapheresis can be performed at the same site where the participant is being treated, the donor is considered eligible.","ALL","18 Years",{"count":20,"type":21},310,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This is a multi-center, non-randomized, concurrent controlled, multi-arm, Phase 1 interventional, open-label, biologic assignment-based umbrella study evaluating the feasibility, safety and preliminary efficacy of an escalating dose regimen of up to 2 doses of TSC-100 and TSC-101 in patients with AML, MDS, or ALL following HCT from a haploidentical donor, MMUD, or MUD",[27,28],"AML","MDS",[30,31,27,28,32,33,34,35,36,37,38,39,40,41,42,43,44,45,46,47,17,48],"HA-2","TSC-101","Adoptive Cell Therapy","T-cell receptor","T lymphocyte","TCR-engineered T cells","bone marrow transplant","haploidentical","allogeneic stem cell transplant","BMT","RIC","HSCT","Hematopoietic stem cell transplantation","ALLOHA-2","Mismatched unrelated donors MMUD","ALLOHA","HA-1","TSC-100","Reduced Intensity Conditioning","RECRUITING","2026-07-10",{"date":52,"type":53},"2026-07-13","ACTUAL",{"date":55,"type":53},"2022-11-01",{"date":57,"type":21},"2028-06",{"name":59,"class":60},"TScan Therapeutics, Inc.","INDUSTRY",21,{"id":63,"slug":64,"hasResults":12,"nctId":65,"briefTitle":66,"officialTitle":67,"acronym":4,"eligibilityCriteria":68,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":69,"targetDuration":4,"studyType":22,"phases":71,"briefSummary":72,"conditions":73,"keywords":76,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":83,"completionDateStruct":85,"leadSponsor":87,"locationsCount":89},"100641694","phase-1-a-study-of-crd3874-si-in-people-with-leukemia-100641694","NCT07661095","A Study of CRD3874-SI in People With Leukemia","A Phase 1 Study of the STING Agonist CRD3874-SI for Relapsed and Refractory Acute Myeloid Leukemia","Inclusion Criteria:\n\n* Documentation of Disease\n\n  o Participant has relapsed or refractory acute myeloid leukemia, defined as bone marrow blasts ≥ 5%, and\u002For reappearance of blasts in the blood in at least 2 peripheral blood samples at least one week apart, and\u002For development of extramedullary disease; or, no CR, CRh or CRi at response assessment after at least 1 line of therapy, as defined by standardized European LeukemiaNet 2022 Criteria. Patients must have failed treatment with available therapies known to be active for treatment of their AML.\n* Participant must be ≥ 18 years of age at the time of signing the informed consent form (ICF).\n* Participant must weigh at least 40 kg\n* Participant has an Eastern Cooperative Oncology Group (ECOG) performance status score 0-2 (See Appendix I for performance status criteria)\n* For patients with known HIV, HBV, and\u002For HCV infection \\[HIV, HBV, and HCV testing do not need to be performed as part of the study; the below language provides guidelines for inclusivity of patients with known HIV, HBV, and\u002For HCV infection\\]:\n\n  * HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.\n  * For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.\n  * Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.\n* Required Organ Function\n\n  * Serum aspartate aminotransferase\u002Fserum glutamic oxaloacetic transaminase (AST\u002FSGOT) and alanine aminotransferase (ALT\u002FSGPT) ≤ 3 x ULN, unless considered due to leukemic organ involvement.\n  * Serum total bilirubin \\\u003C 1.5 x ULN. Higher levels are acceptable if these can be attributed to ineffective erythropoiesis, leukemia organ involvement or Gilbert's syndrome.\n  * Calculated creatinine clearance (CrCl) ≥ 60 mL\u002Fmin by Cockcroft-Gault formula or CKD-EPI 2021 or estimated glomerular filtration rate 60 mL\u002Fmin or greater based on local institutional practice for age-appropriate determination (e.g, Schwartz formula for pediatric patients or Cockcroft Gault formula for adults).\n  * Adequate cardiac function defined as ejection fraction of ≥ 50% by echocardiogram.\n\nExclusion Criteria:\n\n* Participants with acute promyelocytic leukemia\n* Participants with isolated myeloid sarcoma\n* Blast phase of chronic myeloid leukemia\n* Known active central nervous system leukemia\n* Participants with immediate life-threatening, severe complications of leukemia such as uncontrolled bleeding, pneumonia with hypoxia or shock, disseminated intravascular coagulation, or uncontrolled tumor lysis syndrome.\n* Participant has presence of any other condition that may increase the risk associated with study participation, and in the opinion of the treating investigator, would make the patient inappropriate for entry into the study.\n* Participants with concurrent other malignancy that will confound interpretation of study endpoints.\n* Participants who have received other anti-leukemia therapy within 5 half-lives of the agent or 14 days, whichever is sooner, prior to study treatment and if toxicity related to said agent has not resolved; exceptions of acceptable concomitant therapies are listed below\n\n  1. Concomitant cytoreductive therapy in the form of hydroxyurea, corticosteroids, or cytarabine is permitted.\n  2. Concomitant therapy in the form of intrathecal chemotherapy for CNS treatment, is permitted.\n  3. Radiation therapy is not permitted except for localized palliative radiation to focal lesions after discussion with the Medical Monitor for patients who have progressed but remain on the study due to perceived clinical benefit per Investigator assessment\n* Participants with active graft versus host disease (GVHD) of grade 2 or higher requiring systemic treatment. Skin GVHD solely managed with topical corticosteroids would not be exclusionary.\n* Known prior severe hypersensitivity to an investigational product or any component of the study drug therapy's formulations including polyethylene glycol (PEG; National Cancer Institute Common Terminology Criteria for Adverse Events \\[NCI CTCAE\\] v6.0 Grade ≥ 3)\n* Prior organ transplantation, other than allogeneic or autologous hematopoietic stem cell transplantation.\n* Received a live vaccine within 30 days of the planned start of study drug.\n\n  a. (Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however, intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines and are not allowed.)\n* Evidence of clinically significant immunosuppression including the following:\n\n  a. Primary immunodeficiency state such as SCID b. Concurrent opportunistic infection c. Receiving systemic immunosuppressive therapy (\\>2 weeks) including oral steroid doses \\> 10 mg\u002Fday of prednisone or equivalent within seven days prior to enrollment. In the setting of non-immune mediated indications for use, chronic\u002Factive low dose steroid use (equivalent to ≤ 10 mg\u002Fday prednisone) may be permitted at the discretion of the Principal Investigator i. (Note: Other steroid formulations or steroid use for other indications may be permitted and include: 1) Intranasal, inhaled, ocular, or topical steroids, or local steroid injection (e.g., intra-articular injection); 2) Systemic corticosteroids at physiologic doses ≤ 10 mg\u002Fday of prednisone or equivalent; 3) Steroids as premedication for hypersensitivity reactions (e.g., CT scan premedication)\n* History or evidence of symptomatic autoimmune disease (e.g., pneumonitis, glomerulonephritis, vasculitis, or other), or history of active autoimmune disease that has required systemic treatment (i.e., use of corticosteroids, immunosuppressive drugs or biological agents used for treatment of autoimmune diseases) in past two years prior to enrollment\n\n  a. (Note: Replacement therapy \\[e.g., thyroxine for hypothyroidism, insulin for diabetes or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency\\] is not considered a form of systemic treatment for autoimmune disease.)\n* Evidence of clinically significant interstitial lung disease, history of interstitial lung disease, or active, noninfectious pneumonitis related to prior immunotherapy treatment\n* Participant has significant active cardiac disease within 6 months prior to start of study treatment, including New York Heart Association (NYHA) class III or IV congestive heart failure; acute coronary syndrome (ACS); and\u002For stroke.\n* Participant has QTc interval (i.e., Fridericia's correction \\[QTcF\\]) ≥ 470 ms. Patients with a QTcF over 470 ms due to a bundle branch block or a pacemaker may participate in the study with approval of the study principal investigator.\n* Participant has active viral infection with human immunodeficiency virus (HIV), or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV)\n* Participant is known to have dysphagia, short-gut syndrome, gastroparesis, or other conditions that limit the ingestion or gastrointestinal absorption of drugs administered orally.\n* Participant has active uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs\u002Fsymptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and\u002For other treatment).\n* Participant with active use of strong or moderate CYP3A4 inhibitors.\n* Female participant who is pregnant or lactating.\n* Because STING agonist agents impact immune and cellular functioning posing potential risk for impacting normal embryonic development, and because other therapeutic agents used in this trial are known to be teratogenic, participants of child-bearing potential must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during study therapy and for 6 months (females) or 3 months (males) following the completion of study therapy. Male or female participants not willing to comply with contraceptive requirements will be excluded, which adequate contraception (hormonal or barrier method of birth control; abstinence) during study therapy and for 6 months (females) or 3 months (males) following the completion of study therapy",{"count":70,"type":21},24,[24],"The purpose of this study is to find out whether CRD3874-SI is a safe treatment for participants with acute myeloid leukemia (AML).",[74,27,75],"Acute Myeloid Leukemia","Refractory Acute Myeloid Leukemia",[74,27,75,77,78,79],"CRD3874-SI","Memorial Sloan Kettering Cancer Center","26-144","2026-06-16",{"date":82,"type":53},"2026-06-22",{"date":84,"type":53},"2026-06-15",{"date":86,"type":21},"2028-06-15",{"name":78,"class":88},"OTHER",7,{"id":91,"slug":92,"hasResults":12,"nctId":93,"briefTitle":94,"officialTitle":94,"acronym":95,"eligibilityCriteria":96,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":97,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":100,"conditions":101,"keywords":146,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":84,"lastUpdatePostDateStruct":156,"startDateStruct":158,"completionDateStruct":160,"leadSponsor":162,"locationsCount":164},"100289631","familial-investigations-of-childhood-cancer-predisposition-100289631","NCT03050268","Familial Investigations of Childhood Cancer Predisposition","SJFAMILY","NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown below, you may enroll regardless of the results of your clinical genetic testing.\n\nDEFINITION OF FAMILIAR CANCER FOR THIS PROTOCOL:\n\nIn this protocol, the definition of \"Familial Cancer\" is met if any of the following is present:\n\n* An individual with a history of cancer diagnosed under 26 years of age who has at least one first, second or third degree relative with a history of cancer diagnosed under 51 years of age; OR\n* An individual who has been diagnosed with more than one cancer, at least one of which was diagnosed under 26 years of age; OR\n* An individual with a clinical or molecular diagnosis of a known cancer predisposition syndrome; OR\n* An individual with a congenital cancer diagnosed before 6 months of age; OR\n* An individual with a rare pediatric cancer or tumor diagnosed before 26 years of age\n\nº Excluding human papilloma virus-associated cervical cancer and non-melanoma skin cancer occurring in adults.\n\nINCLUSION CRITERIA:\n\n* An individual who meets this protocol's definition of \"Familial Cancer,\" as above.\n* Biologic relatives of an individual meeting this protocol's definition of \"Familial Cancer,\" who are either affected or unaffected by cancer.\n\nEXCLUSION CRITERIA:\n\n* An inability or unwillingness of the research participant or his\u002Fher legally authorized representative (LAR) to provide written informed consent.\n* The participant has received allogeneic bone marrow transplantation and has NO pre-transplant germline (cancer-unaffected) DNA available AND is unwilling to provide a skin sample.",{"count":98,"type":21},1500,"OBSERVATIONAL","NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown in the Eligibility Section, you may enroll regardless of the results of your clinical genetic testing.\n\nWhile it is well recognized that hereditary factors contribute to the development of a subset of human cancers, the cause for many cancers remains unknown. The application of next generation sequencing (NGS) technologies has expanded knowledge in the field of hereditary cancer predisposition. Currently, more than 100 cancer predisposing genes have been identified, and it is now estimated that approximately 10% of all cancer patients have an underlying genetic predisposition.\n\nThe purpose of this protocol is to identify novel cancer predisposing genes and\u002For genetic variants. For this study, the investigators will establish a Data Registry linked to a Repository of biological samples. Health information, blood samples and occasionally leftover tumor samples will be collected from individuals with familial cancer. The investigators will use NGS approaches to find changes in genes that may be important in the development of familial cancer. The information gained from this study may provide new and better ways to diagnose and care for people with hereditary cancer.\n\nPRIMARY OBJECTIVE:\n\n* Establish a registry of families with clustering of cancer in which clinical data are linked to a repository of cryopreserved blood cells, germline DNA, and tumor tissues from the proband and other family members.\n\nSECONDARY OBJECTIVE:\n\n* Identify novel cancer predisposing genes and\u002For genetic variants in families with clustering of cancer for which the underlying genetic basis is unknown.",[102,103,104,27,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123,124,125,28,126,127,128,129,130,131,132,133,134,135,136,137,138,139,140,141,142,143,144,145],"Acute Leukemia","Adenomatous Polyposis","Adrenocortical Carcinoma","BAP1 Tumor Predisposition Syndrome","Carney Complex","Choroid Plexus Carcinoma","Constitutional Mismatch Repair Deficiency Syndrome","Diamond-Blackfan Anemia","DICER1 Syndrome","Dyskeratosis Congenita","Emberger Syndrome","Familial Acute Myeloid Leukemia","Familial Adenomatous Polyposis","Fanconi Anemia","Familial Cancer","Familial Wilms Tumor","Familial Neuroblastoma","GIST","Hereditary Breast and Ovarian Cancer","Hereditary Paraganglioma-Pheochromocytoma Syndrome","Hodgkin Lymphoma","Juvenile Polyposis","Li-Fraumeni Syndrome","Lynch Syndrome","Melanoma Syndrome","Multiple Endocrine Neoplasia Type 1","Multiple Endocrine Neoplasia Type 2","Neuroblastoma","Neurofibromatosis Type 1","Neurofibromatosis Type II","Nevoid Basal Cell Carcinoma Syndrome","Non Hodgkin Lymphoma","Noonan Syndrome and Other Rasopathy","Overgrowth Syndromes","Pancreatic Cancer","Peutz-Jeghers Syndrome","Pheochromocytoma\u002FParaganglioma","PTEN Hamartoma Tumor Syndrome","Retinoblastoma","Rhabdoid Tumor Predisposition Syndrome","Rhabdomyosarcoma","Rothmund-Thomson Syndrome","Tuberous Sclerosis","Von Hippel-Lindau Disease",[147,148,149,150,151,152,153,154,155],"Familial cancer","Genetic predisposition","Heritable disease","Cancer risk","Genome analysis","Genetic modifiers","Next generation sequencing (NGS)","Genetic counseling","DNA",{"date":157,"type":53},"2026-06-17",{"date":159,"type":53},"2017-04-06",{"date":161,"type":21},"2037-03-31",{"name":163,"class":88},"St. Jude Children's Research Hospital",1,{"id":166,"slug":167,"hasResults":12,"nctId":168,"briefTitle":169,"officialTitle":170,"acronym":4,"eligibilityCriteria":171,"healthyVolunteers":12,"sex":17,"minAge":172,"maxAge":173,"enrollmentInfo":174,"targetDuration":4,"studyType":22,"phases":176,"briefSummary":178,"conditions":179,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":180,"lastUpdatePostDateStruct":181,"startDateStruct":183,"completionDateStruct":185,"leadSponsor":187,"locationsCount":164},"100630639","venetoclax-azacitidine-and-liposomal-mitoxantrone-for-newly-diagnosed-aml-100630639","NCT07490288","Venetoclax, Azacitidine and Liposomal Mitoxantrone for Newly Diagnosed AML","A Single-Arm, Open-Label Study of Venetoclax, Azacitidine, and Liposomal Mitoxantrone (VAM) as Induction Therapy in Newly-diagnosed Adult Patients With Acute Myeloid Leukemia (AML)","Inclusion Criteria:\n\n* Patients diagnosed with AML according to the WHO (2022) or ICC criteria, or with MDS\u002FAML as defined by ICC (with 10%-20% blasts in the bone marrow)\n* Age ≥ 14 years, male or female.\n* Eastern Cooperative Oncology Group Performance Status (ECOG-PS) score of 0-2.\n* Meet the following laboratory requirements (tests must be performed within 7 days prior to treatment):\n\n  i. Total bilirubin ≤ 1.5 times the upper limit of normal (ULN) for the corresponding age group.\n\nii. AST and ALT ≤ 2.5 times ULN for the corresponding age group. iii. Serum creatinine \\\u003C 1.5 times ULN for the corresponding age group. iv. Cardiac enzymes \\\u003C 2 times ULN for the corresponding age group. v. Left ventricular ejection fraction (LVEF) within the normal range as measured by echocardiography (ECHO).\n\nExclusion Criteria:\n\n* Acute promyelocytic leukemia with PML::RARA fusion gene.\n* Acute myeloid leukemia with RUNX1::RUNX1T1 fusion gene.\n* Acute myeloid leukemia with BCR::ABL1 fusion gene.\n* Previously treated patients (defined as having received prior induction chemotherapy for AML\u002FMDS; prior use of cytoreductive agents like hydroxyurea is allowed).\n* Concurrent active malignancy of other organs (requiring treatment).\n* Active cardiac disease, defined as one or more of the following:\n\n  i. History of uncontrolled or symptomatic angina. ii. Myocardial infarction within 6 months prior to study enrollment. iii. History of clinically significant arrhythmia requiring medication or causing severe symptoms.\n\niv. Uncontrolled or symptomatic congestive heart failure (\\> New York Heart Association \\[NYHA\\] Class 2).\n\n* Active, uncontrolled infectious diseases (e.g., untreated tuberculosis, pulmonary aspergillosis).\n* Any other condition that, in the opinion of the investigator, makes the patient unsuitable for study participation.","14 Years","100 Years",{"count":175,"type":21},27,[177],"NA","This is a single-arm, open-label clinical trial evaluating the safety and preliminary efficacy of a novel induction regimen combining Venetoclax, Azacitidine, and Liposomal Mitoxantrone (VAM) in patients with newly diagnosed Acute Myeloid Leukemia (AML) who are eligible for intensive chemotherapy.\n\nThe study plans to enroll 27 participants. Patients will receive VAM induction therapy, followed by three cycles of intermediate-dose cytarabine consolidation. Allogeneic hematopoietic stem cell transplantation is recommended for high-risk or MRD-positive patients in remission.",[27],"2026-06-08",{"date":182,"type":53},"2026-06-09",{"date":184,"type":53},"2026-05-08",{"date":186,"type":21},"2029-03-01",{"name":188,"class":88},"Institute of Hematology & Blood Diseases Hospital, China",{"id":190,"slug":191,"hasResults":12,"nctId":192,"briefTitle":193,"officialTitle":194,"acronym":4,"eligibilityCriteria":195,"healthyVolunteers":12,"sex":17,"minAge":196,"maxAge":197,"enrollmentInfo":198,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":200,"conditions":201,"keywords":217,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":221,"lastUpdatePostDateStruct":222,"startDateStruct":224,"completionDateStruct":226,"leadSponsor":228,"locationsCount":230},"100526212","pharmacokinetic-study-of-venetoclax-tablets-crushed-and-dissolved-into-a-solution-100526212","NCT06131801","Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution","A Pharmacokinetic Study of Venetoclax Tablets Crushed and Dissolved Into a Solution in Children and Young Adults With Hematologic Malignancies","Inclusion Criteria:\n\n* Age: Patients must be \\\u003C39 years of age at time of study enrollment\n* Diagnosis: Patients may have a diagnosis of any hematologic malignancy\n* Central access: Patients must have an existing venous or arterial access line for PK blood draws\n* Weight requirement: Patients must weigh at least 5.5 kg at the time of enrollment\n* Venetoclax: Patients must be receiving any dose of venetoclax given as a solution made from crushed tablets by mouth (PO) or via nasogastric (NG), or G-tube as prescribed by their treating oncologist.\n* Concurrent chemotherapy medications: Patients may receive venetoclax as a single agent or in combination with any other chemotherapeutic agents.\n\nExclusion Criteria:\n\n* Pregnant women are excluded from this study because venetoclax has the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with venetoclax, breastfeeding should be discontinued if the mother is treated with venetoclax.\n* Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method while on study treatment and for six months following completion.","0 Years","38 Years",{"count":199,"type":21},30,"The use of venetoclax-based therapies for pediatric patients with relapsed or refractory malignancies is increasingly common outside of the clinical trial setting. For patients who cannot swallow tablets, it is common to crush the tablets and dissolve them in liquid to create a solution. However, no PK data exists in adults or children using crushed tablets dissolved in liquid in this manner, and as a result, the venetoclax exposure with this solution is unknown.\n\nPrimary Objectives\n\n• To determine the pharmacokinetics of venetoclax when commercially available tablets are crushed and dissolved into a solution\n\nSecondary Objectives\n\n* To evaluate the safety of crushed venetoclax tablets administered as an oral solution\n* To determine the pharmacokinetics of venetoclax solution in patients receiving concomitant strong and moderate CYP3A inhibitors\n* To determine potential pharmacokinetic differences based on route of venetoclax solution administration (ie. PO vs NG tube vs G-tube)\n* To determine the concentration of venetoclax in cerebral spinal fluid when administered as an oral solution",[202,203,204,205,17,206,27,207,208,209,133,122,210,211,212,213,214,215,216],"Hematologic Malignancy","Leukemia","Lymphoma","Acute Lymphocytic Leukemia","Acute Myelogenous Leukemia","Chronic Myelogenous Leukemia","CML","Myeloproliferative Neoplasm","Diffuse Large B Cell Lymphoma","Follicular Lymphoma","Burkitt Lymphoma","T-cell Lymphoma","B Cell Lymphoma","Peripheral T Cell Lymphoma","Cutaneous B-Cell Lymphoma",[218,219,220],"Venetoclax","Pediatric AML","Pediatric Relapsed\u002FRefractory AML","2026-06-02",{"date":223,"type":53},"2026-06-04",{"date":225,"type":53},"2023-11-15",{"date":227,"type":21},"2027-12-01",{"name":229,"class":88},"Children's Hospital Medical Center, Cincinnati",5,{"id":232,"slug":233,"hasResults":12,"nctId":234,"briefTitle":235,"officialTitle":235,"acronym":4,"eligibilityCriteria":236,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":237,"targetDuration":4,"studyType":22,"phases":239,"briefSummary":241,"conditions":242,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":243,"lastUpdatePostDateStruct":244,"startDateStruct":245,"completionDateStruct":247,"leadSponsor":249,"locationsCount":164},"100632076","phase-1-phase1b2-trial-of-aza--apg1252-in-patients-with-high-risk-aml-100632076","NCT07508982","Phase1b\u002F2 Trial Of AZA + APG1252 In Patients With High-Risk AML","Inclusion Criteria:\n\n1. Part I, Lead-in phase and Part II, Cohort A:\n\n   Patients with relapsed and\u002For refractory AML Patients with high-risk MDS\u002FAML who have had prior therapy will also be included\n2. Part II, Cohort B\n\n   Patients with untreated, newly diagnosed AML of the following subtypes:\n   * AML-M6 or AML-M7 by FAB or having erythroid or megakaryocytic differentiation by WHO 2022 classification\n   * High-risk MDS\u002FAML with erythroid differentiation and no prior therapy\n   * AML with MECOM rearrangement, including, but not limited to t(3;3), inv(3q), confirmed by conventional karyotype or FISH for MECOM rearrangement.\n3. Age \\>\u002F= 18 years. Because no dosing or adverse event data are currently available on the use of APG1252 in combination with AZA in patients \\\u003C18 years of age, children are not included in this study at this time.\n4. Adequate organ function as defined below:\n\n   Liver function (bilirubin \\\u003C 2mg\u002FdL, AST and ALT \\\u003C3 x ULN - or ≤5 x ULN if related to leukemic involvement) Kidney function (estimated creatinine clearance \\> 50 mL\u002Fmin). Known cardiac ejection fraction of \\> or = 45% within the past 3 months\n5. ECOG performance status of ≤ 2.\n6. A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.\n7. 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.\n\nThe effects of APG1252 on the developing human fetus are unknown. For this reason and because BCL-2\u002FBCL-XL inhibitors as well as other therapeutic agents used in this trial are 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. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:\n\n* Postmenopausal (no menses in greater than or equal to 12 consecutive months).\n* History of hysterectomy or bilateral salpingo-oophorectomy.\n* Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).\n* History of bilateral tubal ligation or another surgical sterilization procedure.\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.\n\nMen 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 APG1252 administration. Ability to understand and the willingness to sign a written informed consent document.\n\nExclusion Criteria:\n\n1. Pregnant women are excluded from this study because the agent used in this study has the potential for teratogenic or abortifacient effects. Because there is a potential risk for adverse events in nursing infants secondary to treatment of the mother with the chemotherapy agents, breastfeeding should also be avoided. 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. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:\n\n   * Postmenopausal (no menses in greater than or equal to 12 consecutive months).\n   * History of hysterectomy or bilateral salpingo-oophorectomy.\n   * Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).\n   * History of bilateral tubal ligation or another surgical sterilization procedure.\n2. Uncontrolled intercurrent illness including, but not limited to active uncontrolled infection, symptomatic congestive heart failure (NYHA Class III or IV), unstable angina pectoris, clinically significant cardiac arrhythmia, or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements.\n3. Patient with documented hypersensitivity to any of the components of the therapy program.\n4. Patients with known active, uncontrolled CNS leukemia will not be eligible.\n5. Patients with prior treatment with a BCL-XL inhibitor will not be eligible.\n6. Men and women of childbearing potential who do not practice contraception. Women of childbearing potential and men must agree to use contraception prior to study entry and for the duration of study participation.\n7. Known history of human immunodeficiency virus (HIV) infection (HIV 1\u002F2 antibodies) unless HIV RNA is undetectable by PCR.\n8. Active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection that requires treatment or at risk for HBV reactivation. Hepatitis B virus DNA and HCV RNA must be undetectable upon testing. At risk for HBV reactivation is defined as hepatitis B surface antigen positive or anti-hepatitis B core antibody positive. Prior test results obtained as part of standard of care that confirm a subject is immune and not at risk for reactivation (ie, hepatitis B surface antigen negative, surface antibody positive) may be used for purposes of eligibility and tests do not need to be repeated. Subjects with prior positive serology results must have negative polymerase chain reaction results. Subjects whose immune status is unknown or uncertain must have results confirming immune status before enrollment.",{"count":238,"type":21},52,[24,240],"PHASE2","This is a phase Ib\u002FII study that aims to investigate the safety, tolerability and explore the efficacy of BCL- XL inhibition in participants with high-risk AML.",[27],"2026-05-29",{"date":221,"type":53},{"date":246,"type":53},"2026-05-22",{"date":248,"type":21},"2031-12-12",{"name":250,"class":88},"M.D. Anderson Cancer Center",{"id":252,"slug":253,"hasResults":12,"nctId":254,"briefTitle":255,"officialTitle":256,"acronym":4,"eligibilityCriteria":257,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":258,"enrollmentInfo":259,"targetDuration":4,"studyType":22,"phases":260,"briefSummary":261,"conditions":262,"keywords":264,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":267,"lastUpdatePostDateStruct":268,"startDateStruct":270,"completionDateStruct":272,"leadSponsor":274,"locationsCount":164},"100640378","phase-1-allogeneic-car-tct0890b-in-nkg2dl-rr-aml-100640378","NCT07617285","Allogeneic CAR-T(CT0890B) in NKG2DL+ R\u002FR AML","A Phase I Study to Evaluate the Safety and Efficacy of Allogeneic CAR-T Cells (CT0890B) in Patients With NKG2DL-Positive Relapsed\u002FRefractory Acute Myeloid Leukemia","Inclusion Criteria:\n\n1. Age 18-70 years (inclusive), male or female.\n2. Relapsed or refractory acute myeloid leukemia (R\u002FR AML) diagnosed according to the 2022 World Health Organization classification or ELN criteria, with confirmed NKG2D ligand-positive disease.\n3. Bone marrow blasts ≥5% by morphology.\n4. Estimated life expectancy \\>12 weeks.\n5. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.\n6. Adequate organ function without ongoing supportive care, defined as:\n\n   1. Cardiac: left ventricular ejection fraction (LVEF) ≥50%;\n   2. Hepatic: ALT and AST ≤2.5 × upper limit of normal (ULN), and total bilirubin ≤2 × ULN;\n   3. Renal: creatinine clearance ≥30 mL\u002Fmin (calculated using the Cockcroft-Gault formula);\n   4. Coagulation: activated partial thromboplastin time (APTT) ≤1.5 × ULN and prothrombin time (PT) ≤1.5 × ULN.\n\n   c) Renal: creatinine clearance ≥30 mL\u002Fmin (calculated using the Cockcroft-Gault formula); d) Coagulation: activated partial thromboplastin time (APTT) ≤1.5 × ULN and prothrombin time (PT) ≤1.5 × ULN.\n\nExclusion Criteria:\n\n1. Participants were diagnosed with acute promyelocytic leukemia (APL), BCR-ABL positive leukemia (chronic myeloid leukemia in acute phase), central nervous system leukemia;\n2. Participants with a history of epilepsy or other central nervous system disease;\n3. Participants who have previously received autologous or allogeneic CAR-T therapy;\n4. Participants who have received autologous stem cell transplantation or allogeneic stem cell transplantation within 12 weeks\n5. Participants who have received prior immunotherapy targeting NKG2DL;\n6. Participant has clinically significant active GVHD or is receiving systemic corticosteroids for GVHD;\n7. Participant has any of the following at screening:\n\n1)Active, uncontrolled systemic infection or requiring intravenous anti-infective agents 2)Any of the following cardiac conditions, including:\n\n1. New York Heart Association Class III-IV heart failure;\n2. History of myocardial infarction, coronary artery bypass grafting, or unstable angina within 6 months prior to Qinglin;\n3. History of uncontrolled arrhythmia of significant clinical significance (as judged by the investigator), such as ventricular arrhythmia;\n4. History of severe nonischemic ardiomyopathy;\n5. Other cardiac disease that the investigatorbelieve could jeopardize the participant 's well-being or compromise participation in this clinical trial; 3) Active bleeding of clinical significance as judged by the investigator; 4)Requiring supplemental oxygen to maintain oxygen saturation\\> 92%; 5)Patients with severe chronic obstructive pulmonary disease (COPD) or other lung diseases that cannot tolerate CAR-T treatment as judged by the investigator;","70 Years",{"count":175,"type":21},[24],"A Clinical Study to Investigate the Safety and Efficacy of CT0890B in Patients with Relapsed\u002FRefractory Acute Myeloid Leukemia.",[27,263],"Refractory\u002FRelapse Acute Myeloid Leukemia",[265,266,27],"CT0890B","Universal CAR-T","2026-05-23",{"date":269,"type":53},"2026-06-01",{"date":271,"type":21},"2026-05-07",{"date":273,"type":21},"2029-12-31",{"name":275,"class":88},"Peking University People's Hospital",{"id":277,"slug":278,"hasResults":12,"nctId":279,"briefTitle":280,"officialTitle":280,"acronym":4,"eligibilityCriteria":281,"healthyVolunteers":12,"sex":17,"minAge":282,"maxAge":283,"enrollmentInfo":284,"targetDuration":4,"studyType":22,"phases":286,"briefSummary":287,"conditions":288,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":289,"lastUpdatePostDateStruct":290,"startDateStruct":292,"completionDateStruct":294,"leadSponsor":296,"locationsCount":164},"100590918","phase-2-a-randomized-study-to-compare-post-transplant-cyclophosphamide-sirolimus-ruxolitinib-and-post-transplant-cyclophosphamide-sirolimus-mycophenolate-mofetil-to-prevent-graft-versus-host-disease-100590918","NCT06973668","A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease","Inclusion Criteria:\n\n1. Age ≥ 65 and \\\u003C 75 years are eligible if they have one of the following diseases.\n\n   1. Acute Myeloid Leukemia\n   2. Myelodysplastic syndrome\n   3. Chronic myelomonocytic leukemia\n2. Available HLA-identical or haploidentical related donor or a 7\u002F8 or 8\u002F8 HLA matched unrelated donor.\n3. Peripheral blood stem cells as a graft source\n4. Subject must voluntarily sign an informed consent.\n5. Adequate organ function per local laboratory reference range as follows: - Aspartate transaminase (AST) and alanine transaminase (ALT) \\\u003C 3.0X ULN - Total Bilirubin \\\u003C1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin) - Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 40 mL\u002Fmin\u002F1.73 m2 (as reported in epic using 2021 CKD-EPI creatinine equation)\n\n   * DLCO corrected for Hgb, if applicable) ≥ 50% of predicted\n   * Ejection Fraction ≥ 50%\n   * The effects on the developing human fetus are unknown. For this reason and as well as other therapeutic agents used in this trial are 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. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:\n\n     * Postmenopausal (no menses in greater than or equal to 12 consecutive months).\n     * History of hysterectomy or bilateral salpingo-oophorectomy.\n     * Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).\n     * History of bilateral tubal ligation or another surgical sterilization procedure.\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.\n\nMen 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 study agent administration.\n\nExclusion Criteria:\n\n1. Subject is known to be positive for HIV.\n2. Subject has acute promyelocytic leukemia.\n3. Subject has known active CNS involvement with AML.\n4. Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score of \\>5\n5. Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:\n\n   1. Uncontrolled and\u002For active systemic infection (viral, bacterial or fungal)\n   2. Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or subjects with positive anti-HBc antibody but negative Hep B DNA may participate.\n6. Cardiac history of CHF requiring treatment or Ejection Fraction \\\u003C 50% or unstable angina or MI within 1 year of study entry\n7. Major adverse cardiac events such as MI\u002Fstroke and pulmonary embolism (PE)\u002Fdeep vein thrombosis (DVT) within 6 months. Recent history of Central line-associated DVT may be allowed after discussion with PI.\n8. Current and\u002For history of active TB\n9. White Blood Cell count \\> 25 X 109 \u002FL.\n10. Pregnant women are excluded from this study because the study agent has unknown potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with the study agent. These potential risks may also apply to other agents used in this study.","65 Years","75 Years",{"count":285,"type":21},80,[240],"The goal of this clinical research study is to compare the effects of these drug combinations (cyclophosphamide, sirolimus, and MMF vs cyclophosphamide, sirolimus, and ruxolitinib) on the prevention of GVHD after a stem cell transplant.",[27],"2026-05-18",{"date":291,"type":53},"2026-05-20",{"date":293,"type":53},"2025-07-22",{"date":295,"type":21},"2030-01-31",{"name":250,"class":88},{"id":298,"slug":299,"hasResults":12,"nctId":300,"briefTitle":301,"officialTitle":302,"acronym":303,"eligibilityCriteria":304,"healthyVolunteers":12,"sex":17,"minAge":172,"maxAge":4,"enrollmentInfo":305,"targetDuration":4,"studyType":22,"phases":307,"briefSummary":308,"conditions":309,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":313,"startDateStruct":315,"completionDateStruct":317,"leadSponsor":319,"locationsCount":164},"100563105","phase-1-venetoclax-in-combination-with-ivosidenib-and-azacitidine-for-newly-diagnosed-idh1-mutated-aml-100563105","NCT06611839","Venetoclax in Combination With Ivosidenib and Azacitidine for Newly Diagnosed IDH1-Mutated AML","A Multicenter, Single-Arm Clinical Study of the Venetoclax, Ivosidenib, and Azacitidine Triple-Drug Regimen in the Treatment of Chemotherapy-eligible Adult Patients With IDH1-Mutated Acute Myeloid Leukemia.","IDH1-AML-2024","Inclusion Criteria:\n\n1. Patients who meet AML according to WHO (2022) or AML and MDS\u002FAML defined by ICC standards with IDH1 mutations detected by PCR or second-generation sequencing.\n2. Age ≥14 years old, male or female.\n3. The physical status assessment (ECOG-PS) of the Eastern Oncology Collaboration group was 0-2 points.\n4. Fulfill the requirements of the following laboratory tests (performed within 7 days prior to treatment) :\n\n   1. Total bilirubin ≤ 1.5 times the upper limit of normal value (same age);\n   2. AST and ALT≤ 2.5 times the upper limit of normal value (same age);\n   3. Blood creatinine \\< 2 times the upper limit of normal (same age);\n   4. Myocardial enzymes \\< 2 times the upper limit of normal (same age);\n   5. Left ventricular ejection fraction \\>50% by measure of echocardiogram (ECHO) Informed consent must be signed before the commencement of all specific study procedures, and is signed by the patient himself or his immediate family. Considering the patient\\'s condition, if the patient\\'s signature is not conducive to the treatment of the condition, the informed consent shall be signed by the legal guardian or the patient\\'s immediate family.\n\nExclusion Criteria:\n\nSubjects who meet any of the following criteria are excluded from the study:\n\n1. Acute promyelocytic leukemia with PML-RARA fusion gene\n2. Acute myeloid leukemia with RUNX1-RUNX1T1 or CBFB-MYH11 fusion gene\n3. Acute myeloid leukemia with BCR-ABL fusion gene\n4. Treated patients (but can receive hydroxyurea or cytarabine to lower tumor burden).\n5. Concurrent malignant tumors of other organs (those requiring treatment).\n6. Active heart disease, defined as one or more of the following:\n\n   1. A history of uncontrolled or symptomatic angina;\n   2. Myocardial infarction less than 6 months after enrollment;\n   3. Have a history of arrhythmia requiring drug treatment or severe clinical symptoms;\n   4. Uncontrolled or symptomatic congestive heart failure (\\> NYHA level 2);\n7. Serious infectious diseases (uncured tuberculosis, pulmonary aspergillosis).\n8. Those who were not considered suitable for inclusion by the researchers.",{"count":306,"type":21},23,[24,240],"Venetoclax can bind to the BCL-2 protein, thereby initiating the apoptosis program and exerting anti-AML effects. The induction regimen combining venetoclax with hypomethylating agents (HMA) significantly improves the remission rate (over 60%) in elderly unfit AML patients and markedly prolongs survival in those achieving complete remission. Isocitrate dehydrogenase (IDH) 1 and 2 are involved in the citric acid cycle. Approximately 20% of AML patients carry IDH1 or IDH2 mutations, which lead to the reduction of α-ketoglutarate to 2-hydroxyglutarate (2-HG). 2-HG can cause histone methylation and inhibit TET2 activity, resulting in DNA hypermethylation, thereby affecting gene expression and cell differentiation. IDH mutations are more common in elderly patients and are often associated with cytogenetic abnormalities; they may also co-occur with FLT3-ITD, NPM1, or DNMT3A mutations. Ivosidenib is an IDH1 inhibitor, and previous studies have confirmed its safety and efficacy in AML treatment. According to adult AML treatment guidelines, IDH-mutated patients eligible for intensive chemotherapy may receive IDH inhibitors during induction therapy. Based on the study by Montesinos et al. on the role of ivosidenib and azacitidine in IDH-mutated AML, for patients ineligible for intensive chemotherapy, a new treatment option has been added: IDH1-mutated AML patients may receive ivosidenib (500 mg, days 1-28) combined with azacitidine (75 mg\u002Fm²\u002Fday for 7 days) in 28-day cycles, or ivosidenib monotherapy. Recent studies have shown that a triple-drug regimen comprising ivosidenib, venetoclax, and azacitidine demonstrates excellent efficacy and safety. In chemotherapy-ineligible patients, the triple regimen achieved a composite complete remission rate (CRc) of 86% and an overall response rate (ORR) of 92%. At a median follow-up of 27.4 months, the 2-year overall survival (OS) was 72%, and the 2-year event-free survival (EFS) was 72%. Therefore, this study aims to conduct a multicenter, single-arm clinical trial to preliminarily evaluate the long-term efficacy of this combination in adult AML.",[27,310,311],"IDH1 Mutation","Treatment","2026-05-10",{"date":314,"type":53},"2026-05-13",{"date":316,"type":53},"2025-10-17",{"date":318,"type":21},"2028-10-01",{"name":188,"class":88},{"id":321,"slug":322,"hasResults":12,"nctId":323,"briefTitle":324,"officialTitle":325,"acronym":326,"eligibilityCriteria":327,"healthyVolunteers":12,"sex":17,"minAge":172,"maxAge":4,"enrollmentInfo":328,"targetDuration":4,"studyType":22,"phases":330,"briefSummary":331,"conditions":332,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":312,"lastUpdatePostDateStruct":334,"startDateStruct":335,"completionDateStruct":337,"leadSponsor":339,"locationsCount":164},"100559265","phase-1-the-efficacy-of-triple-regimen-in-newly-diagnosed-aml-patients-with-flt3-mutation-100559265","NCT06561880","The Efficacy of Triple Regimen in Newly Diagnosed AML Patients With FLT3 Mutation","The Efficacy of a Triple Regimen Including Gilteritinib, Venetoclax, and Azacitidine in Newly Diagnosed Fit AML Patients With FLT3 Mutation","FLT3AML-2024","Inclusion Criteria:\n\n1. MDS\u002FAML patients WHO meet AML and ICC definitions according to WHO (2022) or ICC standards (10%-20% of bone marrow naive cells) and have FLT3-TKD or ITD mutations detected by PCR or second-generation sequencing.\n2. Age ≥15 years old, male or female.\n3. The physical status assessment (ECOG-PS) of the Eastern Oncology Collaboration group was 0-2 points.\n4. Pass the requirements of the following laboratory tests (performed within 7 days before treatment) :\n\n1\\) Total bilirubin ≤ 1.5 times the upper limit of normal value (same age); 2) AST and ALT≤ 2.5 times the upper limit of normal value (same age); 3) Blood creatinine \\\u003C 2 times the upper limit of normal (same age); 4) Myocardial enzymes \\\u003C 2 times the upper limit of normal (same age); 5) Echocardiography (ECHO) was performed to determine the ejection fraction of the heart within the normal range.\n\nExclusion Criteria:\n\n1. Acute promyelocytic leukemia with PML-RARA fusion gene\n2. Acute myeloid leukemia with RUNX1-RUNX1T1 or CBFB-MYH11 fusion gene\n3. Acute myeloid leukemia with BCR-ABL fusion gene\n4. Have treated patients (those who have previously received induction chemotherapy but can receive hydroxyurea down-cell therapy).\n5. Concurrent malignant tumors of other organs (those requiring treatment).\n6. Active heart disease, defined as one or more of the following:\n\n1\\) A history of uncontrolled or symptomatic angina; 2) Myocardial infarction less than 6 months after enrollment; 3) Have a history of arrhythmia requiring drug treatment or severe clinical symptoms; 4) Uncontrolled or symptomatic congestive heart failure (\\> NYHA level 2); 5) The ejection fraction is lower than the lower limit of the normal range. 7. Serious infectious diseases (uncured tuberculosis, pulmonary aspergillosis). 8. Those who were not considered suitable for inclusion by the researchers.",{"count":329,"type":21},66,[24,240],"The FMS tyrosine kinase 3 (FLT3) gene mutation occurs in 30% of newly diagnosed AML patients, leading to a higher relapse rate and mortality rate. In the past, multi-drug combination chemotherapy regimens had limited efficacy in newly diagnosed AML patients with FLT3 mutations, especially in those with FLT3-ITD. However, the FLT3 inhibitors greatly improved the survival of AML patients with FLT3 mutations. Although several studies have focused on the effectiveness of FLT3 inhibitor combination therapy for FLT3-mutated AML, further studies are needed to determine the optimal regimen and dosage. A triple regimen consisting of Gilteritinib, Venetoclax, and Azacitidine had shown good efficacy in unfit newly diagnosed FLT3-mutated AML patients. This clinical trial aims to determine the optimal triple regimen and investigate its efficacy in newly diagnosed fit FLT3-mutated AML patients.",[333,27],"FLT3 Gene Mutation",{"date":314,"type":53},{"date":336,"type":53},"2024-10-08",{"date":338,"type":21},"2027-08-31",{"name":188,"class":88},{"id":341,"slug":342,"hasResults":12,"nctId":343,"briefTitle":344,"officialTitle":345,"acronym":4,"eligibilityCriteria":346,"healthyVolunteers":347,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":348,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":350,"conditions":351,"keywords":353,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":184,"lastUpdatePostDateStruct":359,"startDateStruct":361,"completionDateStruct":363,"leadSponsor":365,"locationsCount":164},"100638844","biomarkers-in-bone-marrow-supernatant-for-predicting-aml-chemosensitivity-100638844","NCT07587944","Biomarkers in Bone Marrow Supernatant for Predicting AML Chemosensitivity","Bone Marrow Microenvironment Signatures for Predicting AML Prognosis and Resistance","Inclusion Criteria:\n\n1. Clinical diagnosis aligns with the \"Chinese guidelines for diagnosis and treatment of adult acute myeloid leukemia (not APL) (2023)\";\n2. All patients are experiencing their first onset of the disease and have not received any related chemotherapy prior to the study;\n3. Patients participate in the study accompanied by family members and sign informed consent documents.\n\nExclusion Criteria:\n\n1. Patients with concurrent malignancies requiring treatment;\n2. Presence of infectious diseases, including SARS, viral hepatitis, or HIV\u002F AIDS;\n3. Major surgery performed within the last 21 days;\n4. Performance Status (PS) score \\>3;\n5. Severe liver or kidney dysfunction or serious infection;\n6. Severe psychiatric conditions that impair understanding of the study protocol or voluntary withdrawal.",true,{"count":349,"type":21},405,"Chemoresistance in acute myeloid leukemia (AML) is closely associated with the bone marrow microenvironment. Elevated levels of IL-6, leptin, fumarate, and other factors within the bone marrow microenvironment have been shown to enhance oxidative phosphorylation or antioxidant capacity in AML cells, thereby inducing chemoresistance. To explore their potential as prognostic biomarkers or therapeutic targets, this study plans to enroll 405 newly diagnosed AML patients meeting the criteria of the Chinese Guidelines for the Diagnosis and Treatment of Adult Acute Myeloid Leukemia (2023 Edition), along with 81 sex- and age-matched healthy controls. By analyzing the levels of IL-6, leptin, fumarate, and other factors in patient bone marrow supernatant, we will evaluate their associations with treatment response (primary endpoints: overall survival \\[OS\\] and overall response rate \\[ORR\\] after one cycle of chemotherapy) and prognosis. Furthermore, patient-derived xenograft (PDX) mouse models established from primary AML cells will be used to validate their roles in chemoresistance, aiming to provide a basis for therapies targeting the bone marrow microenvironment.",[27,352],"Adult",[27,354,355,356,357,358],"Chemosensitivity","Biomarker","Fumarate","Leptin","IL-6",{"date":360,"type":53},"2026-05-14",{"date":362,"type":53},"2025-12-01",{"date":364,"type":21},"2030-01-01",{"name":366,"class":88},"Fujian Medical University Union Hospital",{"id":368,"slug":369,"hasResults":12,"nctId":370,"briefTitle":371,"officialTitle":372,"acronym":373,"eligibilityCriteria":374,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":375,"targetDuration":4,"studyType":22,"phases":377,"briefSummary":378,"conditions":379,"keywords":383,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":390,"lastUpdatePostDateStruct":391,"startDateStruct":393,"completionDateStruct":395,"leadSponsor":397,"locationsCount":164},"100528289","phase-1-pilot-study-of-memory-like-natural-killer-ml-nk-cells-after-tcr-t-cell-depleted-haploidentical-transplant-in-aml-100528289","NCT06158828","Pilot Study of Memory-like Natural Killer (ML NK) Cells After TCRαβ T Cell Depleted Haploidentical Transplant in AML","A Phase I\u002FII Pilot Study of Memory-like NK Cells to Consolidate TCRαβ T Cell Depleted Haploidentical Transplant in High-risk AML","ABCD-NK","Patient Inclusion Criteria - Cohort 1:\n\n1. High risk acute myeloid leukemia (AML) in either:\n\n   1. Complete remission (CR) defined by \\\u003C 5% marrow blasts by morphology in the context of hematological recovery (ANC ≥ 0.5× 10\\^9\u002FL, platelet count ≥ 50 × 10\\^9\u002FL).\n   2. Morphological leukemia free state (MLFS) defined by the absence of hematological recovery and \\\u003C 5% marrow blasts by morphology\n2. Patients must further meet one of the below for inclusion into the study:\n\n   1. De novo AML in CR1 with any of the following high-risk features:\n\n      * MRD ≥ 1% after first induction course\n      * MRD ≥ 0.1% after second induction course\n      * RPN1-MECOM\n      * RUNX1-MECOM\n      * NPM1-MLF1\n      * DEK-NUP214\n      * KAT6A-CREBBP (if ≥ 90 days at diagnosis)\n      * FUS-ERG\n      * KMT2A-AFF1\n      * KMT2A-AFDN\n      * KMT2A-ABI1\n      * KMT2A-MLLT1\n      * 11p15 rearrangement (NUP98 - any partner gene)\n      * 12p13.2 rearrangement (ETV6 - any partner gene)\n      * Deletion 12p to include 12p13.2 (loss of ETV6)\n      * Monosomy 5\u002FDel(5q) to include 5q31 (loss of EGR1)\n      * Monosomy 7\n      * 10p12.3 rearrangement (MLLT10b - any partner gene)\n      * FLT3\u002FITD with allelic ratio \\> 0.1%, without bZIP CEBPA or NPM1\n      * RAM phenotype as evidenced by flow cytometry\n      * Other high-risk features not explicitly stated here, after discussion\u002Fapproval with protocol PI.\n   2. De novo AML in ≥ CR2\n   3. Therapy-related AML in CR1\n   4. AML evolving from myelodysplastic syndrome (MDS)\n3. One prior hematopoietic cell transplant is allowed, provided remission criteria as defined above are met.\n\nPatient Inclusion Criteria - Cohort 2:\n\n1. High risk acute myeloid leukemia (AML) defined by either of the following:\n\n   1. Treatment refractory disease: AML that is not in complete remission despite prior standard or salvage therapies.\n   2. Multiply relapsed disease: AML that has relapsed after 2 or more hematopoietic cell transplantations.\n2. BM disease burden: Less than 25% bone marrow blasts by morphology must be present (M2 marrow), irrespective of peripheral hematological recovery.\n\nPatient Inclusion Criteria - Both Cohorts:\n\n1. Less than or equal to 40 years of age.\n2. Lansky (\\\u003C16 years) or Karnofsky (≥16 years) performance status of \\>60%.\n3. Adequate organ function as defined below:\n\n   1. Total bilirubin ≤ 3 x IULN for age\n   2. AST(SGOT)\u002FALT(SGPT) ≤ 5 x IULN for age\n   3. GFR ≥ 60 mL\u002Fmin\u002F1.73m2 as estimated by (1) updated Schwartz formula for ages 1-17 years or Cockcroft-Gault formula for ages ≥ 18 years, (2) 24-hour creatinine clearance, or (3) renal scintigraphy. If GFR is abnormal for age based on updated Schwartz or Cockcroft-Gault formula, accurate measurement should be obtained by either 24-hour creatinine clearance or renal scintigraphy.\n   4. Renal function may also be estimated by serum creatinine based on age\u002Fgender. A serum creatinine \\\u003C 2 x IULN for age\u002Fgender is required for inclusion on this protocol.\n4. Adequate cardiac function, defined by left ventricular ejection fraction (LVEF) at rest ≥50% or shortening fraction (SF) ≥27% (via echocardiogram or MUGA).\n5. Adequate pulmonary function, defined by:\n\n   1. FEV1, FVC, and DLCO ≥50% of predicted.\n   2. O2 saturation ≥ 92% on room air by pulse oximetry and no supplemental O2 at rest for children \\\u003C 8 years of age or those unable to perform pulmonary function testing (PFT). For children unable to perform PFT, a high-resolution CT chest should be obtained.\n6. The effects of these treatments on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 24 months following transplant. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.\n7. Ability to understand and willingness to sign an IRB approved written informed consent document, or patient has a guardian who has the ability to understand and willingness to sign an IRB approved written informed consent document.\n8. Available familial haploidentical donor. The HCT donor must be available and willing to undergo 2 leukapheresis procedures: (I) one mobilized collection for the HPC graft and (II) one non-mobilized leukapheresis collection for the manufacturing of ML NK cells.\n9. Donor and recipient must be identical at a minimum of one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA- DQB1. A minimum of 5\u002F10 match is required and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.\n\nPatient Exclusion Criteria - Both Cohorts\n\n1. Active GvHD. If patient had prior GvHD, patient must be off immunosuppression for at least 3 months prior to starting study treatment.\n2. Active non-hematologic malignancy. History of other malignancy is acceptable as long as therapy has been completed and there is no current evidence of disease.\n3. Currently receiving any other investigational agents at the time of transplant.\n4. Active CNS or extramedullary disease. History of CNS or extramedullary disease currently in remission is acceptable.\n5. A history of allergic reactions attributed to compounds of similar chemical or biologic composition to agents used in the study.\n6. Inability to discontinue medications that are likely to interfere with ML NK cell activity, i.e., glucocorticoids and other immunosuppressants.\n7. Presence of significant anti-donor HLA antibodies per institutional standards. Anti-donor HLA - Antibody Testing is defined as a positive crossmatch test of any titer (by complement dependent cytotoxicity or flow cytometric testing) or the mean fluorescence intensity (MFI) of any anti-donor HLA antibody by solid phase immunoassay \\> 3000.\n8. Presence of a second major disorder deemed a contraindication for HCT.\n9. Patients with Fanconi Anemia or Down Syndrome.\n10. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection (bacterial, viral with clinical instability, or fungal), symptomatic congestive heart failure, or unstable cardiac arrhythmia.\n11. Pregnant and\u002For breastfeeding. Women of childbearing potential must have a negative pregnancy test within 14 days of the start of conditioning.\n\nDonor Eligibility Criteria - Both Cohorts\n\n1. The preferred donor should be an adult aged 18 years or older. However, in circumstances where no suitable adult donor is available, consideration may be given to a minor donor aged 12 years or older. This exception only applies when all identified, otherwise eligible adult donors meet one or more of the following criteria:\n\n   * A medical condition that poses unacceptable risk, including autoimmune disease, infection, hematologic disorder, malignancy or a pathogenic germline mutation.\n   * Comorbidities that preclude safe administration of granulocyte colony-stimulating factor (G-CSF), placement of a pheresis catheter and\u002For stem cell collection.\n   * Served as donor in prior haploidentical HCT.\n   * Significant psychosocial or logistical barriers.\n2. Donor must be HLA haploidentical (≥ 5\u002F10 and ≤ 9\u002F10 allele match at the -A, -B, -C, DRB1 and DQ loci) by high resolution typing and related to the patient.\n3. Donor must meet the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT).\n4. Donor must be available and willing to undergo one mobilized and one non-mobilized leukapheresis procedure.\n5. Donor may not be pregnant and\u002For breastfeeding. Women of childbearing potential must have a negative pregnancy test within 7 days prior to initiation of recipient's conditioning regimen, within 7 days of donor stem cell mobilization regimen and prior to second non-mobilized leukapheresis..\n6. Donor must be able to understand and willing to sign an IRB-approved written informed consent document.",{"count":376,"type":21},68,[24,240],"This trial represents a single institution phase I\u002FII pilot study with the primary objective of establishing the safety and feasibility of generating and infusing ML NK cells after TCRαβ haplo-HCT.",[380,381,382,74],"AML, Childhood","Aml","Acute Myeloid Leukemia, Pediatric",[384,385,386,387,388,389],"high-risk AML","haploidentical transplant","high-risk acute myeloid leukemia","AML from MDS","memory-like natural killer cells","ML NK cells","2026-04-29",{"date":392,"type":53},"2026-05-05",{"date":394,"type":53},"2024-11-15",{"date":396,"type":21},"2030-05-31",{"name":398,"class":88},"Washington University School of Medicine",{"id":400,"slug":401,"hasResults":12,"nctId":402,"briefTitle":403,"officialTitle":404,"acronym":405,"eligibilityCriteria":406,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":407,"targetDuration":4,"studyType":22,"phases":409,"briefSummary":410,"conditions":411,"keywords":412,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":414,"lastUpdatePostDateStruct":415,"startDateStruct":416,"completionDateStruct":418,"leadSponsor":420,"locationsCount":164},"100437507","phase-2-cardioprotection-in-aml-100437507","NCT04977180","Cardioprotection in AML","Phase II Trial of Cardioprotective Prophylaxis With Combination of Beta Blocker and Angiotensin-Converting Enzyme Inhibitors During Intensive Chemotherapy for Patients With Newly Diagnosed Acute Myeloid Leukemia","AML 001","Inclusion Criteria:\n\n1. Signed informed consent obtained prior to conducting any study-specific screening procedures.\n2. Willing and able to understand the nature of this study and to comply with both the study as well as follow-up procedures for the duration of the study.\n3. Age ≥ 18 years old with newly-diagnosed Acute Myeloid Leukemia (AML)\n4. ECOG performance status must be ≤ 2\n5. Planning to receive initial induction therapy containing an anthracycline for AML. Participants may have started initial induction therapy if anthracycline has not yet been administered.\n6. Adequate organ function as evidenced by the following laboratory findings:\n\n   1. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) or \\\u003C 3 x ULN for patients with Gilbert's Syndrome\n   2. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 2.5 x ULN\n   3. Creatinine clearance \\> 60 mL\u002Fmin\n7. Ability to take oral medication and a willingness to adhere to the beta blocker and lisinopril regimen\n8. Echocardiogram demonstrating an ejection fraction ≥ 50% prior to the initiation of induction chemotherapy\n9. For females of reproductive potential and males: Agree to abstain from sexual activity or use reliable contraception while undergoing treatment with chemotherapy and\u002For ACE inhibitors due to the risk of teratogenicity to the fetus.\n\nExclusion Criteria:\n\n1. Ongoing use of any beta blocker, ACEi, or angiotensin II receptor agonist (ARB) at the time of pre-enrollment screening.\n2. Uncontrolled, intercurrent illnesses including but not limited to symptomatic unstable angina pectoris, cardiac arrhythmias not well controlled with medications, myocardial infarction in the 6 months preceding registration or psychiatric illness\u002Fsocial situations that would limit compliance with study requirements as determined by the study personnel, all at the discretion of the treating oncologist.\n3. Patient receiving concurrent investigational agents, or those who have received an investigational agent within one week of registration.\n\nException - Participants may receive concurrent investigational agents, or have done so within one week of registration if:\n\n* The side effects of the drug are well studied and well known AND\n* The drug is not known to be cardioprotective or cardiotoxic\n\n  4\\. Females who are pregnant or lactating.\n\n  5\\. Life-threatening illnesses other than AML, uncontrolled medical conditions or organ system dysfunction that, in the investigator's opinion, could compromise the patient's safety or study outcomes.\n\n  6\\. Active, untreated and\u002For severe infections as determined by the treating oncologist.\n\n  7\\. History of hematopoietic stem cell transplant (HSCT) with active graft vs host disease, immunosuppression other than low-dose prednisone (≤ 5mg) or calcineurin inhibitors within the four weeks preceding registration\n\n  8 Moderate or severe mitral or aortic valve disease, as determined by echocardiography\n\n  9\\. Congestive heart failure as clinically diagnosed by treating oncologist at the time of presentation for induction chemotherapy, or documented diagnosed by a previous physician.\n\n  10\\. History of (repaired or unrepaired) congenital heart disease that precludes recommendation for or administration of additional anthracyclines\n\n  11\\. Significant liver disease, including cirrhosis or history of transplant or hepatorenal syndrome)\n\n  12\\. Bradycardia (defined as baseline resting heart rate ≤ 60 beats per minute) or third degree atrioventricular heart block at presentation for induction chemotherapy.\n\n  13\\. Baseline resting systolic blood pressure \\\u003C 95mmHg at presentation for induction chemotherapy.\n\n  14\\. Documented allergy to beta blockers or ACE inhibitors.",{"count":408,"type":21},70,[240],"Patients with acute myeloid leukemia (AML) often receive a drug called daunorubicin. Daunorubicin is a type of drug called an anthracycline, which increases the risk of some damage to the heart. Beta blockers and angiotensin-converting enzyme inhibitors (ACEi) are two types of drugs that are often used (and are FDA approved) to treat the type of damage to the heart caused by anthracyclines. They have also been used in some populations to prevent this type of heart damage. In this study, participants will be randomly assigned to either preventively take a beta blocker and ACEi or not to receive these. The primary purpose of the study is to look at how often people in each group develop this type of heart damage. The study investigators will also collect data about your quality of life and other changes in your heart function.\n\nFrequency and severity of anthracycline-induced cardiotoxicity among patients receiving acute myeloid leukemia (AML) chemotherapy is unknown. We hypothesize that up-titrating study agents to maximum tolerated dosage at the time of induction (starting treatment for AML) will prevent the development of systolic dysfunction as determined on serial echocardiography.",[27,74],[413],"cardioprotection","2026-04-28",{"date":392,"type":53},{"date":417,"type":53},"2022-03-04",{"date":419,"type":21},"2028-09",{"name":421,"class":88},"University of Virginia",{"id":423,"slug":424,"hasResults":12,"nctId":425,"briefTitle":426,"officialTitle":426,"acronym":4,"eligibilityCriteria":427,"healthyVolunteers":12,"sex":17,"minAge":428,"maxAge":283,"enrollmentInfo":429,"targetDuration":4,"studyType":22,"phases":431,"briefSummary":432,"conditions":433,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":435,"lastUpdatePostDateStruct":436,"startDateStruct":438,"completionDateStruct":440,"leadSponsor":442,"locationsCount":164},"100630889","phase-2-mt2025-35-allogeneic-hematopoietic-stem-cell-transplantation-using-reduced-intensity-conditioning-treosulfan-and-fludarabine-with-post-transplant-cytoxan-ptcy-for-the-treatment-of-hematological-diseases-100630889","NCT07493538","MT2025-35 Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning Treosulfan and Fludarabine, With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases","Inclusion Criteria:\n\n* Patients 2-75 years of age\n* ≤7 5 years of age: Karnofsky score ≥ 70% (≥ 16 years) or Lansky play score ≥ 50 (\\\u003C 16 years) with appropriate organ criteria as below (in other inclusion criteria)\n* 5\u002F6 or 6\u002F6 related donor, OR a 5-8\u002F8 HLA-A, B, C, DRB1 allele match unrelated donor, OR a haplotype (at least 5\u002F10) related donor\n* adequate liver (no decompensated liver failure, Child Pugh A, AST\u002FALT \\\u003C5X ULN) and renal function (creatinine \\\u003C2.0)\n* absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%\n* DLCO FEV1, FVC ≥ 40% predicted, and absence of O2 requirement\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding\n* Evidence of untreated\u002Funcontrolled HIV infection\n* Untreated active serious infection\n* Active CNS malignancy\n* CML in blast crisis not in a complete remission by abnormal blast count.\n* Less than 3 months since prior myeloablative transplant","2 Years",{"count":430,"type":21},132,[240],"This is a Phase II study following subjects proceeding with Treosulfan (36g\u002Fm2) preparative regimen followed by a related, unrelated, or partially matched family donor stem cell infusion, with post-transplant cyclophosphamide (PTCy) at 40mg\u002Fkg, tacrolimus and MMF for GVHD prophylaxis.",[27,28,102,74,434],"Myelodysplastic Syndromes","2026-04-13",{"date":437,"type":53},"2026-04-16",{"date":439,"type":53},"2026-04-10",{"date":441,"type":21},"2035-03",{"name":443,"class":88},"Masonic Cancer Center, University of Minnesota",{"id":445,"slug":446,"hasResults":12,"nctId":447,"briefTitle":448,"officialTitle":449,"acronym":4,"eligibilityCriteria":450,"healthyVolunteers":12,"sex":17,"minAge":172,"maxAge":283,"enrollmentInfo":451,"targetDuration":4,"studyType":22,"phases":453,"briefSummary":455,"conditions":456,"keywords":4,"overallStatus":457,"whyStopped":4,"lastUpdateSubmitDate":435,"lastUpdatePostDateStruct":458,"startDateStruct":460,"completionDateStruct":461,"leadSponsor":463,"locationsCount":4},"100634448","phase-3-azacitidine--venetoclax-vs-azacitidineas-maintenance-therapy-in-aml-100634448","NCT07539818","Azacitidine + Venetoclax VS Azacitidineas Maintenance Therapy in AML","A Study of Azacitidine With or Without Venetoclax as Maintenance Therapy in Acute Myeloid Leukemia","Inclusion Criteria:\n\n* Diagnosis of AML per WHO 2022 or ICC criteria, or MDS\u002FAML (10-20% blasts) per ICC\n* Age ≥14 and \\\u003C75 years\n* ECOG performance status 0-2\n* First complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation\n* Received at least 2 cycles of intermediate- or high-dose cytarabine (cumulative dose ≥6 g\u002Fm² per cycle)\n* Time from first CR\u002FCRi to enrollment ≤10 months, and time from last treatment to enrollment ≤3 months\n* MRD-negative or low-level MRD detectable; MRD-positive patients are excluded\n* Signed informed consent\n\nExclusion Criteria:\n\n* Acute promyelocytic leukemia with PML-RARA\n* AML with BCR-ABL\n* Active central nervous system involvement\n* Prior allogeneic hematopoietic stem cell transplantation\n* Planned allogeneic HSCT within 6 months\n* Deemed unsuitable by investigator",{"count":452,"type":21},788,[454],"PHASE3","This is a prospective, multicenter, randomized, open-label, phase III trial evaluating the efficacy and safety of azacitidine plus venetoclax versus azacitidine alone as maintenance therapy in patients with acute myeloid leukemia (AML) who have achieved first complete remission (CR) or CR with incomplete count recovery (CRi) after induction and consolidation. Eligible patients aged 14 to 74 years are randomized 1:1 to receive either azacitidine 50 mg\u002Fm²\u002Fday on days 1-5 every 6 weeks for up to 12 cycles, or the same azacitidine regimen combined with venetoclax 400 mg on days 1-7 per cycle. The primary endpoint is disease-free survival (DFS). Secondary endpoints include overall survival (OS), relapse-free survival (RFS), cumulative incidence of relapse (CIR), minimal residual disease (MRD) conversion rate, and safety. A total of 788 patients are planned with stratification by prior venetoclax exposure and MRD status.",[27],"NOT_YET_RECRUITING",{"date":459,"type":53},"2026-04-20",{"date":390,"type":21},{"date":462,"type":21},"2031-04-01",{"name":188,"class":88},{"id":465,"slug":466,"hasResults":12,"nctId":467,"briefTitle":468,"officialTitle":469,"acronym":4,"eligibilityCriteria":470,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":471,"targetDuration":4,"studyType":22,"phases":473,"briefSummary":474,"conditions":475,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":439,"lastUpdatePostDateStruct":483,"startDateStruct":485,"completionDateStruct":487,"leadSponsor":489,"locationsCount":491},"100516228","phase-1-safety-and-tolerability-of-ziftomenib-combinations-in-patients-with-relapsedrefractory-acute-myeloid-leukemia-100516228","NCT06001788","Safety and Tolerability of Ziftomenib Combinations in Patients With Relapsed\u002FRefractory Acute Myeloid Leukemia","Phase 1 Study to Determine the Safety and Tolerability of Ziftomenib Combinations for the Treatment of KMT2A-rearranged or NPM1-mutant Relapsed\u002FRefractory Acute Myeloid Leukemia","Key Inclusion Criteria:\n\n* Has been diagnosed with relapsed\u002Frefractory AML.\n* Has a documented NPM1 mutation or KMT2A rearrangement.\n* Has a documented FLT3 mutation (cA-3 only).\n* Has an Eastern Cooperative Oncology Group (ECOG) Performance status ≤ 2.\n* Has adequate hepatic and renal function as defined per protocol.\n* Has an ejection fraction above a protocol defined limit.\n* Participant, or legally authorized representative, must be able to understand and provide written informed consent prior to the first screening procedure.\n* Has agreed to use contraception as defined per protocol.\n\nKey Exclusion Criteria:\n\n* Has a diagnosis of acute promyelocytic leukemia or blast chronic myeloid leukemia.\n* Has clinically active central nervous system leukemia.\n* Has an active and uncontrolled infection.\n* Has a mean corrected QT interval (QTcF) \\> 480ms.\n* Has uncontrolled intercurrent illness, including, but not limited to protocol defined cardiac disease.\n* Has received radiation, chemotherapy, immunotherapy, or any other anticancer therapy including investigational therapy \\\u003C14 days or within 5 drug half-lives prior to the first dose of study intervention.\n* Has had major surgery within 4 weeks prior to the first dose of study intervention.\n* Has received a hematopoietic stem cell transplant (HSCT) and has not previously had adequate recovery per protocol defined criteria.\n* Has active graft-versus-host disease (GvHD) and or on immunosuppressive drugs for the treatment of GvHD\n* Participant is pregnant or lactating.",{"count":472,"type":21},171,[24],"The safety, tolerability, and antileukemic response of ziftomenib in combination with standard of care treatments for patients with relapsed\u002Frefractory acute myeloid leukemia will be examined with the following agents: FLAG-IDA, low-dose cytarabine, and gilteritinib.",[27,476,202,477,478,479,203,74,480,481,102,482],"AML With Mutated NPM1","KMT2Ar","NPM1 Mutation","MLL Rearrangement","Leukemia, Myeloid","Leukemia, Myeloid, Acute","Neoplasms by Histologic Type",{"date":484,"type":53},"2026-04-14",{"date":486,"type":53},"2024-02-22",{"date":488,"type":21},"2027-08",{"name":490,"class":60},"Kura Oncology, Inc.",45,{"id":493,"slug":494,"hasResults":12,"nctId":495,"briefTitle":496,"officialTitle":497,"acronym":4,"eligibilityCriteria":498,"healthyVolunteers":12,"sex":17,"minAge":499,"maxAge":283,"enrollmentInfo":500,"targetDuration":4,"studyType":22,"phases":502,"briefSummary":503,"conditions":504,"keywords":507,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":510,"lastUpdatePostDateStruct":511,"startDateStruct":512,"completionDateStruct":514,"leadSponsor":516,"locationsCount":164},"100519771","phase-1-uab-2419-cd34-selection-using-the-automated-clinimacs-prodigy-100519771","NCT06047886","UAB 2419-CD34 Selection Using the Automated CliniMACS Prodigy","Feasibility Study of CD34 Selection for GVHD Prophylaxis Using the Automated CliniMACS","Inclusion Criteria:\n\n1. AML in morphologic remission with intermediate\u002Fhigh-risk features or relapsed disease 1 or 2\n2. ALL in morphologic remission with high-risk features or relapsed disease 1 or 2\n3. Lymphoid malignancies in CR or PR (e.g. non-Hodgkin's lymphoma, prolymphocytic leukemia, CLL)\n4. Myelodysplastic syndromes with \\\u003C=10% blasts\n5. CML in morphologic remission after blast phase or accelerated phase\n6. Primary myelofibrosis with \\\u003C=10% blasts \\^morphologic remission is defined as \\\u003C5% blasts on the bone marrow biopsy. Negative test for donor-specific antibody within 28 days of starting conditioning regimen, or adequate for standard desensitization protocol.\n\nExclusion Criteria:\n\n1. Non-compliant patients.\n2. No appropriate caregivers identified.\n3. Uncontrolled medical or psychiatric disorders which may preclude patients to undergo clinical studies (Discretion of the attending physician).\n4. Patients with known allergy to DMSO.\n5. Pregnant or breastfeeding women","4 Weeks",{"count":501,"type":21},50,[24],"Patients with graft failure or delayed engraftment may benefit from a hematopoietic stem cell boost or an additional hematopoietic stem cell transplantation procedure. In such settings standard immune suppression strategies are avoided due to their myelosuppressive nature. Therefore those patients are at increased risk of graft versus host disease, and the infusion of a CD34 selected graft would reduce such a risk. The infusion of CD34 selected graft using CliniMACS plus is currently FDA FDA-approved indication for acute myeloid leukemia. However, the use of the Prodigy would streamline the processing, in terms of hands-off procedure, allowing to provision of this product to the patients without strains on the cell therapy lab team. This procedure has been demonstrated safe and effective in several single-center studies and is currently in advanced phase investigation in several studies for malignant and non-malignant conditions.",[27,17,505,434,208,506],"Lymphoid Malignancies","Primary Myelofibrosis",[508,509],"hematologic malignancies","graft failure","2026-04-08",{"date":435,"type":53},{"date":513,"type":53},"2025-04-22",{"date":515,"type":21},"2029-12",{"name":517,"class":88},"University of Alabama at Birmingham",{"id":519,"slug":4,"hasResults":12,"nctId":13,"briefTitle":520,"officialTitle":521,"acronym":4,"eligibilityCriteria":522,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":523,"targetDuration":4,"studyType":22,"phases":525,"briefSummary":25,"conditions":526,"keywords":528,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":529,"lastUpdatePostDateStruct":530,"startDateStruct":531,"completionDateStruct":532,"leadSponsor":534,"locationsCount":535},"100475667","A Study of TSC-100 and TSC-101 in AML, ALL and MDS in Patients Undergoing Allogeneic Peripheral Blood Stem Transplantation","A Controlled Multi-Arm Phase 1 Umbrella Study Evaluating the Safety and Feasibility of T-Cell Receptor Engineered Donor T-Cells Targeting HA1 (TSC-100) or HA2 (TSC-101) in HLA-A0201 Positive Patients Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation","Inclusion Criteria:\n\n* Male or female aged ≥ 18 years at the time of signing the informed consent.\n* Eastern Cooperative Oncology Group (ECOG)-PS ≤ 2 at the time of the screening visit.\n* Contraceptive use by male and female participants must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n* Male Participants:\n* A male participant must agree to use a highly effective contraceptive as detailed in Appendix 4 of this protocol during the intervention period and for at least 12 months after the last dose of study intervention and refrain from donating sperm during this period.\n* Female Participants:\n* A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:\n* Not a woman of childbearing potential (WOCBP) OR\n* A WOCBP who agrees to follow the contraceptive guidance during the intervention period and for at least 12 months after the last dose of study intervention.\n* Preparing to undergo allogeneic HCT for either of the following:\n* AML\n* MDS\n* ALL\n* Participants in the treatment arms must express HLA-A\\*0201. Participants in the control arm may express any HLA type.\n* Having the HA1+\u002F- or HA-1+\u002F+ (HA-1 positive) genotype to be eligible for TSC-100 treatment.\n* Having the HA2+\u002F- HA-2+\u002F+ (HA-2 positive) genotype to be eligible for TSC-101 treatment.\n* Having a haploidentical donor, MMUD, or MUD for HCT who is adequately HLA-matched by institutional standards and meets the donor inclusion criteria.\n* Considered to be clinically indicated for haploidentical donor, MMUD, or MUD transplantation at the discretion of the treating investigator.\n* Considered to be clinically indicated for RIC at the discretion of the treating investigator.\n* Considered to be clinically indicated for peripheral blood stem cell transplantation at the discretion of the treating investigator.\n* Organ function parameters for transplant eligibility are met per institutional standards.\n* Capable of giving signed informed consent - which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n* Participants must provide consent for mandatory study procedures including bone marrow biopsy and blood sampling for research analyses in the ICF.\n* Participants must agree to participate in long-term follow-up for up to 15 years post initial product treatment if they are enrolled in the study and receive the investigational Tcell infusion.\n\nDonor Inclusion Criteria :\n\n* Male or female aged ≥ 18 years at the time of signing the informed consent.\n* Able to undergo peripheral blood stem cell (PBSC) collection and up to 2 rounds of leukapheresis (for TSC-100 or TSC101 manufacturing for treatment arms only, and f for stem cell collection for both treatment arms and the control arm).\n* Donors matched to TSC-100 participants should be HA-1-\u002F- (negative) and\u002For negative for all HLA-A\\*02 alleles\n* Donors matched to TSC-101 participants should be negative for all HLA-A\\*02 alleles\n* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the ICF and in this protocol.\n\nExclusion Criteria:\n\n* Medical or psychological conditions that would make the participant an unsuitable candidate for cell therapy including another concurrent uncontrolled malignancy or active CNS disease.\n* The presence of organ toxicities will not necessarily exclude participants from enrolling on the protocol at the discretion of the PI; however, a delay in the infusion of HA1\u002FHA2 TCRT cells may be required at the discretion of the treating investigator\n* Participants with levels of donor-specific HLA antibodies that are considered by the treating investigator to be high enough to warrant desensitization protocols and who have no alternate donors.\n* Participants who meet inclusion criteria for TSC-101 but who are also positive for HLAA\\*02:07.\n* Participants with evidence of clinically significant infection or uncontrolled viral r reactivation of cytomegalovirus (CMV), Epstein-Barr virus (EBV), Adenovirus, BK virus (BKV), or human herpesvirus 6 (HHV-6).\n* Participants with active cardiac disease, defined as:\n* Uncontrolled or symptomatic angina within the past 3 months.\n* History of clinically significant arrhythmias (such as ventricular tachycardia, ventricular fibrillation, torsades de pointes). Atrial fibrillation with controlled ventricular response on treatment is not an exclusion.\n* Myocardial infarction \\\u003C 3 months from study entry.\n* Uncontrolled or symptomatic congestive heart failure.\n* Prior allogeneic HCT.\n* Participants who have a history of hypersensitivity to murine proteins.\n* Enrollment on a concomitant trial with a novel investigational agent.\n* Use of anti-thymocyte globulin, alemtuzumab, or other in vivo T-cell depleting agents from Day -14 through end of study.\n\nDonor Exclusion Criteria :\n\n* Donors for TSC-100 positive for any HLA-A\\*02 allele would be excluded unless they are HA-1 negative. If donors with any HLA-A\\*02 allele are considered for patients eligible for TSC-100, the donor would undergo HA-1 testing to ensure that the donor is HA-1 negative (40% probability).\n* Donors for TSC-101 positive for any HLA-A\\*02 allele are excluded regardless of HA- 2 status.\n* Donors who test positive for any of the following: HIV-1, HIV-2, human T-lymphotropic virus (HTLV)-1, HTLV-2 seropositive or with active hepatitis B or hepatitis C virus infection, syphilis, West Nile virus through central lab testing. Donors who screen positive for risk of CreutzfeldtJakob disease or Zika virus infection using donor history questionnaires will also be excluded. Donors with evidence of past CMV or EBV infections will be allowed.\n* Related donor residing outside of the United States of America (USA). If the donor screening, testing and leukapheresis can be performed at the same site where the participant is being treated, the donor is considered eligible.",{"count":524,"type":21},75,[24],[27,434,527],"ALL, Adult",[46,30,47,31,27,28,17,32,33,34,35,36,37,38,39,48,40,41,42,45,44],"2026-04-02",{"date":510,"type":53},{"date":55,"type":53},{"date":533,"type":21},"2026-06",{"name":59,"class":60},15,{"id":537,"slug":538,"hasResults":12,"nctId":539,"briefTitle":540,"officialTitle":540,"acronym":4,"eligibilityCriteria":541,"healthyVolunteers":12,"sex":17,"minAge":542,"maxAge":543,"enrollmentInfo":544,"targetDuration":4,"studyType":22,"phases":546,"briefSummary":547,"conditions":548,"keywords":554,"overallStatus":457,"whyStopped":4,"lastUpdateSubmitDate":560,"lastUpdatePostDateStruct":561,"startDateStruct":562,"completionDateStruct":564,"leadSponsor":566,"locationsCount":164},"100632643","mynavigate-a-guide-to-after-treatment-effects-for-adolescents-and-young-adults-100632643","NCT07516353","my.naviGATE: A Guide to After-Treatment Effects for Adolescents and Young Adults","Inclusion Criteria:\n\n* Patients aged 15-24 who are currently undergoing cancer treatment at one of the three participating sites: DFCI, CNH, or RPOCH.\n* Patients must have initiated and be actively receiving cancer directed therapy for a newly diagnosed cancer.\n* Patients must be actively receiving cancer directed therapy, between the time of diagnosis and end of therapy for the following diagnoses: 1) Sarcomas: including osteosarcoma, Ewing sarcoma, and rhabdomyosarcoma; 2) Acute Myeloid Leukemia (AML); 3) Acute Lymphoblastic Leukemia\u002FLymphoma (ALL); 4) Hodgkin and mature B-cell Lymphomas; and 5) Medulloblastoma.\n* Patients whose treatment includes alkylators, anthracyclines, and\u002For radiation.\n\nInclusion of Children:\n\n• This study is designed to keep AYAs aged 15-24 engaged in cancer survivorship care and therefore a subset of participants will be \\\u003C18 years of age. The rationale for inclusion of children in this study is that AYA cancer survivors are particularly vulnerable to loss to follow-up and consequently lack of risk-based survivorship care, resulting in increased risk for preventable toxicity. Many existing interventions to improve engagement in survivorship screening and care are introduced after treatment completion and in long-term survivorship. Yet, for patients who are unaware of their late effect risks, and\u002For those who move frequently and are lost to follow-up, this may be too late. This study is designed to improve awareness of and engagement in risk-informed survivorship care for AYAs. This study presents no more than minimal risk to participants.\n\nExclusion Criteria:\n\n* Patients who are unwilling to give informed consent or assent to participate will be excluded. For patients under 18, patients whose guardians do not give informed consent will be excluded.\n* Patients with no chance of cure as identified by the AYA's physician, will be excluded given that issues of survivorship are not relevant and may be distressing to this population. Similarly, patients with relapsed or refractory disease will also be excluded.\n* Patients who are non-English-speaking and -reading will be excluded as the digital tool is being developed initially in English.\n* We will seek physician permission before offering enrollment to patients. If the provider team requests that the patient not be approached to participate, the patient will be excluded.","15 Years","24 Years",{"count":545,"type":21},143,[177],"This study aims to design and test a novel, personalized digital intervention-my.naviGATE-for adolescent and young adults (AYA) with cancer. my.naviGATE is a mobile app that provides personalized survivorship education, access to virtual peer navigation, and responsive participant-reported outcomes (PROs).",[549,550,551,142,74,381,17,552,553],"Sarcoma","Osteosarcoma","Ewing Sarcoma","Medulloblastoma","Acute Lymphoblastic Leukemia ,Lymphomas",[555,556,557,558,559,549,550,551,142,74,381,17,552],"Cancer Survivorship","Adolescent and Young Adult Cancer","Late Effects of Cancer Treatment","Hodgkin and mature B-cell Lymphomas","Acute Lymphoblastic Leukemia\u002FLymphoma","2026-03-31",{"date":510,"type":53},{"date":563,"type":21},"2026-08-20",{"date":565,"type":21},"2030-12-31",{"name":567,"class":88},"Dana-Farber Cancer Institute",{"id":569,"slug":570,"hasResults":12,"nctId":571,"briefTitle":572,"officialTitle":573,"acronym":4,"eligibilityCriteria":574,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":575,"enrollmentInfo":576,"targetDuration":4,"studyType":22,"phases":577,"briefSummary":578,"conditions":579,"keywords":600,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":560,"lastUpdatePostDateStruct":613,"startDateStruct":615,"completionDateStruct":617,"leadSponsor":619,"locationsCount":164},"100495785","phase-2-mt2021-08t-cell-receptor-alphabeta-depletion-pbsc-transplantation-for-heme-malignancies-100495785","NCT05735717","MT2021-08T Cell Receptor Alpha\u002FBeta Depletion PBSC Transplantation for Heme Malignancies","Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha\u002FBeta Depletion (A\u002FB TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults With Hematological Malignancies","Inclusion Criteria:\n\n* Histological confirmation of hematological malignancies\n* Acute leukemias\n* Acute Myeloid Leukemia (AML) and related precursor neoplasms\n* Favorable risk AML is defined as having one of the following:\n* Acute lymphoblastic leukemia (ALL)\u002Flymphoma\n* Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features.\n* Age 60 years of age or younger at the time of consent\n* Karnofsky performance status ≥ 70% or Lansky play score 50% for ≤16 years of age.\n* Adequate organ function\n\nExclusion Criteria:\n\n* Pregnant or breastfeeding.\n* Active uncontrolled infection within 1 week of starting preparative therapy\n* Known seropositive for HIV or known active Hepatitis B or C infection with detectable viral load by PCR.\n* Any prior autologous or allogeneic transplant\n* CML blast crisis\n* Active central nervous system malignancy","60 Years",{"count":408,"type":21},[240],"This is a phase II, open-label, prospective study of T cell receptor alpha\u002Fbeta depletion (TCR α\u002Fβ TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies. This is a safety\u002Ffeasibility study of the investigational procedure\u002Fproduct.",[202,102,580,74,581,27,582,583,584,585,586,587,588,589,590,591,592,593,594,595,596,597,598,599],"Remission","Acute Lymphoblastic Leukemia","TP53","Intrachromosomal Amplification of Chromosome 21","Cytogenetic Abnormality","CNS Leukemia","Minimal Residual Disease","Myelodysplasia","Juvenile Myelomonocytic Leukemia","Somatic Mutation","PTPN11 Gene Mutation","N-RAS Gene Amplification","Neurofibromatosis 1","NF1 Mutation","CBL Gene Mutation","Monosomy 7","Chromosome Abnormality","Fetal Hemoglobin","Lymphoblastic Lymphoma","High Grade Non-Hodgkin's Lymphoma, Adult",[601,602,603,604,605,606,607,608,609,610,611,612],"Bu","Flu","G-CSF","GFSR","aGVHD","HCT","MAC","Mel","PBSCT","PTLD","RECIST","TCR",{"date":614,"type":53},"2026-04-06",{"date":616,"type":53},"2023-05-11",{"date":618,"type":21},"2030-11-30",{"name":443,"class":88},{"id":621,"slug":622,"hasResults":12,"nctId":623,"briefTitle":624,"officialTitle":625,"acronym":4,"eligibilityCriteria":626,"healthyVolunteers":12,"sex":17,"minAge":172,"maxAge":575,"enrollmentInfo":627,"targetDuration":4,"studyType":22,"phases":629,"briefSummary":630,"conditions":631,"keywords":4,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":633,"lastUpdatePostDateStruct":634,"startDateStruct":636,"completionDateStruct":638,"leadSponsor":640,"locationsCount":164},"100573301","phase-3-standard-dose-vs-intermediate-dose-cytarabine-induction-in-the-treatment-of-acute-myeloid-leukemia-with-runx1-runx1t1-100573301","NCT06744504","Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1","Anthracycline-based Standard-dose vs Intermediate-dose Cytarabine Induction in the Treatment of Acute Myeloid Leukemia With RUNX1-RUNX1T1: a Prospective, Randomized, Controlled Phase III Clinical Trial","Inclusion Criteria:\n\n1. AML conforming to WHO (2022) or ICC standards\n2. Possessing the RUNX1::RUNX1T1 fusion gene\n3. Age ranging from 14 to 60 years old, regardless of gender.\n4. The performance status assessment of the Eastern Cooperative Oncology Group (ECOG-PS) being 0 - 2.\n5. Meeting the requirements of the following laboratory examination indicators (conducted within 7 days before treatment):\n\n1\\) Total bilirubin ≤ 1.5 times the upper limit of the normal value for the same age group; 2) AST and ALT ≤ 2.5 times the upper limit of the normal value for the same age group; 3) Serum creatinine \\\u003C 2 times the upper limit of the normal value for the same age group; 4) Cardiac enzymes \\\u003C 2 times the upper limit of the normal value for the same age group; 5) The cardiac ejection fraction determined by echocardiography (ECHO) \\> 50%. An informed consent form must be signed before the commencement of all specific research procedures, either by the patient themselves or their immediate relatives. Considering the patient's condition, if the patient's signature is not conducive to the treatment of the disease, the informed consent form shall be signed by the legal guardian or the immediate relatives of the patient.\n\nExclusion Criteria:\n\n1. Acute promyelocytic leukemia accompanied by PML-RARA fusion gene.\n2. Acute myeloid leukemia featuring BCR-ABL fusion gene.\n3. Patients undergoing retreatment (but can receive cytoreductive therapy with hydroxyurea and cytarabine).\n4. Individuals concurrently having malignant tumors in other organs (requiring treatment).\n5. Active cardiac disorders, defined as one or more of the following:\n\n1\\) A history of uncontrolled or symptomatic angina pectoris; 2) Myocardial infarction less than 6 months from study enrollment; 3) A history of significant arrhythmia requiring medication or presenting with severe clinical symptoms; 4) Uncontrolled or symptomatic congestive heart failure (\\> NYHA Class 2)\n\n6\\. Severe infectious diseases (untreated tuberculosis, pulmonary aspergillosis).\n\n7\\. Individuals deemed ineligible for enrollment by the investigator.",{"count":628,"type":21},300,[454],"Leukemia is one of the common malignant tumors that threaten human health. Although the efficacy of AML treatment has improved significantly in recent years, it remains one of the major diseases threatening human health. Current research on AML treatment mainly has two directions. One is the addition of new targeted therapy drugs, and the other research direction is to enhance the intensity of AML chemotherapy, including the use of large doses of anthracycline drugs or the use of high-dose cytarabine treatment.\n\nSince the 1990s, induction remission has been achieved by using anthracyclines in combination with high-dose cytarabine. The ECOG (Eastern Cooperative Oncology Group) contends that high-dose induction chemotherapy fails to enhance the bone marrow remission rate but elevates the chemotherapy-related mortality rate. Bradstock and the Australian Group also noted that although it does not increase the bone marrow remission rate, it can result in longer survival time and disease-free survival time. The clinical study from EORTC-GIMEMA AML-12 discovered that AML patients under the age of 45 could benefit from induction therapy incorporating high-dose cytarabine. In our previous randomized controlled clinical trials, it was found that the HAD and DA regimens containing intermediate-dose cytarabine could enhance the complete remission rate and improve the overall survival of adult AML. However, the degree of benefit varies among different AML subgroups.\n\nThe abnormalities of RUNX1-RUNX1T1 and CBFβ-MYH11 respectively involve a subunit of CBF (core binding factor), thus the two are collectively called CBF leukemia. Previous retrospective studies show that this type of leukemia benefits from intensified treatment regimens such as FLAG. However, at present, there is a lack of prospective randomized controlled clinical studies to confirm this. Therefore, in this study, we intend to further verify through a prospective randomized controlled clinical trial whether the induction treatment regimen containing intermediate-dose cytarabine can improve the long-term efficacy of adult RUNX1-RUNX1T1 acute myeloid leukemia.",[27,632],"RUNX1-RUNX1T1 Fusion Protein Expression","2026-03-24",{"date":635,"type":53},"2026-03-27",{"date":637,"type":53},"2025-01-10",{"date":639,"type":21},"2029-12-01",{"name":188,"class":88},{"id":642,"slug":643,"hasResults":12,"nctId":644,"briefTitle":645,"officialTitle":646,"acronym":647,"eligibilityCriteria":648,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":649,"enrollmentInfo":650,"targetDuration":4,"studyType":22,"phases":652,"briefSummary":653,"conditions":654,"keywords":655,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":657,"lastUpdatePostDateStruct":658,"startDateStruct":659,"completionDateStruct":661,"leadSponsor":662,"locationsCount":664},"100580206","phase-1-cer-1236-in-patients-with-acute-myeloid-leukemia-aml-myelodysplastic-syndrome-mds-and-myelofibrosis-mf-100580206","NCT06834282","CER-1236 in Patients With Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS), and Myelofibrosis (MF)","Phase 1\u002F1b First-in-human Study of Autologous Chimeric Engulfment Receptor T-Cell CER-1236 in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Myelofibrosis (CertainT-1)","CertainT-1","Inclusion Criteria:\n\n* Patients need to have a confirmed diagnosis of de novo or secondary AML, or myelodysplastic syndrome (MDS)\u002FAML with 10% to 19% blasts, per the International Consensus Classification 2022 or the WHO 2022 classification.\n* Absolute lymphocyte count \\>0.3 x 109\u002FL prior to apheresis.\n* Eastern cooperative oncology group (ECOG) performance status 0 to 1.\n\nExclusion Criteria:\n\n* Prior therapy with a permanently integrated, genetically modified cell product.\n* No measurable leukemia on the screening bone marrow evaluation prior to any bridging therapy.\n* Active autoimmune disease or history of autoimmune disease requiring treatment within the prior 2 years. Patients with history of autoimmune thyroiditis or type 1 diabetes well controlled on replacement regimen are eligible.\n* A known hypersensitivity or severe allergy to fludarabine, cyclophosphamide, or study drug components or diluents.\n* Any other medical, psychological, or social condition that may interfere with study participation or compliance, or compromise patient safety in the opinion of the physician.\n* Primary immunodeficiency disorder.","85 Years",{"count":651,"type":21},18,[24],"This is a first in human, multi center, open label, phase 1\u002F1b study to evaluate the safety and preliminary efficacy of CER-1236 in patients with relapsed\u002Frefractory (R\u002FR), measurable residual disease (MRD) positive acute myeloid leukemia (AML), or TP53mut disease.",[27,74,75],[656],"Relapsed Acute Myeloid Leukemia","2026-03-23",{"date":635,"type":53},{"date":660,"type":53},"2025-04-07",{"date":273,"type":21},{"name":663,"class":60},"CERo Therapeutics Holdings, Inc.",4,{"id":666,"slug":667,"hasResults":12,"nctId":668,"briefTitle":669,"officialTitle":670,"acronym":4,"eligibilityCriteria":671,"healthyVolunteers":12,"sex":17,"minAge":18,"maxAge":283,"enrollmentInfo":672,"targetDuration":4,"studyType":22,"phases":674,"briefSummary":675,"conditions":676,"keywords":679,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":657,"lastUpdatePostDateStruct":681,"startDateStruct":683,"completionDateStruct":685,"leadSponsor":687,"locationsCount":689},"100540176","phase-1-revumenib-in-combination-with-73--midostaurin-in-aml-100540176","NCT06313437","Revumenib in Combination With 7+3 + Midostaurin in AML","A Phase I Trial of Revumenib in Combination With 7+3 (7 Days of Cytarabine and 3 Days of Daunorubicin) + Midostaurin Induction Chemotherapy for the Frontline Treatment of NPM1 and FLT3 Mutated AML","Inclusion Criteria:\n\n* Patients with AML who are newly diagnosed according to the WHO 2022 Classification and previously untreated except for hydroxyurea. ATRA pretreatment for suspected APL for less than 5 days is allowed. Eligible patients with AML arising from an antecedent hematologic disease (AHD) including MDS, may have been treated for their prior hematologic disease (except for allogenic transplant).\n* Patients must be ≥ 18 and \\\u003C 75 years old.\n* Eastern Cooperative Oncology Group (ECOG) Performance status of 0 to 2.\n* Presence of FLT3-ITD and\u002For TKD mutation(s) AND NPM1 mutation in bone marrow or peripheral blood\n* Dose escalation phase only: Presence of any of the following adverse risk genetic characteristics:\n\n  * 2022 ELN adverse risk genetic features:\n\n    * t(6;9)(p23.3;q34.1)\u002FDEK::NUP214\n    * t(v;11q23.3)\u002FKMT2A-rearranged\n    * t(9;22)(q34.1;q11.2)\u002FBCR::ABL1\n    * t(8;16)(p11.2;p13.3)\u002FKAT6A::CREBBP\n    * inv(3)(q21.3q26.2) or t(3;3)(q21.3;q26.2)\u002F GATA2, MECOM(EVI1)\n    * t(3q26.2;v)\u002FMECOM(EVI1)-rearranged\n    * -5 or del(5q); -7; -17\u002Fabn(17p)\n    * Complex karyotype, monosomal karyotype\n    * Mutations in either one of these genes: ASXL1, BCOR, EZH2, RUNX1, SF3B1, SRSF2, STAG2, U2AF1, and\u002For ZRSR2\n    * Mutated TP53\n  * NPM1 + FLT3-ITD + DNMT3A mutation\n* LVEF ≥ 50% by MUGA or ECHO at screening.\n* Adequate renal function as demonstrated by a calculated creatinine clearance ≥ 60 mL\u002Fmin; determined by the Cockcroft Gault formula.\n* Adequate liver function as demonstrated by:\n\n  * aspartate aminotransferase (AST) ≤ 2.5 × ULN\\*\n  * alanine aminotransferase (ALT) ≤ 2.5× ULN\\*\n  * total bilirubin ≤ 1.5 × ULN\\* \\* Unless considered due to leukemic organ involvement. Note: Subjects with Gilbert's Syndrome may have a total bilirubin \\> 1.5 × ULN per discussion with the Sponsor-Investigator\n* Resolution of adverse reactions to prior drug therapy (such as hydroxyurea) to ≤ grade 1\n* Eligible for intensive cytarabine\u002Fdaunorubicin (7+3) chemotherapy based on the opinion of the treating physician.\n* Male subjects must agree to refrain from unprotected sex and sperm donation from initial study drug administration until 90 days after the last dose of study drug.\n* Females of childbearing potential (i.e., not postmenopausal for at least 1 year or not surgically sterile) must have negative results by a serum or urine pregnancy test performed within 7 days of day 1.\n* Ability to understand and the willingness to sign a written informed consent document. (Providing consents in as many languages as possible is encouraged)\n* Consolidation should occur between 1-4 weeks following count recovery after induction and remission (must be confirmed by labs to document maximal response) is established. Subjects will receive medium intensity cytarabine -based consolidation in combination with midostaurin and revumenib if the following criteria are fulfilled.\n\n  * an induction response \\\u003C 5% blasts in the bone marrow and ANC \\>1000 and PLT \\>75000 for whom documented path report is submitted.\n  * sufficiently fit (performance status \\\u003C3)\n  * resolution of any adverse reactions to no greater than grade 1 severity\n\nExclusion Criteria:\n\n* Subject has acute promyelocytic leukemia, inversion (16), t(8;21) AML as described below. Contact Sponsor-Investigator with questions. Inversion 16 and t(8;21): CBF chromosomal abnormalities may be assessed by molecular (PCR), metaphase cytogenetics, or FISH.\n* Subject has known active CNS involvement with AML.\n* Subject has received a strong CYP3A4 inducer (APPENDIX C) within 7 days prior to the initiation of study treatment\n* Strong CYP3A4 inhibitors (APPENDIX C) are contraindicated except strong CYP3A4 inhibitor antifungal azole medications (systemic itraconazole, ketoconazole, posaconazole, voriconazole). For strong CYP3A4 inhibitor antifungal azole medications, the starting dose of revumenib has to be adjusted (Table 1).\n* QTc using Fridericia's correction \\[QTcF\\]) \\> 450 msec. Drugs that prolong QTc should be avoided if possible. A list of common QTc prolonging drugs and alternatives that are not QTc prolonging can be found in APPENDIX D.\n* Subject has tested positive for HIV (due to potential drug-drug interaction between antiretroviral medications and Midostaurin\u002Frevumenib). Note: HIV testing is not required.\n* Subject is known to be positive for hepatitis B or C infection with the exception of those with an undetectable viral load within 3 months. (Hepatitis B or C testing is not required). Subjects with serologic evidence of prior vaccination to HBV \\[i.e., HBs Ag-, and antiHBs+\\] are allowed.\n* Subject has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or Star fruit within 3 days prior to the initiation of study treatment.\n* Subject has a cardiovascular disability status of New York Heart Association Class ≥ 2. Class 2 is defined as cardiac disease in which patients are comfortable at rest but ordinary physical activity results in fatigue, palpitations, dyspnea, or anginal pain.\n* Subject has a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, cardiovascular disease, or any other medical condition that in the opinion of the investigator would adversely affect his\u002Fher participating in this study.\n* Subject has chronic respiratory disease that requires continuous oxygen use.\n* Subject has a malabsorption syndrome or other condition that precludes enteral route of administration.\n* Subject exhibits evidence of other clinically significant uncontrolled condition(s) including, but not limited to uncontrolled systemic infection.\n* Subject has a history of other malignancies prior to study entry, with the exception of:\n\n  * Adequately treated in situ carcinoma of the cervix uteri or carcinoma in situ of breast;\n  * Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin;\n  * Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent.\n  * Prior malignancies treated with (surgery+\u002F- chemotherapy+\u002F- radiation) that have remained disease free for at least two years after completion of therapy\n* Subject treated with any form of chemotherapy, immunotherapy, or investigative agent within 1 month of enrollment.\n* Patients who have had prior exposure to a menin inhibitor.",{"count":673,"type":21},22,[24],"This research is being conducted to determine a safe and effective dose of revumenib that can be given in combination with standard induction (initial therapy to induce a remission) + FLT3 targeted therapy (midostaurin) and a single cycle of post-remission therapy + FLT3 targeted therapy (midostaurin) to participants with newly diagnosed Nucleophosmin (NPM1) and FMS-like tyrosine kinase 3 (FLT3) mutated Acute Myeloid Leukemia (AML).\n\nThe names of the study drugs involved in this study are:\n\n* Revumenib (SNDX-5613) (a type of menin inhibitor)\n* Midostaurin (a type of multi-kinase including FLT3 inhibitor)\n* Cytarabine (a type of antineoplastic agent)\n* Daunorubicin (a type of antineoplastic agent)",[74,677,678,27,203],"AML, Adult","AML With Gene Mutations",[74,677,680,27,203],"AML with Gene Mutations",{"date":682,"type":53},"2026-03-25",{"date":684,"type":53},"2024-12-06",{"date":686,"type":21},"2028-03-02",{"name":688,"class":88},"Richard Stone, MD",2,{"id":691,"slug":692,"hasResults":12,"nctId":693,"briefTitle":694,"officialTitle":694,"acronym":695,"eligibilityCriteria":696,"healthyVolunteers":12,"sex":17,"minAge":697,"maxAge":698,"enrollmentInfo":699,"targetDuration":4,"studyType":22,"phases":701,"briefSummary":702,"conditions":703,"keywords":707,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":708,"lastUpdatePostDateStruct":709,"startDateStruct":711,"completionDateStruct":713,"leadSponsor":715,"locationsCount":689},"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":700,"type":21},37,[240],"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)",[74,434,704,28,705,706,381],"Myeloid Malignancies","Inherited Bone Marrow Failure Syndrome","Myeloid Neoplasm",[74,434,704,28,705,706,27],"2026-03-17",{"date":710,"type":53},"2026-03-19",{"date":712,"type":53},"2023-04-19",{"date":714,"type":21},"2029-09-01",{"name":716,"class":88},"Franziska Wachter",{"id":718,"slug":719,"hasResults":12,"nctId":720,"briefTitle":721,"officialTitle":722,"acronym":4,"eligibilityCriteria":723,"healthyVolunteers":12,"sex":17,"minAge":4,"maxAge":724,"enrollmentInfo":725,"targetDuration":4,"studyType":22,"phases":727,"briefSummary":728,"conditions":729,"keywords":736,"overallStatus":49,"whyStopped":4,"lastUpdateSubmitDate":745,"lastUpdatePostDateStruct":746,"startDateStruct":748,"completionDateStruct":750,"leadSponsor":752,"locationsCount":164},"100411935","phase-2-cord-blood-transplant-in-children-and-young-adults-with-blood-cancers-and-non-malignant-disorders-100411935","NCT04644016","Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders","Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders","Participant Inclusion Criteria:\n\nAge and Donor Status:\n\nPatients with age ≤ 21 years at time of consent with no available and suitably matched related or unrelated donor in the required time period.\n\nDiagnoses :\n\nI. Acute myelogenous leukemia (AML) :\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * Known prior diagnosis of myelodysplasia (MDS) or myeloproliferative disorder (MPS).\n  * Therapy-related AML (t-AML).\n  * White cell count at presentation \\> 100,000.\n  * Presence of extramedullary leukemia at diagnosis.\n  * Any unfavorable subtype by FAB or WHO classification.\n  * High-risk cytogenetics (e.g. those associated with MDS, abnormalities of 5, 7, 8, complex karyotype) or high-risk molecular abnormalities.\n  * Requirement for 2 or more inductions to achieve CR1.\n  * Presence of Minimal Residual Disease (MRD+) by cytogenetics, flow cytometry or molecular methods after induction.\n  * Any patient with newly diagnosed AML with intermediate risk cytogenetics who elects allograft with curative intent over consolidation chemotherapy.\n  * Any patient unable to tolerate consolidation chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed AML with less than 10% blasts by bone marrow morphology. Patients with cytogenetic, flow cytometric, or molecular abnormalities in ≤ 10% of cells are eligible.\n\nII. Acute lymphoblastic leukemia (ALL):\n\n* Complete first remission (CR1) at high risk for relapse such as any of the following:\n\n  * White cell count at presentation \\> 30,000 for B-cell lineage and \\> 100,000 for T-cell lineage.\n  * Presence of any high-risk cytogenetic abnormalities such as t (9;22), t (1;19), t (4;11) or other MLL rearrangements (11q23) or other high-risk molecular abnormality.\n  * Failure to achieve complete remission (CR) after four weeks of induction therapy.\n  * Persistence or recurrence of MRD on therapy.\n  * Any patient unable to tolerate consolidation and\u002For maintenance chemotherapy as would have been deemed appropriate by the treating physician.\n  * Other high-risk features not defined above.\n* Complete second remission (CR2).\n* Primary refractory or relapsed ALL with MRD disease after antibody therapy (e.g., blinatumomab, inotuzumab, other) and\u002For CAR-T cell therapy.\n\nIII. Other acute leukemias:\n\nLeukemias of ambiguous lineage or of other types (e.g. blastic plasmacytoid dendritic cell neoplasm) with less than 5% blasts by BM morphology. Patients with persistent\u002Frelapsed disease with cytogenetic, flow cytometric or molecular aberrations in ≤ 5% of cells are eligible.\n\nIV. Myelodysplastic Syndrome (MDS) \u002F Myeloproliferative Disorders (MPD) other than myelofibrosis:\n\n* International prognostic scoring system (IPSS) risk score of INT-2 or high risk at the time of diagnosis.\n* Any IPSS risk category if life-threatening cytopenia(s) exists.\n* Any IPSS risk category with karyotype or genomic changes that indicate high risk for progression to acute myelogenous leukemia.\n* MDS\u002F myeloproliferative disorder overlap syndromes without myelofibrosis.\n* MDS\u002F MPD patients must have less than 10% bone marrow myeloblasts and ANC ≥ 0.2 (growth factor supported if necessary) at transplant work-up.\n\nV. Non-Hodgkin lymphoma (NHL) or Hodgkin lymphoma (HL) at high-risk of relapse or progression if not in remission:\n\n* Eligible patients with aggressive histology (such as, but not limited to, diffuse large B-cell NHL, mantle cell NHL, and T-cell histology) in CR.\n* Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) will have 2nd or subsequent progression with stable disease\u002F CR\u002F PR with no single lesion equal to or more than 5 cm.\n* Eligible patients with HL will be those without progression of disease (POD) after salvage chemotherapy with no single lesion ≥ 5 cm.\n\nVI. Inherited Metabolic Disorders \\[also see EBMT Handbook for discussion on patient eligibility for allogeneic transplant; in general, patients are considered early in the disease course, before they develop neurologic symptoms (46)\\]:\n\n* Hurler Syndrome\n* Hunter (MPS 2 - early disease)\n* Sly syndrome (MPSVIII)\n* α-Mannosidosis\n* X- ALD\n* Osteopetrosis\n* Metachromatic Leukodystrophy\n* Globoid (GLD)\n\nVII. Non-Malignant disorders (other) \\[also see EBMT Handbook for criteria for transplant (46)\\]\n\n* Hemoglobinopathies\n* Bone Marrow Failure syndromes\n* Immunodeficiencies, including HLH\n\nOrgan Function and Performance Status Criteria:\n\n* Karnofsky or Lansky score ≥ 70% (see Appendix)\n* Bilirubin ≤ 1.5 mg\u002FdL (unless benign congenital hyperbilirubinemia).\n* ALT ≤ 3 x upper limit of normal.\n* Pulmonary function (spirometry and corrected DLCO) ≥ 50% predicted (corrected for hemoglobin) .\n* Left ventricular ejection fraction ≥ 50%.\n* Age-adjusted Hematopoietic Cell Transplantation-Comorbidity Index (aaHCT-CI) less than or equal to 7.\n* Renal: serum creatinine ≤ 1.5x normal for age. If serum creatinine is outside the normal range, then CrCl \\> 50 mL\u002Fmin\u002F1.73m2 (calculated or estimated) or GFR (mL\u002Fmin\u002F1.72m2) \\>30% of predicted normal for age.\n\nNormal GFR in Children and Young Adults (Age) : Mean GFR +- SD (mL\u002Fmin\u002F1.73 m2)\n\n1 week: 40.6 + \u002F - 14.8 2-8 weeks: 65.8 + \u002F - 24.8 \\>8 weeks: 95.7 + \u002F - 21.7 2-12 years: 133.0 + \u002F - 27.0 13-21 years (males): 140.0 + \u002F - 30.0 13-21 years (females: 126.0 + \u002F - 22.0\n\nGFR, glomerular filtration rate; SD, standard deviation greater than 2 years old: Normal GFR is 100 mL\u002F min. Infants: GFR must be corrected for body surf ace area.\n\nFor metabolic diseases: disease status to be evaluated according to EBMT Handbook \\[45\\].\n\nGraft Criteria\n\nCB units will be selected according to the current MSKCC unit selection algorithm. High resolution 8 allele HLA typing and recipient HLA antibody profile will be performed. Cord unit selection will occur based on HLA-match, total nucleated cell (TNC) and CD34+ cell dose adjusted per patient body weight. The cord bank of origin will also be considered. Donor specific HLA antibodies, if present, will also be taken into consideration and may influence the selection of the graft. CB graft will consist of one or two CB units (CBU) based on MSKCC selection algorithm.\n\n* Each CB unit must be at least 3\u002F8 HLA-matched to the patient considering high-resolution 8-allele HLA typing.\n* For malignant diseases follow MSKCC CBU selection algorithm\n* For non-malignant diseases, CBU will be required to have \\> 5 x 107 TNC\u002Fkg; high HLA allele level match is preferable\n\nParticipant Exclusion Criteria:\n\n* Inadequate performance status\u002F organ function.\n* Advanced metabolic disease (EBMT handbook).\n* Active CNS leukemic involvement.\n* Indolent NHL or Hodgkin lymphoma with progression of disease after most recent salvage chemotherapy.\n* Diagnosis of myelofibrosis or other malignancy with moderate-severe bone marrow fibrosis.\n* Autologous stem cell transplant within the preceding 6 months.\n* Any prior allogeneic stem cell transplant.\n* Active and uncontrolled infection (bacterial\u002Ffungal\u002Fviral) at time of transplantation.\n* HIV infection.\n* Seropositivity for HTLV-1.\n* Pregnancy or breast feeding.\n* Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, long-term follow-up, and research tests.","21 Years",{"count":726,"type":21},31,[240],"This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic disorders who do not have a matched related donor for allogeneic transplantation.",[27,17,28,730,731,732,733,734,735],"MPD Withou Myelofibrosis","NHL or HL","Inherited Metabolic Disorders","Hemoglobinopathies","Bone Marrow Failure","HLH",[737,27,587,28,738,739,740,741,742,743,744,78],"Acute myelogenous leukemia","Myeloproliferative Disorder","Therapy-Related AML and MDS","Therapy-Related Acute Myeloid Leukemia","T-AML","High-risk cytogenetics","Cord Blood Transplantation","20-480","2026-03-12",{"date":747,"type":53},"2026-03-13",{"date":749,"type":53},"2020-11-20",{"date":751,"type":21},"2026-12-20",{"name":78,"class":88}]