^
    1d
    Venetoclax Plus Hypomethylating Agents and Subcutaneous Cytarabine for CEBPA-Mutated AML (clinicaltrials.gov)
    P1/2, N=29, Recruiting, The First Affiliated Hospital of Soochow University
    New P1/2 trial
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    CEBPA (CCAAT Enhancer Binding Protein Alpha)
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    Venclexta (venetoclax) • cytarabine • azacitidine
    1d
    A Study of Gilteritinib in Combination With Ivosidenib or Enasidenib in People With Acute Myeloid Leukemia (AML) (clinicaltrials.gov)
    P1, N=36, Recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Feb 2026 --> Feb 2027 | Trial primary completion date: Feb 2026 --> Feb 2027
    Trial completion date • Trial primary completion date
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    FLT3 (Fms-related tyrosine kinase 3) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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    IDH1 mutation • IDH2 mutation • FLT3 mutation
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    Xospata (gilteritinib) • Tibsovo (ivosidenib) • Idhifa (enasidenib)
    1d
    ALLG AMLM28-A3. A randomised study of ambulatory management compared to standard of care for fever related hospitalisation in patients with AML receiving Venetoclax and Azacitidine – a domain of Achieving Durable remissions via Adaptive Pro-survival Targeting in AML (ADAPT) (ACTRN12626000278336)
    P=N/A, N=100, Not yet recruiting, Australasian Leukaemia and Lymphoma Group
    New trial
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    Venclexta (venetoclax) • azacitidine
    1d
    SC-Het-GM-CCS: Symptom Cluster Heterogeneity and Gut Microbiota Mechanisms in Childhood Cancer Survivors (clinicaltrials.gov)
    P=N/A, N=600, Recruiting, The Children's Hospital of Zhejiang University School of Medicine
    New trial
    1d
    A Study of BL-M11D1 in Combination With Cytarabine + Daunorubicin or Venetoclax + Azacitidine in Patients With Acute Myeloid Leukemia (clinicaltrials.gov)
    P2/3, N=216, Not yet recruiting, Sichuan Baili Pharmaceutical Co., Ltd. | N=80 --> 216 | Initiation date: Dec 2025 --> Mar 2026
    Enrollment change • Trial initiation date
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    Venclexta (venetoclax) • cytarabine • azacitidine • daunorubicin • BL-M11D1
    1d
    Mislocalisation of FLT3-ITD receptor contributes to MV4-11 leukaemia cell resistance to antibody-drug conjugate. (PubMed, J Enzyme Inhib Med Chem)
    To evaluate the impact of this trafficking difference, we synthesised an anti-FLT3 mAb-MMAE, linked via a Val-Cit-PAB linker at the Fc N-glycan, which exhibited lower cytotoxicity in MV4-11 than THP-1 cells, indicating that the impaired lysosomal trafficking of FLT3-ITD limits drug release and reduces ADC potency. These findings highlight that effective lysosomal targeting is essential for ADC activity and suggest that optimising linker design or restoring lysosome trafficking may enhance FLT3-targeted ADC in AML.
    Journal
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    FLT3 (Fms-related tyrosine kinase 3)
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    FLT3-ITD mutation • FLT3 mutation
    1d
    Evaluation of Drugs with Selective Inhibitors Targeting the Anti-Apoptotic Protein B-cell Lymphoma 2 (BCL-2) with Pro-Apoptotic and Antineoplastic Activities in Grade IV Glioblastoma. (PubMed, Turk Neurosurg)
    Venetoclax, navitoclax, and obatoclax represented significant advances in apoptosis-targeted therapy, with venetoclax emerging as the most clinically successful agent. However, resistance mechanisms and side effects were significant challenges, necessitating further preclinical and clinical studies to optimize the therapeutic potential of these agents.
    Journal
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    BCL2 (B-cell CLL/lymphoma 2) • MCL1 (Myeloid cell leukemia 1) • BCL2L1 (BCL2-like 1)
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    Venclexta (venetoclax) • navitoclax (ABT 263) • obatoclax (GX 15-070)
    1d
    Impact of FMS-like tyrosine kinase 3 inhibitor maintenance on post-transplant outcomes in acute myeloid leukemia with FMS-like tyrosine kinase 3 mutations: a real-world German registry analysis highlighting sorafenib. (PubMed, Haematologica)
    We analyzed 523 adults with FLT3-ITD AML in first complete remission who underwent allo-HCT between 2011 and 2023 in 13 German transplant centers participating in the national DRST registry; 22% received FLT3i maintenance (sorafenib 49%, midostaurin 37%, gilteritinib 5%, unknown 9%). Sorafenib maintenance (n=50) demonstrated superior efficacy with 5-year OS of 85% versus 62% (HR 2.979, p=0.0045) and RFS of 84% versus 55% (HR 2.771, p=0.0043) compared to no maintenance. These real-world findings, while limited by the retrospective design and potential selection bias, align with randomized trial data and support the use of FLT3i maintenance as part of post-transplant care for FLT3-ITD AML.
    Journal • Real-world evidence
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    FLT3 (Fms-related tyrosine kinase 3)
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    FLT3 mutation
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    sorafenib • Xospata (gilteritinib) • midostaurin
    1d
    Aberrant fucosylation of extracellular vesicles remodels the vascular microenvironment and promotes chemoresistance in myelodysplastic syndromes and acute myeloid leukemia. (PubMed, Haematologica)
    Here, we show that small extracellular vesicles (sEVs) derived from leukemic cells resistant to decitabine (DAC-R), a commonly used HMA, promote vascular permeability by downregulating tight junction proteins, including ZO-1, occludin, and claudin5, in endothelial cell monolayers...Functionally, vascular remodeling driven by fucosylated sEVs promotes leukemic dissemination, suggesting that disruption of vascular homeostasis represents an additional layer of therapeutic resistance. These findings define a TWIST1-FUT4-LeX-ICAM3 axis and highlight glycosylation as a critical mediator of vascular microenvironment remodeling in MDS/AML.
    Journal
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    TWIST1 (Twist Family BHLH Transcription Factor 1) • TJP1 (Tight Junction Protein 1) • CLDN5 (Claudin 5) • FUT4 (Fucosyltransferase 4) • OCLN (Occludin)
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    decitabine
    2d
    Trial of Novel Anti-leukemia Agents in Flu/Mel RIC Transplant for Myeloid Malignancies (clinicaltrials.gov)
    P1, N=20, Recruiting, University of Alabama at Birmingham | Not yet recruiting --> Recruiting | Initiation date: Feb 2026 --> Jul 2025
    Enrollment open • Trial initiation date
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    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • HLA-B (Major Histocompatibility Complex, Class I, B) • HLA-DPB1 (Major Histocompatibility Complex, Class II, DP Beta 1) • HLA-C (Major Histocompatibility Complex, Class I, C)
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    Venclexta (venetoclax) • decitabine • melphalan
    2d
    VICEROY: A Study of Gilteritinib, Venetoclax and Azacitidine as a Combined Treatment for People Newly Diagnosed With Acute Myeloid Leukemia (clinicaltrials.gov)
    P1/2, N=70, Active, not recruiting, Astellas Pharma Global Development, Inc. | Recruiting --> Active, not recruiting
    Enrollment closed
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    FLT3 (Fms-related tyrosine kinase 3)
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    FLT3 mutation
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    Venclexta (venetoclax) • Xospata (gilteritinib) • azacitidine
    2d
    HAP2HCT: TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies (clinicaltrials.gov)
    P2, N=69, Active, not recruiting, St. Jude Children's Research Hospital | N=140 --> 69 | Trial completion date: Aug 2025 --> Jun 2026
    Enrollment change • Trial completion date
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    FLT3 (Fms-related tyrosine kinase 3) • RUNX1 (RUNX Family Transcription Factor 1) • KMT2A (Lysine Methyltransferase 2A) • ETV6 (ETS Variant Transcription Factor 6) • WT1 (WT1 Transcription Factor) • CREBBP (CREB binding protein) • NUP98 (Nucleoporin 98 And 96 Precursor 2) • NSD1 (Nuclear Receptor Binding SET Domain Protein 1) • HOXA9 (Homeobox A9) • NUP214 (Nucleoporin 214) • CBFA2T3 (CBFA2/RUNX1 Partner Transcriptional Co-Repressor 3) • GLIS2 (GLIS Family Zinc Finger 2) • KAT6A (Lysine Acetyltransferase 6A) • KDM5A (Lysine Demethylase 5A) • DEK (DEK Proto-Oncogene) • AFDN (Afadin, Adherens Junction Formation Factor) • MLLT10 (MLLT10 Histone Lysine Methyltransferase DOT1L Cofactor)
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    FLT3-ITD mutation
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    cyclophosphamide • Blincyto (blinatumomab) • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)