[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"npm1-mutation\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:npm1-mutation":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,50,73,100,133,159,199],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":31,"overallStatus":37,"whyStopped":4,"lastUpdateSubmitDate":38,"lastUpdatePostDateStruct":39,"startDateStruct":42,"completionDateStruct":44,"leadSponsor":46,"locationsCount":49},"100644306","phase-2-study-on-the-efficacy-and-safety-of-va-regimen-compared-to-37-regimen-in-newly-diagnosed-aml-with-npm1-or-idh1idh2-mutations-100644306",false,"NCT07664839","Study on the Efficacy and Safety of VA Regimen Compared to \"3+7\" Regimen in Newly Diagnosed AML With NPM1 or IDH1\u002FIDH2 Mutations","A Prospective, Multicenter, Randomized Controlled, Open Label, Non-Inferiority Study Comparing the Efficacy and Safety of the VA Regimen (Venetoclax Combined With Azacitidine) With the \"3+7\" Regimen in the Treatment of Newly Diagnosed AML Patients With NPM1 or IDH1\u002FIDH2 Mutations","Inclusion Criteria:\n\n* Age ≥ 65 years old ≥ 18 years old;\n* Diagnosed as acute myeloid leukemia (non APL) (diagnostic criteria refer to the 2022 ELN classification system);\n* Initial diagnosis accompanied by NPM1 mutations (A, B, D types and rare types are all acceptable) and\u002For IDH1\u002FIDH2 mutations;\n* Have not received any other induction therapy before (except hydroxyurea);\n* Physical fitness status score (ECOG PS) 0-3;\n* Having sufficient organ function, defined as follows:\n\n  1. Liver function: serum total bilirubin ≤ 3 x upper limit of normal range (ULN), aspartate aminotransferase (AST), alanine aminotransferase (ALT), and alkaline phosphatase (ALP) ≤ 3 x ULN, unless considered to be caused by leukemia;\n  2. Renal function: endogenous creatinine clearance rate ≥ 30ml\u002Fmin;\n  3. Heart function: NYHA classification ≤ 2 points;\n* Participants must have the ability to understand and be willing to participate in this study, and sign an informed consent form.\n\nExclusion Criteria:\n\n* Acute promyelocytic leukemia;\n* Merge extramedullary infiltration such as central nervous system leukemia;\n* Have a clear history of CMML or MDS, and later progress to AML; Or have a history of malignant tumors;\n* There is uncontrolled active infection (including bacterial, fungal, or viral infections);\n* Pregnant or lactating women;\n* Researchers determine that participants are not suitable to participate in this experiment","ALL","18 Years","65 Years",{"count":20,"type":21},148,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","This prospective, multicenter, randomized, open-label, non-inferiority clinical study aims to compare the efficacy and safety of VA regimen (venetoclax combined with azacitidine) versus conventional \"3+7\" chemotherapy regimen in adult patients aged 18 to 65 years with newly diagnosed acute myeloid leukemia (AML) carrying NPM1, IDH1 or IDH2 gene mutations.\n\nThe primary goal of this trial is to check whether the VA treatment can reach a non-inferior composite complete remission rate at the end of the induction treatment cycle, which is the key primary endpoint of this research. Several secondary clinical outcomes will also be evaluated in this study, including the rate of minimal residual disease (MRD) negativity after remission, duration of remission, 1-year event-free survival rate and 1-year overall survival rate of enrolled patients. In addition, the safety and treatment-related side effects occurring during the whole induction treatment phase will be systematically collected and compared between two groups as another important secondary assessment.\n\nEligible enrolled participants will be randomly split into two study groups: patients in experimental group will receive venetoclax plus azacitidine (VA regimen), while patients in control group will receive standard \"3+7\" induction chemotherapy following conventional clinical protocol. All subjects will complete regular disease assessment, laboratory examinations and scheduled follow-up visits as required by trial design during treatment and post-treatment observation period.\n\nResearchers will collect and analyze all above clinical outcome data from all participants, to verify the non-inferior efficacy and relative safety of VA regimen for this specific subtype of newly diagnosed AML patients.",[27,28,29,30],"Acute Myeloid Leukemia","NPM1 Mutation","IDH1 Mutation","IDH2 Mutation",[32,33,34,35,36],"Acute myeloid leukemia","Venetoclax","NPM1 mutation","IDH1 mutation","IDH2 mutation","NOT_YET_RECRUITING","2026-06-23",{"date":40,"type":41},"2026-06-24","ACTUAL",{"date":43,"type":21},"2026-07-01",{"date":45,"type":21},"2032-05-31",{"name":47,"class":48},"Shen yang","OTHER",1,{"id":51,"slug":52,"hasResults":11,"nctId":53,"briefTitle":54,"officialTitle":55,"acronym":56,"eligibilityCriteria":57,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":58,"targetDuration":4,"studyType":60,"phases":4,"briefSummary":61,"conditions":62,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":49},"100599167","observational-study-on-apl-like-acute-myeloid-leukemia-distinct-phenotype-and-early-vascular-complications-100599167","NCT07080970","Observational Study on APL-like aCute Myeloid Leukemia: disTInct Phenotype and Early VAscular complicaTions","Observational Study on APL-like Subset Within NPM1-mutated Acute Myeloid Leukemia: a Distinct Phenotypic Signature Correlating With Early-onset Vascular Complications. ACTIVATE (APL-like aCute Myeloid Leukemia: disTInct Phenotype and Early VAscular complicaTions)","ACTIVATE","Inclusion Criteria:\n\n* Patients with de novo AML, untreated, newly diagnosed, according to WHO\u002FICC 2022 criteria from January 2015 onwards.\n* Presence of NPM1 mutation.\n* Availability of immunophenotypic characterization at diagnosis\n* Age \\>= 18 years\n* Signed written informed consent according to ICH\u002FEU\u002FGCP and national local laws (if applicable)\n\nExclusion Criteria:\n\n* No specific exclusion criteria are provided once eligibility criteria are met.",{"count":59,"type":21},220,"OBSERVATIONAL","This is a multicenter observational study with a retrospective and a prospective cohort investigating clinically and biologically the APL-like subset as a potential predictor of coagulopathy and susceptibility to early vascular events.",[27,28],"RECRUITING","2026-06-17",{"date":66,"type":41},"2026-06-22",{"date":68,"type":21},"2026-07",{"date":70,"type":21},"2028-11",{"name":72,"class":48},"Gruppo Italiano Malattie EMatologiche dell'Adulto",{"id":74,"slug":75,"hasResults":11,"nctId":76,"briefTitle":77,"officialTitle":78,"acronym":4,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":16,"minAge":80,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":60,"phases":4,"briefSummary":83,"conditions":84,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":49},"100635977","a-real-world-study-on-the-efficacy-and-safety-of-menin-inhibitors-as-maintenance-after-allo-hsct-100635977","NCT07559695","A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance After Allo-HSCT","A Real-world Study on the Efficacy and Safety of Menin Inhibitors as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation","Inclusion Criteria:\n\n1. Age ≥ 15 years.\n2. Patients diagnosed with acute leukemia (including AML, ALL, and MPAL) according to the World Health Organization (WHO 2022) criteria.\n3. Must meet one of the following characteristics: a. Harboring an NPM1 gene mutation (without concurrent FLT3-ITD or FLT3-TKD mutation); b. Harboring a KMT2A gene rearrangement or KMT2A-PTD; c. Harboring a NUP98 gene rearrangement; d. Other acute leukemia subtypes dependent on the menin-KMT2A interaction, if evidenced, may be enrolled upon discussion with and approval from the principal research team.\n4. Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT), with ≥ 30 days elapsed since the date of graft infusion.\n5. Received menin inhibitor maintenance therapy after allo-HSCT and meets the following conditions: a. Received at least ≥ 2 complete cycles (7 days per cycle) of menin inhibitor therapy, or cumulative medication duration ≥ 14 days; b. Patient was in a state of CR\u002FCRh\u002FCRi at the initiation of maintenance therapy.\n6. No evidence of leukemia relapse during menin inhibitor maintenance therapy, defined as: a. Bone marrow blasts \\\u003C 5%, and blasts do not exhibit morphological features of acute leukemia (e.g.Auer rods); b. No evidence of extramedullary leukemia (e.g. CNS leukemia or myeloid sarcoma).\n7. The menin inhibitors used include but are not limited to: Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors.\n8. Capable of understanding and voluntarily signing the informed consent form.\n9. Complete clinical data.\n\nExclusion Criteria:\n\n1. Presence of any of the following at the initiation of menin inhibitor maintenance therapy (including within 28 days prior to starting treatment): a. Morphologic relapse in bone marrow (bone marrow blasts ≥ 5%); b. Presence of leukemic cells in peripheral blood.\n2. Active infection that is deemed uncontrolled by the investigator.\n3. Severe organ dysfunction, including: a. Hepatic impairment: ALT or AST ≥ 5 × ULN (Upper Limit of Normal), or total bilirubin ≥ 3 × ULN; b. Severe renal impairment: eGFR \\\u003C 30 ml\u002Fmin\u002F1.73 m²; c. Cardiac dysfunction: NYHA (New York Heart Association) Class III-IV.\n4. Concurrent acute graft-versus-host disease (aGVHD) ≥ Grade 2 or chronic graft-versus-host disease (cGVHD) ≥ Grade 3, requiring corticosteroids ≥ 1 mg\u002Fkg and ≥ 3 types of immunosuppressive therapy (including CNI, ruxolitinib, belumosudil, etc.).\n5. History of other malignancies requiring ongoing treatment (except for malignancies that have undergone curative treatment or are assessed to be in complete remission and require no systemic maintenance therapy or radiotherapy).\n6. Any gastrointestinal disorder that may affect the intake or absorption of oral medications (e.g.dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal GVHD, etc).\n7. Patients deemed unsuitable for inclusion in this study by the investigator.\n8. Severely missing clinical data, precluding efficacy or safety assessment.\n9. Receipt of other maintenance therapies, including but not limited to hypomethylating agents, donor lymphocyte infusion (DLI), or other specific small-molecule targeted drugs.","15 Years",{"count":82,"type":21},20,"The objective of this observational study is to evaluate the efficacy and safety of menin inhibitor maintenance therapy in patients with acute leukemia who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).\n\nEligible patients will receive menin inhibitor maintenance therapy as part of their routine clinical practice. Acceptable agents include, but are not limited to, Revumenib, BN104, Ziftomenib, HMPL-506, or other menin-KMT2A interaction inhibitors. This study imposes no additional interventions on clinical management. The specific menin inhibitor, initiation timing, dose adjustments, and treatment duration are determined at the investigator's discretion based on the patient's individual condition and clinical circumstances.\n\nPatients will enter the follow-up phase upon initiation of menin inhibitor maintenance therapy. Efficacy and safety will be assessed at every cycle during the treatment period. Following the completion of treatment, survival follow-up visits will be conducted every three cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.",[85,86,87,88,89,28,90],"Menin Inhibitors","Acute Leukemia","Post Hematopoietic Stem Cell Transplantation","Maintenance Therapy","KMT2A Rearrangement","NUP98 Gene Rearrangement","2026-04-24",{"date":93,"type":41},"2026-04-30",{"date":95,"type":41},"2025-11-01",{"date":97,"type":21},"2029-12-31",{"name":99,"class":48},"The First Affiliated Hospital of Soochow University",{"id":101,"slug":102,"hasResults":11,"nctId":103,"briefTitle":104,"officialTitle":105,"acronym":4,"eligibilityCriteria":106,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":107,"targetDuration":4,"studyType":22,"phases":109,"briefSummary":111,"conditions":112,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":122,"lastUpdatePostDateStruct":123,"startDateStruct":125,"completionDateStruct":127,"leadSponsor":129,"locationsCount":132},"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":108,"type":21},171,[110],"PHASE1","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.",[113,114,115,116,28,117,118,27,119,120,86,121],"AML","AML With Mutated NPM1","Hematologic Malignancy","KMT2Ar","MLL Rearrangement","Leukemia","Leukemia, Myeloid","Leukemia, Myeloid, Acute","Neoplasms by Histologic Type","2026-04-10",{"date":124,"type":41},"2026-04-14",{"date":126,"type":41},"2024-02-22",{"date":128,"type":21},"2027-08",{"name":130,"class":131},"Kura Oncology, Inc.","INDUSTRY",45,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":140,"targetDuration":4,"studyType":22,"phases":142,"briefSummary":143,"conditions":144,"keywords":146,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":149,"lastUpdatePostDateStruct":150,"startDateStruct":152,"completionDateStruct":154,"leadSponsor":156,"locationsCount":158},"100549905","phase-1-ziftomenib-maintenance-post-allo-hct-100549905","NCT06440135","Ziftomenib Maintenance Post Allo-HCT","An Open Label Phase I Study of Ziftomenib as Maintenance Therapy Following Allogeneic Hematopoietic Cell Transplantation","Inclusion Criteria:\n\n* 18 years or older.\n* Pathologically confirmed diagnosis of acute myeloid leukemia (AML).\n* Complete remission (CR) or complete remission with incomplete count recovery (CRi) at screening.\n\n  * Complete remission (CR):\n\n    * no circulating blasts in peripheral blood and \\\u003C5% blasts in bone marrow\n    * no extramedullary disease\n    * platelet count ≥100 x 10(9)\u002FL and\u002For absolute neutrophil count ≥1000\u002FµL\n  * Complete remission with incomplete count recovery (CRi):\n\n    * no circulating blasts in peripheral blood and \\\u003C5% blasts in bone marrow\n    * no extramedullary disease\n    * platelet count \\\u003C100 x 10(9)\u002FL and\u002For absolute neutrophil count \\\u003C1000\u002FµL\n* Presence of at least one of the following molecular mutations:\n\n  * KMT2A rearrangement\n\n    * Eligibility and enrollment will be based on local mutational testing.\n    * The presence of a KMT2A rearrangement (excluding partial tandem duplication \\[PTD\\]) at the time of initial diagnosis or any other time thereafter is sufficient.\n    * Participants may receive additional treatment for AML between consent and transplant.\n  * NPM1 mutation\n\n    * Eligibility and enrollment will be based on local mutational testing.\n    * For participants being transplanted in CR1, the presence of a NPM1 mutation at screening is necessary for the purposes of eligibility.\n    * For participants being transplanted in greater than or equal to CR2, the presence of a NPM1 mutation at the time of consent is not necessary for eligibility and its presence at the time of initial diagnosis or any other time thereafter is sufficient.\n    * Participants may receive additional treatment for AML between consent and transplant.\n* Treatment with a menin inhibitor prior to transplant is permitted. However, patients who experienced AML relapse or progression while being treated with a menin inhibitor prior to transplant are ineligible.\n* Will undergo first allogeneic HCT for their malignancy.\n* Transplantation will be performed with the use of conventional myeloablative (MAC) or reduced intensity conditioning (RIC).\n* HCT Donor will be one of the following:\n\n  * 5\u002F6 or 6\u002F6 (HLA-A, B, DR) matched related donor\n  * 7\u002F8 or 8\u002F8 (HLA-A, B, DR, C) matched unrelated donor. Matching in the unrelated setting must be at the allele level.\n  * Haploidentical related donor, defined as ≥ 3\u002F6 (HLA-A, B, DR) matched\n  * ≥ 4\u002F6 (HLA-A, B, DR) umbilical cord blood (UCB). Matching in the UCB setting is at the antigen level. Recipients may receive either one or two UCB units. In the case of 2 UCB units, both units must have been at least 4\u002F6 matched with the recipient.\n* Any non-investigational GVHD prophylaxis regimen is allowed.\n* Eastern Cooperative Oncology Group (ECOG) performance status 0-2.\n* Participants must have normal organ and function as defined below:\n\n  * AST (SGOT), ALT (SGPT) and Alkaline phosphatase \\\u003C 3x institutional upper limit of normal (ULN)\n  * Total bilirubin \\\u003C 1.5 x institutional ULN (with the exception of subjects with a history of Gilbert's syndrome, for which the total bilirubin must be \\\u003C 5 x ULN)\n  * Calculated creatinine clearance ≥ 30 mL\u002Fmin (Cockcroft-Gault formula)\n* LVEF must be ≥50%, as measured by MUGA scan or echocardiogram.\n* Female patients of childbearing potential must have a negative pregnancy test, as measured by serum or urine testing.\n* The effects of ziftomenib on the developing human fetus are unknown. For this reason women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) during the entire study treatment period and through 6 months after the last dose of treatment.\n* Ability to understand and the willingness to sign a written informed consent document.\n\nExclusion Criteria:\n\n* History of other malignancy(ies) unless\n\n  * the participant has been disease-free for at least 2 years and is deemed by the investigator to be at low risk of recurrence of that malignancy, or\n  * the cancer has been deemed indolent with no progression over the last 2 years, and deemed by the investigator to be at low risk for further progression during the course of study and follow-up\n  * the only prior malignancy was cervical cancer in situ and\u002For basal cell or squamous cell carcinoma of the skin\n* Known diagnosis of active hepatitis B or hepatitis C\n* Current or history of congestive heart failure New York Heart Association (NHYA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF \\\u003C 50%, as measured by multigated acquisition (MUGA) scan or echocardiogram)\n* Current or history of ventricular or life-threatening arrhythmias or diagnosis of long-QT syndrome\n* Systemic uncontrolled infection\n* Known dysphagia, short-gut syndrome, gastroparesis, or other condition(s) that limits the ingestion or gastrointestinal absorption of drugs administered orally\n* Uncontrolled hypertension (systolic blood pressure \\[BP\\] \\> 180 mmHg or diastolic BP \\> 100 mmHg)\n* QTc interval (i.e., Friderica's correction \\[QTcF\\]) ≥ 480 ms or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome) at screening\n* Uncontrolled intercurrent illness that would limit compliance with study requirements.\n* Persons who are pregnant or lactating.",{"count":141,"type":21},22,[110],"The purpose of this study is to test the safety, effects, and recommended dose of an investigational drug, ziftomenib, in addition to the standard treatment on blood cancer with Allogeneic Hematopoietic Cell Transplantation (allo-HCT). This study plans to learn more about ziftomenib, which targets and inhibits negative interactions within cancer cells related to AML, when given after allo-HCT, to determine if it improves outcomes following allo-HCT.\n\nThe name of the study drug involved in this study is:\n\n• Ziftomenib",[27,145,28,89],"Acute Myeloid Leukemia in Remission",[27,147,148,28,89],"Allogeneic hematopoietic cell transplantation (HCT)","Allo-HCT","2026-03-17",{"date":151,"type":41},"2026-03-19",{"date":153,"type":41},"2024-06-11",{"date":155,"type":21},"2027-09-01",{"name":157,"class":48},"Massachusetts General Hospital",2,{"id":160,"slug":161,"hasResults":11,"nctId":162,"briefTitle":163,"officialTitle":164,"acronym":4,"eligibilityCriteria":165,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":166,"targetDuration":4,"studyType":22,"phases":168,"briefSummary":169,"conditions":170,"keywords":177,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":190,"lastUpdatePostDateStruct":191,"startDateStruct":193,"completionDateStruct":195,"leadSponsor":197,"locationsCount":198},"100495744","phase-1-a-study-to-investigate-the-safety-and-tolerability-of-ziftomenib-in-combination-with-venetoclaxazacitidine-venetoclax-73-or-73quizartinib-in-patients-with-aml-100495744","NCT05735184","A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax\u002FAzacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML","Phase 1 Study of Venetoclax\u002FAzacitidine or Venetoclax in Combination With Ziftomenib or Standard Induction Cytarabine\u002FDaunorubicin (7+3) Chemotherapy in Combination With Ziftomenib for the Treatment of Patients With Acute Myeloid Leukemia","Key Inclusion Criteria:\n\n* Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed\u002Frefractory AML\n\n  * Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2\n* Adequate liver, renal, and cardiac function according to protocol defined criteria\n* A female of childbearing potential must agree to use adequate contraception as well as a double barrier method from the time of screening through 180 days following the last dose of study intervention. A male of childbearing potential must agree to use abstinence or use a double barrier method of contraception from the time of screening through 180 days following the last dose of study intervention\n\n  * Female patients of childbearing potential who receive quizartinib in Arm C should use a highly effective method of contraception during quizartinib treatment and for 7 months after the last dose\n\nKey Exclusion Criteria:\n\n* Diagnosis of either acute promyelocytic leukemia or blast phase chronic myeloid leukemia\n* Known history of BCR-ABL alteration\n* Advanced malignant hepatic tumor\n* Administration of live attenuated vaccines within 14 days prior to, during, or after treatment until B-cell recovery\n* Active central nervous system (CNS) involvement by AML.\n* Clinical signs\u002Fsymptoms of leukostasis or WBC \\> 25,000 \u002F microliter. Hydroxyurea and\u002For leukapheresis and\u002For up to 2 doses of cytarabine if used per institutional SOC for control of leukocytosis are permitted to meet this criterion\n* Not recovered to Grade ≤1 (NCI-CTCAE v5.0) from all nonhematological toxicities except for alopecia\n* Known clinically active human immunodeficiency virus, active hepatitis B or active hepatitis C infection\n* For newly diagnosed cohorts: received prior chemotherapy for leukemia, except hydroxyurea and\u002For leukapheresis and\u002For up to 2 doses of cytarabine per institutional standards to control leukocytosis, or prior treatment with all-transretinoic acid for initially suspected acute promyelocytic leukemia\n* For relapsed\u002Frefractory cohorts: received chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational \\\u003C 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug\n* Uncontrolled intercurrent illness including, but not limited to, cardiac illness as defined in the protocol\n* Mean QT interval corrected for heart rate by Fredericia's formula (QTcF)\n\n  * Arm A and Arm B: \\>480 ms on triplicate ECGs\n  * Arm C: \\>450 ms on triplicate ECGs\n* Uncontrolled infection\n* Women who are pregnant or lactating\n* An active malignancy and currently receiving chemotherapy for that malignancy or disease that is uncontrolled\u002Fprogressing\n* Patients who have active GVHD requiring \\>0.5 mg\u002Fkg prednisone or any new or increase in immunosuppressants in the prior 2 weeks for GVHD treatment",{"count":167,"type":21},420,[110],"Ziftomenib is an investigational drug in development for the treatment of patients with acute myeloid leukemia (AML) with certain genetic alterations.\n\nThis protocol has 3 separate arms that will investigate the benefits and risks of adding ziftomenib to standard-of-care (SOC) drug treatments in patients who have AML with certain genetic mutations. Both newly diagnosed and relapsed refractory patients with AML will be assigned to different cohorts based on specific study criteria and physician discretion.\n\nThe purpose of this study is to assess the safety, tolerability, and early signs of efficacy of ziftomenib in combination with SOC drugs to treat AML.",[27,171,172,114,173,174,28,116,175,176],"Mixed Lineage Leukemia Gene Mutation","Refractory AML","Acute Myeloid Leukemia Recurrent","Acute Myeloid Leukemia, in Relapse","Myeloid Sarcoma","Nucleophosmin 1-mutated Acute Myeloid Leukemia",[118,178,113,179,180,181,182,86,183,184,185,186,187,188,34,172,174,189],"Myeloid","Hematological malignancy","KMT2A","NPM1","Menin","Newly diagnosed AML","Untreated AML","venetoclax","cytarabine","daunorubicin","KMT2A-r","quizartinib","2026-03-11",{"date":192,"type":41},"2026-03-13",{"date":194,"type":41},"2023-07-18",{"date":196,"type":21},"2030-04",{"name":130,"class":131},44,{"id":200,"slug":201,"hasResults":11,"nctId":202,"briefTitle":203,"officialTitle":204,"acronym":4,"eligibilityCriteria":205,"healthyVolunteers":11,"sex":16,"minAge":206,"maxAge":4,"enrollmentInfo":207,"targetDuration":4,"studyType":22,"phases":209,"briefSummary":210,"conditions":211,"keywords":4,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":212,"lastUpdatePostDateStruct":213,"startDateStruct":215,"completionDateStruct":217,"leadSponsor":219,"locationsCount":49},"100600746","phase-2-phase-2-trial-of-bn104-as-post-hsct-maintenance-in-acute-leukemia-100600746","NCT07101497","Phase 2 Trial of BN104 as Post-HSCT Maintenance in Acute Leukemia","Phase 2 Trial of Menin Inhibitor BN104 as Post Hematopoietic Stem Cell Transplantation Maintenance in Patients With Acute Leukemia","Inclusion Criteria:\n\n* Male or female patients.\n* Adult and adolescent patients aged ≥12 years who must weight ≥35 kg.\n* Diagnosed with acute myeloid leuekmia, acute lymphoblastic leukemia or ambiguous acute leukemia according to the World Health Organization classification of hematologic neoplams (WHO 2022).\n* Intermediate or high risk accroding to the ELN risk stratification.\n* Harboring one of the following genetic aberrations: a. somatic NPM1 mutation (without FLT3-ITD\u002FTKD co-mutations); b. KMT2A rearrangement\u002FKMT2A-PTD; c. NUP98 rearrangement; d. other genetic alterations dependent on menin-KMT2A.\n* Received allogenic hematopoietic stem cell transplantation within 30-180 days at the initiation of BN104 maintenance therapy.\n* Achieved full donor chimerism and hematologic recovery, with acute neuthrophil count (ANC) ≥1.0×10⁹\u002FL, platelets ≥75×10⁹\u002FL (no red blood cells \u002Fplatelets transfusion within 7 days, no G-CSF or GM-CSF within 72 hours).\n* Complete hematological remission (CHR) after first allo-SCT. CHR must be confirmed by bone marrow analysis within 14 days before entering the study (CHR criteria are: \"\\\u003C 5% marrow blasts, no peripheral blasts, blood platelet count \\> 75×10⁹\u002FL, WBC count \\> 3.5 G\u002FL, ANC ≥ 1.0×10⁹\u002FL).\n* No extramedullary leukemia.\n* Eastern Cooperative Oncology Group (ECOG) performance status score 0-2.\n* Adequate organ function\n* Provided informed consent by all patients and the guardians ( aged 12-17 years).\n* Written informed consent.\n* ECOG ≥ 2.\n\nExclusion Criteria:\n\n* Complicated with active and uncontrolled infections.\n* Activation of virus, (e.g., CMV viremia with CMV DNA copies \\> 400 copies\u002Fml, EBV viremia with EBV DNA copies \\> 400 copies\u002Fml, and proof of activation of adenovirus and Human Parvovirus B19 ).\n* Activation of hepatitis B, hepatitis C, or human immunodeficiency virus.\n* Cardiac disease as followings: a. inherited long QT syndrome. b. Congestive heart failure with NYHA ≥ grade 2.\n* ≥ grade 2 acute GVHD or ≥ grade 3 chronic GVHD which requiring systemic therapy.\n* Have received other maintenance therapies (e.g., hypomethylating agents, targetd drugs such as Bcl-2 inhibitors, FLT3 inhibitors, IDH1\u002F2 inhibitors, interferon, interleukin-2, donor lymphocyte infusion and chemotherapy).\n* History of other malignancies which needed systemic treatment (excluding those in stable remission without maintenance therapy).\n* Any gastrointestinal condition that may interfere with oral drug intake or absorption (e.g., dysphagia, gastroparesis, uncontrolled chronic diarrhea, intestinal graft versus host disease.\n* Pregnancy, breastfeeding\n* Hypersensitivity to BN104.","12 Years",{"count":208,"type":21},60,[24],"This is a phase 2, open label, single arm trial. This study aims to assess the efficacy and safety of menin inhibitor BN104 as maintenance therapy in patients with acute leukemia harboring specific genetic alterations who have undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).\n\nEligible patients will be screened at 30-180 days post allo-HSCT. Participants will take BN104 100-200mg orally, twice a day, 28 days a cycle for 24-36 cycles. The primary endpoint is 2-year relapse-free survival rate since enrollment. The secondary endpoints included overall survival, event-free survival, cumulative incidence of relapse, non relpase related mortality and safety.",[85,87,88,86,89,28,90],"2025-07-28",{"date":214,"type":41},"2025-08-03",{"date":216,"type":21},"2025-08-01",{"date":218,"type":21},"2029-12-30",{"name":99,"class":48}]