^
    13d
    A prospective clinical study to evaluate the efficacy of dexitabine in maintenance therapy after hematopoietic stem cell transplantation in juvenile myelomonocytic leukemia (ChiCTR2400091166)
    P=N/A, N=40, Not yet recruiting, The Seventh Affiliated Hospital, Sun Yat-sen University; The Seventh Affiliated Hospital, Sun Yat-sen University
    New trial
    |
    KRAS (KRAS proto-oncogene GTPase) • ABL1 (ABL proto-oncogene 1) • NRAS (Neuroblastoma RAS viral oncogene homolog) • NF1 (Neurofibromin 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    KRAS mutation • NRAS mutation • NF1 mutation • PTPN11 mutation • CBL mutation
    22d
    Increased response to granulocyte-macrophage colony-stimulating factor in peripheral blood cells and transient manifestations mimicking juvenile myelomonocytic leukemia in a male patient with NEMO deficiency caused by a deep intronic pathogenic variant of IKBKG. (PubMed, Immunol Med)
    The patient with NEMO deficiency demonstrated JMML-like manifestation and severe inflammation. PBMCs of the patient demonstrated increased RAS signaling activation with unknown pathophysiology.
    Journal
    |
    CSF2 (Colony stimulating factor 2)
    |
    CSF2 elevation
    25d
    ADVL1521: Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia (clinicaltrials.gov)
    P2, N=10, Active, not recruiting, National Cancer Institute (NCI) | Trial completion date: Sep 2024 --> Oct 2025
    Trial completion date
    |
    NF1 (Neurofibromin 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    NF1 mutation • RAS mutation • PTPN11 mutation • CBL mutation
    |
    Mekinist (trametinib) • omipalisib (GSK2126458)
    2ms
    Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation (clinicaltrials.gov)
    P2, N=170, Active, not recruiting, St. Jude Children's Research Hospital | Recruiting --> Active, not recruiting | N=52 --> 170
    Enrollment closed • Enrollment change
    |
    Rituxan (rituximab) • cyclophosphamide • Blincyto (blinatumomab) • sirolimus • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)
    2ms
    SL03-OHD-104: Phase 1 Study of Shattuck Labs (SL)-172154 in Subjects with MDS or AML (clinicaltrials.gov)
    P1, N=160, Active, not recruiting, Shattuck Labs, Inc. | Recruiting --> Active, not recruiting | Trial completion date: Dec 2025 --> Nov 2024 | Trial primary completion date: Apr 2025 --> Nov 2024
    Enrollment closed • Trial completion date • Trial primary completion date • Combination therapy
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    Venclexta (venetoclax) • azacitidine • SL-172154
    2ms
    TCD17197: First-in-human Study of SAR443579 Infusion in Male and Female Children and Adult Participants With Relapsed or Refractory Acute Myeloid Leukemia (R/R AML), B-cell Acute Lymphoblastic Leukemia (B-ALL), High Risk-myelodysplasia (HR-MDS), or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) (clinicaltrials.gov)
    P1/2, N=169, Recruiting, Sanofi | N=126 --> 169 | Trial completion date: Nov 2026 --> Oct 2030
    Enrollment change • Trial completion date
    |
    IL3RA (Interleukin 3 Receptor Subunit Alpha)
    |
    SAR443579
    2ms
    Transient Myeloproliferative Disorder (TMD), Acute Lymphoblastic Leukemia (ALL), and Juvenile Myelomonocytic Leukemia (JMML) in a Child with Noonan Syndrome: Sequential Occurrence, Single Center Experience, and Review of the Literature. (PubMed, Genes (Basel))
    After an initial response to antimetabolite therapy (6-mercaptopurine), she underwent haploidentical hematopoietic stem cell transplantation (HSCT) and is currently in complete remission. The goal of this review is to gain insight into the various hematological diseases associated with NS, starting from our unique case.
    Review • Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    PTPN11 mutation
    |
    mercaptopurine
    2ms
    A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy (clinicaltrials.gov)
    P1, N=36, Recruiting, Children's Oncology Group | Trial completion date: Apr 2026 --> Jun 2026 | Trial primary completion date: Apr 2026 --> Jun 2026
    Trial completion date • Trial primary completion date • Combination therapy
    |
    cytarabine • methotrexate • leucovorin calcium • fludarabine IV • Rytelo (imetelstat) • Starasid (cytarabine ocfosfate)
    2ms
    CBL mutations in chronic myelomonocytic leukemia often occur in the RING domain with multiple subclones per patient: Implications for targeting. (PubMed, PLoS One)
    Furthermore, we noted an increased percentage of CMML CD34+ stem and progenitor cells expressing CD116 and CD131 in all CBL mutant cases and increased CD116 receptor density compared to healthy controls, similar to CMML overall. In summary, our data demonstrate that CBL mutations are associated with distinct molecular and clinical features in CMML and are potentially targetable with CD116-directed immunotherapy.
    Journal • IO biomarker
    |
    KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • TET2 (Tet Methylcytosine Dioxygenase 2) • CD34 (CD34 molecule)
    |
    KRAS mutation • NRAS mutation • TET2 mutation • CBL mutation
    2ms
    Pitfalls in Diagnosis: JMML versus KMT2A Rearranged Juvenile AML. (PubMed, Case Rep Hematol)
    This case highlights the challenges of diagnosing KMT2A-rearranged monocytic AML and the importance of careful morphological assessment in partnership with cytogenetic and molecular diagnostics to distinguish between KMT2A-rearranged AML and JMML. Moreover, the emerging role of molecular monitoring in AML is highlighted.
    Journal
    |
    KMT2A (Lysine Methyltransferase 2A)
    |
    KMT2A rearrangement • MLL rearrangement
    3ms
    Myeloproliferative Neoplasms (MPNs) Patient Registry (clinicaltrials.gov)
    P=N/A, N=5000, Recruiting, University Health Network, Toronto | Trial completion date: Oct 2025 --> Oct 2027 | Trial primary completion date: Oct 2025 --> Oct 2027
    Trial completion date • Trial primary completion date
    3ms
    Prototypical innate immune mechanism hijacked by leukemia-initiating mutant stem cells for selective advantage and immune evasion in Ptpn11-associated juvenile myelomonocytic leukemia. (PubMed, Res Sq)
    Importantly, pharmacological inhibition of S100a9/S100a8 signaling effectively impeded leukemia development from Ptpn11 E76K/+ mutant stem cells. These findings collectively suggest that JMML tumor-initiating cells exploit evolutionarily conserved innate immune and inflammatory mechanisms to establish clonal dominance.
    Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • S100A8 (S100 Calcium Binding Protein A8) • S100A9 (S100 Calcium Binding Protein A9)
    4ms
    Observation and Management of Juvenile Myelomonocytic Leukemia and Noonan Syndrome-Associated Myeloproliferative Disorder: A Real-World Experience. (PubMed, Cancers (Basel))
    All patients (5/5) treated with 5-azacitidine alone and one of the four treated with 6-mercaptopurine monotherapy had a reduction in mutant variant allele frequency. Among patients who received transplants, 7/13 are alive, and relapse post-transplant occurred in 3/13 with a median time to relapse of 3.55 months. This report provides insight into therapy responses and long-term outcomes across different genetic subsets of JMML and lends insight into the expected time to spontaneous resolution in patients with NS-MPD with germline PTPN11 mutations.
    Journal • Real-world evidence • Real-world
    |
    NRAS (Neuroblastoma RAS viral oncogene homolog) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    azacitidine • mercaptopurine
    4ms
    A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy (clinicaltrials.gov)
    P1, N=36, Recruiting, Children's Oncology Group | Initiation date: Jul 2024 --> Mar 2025
    Trial initiation date • Combination therapy
    |
    cytarabine • methotrexate • leucovorin calcium • fludarabine IV • Rytelo (imetelstat) • Starasid (cytarabine ocfosfate)
    4ms
    Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation (clinicaltrials.gov)
    P2, N=52, Recruiting, St. Jude Children's Research Hospital | Trial completion date: Jul 2025 --> Jul 2026 | Trial primary completion date: Jul 2024 --> Jul 2025
    Trial completion date • Trial primary completion date
    |
    Rituxan (rituximab) • cyclophosphamide • Blincyto (blinatumomab) • sirolimus • melphalan • fludarabine IV • mesna • thiotepa • Neupogen (filgrastim)
    4ms
    Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation (clinicaltrials.gov)
    P1, N=15, Recruiting, Johns Hopkins All Children's Hospital | Trial completion date: Oct 2024 --> Oct 2025 | Trial primary completion date: Jul 2024 --> Jul 2025
    Trial completion date • Trial primary completion date • Combination therapy
    |
    azacitidine • Zolinza (vorinostat)
    4ms
    Chemical genetic screens reveal defective lysosomal trafficking as synthetic lethal with NF1 loss. (PubMed, J Cell Sci)
    Knockdown of a BORC complex subunit using siRNA recapitulated the phenotypes observed with Y102 treatment. Our findings demonstrate that the BORC complex may be a promising therapeutic target for NF1-deficient tumors.
    Journal • Synthetic lethality
    |
    NF1 (Neurofibromin 1)
    4ms
    A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy (clinicaltrials.gov)
    P1, N=36, Recruiting, Children's Oncology Group | Not yet recruiting --> Recruiting
    Enrollment open
    |
    cytarabine • methotrexate • leucovorin calcium • fludarabine IV • Rytelo (imetelstat) • Starasid (cytarabine ocfosfate)
    5ms
    A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies (clinicaltrials.gov)
    P2, N=3, Terminated, St. Jude Children's Research Hospital | N=32 --> 3 | Trial completion date: Apr 2027 --> May 2024 | Recruiting --> Terminated | Trial primary completion date: Apr 2026 --> May 2024; Principal Investigator left institution
    Enrollment change • Trial completion date • Trial termination • Trial primary completion date
    |
    Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • mesna • thiotepa • busulfan • cyclosporine
    5ms
    Immunological and hematological findings as major features in a patient with a new germline pathogenic CBL variant. (PubMed, Am J Med Genet A)
    Functional studies documented enhanced EGF-induced ERK phosphorylation in transiently transfected COS1 cells. The present findings further support the association of pathogenic CBL variants with immunological and hematological manifestations in the context of a presentation with only minor findings reminiscent of NS or a clinically related RASopathy.
    Retrospective data • Journal
    |
    EGF (Epidermal growth factor)
    5ms
    A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in pediatric and young adult (<21year) Patients with High-Risk and Very High-Risk Myeloid Malignancies (ECT-001-CB.007) (clinicaltrialsregister.eu)
    P2, N=15, Cordex Biolohics Inc
    New P2 trial
    |
    TP53 (Tumor protein P53) • HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
    |
    dorocubicel (UM171 cell therapy)
    5ms
    Risk-adapted Donor Lymphocyte Infusion After Allo-HSCT in Children With Hematologic Malignancy (clinicaltrials.gov)
    P1/2, N=50, Completed, St. Petersburg State Pavlov Medical University | Active, not recruiting --> Completed
    Trial completion
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
    6ms
    Vorinostat Dose-escalation After Allogeneic Hematopoietic Cell Transplantation (clinicaltrials.gov)
    P1, N=15, Recruiting, Johns Hopkins All Children's Hospital | Trial completion date: Jun 2024 --> Oct 2024
    Trial completion date
    |
    azacitidine • Zolinza (vorinostat)
    6ms
    A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia (clinicaltrials.gov)
    P1/2, N=58, Recruiting, Therapeutic Advances in Childhood Leukemia Consortium | Not yet recruiting --> Recruiting
    Enrollment open • Tumor mutational burden
    |
    KRAS (KRAS proto-oncogene GTPase) • NRAS (Neuroblastoma RAS viral oncogene homolog) • NF1 (Neurofibromin 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    KRAS mutation • NRAS mutation • NF1 mutation • RAS mutation
    |
    Mekinist (trametinib) • cytarabine • azacitidine • fludarabine IV
    7ms
    The elevation of red blood cell distribution width is an independent prognostic factor for juvenile myelomonocytic leukemia. (PubMed, Blood Sci)
    RDW is an independent prognostic variable in JMML subjects. RDW may be regarded as an inexpensive biomarker to predict the clinical outcome in patients with JMML.
    Journal
    |
    PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11)
    |
    PTPN11 mutation
    7ms
    Identifying Potential SOS1 Inhibitors via Virtual Screening of Multiple Small Molecule Libraries against KRAS-SOS1 Interface. (PubMed, Chembiochem)
    Comparative analysis of the average binding free energies of these predicted potent compounds with known SOS1 small molecule inhibitors revealed that the identified compounds display similar or even superior predicted binding affinities compared to the known inhibitors. These findings offer valuable insights into the potential of these compounds as candidates for further development as effective anti-cancer agents.
    Journal
    |
    KRAS (KRAS proto-oncogene GTPase)
    |
    KRAS mutation
    7ms
    SH2B3 alterations in a novel genetic condition, juvenile myelomonocytic leukemia, and myeloproliferative neoplasia. (PubMed, Haematologica)
    Not available.
    Journal
    |
    SH2B3 (SH2B Adaptor Protein 3)
    |
    SH2B3 alteration
    8ms
    Alpha/Beta T Cell and CD19+ B Cell Depletion in Allogeneic Stem Cell Transplantation in Patients With Malignant Diseases (clinicaltrials.gov)
    P2, N=20, Recruiting, University of Florida | Not yet recruiting --> Recruiting
    Enrollment open
    |
    FLT3 (Fms-related tyrosine kinase 3)
    |
    FLT3 mutation • CBL mutation • Chr t(9;11)
    8ms
    03-OHD-104: Phase 1 Study of Shattuck Labs (SL)-172154 in Subjects With MDS or AML (clinicaltrials.gov)
    P1, N=160, Recruiting, Shattuck Labs, Inc. | N=107 --> 160 | Trial completion date: Oct 2024 --> Dec 2025 | Trial primary completion date: Feb 2024 --> Apr 2025
    Enrollment change • Trial completion date • Trial primary completion date • Combination therapy
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    Venclexta (venetoclax) • azacitidine • SL-172154
    8ms
    HA-1 T TCR T Cell Immunotherapy for the Treatment of Patients With Relapsed or Refractory Acute Leukemia After Donor Stem Cell Transplant (clinicaltrials.gov)
    P1, N=24, Suspended, Fred Hutchinson Cancer Center | Trial completion date: Jul 2025 --> Jul 2028 | Trial primary completion date: Oct 2024 --> Oct 2027
    Trial completion date • Trial primary completion date
    |
    HLA-A (Major Histocompatibility Complex, Class I, A) • CD4 (CD4 Molecule)
    |
    HLA-A*02:01
    |
    fludarabine IV
    8ms
    Long-term remission of infantile Takayasu arteritis associated with germline CBL syndrome after allogeneic hematopoietic stem cell transplantation: A case report and literature review. (PubMed, Transpl Immunol)
    The pathogenesis of vasculitis in CBL syndrome appears to involve dysregulated T cell function and possibly increased angiogenesis. This case advances the understanding of vascular involvement in CBL syndrome and of the genetic, immune, and vascular interplay in TA, offering insights for treating CBL syndrome and broader TA.
    Clinical • Observational data • Retrospective data • Review • Journal
    |
    CBL (Cbl proto-oncogene)
    9ms
    A comprehensive genomic profiling of myeloid malignancies demonstrates mutational spectrum of DNA variants, FLT3-ITDs, and gene fusions (AACR 2024)
    The Oncomine Myeloid Assay is a fast, robust, and reproducible solution for comprehensive genomic profiling of myeloid malignancies. We describe the mutational spectrum of DNA variants and RNA fusions in a range of clinical research samples. (For research use only.
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • ABL1 (ABL proto-oncogene 1) • BCR (BCR Activator Of RhoGEF And GTPase) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • FGFR1 (Fibroblast growth factor receptor 1) • DNMT3A (DNA methyltransferase 1) • MYD88 (MYD88 Innate Immune Signal Transduction Adaptor) • RUNX1 (RUNX Family Transcription Factor 1) • ASXL1 (ASXL Transcriptional Regulator 1) • KMT2A (Lysine Methyltransferase 2A) • TET2 (Tet Methylcytosine Dioxygenase 2) • SRSF2 (Serine and arginine rich splicing factor 2) • CREBBP (CREB binding protein) • CEBPA (CCAAT Enhancer Binding Protein Alpha) • ZMYM2 (Zinc Finger MYM-Type Containing 2) • CALR (Calreticulin) • KAT6A (Lysine Acetyltransferase 6A) • ANKRD26 (Ankyrin Repeat Domain Containing 26)
    |
    FLT3-ITD mutation • ASXL1 mutation • TET2 mutation • SRSF2 mutation • FGFR1 fusion
    |
    Oncomine Myeloid Assay GX • Oncomine Myeloid Research Assay
    9ms
    A predictive classifier of poor prognosis in transplanted patients with juvenile myelomonocytic leukemia: a study on behalf of the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire. (PubMed, Haematologica)
    By integrating genetic information with clinical and hematological features, we have devised a predictive classifier. This classifier effectively identifies a subgroup of patients who are at a heightened risk of unfavorable post-transplant outcomes who would benefit novel therapeutic agents and post-transplant strategies.
    Journal
    |
    ASXL1 (ASXL Transcriptional Regulator 1) • JAK3 (Janus Kinase 3) • SETBP1 (SET Binding Protein 1)
    |
    RAS mutation • ASXL1 mutation • JAK3 mutation
    9ms
    US Study of ECT-001-CB in Pediatric and Young Adult Patients With High-Risk Myeloid Malignancies (clinicaltrials.gov)
    P1/2, N=12, Recruiting, ExCellThera inc. | Trial completion date: Dec 2025 --> Jun 2026 | Trial primary completion date: Dec 2023 --> Jun 2026
    Trial completion date • Trial primary completion date
    |
    HLA-DRB1 (Major Histocompatibility Complex, Class II, DR Beta 1) • CD34 (CD34 molecule)
    |
    cyclophosphamide • clofarabine • fludarabine IV • thiotepa • busulfan • dorocubicel (UM171 cell therapy)
    9ms
    CD45RA Depleted Peripheral Stem Cell Addback for Viral or Fungal Infections Post TCRαβ/CD19 Depleted HSCT (clinicaltrials.gov)
    P=N/A, N=50, Recruiting, Children's Hospital of Philadelphia | N=30 --> 50 | Trial completion date: Jan 2024 --> Jan 2026 | Trial primary completion date: Jan 2024 --> Feb 2025
    Enrollment change • Trial completion date • Trial primary completion date
    |
    cyclophosphamide
    10ms
    A Study to Find the Highest Dose of Imetelstat in Combination With Fludarabine and Cytarabine for Patients With AML, MDS or JMML That Has Come Back or Does Not Respond to Therapy (clinicaltrials.gov)
    P1, N=36, Not yet recruiting, Children's Oncology Group
    New P1 trial
    |
    cytarabine • methotrexate • leucovorin calcium • fludarabine IV • Rytelo (imetelstat) • Starasid (cytarabine ocfosfate)
    10ms
    A Phase 1 Trial of CIML NK Cell Infusion for Myeloid Disease Relapse After Hematopoietic Cell Transplantation (clinicaltrials.gov)
    P1, N=50, Enrolling by invitation, Dana-Farber Cancer Institute | Active, not recruiting --> Enrolling by invitation
    Enrollment open
    |
    IL2 (Interleukin 2)
    |
    cyclophosphamide • fludarabine IV • CIML NK
    10ms
    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=140, Active, not recruiting, St. Jude Children's Research Hospital | Recruiting --> Active, not recruiting
    Enrollment closed
    |
    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)
    |
    FLT3-ITD mutation • WT1 mutation • MLL fusion • NUP98-NSD1 fusion
    |
    cyclophosphamide • Blincyto (blinatumomab) • melphalan • fludarabine IV • thiotepa • Neupogen (filgrastim)
    11ms
    LIMBER: Safety and Tolerability Study of INCB057643 in Participants With Myelofibrosis and Other Advanced Myeloid Neoplasms (clinicaltrials.gov)
    P1, N=216, Recruiting, Incyte Corporation | N=138 --> 216
    Enrollment change • Metastases
    |
    Jakafi (ruxolitinib) • INCB57643
    11ms
    Risk-adapted Donor Lymphocyte Infusion After Allo-HSCT in Children With Hematologic Malignancy (clinicaltrials.gov)
    P1/2, N=50, Active, not recruiting, St. Petersburg State Pavlov Medical University | Recruiting --> Active, not recruiting | Trial completion date: Apr 2023 --> Apr 2024
    Enrollment closed • Trial completion date
    |
    TP53 (Tumor protein P53) • FLT3 (Fms-related tyrosine kinase 3) • KMT2A (Lysine Methyltransferase 2A) • NUP98 (Nucleoporin 98 And 96 Precursor 2)
    |
    TP53 mutation • MLL rearrangement
    11ms
    A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies (clinicaltrials.gov)
    P2, N=32, Recruiting, St. Jude Children's Research Hospital
    Trial completion date • Trial primary completion date
    |
    Jakafi (ruxolitinib) • cyclophosphamide • fludarabine IV • mesna • thiotepa • busulfan • cyclosporine
    11ms
    03-OHD-104: Phase 1 Study of Shattuck Labs (SL)-172154 in Subjects With MDS or AML (clinicaltrials.gov)
    P1, N=107, Recruiting, Shattuck Labs, Inc.
    Trial completion date • Trial primary completion date • Combination therapy
    |
    TP53 (Tumor protein P53)
    |
    TP53 mutation
    |
    Venclexta (venetoclax) • azacitidine • SL-172154