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Gene Symbol CBL
Synonyms C-CBL | CBL2 | FRA11B | NSLL | RNF55
Gene Description CBL, Cbl proto-oncogene, is an E3 ubiquitin-protein ligase involved in cell signaling and ubiquitination of tyrosine kinases (PMID: 23085373). CBL mutations, often resulting in a loss of function, have been identified primarily in myeloid neoplasms, including myelodysplastic syndrome, acute myeloid leukemia, chronic myeloid leukemia, myelofibrosis, and juvenile myelomonocytic leukemia (PMID: 25477533).

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT05658640 Phase Ib/II Cyclophosphamide + Cytarabine + Dexamethasone + Trametinib HEM iSMART-D: Trametinib + Dexamethasone + Chemotherapy in Children With Relapsed or Refractory Hematological Malignancies (HEM-iSMART D) Not yet recruiting NLD 0
NCT04487106 Phase II Azacitidine + Trametinib + Venetoclax Azacitidine, Venetoclax, and Trametinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Higher-Risk Myelodysplastic Syndrome Completed USA 0
NCT04493138 Phase Ib/II Azacitidine + Quizartinib Azacitidine and Quizartinib for the Treatment of Myelodysplastic Syndrome or Myelodysplastic/Myeloproliferative Neoplasm With FLT3 or CBL Mutations Recruiting USA 0
NCT04409639 Phase II Cobimetinib Cobimetinib in Newly Diagnosed or HMA-treated CMML Patients With RAS Pathway Mutations (CONCERTO) Recruiting USA 0
NCT03613532 Phase I Azacitidine + Venetoclax Busulfan + Fludarabine + Venetoclax Decitabine and Cedazuridine Venetoclax Added to Fludarabine + Busulfan Prior to Transplant and to Maintenance Therapy for AML, MDS, and MDS/MPN Recruiting USA 0
NCT04708054 Phase II Busulfan + Cladribine + Fludarabine + Thiotepa + Venetoclax Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS Active, not recruiting USA 0
NCT03190915 Phase II Trametinib Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia Active, not recruiting USA 0
NCT06297629 Phase II Decitabine and Cedazuridine A Phase I/II Trial to Assess the Efficacy and Toxicity of ASTX727 (Oral Decitabine/Cedazuridine) for the Treatment of Hematological Neoplasms After Allogeneic Stem Cell Transplantation Not yet recruiting USA 0
NCT05441514 Phase I Cobimetinib + Enasidenib Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia Recruiting USA 0
NCT06284460 Phase Ib/II ASTX029 ASTX029 + Decitabine and Cedazuridine Phase I/II Study of a Combination of Decitabine and Cedazuridine (ASTX727) and ASTX029, an ERK Inhibitor, for Patients With RAS Pathway Mutant Myelodysplastic Syndromes and Myelodysplastic/Myeloproliferative Neoplasms Not yet recruiting USA 0