[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"refractory-aml\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:refractory-aml":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,8,0,[8,43,92,114,166,202,228,250],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":25,"conditions":26,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100639057","phase-1-adapt-nk-for-high-risk-myeloid-diseases-as-bridge-to-allo-hsct-100639057",false,"NCT07591649","Adapt NK for High Risk Myeloid Diseases as Bridge to Allo HSCT","Safety and Efficacy of Expanded KIR-HLA Mismatched Natural Killer Cell Immunotherapy (AdaptNK) for High-Risk Myeloid Diseases as Bridge to Allogeneic Hematopoietic Stem Cell Transplantation","Inclusion Criteria:\n\n* 18-74 years with Karnofsky score ≥ 70%\n* 75 years and older: KPS ≥ 70%, HCT-CI \\\u003C 5 (excluding history of solid tumor), AND not frail by Fried frailty criteria (see Appendix III)\n* HLA type C1\u002FC1 or C2\u002FC2\n\nNote: For easy determination, the definition of HLA-C ligand group assigments is included below:\n\nHLA-C1 group alleles are defined as HLA-C01, C03, C07, C08, C12, C14, C16 HLA-C2 group alleles are defined as HLA-C02, C04, C05, C06, C15, C17, C18\n\n* adequate liver, renal, pulmonary and cardiac function\n* ability to be off glucocorticoids and other immunosuppressive medications indicated for acute or chronic GVHD for at least 28 days prior to the AdaptNK cell infusion\n* There must be sufficient time between the most recent therapy and the screening bone marrow as delineated below:\n* anti-leukemic systemic cytotoxic chemotherapy - 2 weeks\n* Targeted anti-leukemic agents (FLT-3, IDH, menin inhibitors) - 3 half-lives of the medication\n* Radiotherapy - 1 week\n* donor lymphocyte infusions - 6 weeks\n* hematopoietic growth factors (filgrastim, TPO agonists, EPO) - 1 week\n* biologic therapy (monoclonal antibodies, T-cell engagers) - 2 weeks\n* Immune effector cellular therapy - 4 weeks\n* Intrathecal chemotherapy for treatment of active CNS leukemia - there must be at least two CSF samples negative for leukemia separated by one week before enrollment.\n* WBC shall be \\\u003C 25,000 before infusion. Hydroxyurea is permitted until day -3 to control excess blast proliferation. No other systemic treatment is allowed after the screening bone marrow is performed for inclusion in protocol\n* All prior treatment related toxicities should have resolved to ≤ grade 1 prior to study enrollment\n* agrees to use of adequate contraception from study enrollment to 4 months after cell infusion\n* voluntary written consent\n\nExclusion Criteria:\n\n* Myeloid neoplasms with known or strongly suspected germline background, except DDX41, TP53, or RUNX1.\n* Acute promyelocytic leukemia (APL)\n* myocardial infarction (MI) within previous 6 months of study enrollment\n* pregnant or breastfeeding\n* Active CNS involvement with AML\n* new or progressive pulmonary infiltrates\n* active autoimmune disease requiring immunosuppressive therapy\n* Preexisting inflammatory disease requiring immunosuppressive therapy\n* history of severe asthma and currently on chronic systemic medications\n* HIV-1\u002F2 positivity or hepatitis C\u002FB\n* active systemic infections requiring anti-infective treatment\n* received any investigational agent within the 14 days before the start of study treatment (1st dose of fludarabine)\n* Patients with second malignancies are excluded if they have required systemic cytotoxic chemotherapy within 1 year or if they are not in remission\n* Exception: patients that are on stable dosing of hormonal therapy (e.g. aromatase inhibitor or antiandrogen therapy) for active breast or prostate cancer for 1 year are eligible.\n* Patients with excised basal cell or squamous cell carcinoma of the skin are eligible.\n* Patients with excised carcinoma in situ of the cervix or breast are eligible.\n* Patients with untreated T1a or T1b prostate cancer are eligible.","ALL","18 Years",{"count":19,"type":20},18,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE1","PHASE2","This is a multi-institutional Phase I\u002FII study of an allogeneic KIR-HLA mismatched NK cell infusion (AdaptNK) and a short course of subcutaneous interleukin-2 (IL-2) administered after lymphodepleting chemotherapy \\[cyclophosphamide (CY)\u002Ffludarabine (FLU)\\] in patients with relapsed or refractory acute myelogenous leukemia (AML). AdaptNK is a natural killer (NK) cell product that is enriched for NK cells with an \"adaptive\", or human cytomegalovirus (CMV)-induced, phenotype.",[27,28,29],"Relapsed Adult AML","Refractory AML","Acute Myelogenous Leukemia","RECRUITING","2026-05-15",{"date":33,"type":34},"2026-05-19","ACTUAL",{"date":36,"type":34},"2026-05-08",{"date":38,"type":20},"2035-03-01",{"name":40,"class":41},"Masonic Cancer Center, University of Minnesota","OTHER",1,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":21,"phases":52,"briefSummary":53,"conditions":54,"keywords":64,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":81,"lastUpdatePostDateStruct":82,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":91},"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":51,"type":20},420,[23],"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.",[55,56,28,57,58,59,60,61,62,63],"Acute Myeloid Leukemia","Mixed Lineage Leukemia Gene Mutation","AML With Mutated NPM1","Acute Myeloid Leukemia Recurrent","Acute Myeloid Leukemia, in Relapse","NPM1 Mutation","KMT2Ar","Myeloid Sarcoma","Nucleophosmin 1-mutated Acute Myeloid Leukemia",[65,66,67,68,69,70,71,72,73,74,75,76,77,78,79,28,59,80],"Leukemia","Myeloid","AML","Hematological malignancy","KMT2A","NPM1","Menin","Acute Leukemia","Newly diagnosed AML","Untreated AML","venetoclax","cytarabine","daunorubicin","KMT2A-r","NPM1 mutation","quizartinib","2026-03-11",{"date":83,"type":34},"2026-03-13",{"date":85,"type":34},"2023-07-18",{"date":87,"type":20},"2030-04",{"name":89,"class":90},"Kura Oncology, Inc.","INDUSTRY",44,{"id":93,"slug":94,"hasResults":11,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":99,"enrollmentInfo":100,"targetDuration":4,"studyType":21,"phases":102,"briefSummary":103,"conditions":104,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":105,"lastUpdatePostDateStruct":106,"startDateStruct":108,"completionDateStruct":110,"leadSponsor":112,"locationsCount":42},"100539685","phase-1-cll-1-car-nk-cells-for-relapsedrefractory-aml-100539685","NCT06307054","CLL-1 CAR-NK Cells for Relapsed\u002FRefractory AML","The Safety and Efficacy for Anti-human CLL-1 CAR-NK Cells in Subjects With Relapsed\u002FRefractory Acute Myeloid Leukemia","Inclusion Criteria:\n\n1. Age 18-70 years, gender unrestricted;\n2. Expected survival time exceeds 12 weeks;\n3. ECOG score 0-2;\n4. Meets the 2022 WHO criteria for acute myeloid leukemia and flow cytometry shows ≥70% expression of CLL1 in leukemia cells; or immunohistochemistry shows CLL1 expression ≥50% and meets the following criteria for relapse and refractory:\n\n   1. Relapse criteria: Bone marrow shows primitive cells ≥5% after hematological remission (excluding post-chemotherapy hematopoietic recovery); or at least two peripheral blood samples taken at least one week apart show primitive cells; or extramedullary lesions are present. Early relapse (relapse within 12 months after initial remission) patients can be directly included, while late relapse (relapse after 12 months of initial remission) patients must have undergone salvage chemotherapy according to standard protocols without achieving complete remission. Salvage treatment for all relapsed patients should include at least one course of targeted therapy without achieving remission. Patients who relapse after allogeneic hematopoietic stem cell transplantation, have no other effective treatment options, and have no active grade 2 or higher acute GVHD.\n   2. Refractory criteria: No complete remission after two courses of standard intensive chemotherapy based on the 3+7 regimen, and no complete remission after second-line salvage chemotherapy or treatment including targeted therapy; or no complete remission after one course of purine analog induction chemotherapy (such as FLAG-Ida, CLIA, or similar regimens), and no complete remission after salvage treatment or treatment including targeted therapy; or no complete remission after three cycles of low-intensity treatment based on HMA, including low-intensity regimens containing venetoclax; relapse within 12 months after remission (early relapse); patients for whom the original induction is ineffective after relapse.\n5. Able to establish the required venous access for collection, and no contraindications for leukapheresis;\n6. Liver and kidney function, cardiac and pulmonary function meet the following requirements:\n\n   1. Creatinine clearance (calculated by Cockcroft-Gault formula) ≥ 60 mL\u002Fmin or creatinine ≤ 2.5×ULN;\n   2. Ejection fraction \\>50%, no clinically significant electrocardiogram changes; baseline blood oxygen saturation \\>92%; total bilirubin ≤ 3×ULN; ALT and AST ≤ 3×ULN;\n7. Able to understand and sign the informed consent form.\n\nExclusion Criteria:\n\nAny of the following conditions disqualify a subject from participation in the trial:\n\n1. Confirmed AML with PML-RARA fusion gene;\n2. History of malignancies other than acute myeloid leukemia within 5 years before screening, except adequately treated carcinoma in situ of the cervix, basal cell carcinoma, squamous cell carcinoma of the skin, or prostate cancer after radical surgery, or thyroid cancer after radical surgery;\n3. Uncontrolled active bacterial, viral, or fungal infections requiring treatment; HBsAg or HBcAb positive, with peripheral blood HBV DNA ≥ lower limit of detection; HCV RNA positive in the presence of hepatitis C virus antibodies; positive TRUST test for syphilis; HIV antibody positive;\n4. Significant organ (cardiovascular, pulmonary) dysfunction; active gastrointestinal bleeding within the past 3 months; uncontrolled hypertension or history of hypertensive crisis or hypertensive encephalopathy; significant history or evidence of major cardiovascular risk, including congestive heart failure, unstable angina, clinically significant arrhythmias (such as ventricular fibrillation, ventricular tachycardia, etc.); history of arterial thrombosis within the past 3 months (such as stroke, transient ischemic attack); symptomatic deep vein thrombosis within the past 6 months, history of pulmonary embolism, or history of coronary angioplasty, defibrillation, or any clinical significant complications or diseases that may pose a risk to the subject's safety or interfere with the study evaluation, procedures, or completion;\n5. Any uncontrolled active disease that hinders participation in the trial;\n6. Active, uncontrolled central nervous system involvement, or a history of central nervous system disease requiring treatment (such as epilepsy);\n7. Subjects receiving systemic corticosteroid therapy before screening and deemed by the investigator to require long-term systemic corticosteroid therapy during the study period (excluding inhalation or local use); and subjects who have received systemic corticosteroid therapy within 72 hours before cell infusion (excluding inhalation or local use);\n8. Subjects who have used PD-1 or PD-L1 monoclonal antibodies within 3 months before enrollment\n9. Pregnant or lactating women; and subjects planning to become pregnant within 1 year after infusion, during or after the treatment period;\n10. Uncontrolled active infections (excluding simple urinary tract infections or upper respiratory tract infections);\n11. Subjects who have received CAR-T therapy or other gene-modified cell therapy in the past;\n12. Patients after allogeneic transplantation, with no significant acute or chronic GVHD, and have discontinued immunosuppressive drugs for at least 1 month;\n13. Known allergy to any component of anti-CLL-1 CAR-NK cell infusion or chemotherapy regimen (cyclophosphamide and fludarabine);\n14. Any situation deemed by the investigator to compromise the safety of the subject or interfere with the purpose of the study, or deemed unsuitable for participation by the investigator;\n15. Afflicted with a condition that affects the ability to sign the informed consent form in writing or to comply with the study procedures; unwilling or unable to comply with the study requirements.","70 Years",{"count":101,"type":20},24,[23],"This study is a single-arm, open-label, dose-escalation clinical trial aimed at exploring the safety, tolerability, and pharmacokinetic characteristics of the CLL-1 CAR NK cells, as well as providing preliminary observations on its efficacy in subjects with relapsed\u002Frefractory acute myeloid leukemia.",[27,28],"2025-09-28",{"date":107,"type":34},"2025-09-30",{"date":109,"type":34},"2024-04-01",{"date":111,"type":20},"2027-03-15",{"name":113,"class":41},"Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine",{"id":115,"slug":116,"hasResults":11,"nctId":117,"briefTitle":118,"officialTitle":119,"acronym":4,"eligibilityCriteria":120,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":121,"targetDuration":4,"studyType":21,"phases":123,"briefSummary":125,"conditions":126,"keywords":133,"overallStatus":156,"whyStopped":4,"lastUpdateSubmitDate":157,"lastUpdatePostDateStruct":158,"startDateStruct":160,"completionDateStruct":162,"leadSponsor":164,"locationsCount":4},"100605053","phase-2-radioimmunotherapy-conditioning-with-131i--apamistamab-for-allogeneic-transplant-in-relapserefractory-aml-100605053","NCT07157514","Radioimmunotherapy Conditioning With 131I- Apamistamab for Allogeneic Transplant in Relapse\u002FRefractory AML","An Adaptive, Operationally Seamless Phase II \u002F III Study of 131I-apamistamab-Led Allogeneic Hematopoietic Stem Cell Transplant in Patients With Relapsed or Refractory Acute Myeloid Leukemia With Active Disease","Inclusion Criteria:\n\n1. Have active, relapsed, or refractory AML with ≥5% and ≤20% blasts in the marrow.\n2. 2R\u002FR AML is defined as one of the following: Primary induction failure after ≥2 cycles of therapy, first early relapse after remission \\\u003C6 months, relapse refractory to salvage combination therapy or second or subsequent relapse\n3. Documented CD45 expression by leukemic cells via flow cytometry.\n4. ≥18 years of age and not suitable for myeloablative conditioning regimen.\n5. Circulating blast count \\\u003C10,000\u002Fmm³ (hydroxyurea allowed).\n6. Calculated creatinine clearance (Cockcroft-Gault) \\>50 mL\u002Fmin.\n7. Adequate hepatic function: AST\u002FALT ≤2 × ULN; total bilirubin ≤1.5 × ULN (≤3 × ULN if due to underlying malignancy or Gilbert's).\n8. Karnofsky performance score ≥70.\n9. Expected survival \\>60 days.\n10. Central venous catheter line in place before study treatment.\n11. 8\u002F8 HLA-matched related or unrelated donor (HLA-A, HLA-B, HLA-C, DRB1).\n12. Women of childbearing potential must be surgically sterile or use acceptable contraception through 1-year post-transplant.\n13. Men with partners of childbearing potential must be surgically sterile or use acceptable contraception through 12 weeks after last dose.\n14. Able to understand procedures, provide informed consent, and comply with study requirements.\n\nExclusion Criteria:\n\n1. Positive human anti-mouse antibody (HAMA) at screening.\n2. \\>20% leukemic blasts in marrow.\n3. Prior radiation to maximally tolerated levels of any critical organ.\n4. Active CNS leukemia (blasts in CSF or CNS chloromas).\n5. Prior allogeneic or autologous HSCT.\n6. Candidates suitable for myeloablative conditioning.\n7. Clinically significant cardiac disease, including: NYHA Class III or IV heart failure, Clinically significant arrhythmias (ventricular tachycardia, ventricular fibrillation, Torsade de Pointes), Myocardial infarction with uncontrolled angina within 6 months, Clinically significant congestive heart failure or cardiomyopathy\n8. QTcF \\>450 ms after correction of electrolytes (unless paced rhythm or investigator deems eligible; cardiology consult optional).\n9. Positive HIV, HBV, or HCV test (exceptions: vaccinated HBV, or positive hepatitis markers with adequate organ function).\n10. Active, uncontrolled infection.\n11. Acute promyelocytic leukemia (t\\[15;17\\]).\n12. Active malignancy within 2 years, except: Myelodysplastic syndrome, Treated non-melanoma skin cancer, Completely resected stage 0-1 melanoma (\\>1 year from resection), Carcinoma in situ or cervical intraepithelial neoplasia, Organ-confined prostate cancer without progression\n13. Inability to tolerate diagnostic or therapeutic procedures, particularly radiation isolation.\n14. Received anti-leukemic therapy within 14 days prior to randomization (hydroxyurea allowed up to day of 131I-apamistamab).",{"count":122,"type":20},306,[24,124],"PHASE3","This is a multicenter, open-label study in people aged 18 and older with relapsed or refractory acute myeloid leukemia. It has two parts. In Phase 2, we are testing three radiation dose levels of 131I-apamistamab combined with fludarabine and low-dose whole-body radiation before stem cell transplant to find the safest and most effective dose. In Phase 3, patients will be randomly assigned to receive either this treatment combination or a standard of care regimen before transplant. The main goal is to see if the new approach helps people live longer. Phase 2 will enroll about 60 people, and Phase 3 will enroll about 246 people.",[72,127,29,55,59,128,129,130,131,132,28],"Myeloid Leukemia","Myelogenous Leukemia","Myelogenous Leukemia, Acute","Myelogenous Leukemia in Relapse","Transplant-Related Disorder","Allogeneic Disease",[55,65,134,135,136,137,28,138,139,140,141,142,143,144,145,146,147,148,149,150,151,152,153,154,155],"Acute Myeloid","Acute Bone Marrow Cell Transplant","Transplant","Bone Marrow HCT","Relapsed AML","I-131","Iomab","I131- Apamistamab","Iodine","Iodine-131","131-I AML","Iomab-B","Radioimmunotherapy","Allogeneic Transplant","Radiotherapy","CD45","Anti-CD45 antibody","Apamistamab","Fludarabine","Bone Marrow Transplant","Radiolabeled antibody therapy","131I -apamistamab","NOT_YET_RECRUITING","2025-09-04",{"date":159,"type":34},"2025-09-10",{"date":161,"type":20},"2026-01",{"date":163,"type":20},"2034-02",{"name":165,"class":90},"Actinium Pharmaceuticals",{"id":167,"slug":168,"hasResults":11,"nctId":169,"briefTitle":170,"officialTitle":171,"acronym":172,"eligibilityCriteria":173,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":174,"targetDuration":4,"studyType":21,"phases":176,"briefSummary":177,"conditions":178,"keywords":182,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":192,"lastUpdatePostDateStruct":193,"startDateStruct":195,"completionDateStruct":197,"leadSponsor":199,"locationsCount":201},"100351035","phase-1-clinical-trial-to-evaluate-the-safety-tolerability-pharmacokinetics-and-pharmacodynamics-of-tuspetinib-hm43239-in-patients-with-relapsed-or-refractory-acute-myeloid-leukemia-100351035","NCT03850574","Clinical Trial to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tuspetinib (HM43239) in Patients With Relapsed or Refractory Acute Myeloid Leukemia","A Phase 1\u002F2 Open Label, Multicenter, Dose Escalation and Expansion Study of the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of HM43239 in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML)","TUSCANY","Inclusion Criteria for Parts A\u002FB\u002FC:\n\n* Study participant is defined as having morphologically documented primary or secondary AML, MDS-IB2 (≥ 10% bone marrow blasts), or CMML by the World Health Organization (WHO) criteria (2016), and fulfills one of the following:\n\n  1. Refractory to at least 1 cycle of prior therapy\n  2. Relapsed after achieving remission with a prior therapy\n* Study participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2.\n* Study participant's interval from prior treatment to time of study drug administration is at least 2 weeks for cytotoxic agents (except hydroxyurea given for controlling blast cells), at 4 weeks for biologic or cellular immunotherapies, or least 5 half-lives for prior experimental agents or noncytotoxic agents, including immunosuppressive therapy post hematopoietic stem cell transplantation (HSCT). Upon discussion with the Medical Monitor, a shorter than stated washout period may be considered provided that the study participant has recovered from any clinically relevant safety issue and recovered to Grade ≤ 1 toxicity from prior therapies.\n* Study participant must meet the following criteria as indicated on the clinical laboratory tests.\n\n  1. Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3× institutional upper limit normal (ULN)\n  2. Total serum bilirubin ≤ 1.5× institutional ULN\n  3. Creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of \\> 45 mL\u002Fmin.\n* Study participant is suitable for oral administration of study drug and has minimum life expectancy (≥ 3 months)\n* Female study participants must be either:\n* Of non-childbearing potential\n\n  1. Post-menopausal (defined as at least 1 year without any menses) prior to screening, or\n  2. Documented surgically sterile or status post hysterectomy (at least 1 month prior to screening)\n* Or, if of childbearing potential,\n\n  1. Must have a negative serum or urine pregnancy test at screening (within 72 hours prior to start of treatment), and\n  2. Must use an acceptable highly effective method of birth control starting at screening and throughout the study period and for 90 days after the final study drug administration.\n* Female study participants must not be breastfeeding at screening and during the study period, and for 90 days after the final study drug administration\n* Female study participants must not donate ova starting at screening and throughout the study period, and for 90 days after the final study drug administration.\n* Male study participants and their female spouse\u002Fpartners who are of childbearing potential must be using highly effective contraception starting at screening and continue throughout the study period and for 90 days after the final study drug administration.\n* Male study participants must not donate sperm starting at screening and throughout the study period and for 90 days after the final study drug administration.\n* Study participant agrees not to participate in another interventional study while on treatment.\n\nExclusion Criteria for Parts A\u002FB\u002FC:\n\nStudy participants must not enter the study if any of the following exclusion criteria are met:\n\n* Study participant was diagnosed with acute promyelocytic leukemia (APL).\n* Study participant has known BCR-ABL-positive leukemia.\n* Study participant has an active malignancy other than AML, MDS-IB2, or CMML.\n* Study participant has persistent non-hematological toxicities of ≥ Grade 2 (CTCAE v4.03), with symptoms and objective findings, from prior AML, MDS-IB2, or CMML treatment (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, radiation, or surgery)\n* Study participant has had hematopoietic stem cell transplant (HSCT) and meets any of the following criteria:\n\n  1. Has undergone HSCT within the 2-month period prior to the first study dose\n  2. Has clinically significant graft-versus-host-disease (GVHD) requiring treatment\n  3. Has ≥ Grade 2 persistent non-hematological toxicity related to the transplant\n  4. Had a donor lymphocyte infusion (DLI) ≤ 30 days prior to the first study dose.\n* Study participant has meningeal or central nervous system (CNS) involvement with leukemia or other CNS disease related to underlying and secondary effects of malignancy.\n* Study participant has disseminated intravascular coagulation abnormality (DIC).\n* Study participant has had major surgery within 4 weeks prior to the first study dose.\n* Study participant has had radiation therapy within 4 weeks prior to the first study dose.\n* Study participant has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or study participant with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 3 months prior to study entry results in a left ventricular ejection fraction (LVEF) that is ≥ 45%.\n* Study participant has any of the following cardiac abnormalities of history:\n\n  1. Study participant has any clinically important abnormalities in rhythm, conduction or morphology of resting ECG, e.g., complete left bundle branch block, third-degree heart block, second-degree heart block, or PR interval \\> 250 milliseconds (ms).\n  2. Study participant has a mean QT interval (QTc) by Friderica's method (QTcF) \\> 450 ms in three successive screening measurements.\n  3. Study participant has any factors that increase the risk of QTc prolongation or risk of arrhythmic events, such as congenital long QT, syndrome, family history of long QT syndrome.\n* Study participant is known to have active infection including any identified active COVID-19 infection.\n* Study participant is known to have human immunodeficiency virus infection.\n* Study participant has known active hepatitis B or C, or other active hepatic disorder.\n* Study participant has any condition which, in the Investigator's opinion, makes the study participant unsuitable for study participation.\n* Study participant has a history of Grade 3 or 4 non-hematologic toxicity related to tyrosine kinase inhibitor.\n\nInclusion Criteria for Part D:\n\n* Study participant is defined as having morphologically documented newly diagnosed previously untreated primary or secondary AML by the World Health Organization (WHO) criteria (2016) and considered ineligible to receive intensive chemotherapy.\n\nStudy participants ≥ 75 years of age are considered ineligible to receive intensive chemotherapy if they meet any of the following criteria:\n\n1. ECOG Performance Status of 2 or 3;\n2. Cardiac history of congestive heart failure (CHF) requiring treatment or ejection fraction ≤ 50% or chronic stable angina;\n3. Diffusing capacity of the lung for carbon monoxide (DLCO) ≤ 65% or forced expiratory volume during the first second (FEV1) ≤ 65%;\n4. Creatinine clearance ≥ 30 mL\u002Fmin to \\\u003C 45 ml\u002Fmin;\n5. Moderate hepatic impairment with total bilirubin \\> 1.5 to ≤ 3.0×ULN;\n6. Any other comorbidity that the Investigator judges to be incompatible with intensive chemotherapy must be reviewed and approved by the Medical Monitor during screening and prior to treatment.\n\nStudy participants are considered ineligible to receive intensive chemotherapy based on their age being ≥ 75 years.\n\n* Study participant \\\u003C 75 years has an ECOG performance status ≤ 2; study participant ≥ 75 years has an ECOG performance status 0-2.\n* Study participant must meet the following criteria as indicated on the clinical laboratory tests:\n\n  1. Serum AST and ALT ≤ 3× institutional ULN unless considered due to leukemic organ involvement.\n  2. Total serum bilirubin ≤ 3x institutional ULN unless considered due to leukemic organ involvement for study participants \\\u003C 75 years; total serum bilirubin ≤ 1.5x institutional ULN (or ≤ 3x institutional ULN if documented history of Gilbert's syndrome) unless considered due to leukemic organ involvement for study participants ≥ 75 years.\n  3. Creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of ≥ 30 mL\u002Fmin for study participants \\\u003C 75 years; creatinine clearance as calculated by the Cockcroft-Gault formula or measured by 24-h urine collection of ≥ 45 mL\u002Fmin for study participants ≥ 75 years.\n* Study participant is suitable for oral administration of study drug and has minimum life expectancy (≥ 3 months)\n* Female study participants must be either:\n* Of non-childbearing potential\n\n  1. Post-menopausal (defined as at least 1 year without any menses) prior to screening, or\n  2. Documented surgically sterile or status post hysterectomy (at least 1 month prior to screening)\n* Or, if of childbearing potential,\n\n  1. Must have a negative serum or urine pregnancy test at screening (within 72 hours prior to start of treatment), and\n  2. Must use an acceptable highly effective contraception starting at screening and throughout the study period and for 90 days after the final study drug administration.\n* Female study participants must not be breastfeeding at screening and during the study period, and for 90 days after the final study drug administration.\n* Female study participants must not donate ova starting at screening and throughout the study period, and for 90 days after the final study drug administration.\n* Male study participants and their female spouse\u002Fpartners who are of childbearing potential must be using highly effective contraception starting at screening and continue throughout the study period and for 90 days after the final study drug administration.\n* Male study participants must not donate sperm starting at screening and throughout the study period and for 90 days after the final study drug administration.\n* Study participant agrees not to participate in another interventional study while on treatment.\n\nExclusion Criteria for Part D:\n\nStudy participants must not enter the study if any of the following exclusion criteria are met:\n\n* Study participant was diagnosed with acute promyelocytic leukemia (APL).\n* Study participant has known BCR-ABL-positive leukemia.\n* Study participant has an active malignancy other than AML.\n* Study participant has received treatment with the following:\n\n  1. An HMA, VEN (or other BCL-2 inhibitor), a tyrosine kinase inhibitor (TKI), a FLT3 inhibitor (FLT3i), a hematopoietic stem cell transplant (HSCT), and\u002For a chemotherapeutic agent for antecedent myeloid neoplasm (Note: Prior chemotherapy for solid tumors considered in remission is allowed.)\n  2. CAR-T cell therapy\n  3. Experimental therapies for antecedent myeloid neoplasms\n  4. Current participation in another research or observational study\n* Study participant has persistent non-hematological toxicities of ≥ Grade 2 (CTCAE v4.03), with symptoms and objective findings, from treatment for antecedent myeloid neoplasms (including chemotherapy, kinase inhibitors, immunotherapy, experimental agents, immunosuppressive therapy, radiation, or surgery).\n* Study participant has meningeal or central nervous system (CNS) involvement with leukemia or other CNS disease related to underlying and secondary effects of malignancy.\n* Study participant has disseminated intravascular coagulation abnormality (DIC).\n* Study participant has had major surgery within 4 weeks prior to the first study dose.\n* Study participant has had radiation therapy within 4 weeks prior to the first study dose.\n* Study participant has congestive heart failure New York Heart Association (NYHA) class 3 or 4, or study participant with a history of congestive heart failure NYHA class 3 or 4 in the past, unless a screening echocardiogram or multigated acquisition (MUGA) scan performed within 3 months prior to study entry results in a left ventricular ejection fraction (LVEF) that is ≥ 45%.\n* Study participant has any of the following cardiac abnormalities of history:\n\n  1. Study participant has any clinically important abnormalities in rhythm, conduction or morphology of resting ECG, e.g., complete left bundle branch block, third-degree heart block, second-degree heart block, or PR interval \\> 250 milliseconds (ms).\n  2. Study participant has a mean QT interval (QTc) by Friderica's method (QTcF) \\> 450 ms in three successive screening measurements.\n  3. Study participant has any factors that increase the risk of QTc prolongation or risk of arrhythmic events, such as congenital long QT, syndrome, family history of long QT syndrome.\n* Study participant is known to have active infection, including any identified active COVID-19 infection.\n* Study participant is known to have human immunodeficiency virus infection.\n* Study participant has known active hepatitis B or C, or other active hepatic disorder.\n* Study participant has any condition which, in the Investigator's opinion, makes the study participant unsuitable for study participation, including a chronic respiratory disease that requires continuous oxygen, or a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, hepatic, or cardiovascular disease, or any other medical condition or known hypersensitivity to any of the study medications including excipients of VEN and AZA that in the opinion of the Investigator would adversely affect participating in this study.\n* Study participant exhibits evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal) or other medical conditions (e.g., infection, heart failure, COPD flare, etc.).\n* Study participant has a white blood cell count \\> 25 × 10\\^9\u002FL. (Treatment with hydroxyurea or use of leukapheresis are permitted to meet this criterion.)",{"count":175,"type":20},240,[23,24],"The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 (MDS-IB2), or chronic myelomonocytic leukemia (CMML) that is relapsed or refractory after at least one line of prior therapy, or in study participants with newly diagnosed AML. Tuspetinib will be administered as a single agent or in combination with other drugs (venetoclax or venetoclax plus azacitidine), as specified for each part of the study.",[179,28,27,180,181],"Leukemia, Myeloid, Acute","Myelodysplastic Syndrome With Excess Blasts-2","Chronic Myelomonocytic Leukemia",[183,55,67,184,185,186,187,181,188,189,190,191],"Tuspetinib","Relapsed","Refractory","Myelodysplastic Syndromes with Increased Blasts Grade 2","MDS-IB2","CMML","Venetoclax","Azacitidine","Newly Diagnosed","2025-08-20",{"date":194,"type":34},"2025-08-26",{"date":196,"type":34},"2019-03-11",{"date":198,"type":20},"2027-04",{"name":200,"class":90},"Aptose Biosciences Inc.",34,{"id":203,"slug":204,"hasResults":11,"nctId":205,"briefTitle":206,"officialTitle":207,"acronym":208,"eligibilityCriteria":209,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":210,"targetDuration":4,"studyType":21,"phases":212,"briefSummary":213,"conditions":214,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":218,"lastUpdatePostDateStruct":219,"startDateStruct":221,"completionDateStruct":223,"leadSponsor":225,"locationsCount":227},"100517781","phase-2-study-investigating-the-efficacy-and-safety-of-the-addition-of-oral-azacitidine-to-salvage-treatment-by-gilteritinib-in-subjects-18-years-of-age-with-relapsedrefractory-flt3-mutated-acute-myeloid-leukemia-100517781","NCT06022003","Study Investigating the Efficacy and Safety of the Addition of Oral-azacitidine to Salvage Treatment by Gilteritinib in Subjects ≥18 Years of Age With Relapsed\u002FRefractory FLT3-mutated Acute Myeloid Leukemia","Open-label, Phase 2 Study Investigating the Efficacy and Safety of the Addition of Oral-azacitidine to Salvage Treatment by Gilteritinib in Subjects ≥18 Years of Age With Relapsed\u002FRefractory FLT3-mutated Acute Myeloid Leukemia","OGILAR","Inclusion Criteria:\n\n1. Confirmed diagnosis of acute myeloid leukemia (AML) according to world health organization (WHO) 2016 classification\n2. Presence of FLT3-mutation(s) at inclusion: in case of FLT3-ITD, the ITD\u002Fwt ratio must be \\> 0.05 ; in case of FLT3-TKD, the mutation must be at D835 or I836 position with a VAF \\> 5% by NGS.\n3. Subjects must be primary refractory or relapsed (R\u002FR) to 1st line intensive chemotherapy (ICT) for AML. Hydroxyurea is allowed for the control of peripheral leukemic blasts in patients with leukocytosis.\n\n3a. Primary refractory is defined as no CR or CRi after at least one course of ICT (including \"7+3\", gemtuzumab ozogamycin (GO)-based and CPX-351, including or not midostaurine) or two courses (maximum 4) of AZA and venetoclax 3b. Relapse after 1st line ICT for AML is defined as the first hematologic relapse with bone marrow blasts \\>5% after one line of treatment for AML that includes at least one course of ICT (one line of treatment for AML can include induction, re-induction, consolidation, allogeneic HSCT and maintenance) 3c. Relapse after 1st line non intensive chemotherapy for AML is defined as the first hematologic relapse with bone marrow blasts \\>5% after or during treatment by AZA venetoclax regardless of number of cycles 4. 1st line intensive treatment may or may not include previous treatment by tyrosine kinase inhibitor (TKI) except gilteritinib.\n\n5\\. Patients who never received oral azacitidine 6. Age ≥ 18 years 7. Adequate baseline organ function defined by the criteria below:\n\n* adequate renal function as demonstrated by a creatinine clearance ≥ 50 ml\u002Fmin; calculated by the Cockcroft gault formula or measured by 24-hours urine collection\n* aspartate aminotransferase (AST) ≤ 2.5 × ULN\n* alanine aminotransferase (ALT) ≤ 2.5× ULN\n* bilirubin ≤ 1.5 × ULN\n* adequate cardiac function with LVEF ≥45% 8. ECOG \\\u003C 3 (appendix 1) 9. Absence of any psychological, familial, sociological, or geographical conditions potentially hampering compliance with the study protocol and follow-up schedule 10. Patient is suitable for oral administration of study drug. 11. A female subject is eligible to participate if she is not pregnant and at least one of the following conditions applies: 9a. Not a woman of childbearing potential (WOCBP) as defined in post-menopausal (defined as at least 1 year without any menses) prior to Screening, or documented as surgically sterile (at least 1 month prior to Screening) 9b. WOCBP agrees to follow the contraceptive treatment starting at screening and continue throughout the study period, and for at least 180 days after the final study drug administration 12. Patient must be affiliated to the french social security (health insurance) 13. Signed written informed consent for the study 14. Female subject must agree not to breastfeed starting at screening and throughout the study period, and for 60 days after the final study drug administration.\n\n  15\\. Female subject must not donate ova starting at screening and throughout the study period, and for 180 days after the final study drug administration.\n\n  16\\. A male subject with female partner(s) of childbearing potential must agree to use contraception starting at screening and continue throughout the study period, for at least 120 days after the final study drug administration.\n\n  17\\. Male subject must not donate sperm starting at screening and throughout the study period and for 120 days after the final study drug administration.\n\nExclusion criteria\n\n1\\. Subjects with any of the following current or previous diagnoses:\n\n1a. AML secondary to prior myeloproliferative syndrome (MPN)\n\n1b. Acute promyelocytic leukemia (APL) and core binding factor (CBF) AML\n\n1c. DNA fragility or bone marrow (BM) failure syndromes\n\n1d. Blastic plasmacytoid dendritic cell neoplasm\n\n1e. Acute lymphoblastic leukemia including ambiguous lineage 2. Patients ≥ 3rd line of treatment, HSCT being not considered as a line of treatment 3. Patients previously treated by AZA as single agent for AML are not allowed 4. Subjects that have previously been treated by gilteritinib 5. Subjects that have previously been treated by oral azacitidine 6. Clinically active central nervous system (CNS) leukemia 7. Subjects who have received more than 1 prior allogeneic HSCT 8. Subjects who have relapsed within 100 days after allogeneic HSCT 9. Presence of Grade 2 or above graft-versus-host disease (GVHD), including acute, chronic, or overlap; or escalation of therapy for GVHD within 14 days prior to randomization 10. Subject requires treatment with concomitant drugs that are strong inducers of cytochrome P450 (CYP)3A (Annexe 7) 11. Subject requires treatment with concomitant drugs that are strong inhibitors or inducers of P-gp with the exception of drugs that are considered absolutely essential for the care of the subject (Annexe 7) 12. Severe liver disease (e.g. cirrhosis, non-alcoholic steatohepatitis, sclerosing cholangitis or hyperbilirubinemia) 13. Participant has clinically significant abnormality of coagulation profile, such as disseminated intravascular coagulation (DIC).\n\n14\\. Subject exhibiting evidence of other clinically significant uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal).\n\n15\\. Isolated extramedullary leukemia relapse 16. History of another malignancy within the past 3 years except basal cell carcinoma of the skin or cervix in situ carcinoma 17. Any other serious medical condition, laboratory abnormalities or psychiatric illness that would place the participant at an unacceptable risk or prevent them from giving informed consent 18. Severe medical or mental condition precluding the administration of protocol treatments 19. persons deprived of their liberty by judicial or administrative decision, persons subject to a legal protection measure (guardianship, curatorship, legal protection), persons under psychiatric care 20. Other comorbidity that the physician judges to be incompatible with conventional intensive chemotherapy which must be reviewed and approved by the study medical monitor before study enrolment 21. Subject with Positive HIV test (due to potential drug-drug interactions). HIV testing will be performed at screening, if required per local guidelines or institutional standards. Subject known to be positive for hepatitis B virus (HBV), or hepatitis C virus (HCV) infection. Inactive hepatitis carrier status with undetectable PCR viral load on antivirals (non-exclusionary medications) are not excluded 22. Known hypersensitivity to the study medication 23. Subject has congestive heart failure classified as New York Heart Association Class III and IV unless a screening echocardiogram performed within 3 months prior to study entry results in a left ventricular ejection fraction that is ≥ 45% 24. Subject with mean Fridericia-corrected QT interval (QTcF) \\> 450 ms at screening based on central reading 25. Subject with a history of Long QT Syndrome at screening",{"count":211,"type":20},33,[24],"Approximately 30% of adult AML subjects are refractory to induction therapy. Furthermore, of those who achieve CR, approximately 75% will relapse. FLT3-mutated AML comprise an especially poor prognosis group.\n\nUntil now, there was no established standard for relapsed subjects with FLT3 mutations and less than 20% will achieve CR with subsequent treatment.\n\nIn phase 3 Study ADMIRAL Trial, gilteritinib has resulted in CRc in over 25% of subjects receiving 120 mg\u002Fday before on study HSCT. With this treatment, the median overall survival is at 9.3 months, furthermore, gilteritinib was well tolerated at the proposed doses. This study has been designed for R\u002FR patients for which gilteritinib as single agent has been showed to be superior to high- and low-intensity chemotherapy (Perl, NEJM 2019, Supp Table S4) and patients included in this study will receive this treatment. Beyond high- or low-intensity chemotherapy, other options available are best supportive car or other clinical trials.\n\nThe aim of this study is to assess the efficacy and safety of the addition of oral-azacitidine to salvage treatment by gilteritinib in subjects ≥18 years of age with relapsed\u002Frefractory FLT3-mutated acute myeloid leukemia",[215,28,27,216,217],"AML, Adult","FLT3-TKD Mutation","FLT3-ITD","2025-03-20",{"date":220,"type":34},"2025-03-24",{"date":222,"type":34},"2024-01-13",{"date":224,"type":20},"2027-10",{"name":226,"class":41},"French Innovative Leukemia Organisation",20,{"id":229,"slug":230,"hasResults":11,"nctId":231,"briefTitle":232,"officialTitle":233,"acronym":4,"eligibilityCriteria":234,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":235,"targetDuration":4,"studyType":21,"phases":237,"briefSummary":238,"conditions":239,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":241,"lastUpdatePostDateStruct":242,"startDateStruct":244,"completionDateStruct":246,"leadSponsor":248,"locationsCount":42},"100572765","phase-1-chiglitazar-sodium-combined-with-venetoclax-and-azacitidine-cva-for-the-treatment-of-rr-aml-100572765","NCT06737523","Chiglitazar Sodium Combined With Venetoclax and Azacitidine (CVA) for the Treatment of R\u002FR AML","A Multicenter, Prospective, Single-arm Clinical Study of Chiglitazar Sodium Combined With Venetoclax and Azacitidine (CVA) for the Treatment of Refractory\u002FRelapsed Acute Myeloid Leukemia (R\u002FR AML)","Inclusion Criteria:\n\n1. Acute myeloid leukemia (AML) confirmed by histology. Relapsed or refractory AML, including: a) Refractory disease is defined as no remission after at least one prior treatment. b) Disease relapse is defined as 5% or more blasts in the bone marrow after remission. c) Patients with AML arising from myelodysplastic syndromes (including CMML) or myeloproliferative neoplasms (secondary AML, t-AML) are also eligible;\n2. Age ≥18 years, male or female, with an expected survival of more than 3 months;\n3. Estimated creatinine clearance ≥ 50 mL\u002Fmin;\n4. AST and ALT ≤ 3.0 x ULN (unless considered due to leukemia organ involvement), bilirubin ≤ 3.0 x ULN (unless considered due to leukemia organ involvement);\n5. ECOG ≤ 2;\n6. Subjects are non-pregnant or using contraceptive measures during treatment;\n7. Capable of understanding and voluntarily providing informed consent.\n\nExclusion Criteria:\n\n1. Acute promyelocytic leukemia (APL);\n2. Active central nervous system leukemia;\n3. Patients with clinically significant QTc interval prolongation (males \\> 450 ms; females \\> 470 ms), ventricular tachycardia and atrial fibrillation, second-degree heart block, history of myocardial infarction and congestive heart failure within the year prior to enrollment, and patients with clinically symptomatic coronary heart disease requiring medication;\n4. Active, uncontrolled severe infection;\n5. Other non-myeloid malignancies within the past 2 years;\n6. Mental disorders that would impede study participation;\n7. Previous solid organ transplantation (pre-treatment with SCT is allowed, but not allowed if the patient has GVHD or is still receiving immunosuppressive\u002FGVHD treatment);\n8. Any other conditions that, in the opinion of the investigator, make the patient unsuitable to participate in this trial.",{"count":236,"type":20},30,[23],"To investigate the safety and efficacy of the CVA regimen, composed of Chiglitazar Sodium in combination with Venetoclax and Azacitidine, in the treatment of patients with refractory\u002Frelapsed acute myeloid leukemia (R\u002FR AML).",[55,240,28],"Relapse","2025-02-21",{"date":243,"type":34},"2025-02-24",{"date":245,"type":20},"2025-03-15",{"date":247,"type":20},"2026-12-31",{"name":249,"class":41},"The First Affiliated Hospital of Xiamen University",{"id":251,"slug":252,"hasResults":11,"nctId":253,"briefTitle":254,"officialTitle":255,"acronym":4,"eligibilityCriteria":256,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":17,"enrollmentInfo":257,"targetDuration":4,"studyType":21,"phases":259,"briefSummary":260,"conditions":261,"keywords":266,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":272,"lastUpdatePostDateStruct":273,"startDateStruct":275,"completionDateStruct":277,"leadSponsor":279,"locationsCount":281},"100540447","phase-2-safety-and-efficacy-of-avapritinib-in-relapsed-or-refractory-pediatric-cbf-aml-with-kit-mutation-100540447","NCT06316960","Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation","A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed\u002FRefractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation","Inclusion Criteria:\n\n1. Gender unlimited;\n2. Under 18 years;\n3. Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).\n4. Presence of t(8;21)\u002FRUNX1::RUNX1T1 or inv(16)\u002Ft(16;16)\u002FCBFβ::MYH11;\n5. KIT mutation;\n6. Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;\n7. Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;\n8. No active infections;\n9. Liver function: Tbil ≤2×ULN, ALT\u002FAST ≤3×ULN, creatinine clearance ≥50ml\u002Fmin;\n10. ECOG score \\\u003C2;\n11. Expected survival time \\>12 weeks;\n12. Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.\n\nExclusion Criteria:\n\n1. Have received prior treatment with avapritinib;\n2. Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;\n3. Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);\n4. Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction；\n5. With other malignancies requiring treatment；\n6. Already enrolled in another interventional clinical study；\n7. The researchers determined that the individual is not suitable to participate in this trial.",{"count":258,"type":20},50,[24],"The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.",[262,263,28,264,265],"AML, Childhood","Relapse\u002FRecurrence","Core Binding Factor Acute Myeloid Leukemia","C-KIT Mutation",[267,268,269,270,271],"Avapritinib","KIT","CBF AML","Childhood","Relapsed\u002FRefractory","2024-08-21",{"date":274,"type":34},"2024-08-22",{"date":276,"type":34},"2024-03-01",{"date":278,"type":20},"2027-03",{"name":280,"class":41},"Children's Hospital of Soochow University",12]