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Profile Name KMT2A rearrange
Gene Variant Detail

KMT2A rearrange (unknown)

Relevant Treatment Approaches

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Molecular Profile Indication/Tumor Type Response Type Relevant Treatment Approaches Therapy Name Approval Status Evidence Type Efficacy Evidence References
KMT2A rearrange acute lymphoblastic leukemia sensitive Cytarabine + Romidepsin Preclinical - Cell line xenograft Actionable In a preclinical study, Istodax (romidepsin) enhanced the effects of Cytosar-U (cytarabine) in acute lymphocytic leukemia cell lines harboring a KMT2A rearrangement in culture, and in cell line xenograft models, demonstrating decreased leukemic cells by 73% (PMID: 27443263). 27443263
KMT2A rearrange acute myeloid leukemia sensitive CYC065 Preclinical - Cell line xenograft Actionable In a preclinical study, CYC065 (Fadraciclib) treatment resulted in decreased cell viability in an acute myeloid leukemia cell line harboring a KMT2A rearrangement in culture and inhibited tumor growth in a cell line xenograft model (PMID: 32645016). 32645016
KMT2A rearrange acute leukemia predicted - sensitive Pinometostat Case Reports/Case Series Actionable In a Phase I trial, Pinometostat (EPZ-5676) treatment resulted in complete remission in 2 patients with KMT2A rearranged (both with t(11;19)) acute leukemia (PMID: 29724899; NCT01684150). 29724899
KMT2A rearrange B-cell acute lymphoblastic leukemia not applicable N/A Guideline Prognostic KMT2A rearrangements are associated with a poor prognosis in patients with B-cell acute lymphoblastic leukemia (NCCN.org). detail...
KMT2A rearrange acute lymphoblastic leukemia sensitive Cytarabine + Mocetinostat Preclinical - Cell culture Actionable In a preclinical study, Mocetinostat (MGCD0103) enhanced the effects of Cytosar-U (cytarabine) in acute lymphoblastic leukemia cell lines harboring a KMT2A rearrangement in culture, resulting in cell death (PMID: 27443263). 27443263
KMT2A rearrange acute lymphoblastic leukemia predicted - sensitive CYC065 Preclinical - Cell culture Actionable In a preclinical study, acute lymphoblastic leukemia cell lines harboring KMT2A rearrangements were sensitive to treatment with CYC065 (Fadraciclib) in culture (PMID: 32645016). 32645016
KMT2A rearrange acute myeloid leukemia predicted - sensitive KO-539 Preclinical - Cell line xenograft Actionable In a preclinical study, KO-539 inhibited growth of KMT2A (MLL1)-rearranged acute myeloid leukemia cells in culture, and prolonged survival in cell-line xenograft models (AACR; Mol Cancer Ther 2018;17(1 Suppl):Abstract nr LB-A27). detail...
KMT2A rearrange acute lymphoblastic leukemia sensitive Cytarabine + Panobinostat Preclinical - Cell culture Actionable In a preclinical study, Farydak (panobinostat) enhanced the effects of Cytosar-U (cytarabine) in acute lymphoblastic leukemia cells harboring a KMT2A rearrangement in culture, resulting in cell death (PMID: 27443263). 27443263
KMT2A rearrange acute myeloid leukemia not applicable N/A Guideline Prognostic KMT2A rearrangements (t(v;11q23.3)) are associated with a poor/adverse prognosis in patients with non-APL acute myeloid leukemia (NCCN.org). detail...
KMT2A rearrange acute myeloid leukemia not applicable N/A Clinical Study Prognostic In multiple clinical studies, KMT2A rearrangements, specifically partial tandem duplications, were associated with a poor overall survival in acute myeloid leukemia patients (PMID: 24487413, PMID: 22915647, PMID: 22417203, PMID: 21881046). 21881046 24487413 22417203 22915647
KMT2A rearrange childhood B-cell acute lymphoblastic leukemia not applicable N/A Guideline Prognostic KMT2A rearrangements are associated with a poor prognosis in pediatric patients with B-cell acute lymphoblastic leukemia (NCCN.org). detail...
KMT2A rearrange acute lymphoblastic leukemia sensitive Cytarabine + Dacinostat Preclinical - Cell culture Actionable In a preclinical study, Dacinostat (LAQ824) enhanced the effects of Cytosar-U (cytarabine) in acute lymphoblastic leukemia cell lines harboring a KMT2A rearrangement in culture, resulting in cell death (PMID: 27443263). 27443263
KMT2A rearrange acute lymphoblastic leukemia sensitive Cytarabine Preclinical - Cell line xenograft Actionable In a preclinical study, Cytosar-U (cytarabine) decreased leukemic cells by 66% in acute lymphocytic leukemia cell line xenograft models harboring a KMT2A rearrangement (PMID: 27443263). 27443263
KMT2A rearrange acute lymphoblastic leukemia decreased response Romidepsin Preclinical - Cell line xenograft Actionable In a preclinical study, Istodax (romidepsin) resulted in minimal activity in acute lymphocytic leukemia cell lines harboring a KMT2A rearrangement in culture, and in cell line xenograft models, demonstrating decreased leukemic cells by 16% (PMID: 27443263). 27443263
KMT2A rearrange leukemia predicted - sensitive I-CBP112 + JQ1 Preclinical Actionable In a preclinical study, I-CBP112 sensitized leukemia cells harboring KMT2A fusions to JQ1, resulting in decreased cell growth in culture (PMID: 26552700). 26552700
KMT2A rearrange leukemia predicted - sensitive Doxorubicin + I-CBP112 Preclinical Actionable In a preclinical study, I-CBP112 sensitized leukemia cells harboring KMT2A fusions to Doxorubicin, leading to decreased cell growth in culture (PMID: 26552700). 26552700
Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT04659616 Phase I Cytarabine + Pemigatinib Cytarabine + Daunorubicin + Pemigatinib Pemigatinib in Combination With Chemotherapy for the Treatment of Newly Diagnosed Acute Myeloid Leukemia Recruiting USA 0
NCT01028716 Phase II Filgrastim + Mycophenolate mofetil + Tacrolimus Cyclophosphamide + Fludarabine Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies Recruiting USA 0
NCT04065399 Phase Ib/II SNDX-5613 A Study of SNDX-5613 in R/R Leukemias Including Those With an MLLr/KMT2A Gene Rearrangement or NPM1 Mutation (AUGMENT-101) Recruiting USA 0
NCT03513484 Phase I Azacitidine + Nintedanib Nintedanib and Azacitidine in Treating Participants With HOX Gene Overexpression Relapsed or Refractory Acute Myeloid Leukemia Recruiting USA 0
NCT02141828 Phase I Pinometostat A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients With Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene Completed USA | CAN 0
NCT02419755 Phase II Cytarabine Mercaptopurine Methotrexate Pegaspargase Vorinostat Doxorubicin Bortezomib Dexamethasone Mitoxantrone Prednisone Bortezomib and Vorinostat in Younger Patients With Refractory or Relapsed MLL Rearranged Hematologic Malignancies Terminated USA 0
NCT03613532 Phase I Busulfan + Fludarabine + Venetoclax Venetoclax Added to Fludarabine + Busulfan Prior to Transplant for AML, MDS, and MDS/MPN Recruiting USA 0
NCT03826992 Phase I CPX-351 + Venetoclax Venetoclax Combined With Vyxeos (CPX-351) for Participants With Relapsed or Refractory Acute Leukemia Recruiting USA 0
NCT02828358 Phase II Cytarabine + Hydrocortisone + Leucovorin + Mercaptopurine + Pegaspargase Cyclophosphamide + Cytarabine + Hydrocortisone + Mercaptopurine + Methotrexate Cytarabine + Hydrocortisone + Mercaptopurine + Methotrexate Azacitidine Cytarabine + Daunorubicin + Dexamethasone + Hydrocortisone + Pegaspargase + Thioguanine + Vincristine sulfate liposome Cytarabine + Daunorubicin + Dexamethasone + Hydrocortisone + Methotrexate + Pegaspargase + Prednisolone + Vincristine Sulfate Mercaptopurine + Methotrexate Cyclophosphamide + Cytarabine + Thioguanine Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement Active, not recruiting USA | CAN 2
NCT04752163 Phase Ib/II Cyclophosphamide + Cytarabine + Dexamethasone + DS-1594b + Filgrastim + Mesna + Methotrexate + Vincristine Sulfate DS-1594b + Prednisone + Vincristine Sulfate DS-1594b Azacitidine + DS-1594b + Venetoclax Cytarabine + DS-1594b + Filgrastim + Leucovorin + Methotrexate Cytarabine + DS-1594b + Filgrastim + Leucovorin + Methotrexate + Rituximab DS-1594b With or Without Azacitidine, Venetoclax, or Mini-HCVD for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphoblastic Leukemia Recruiting USA 0
NCT03724084 Phase Ib/II Cytarabine + Daunorubicin + Pinometostat Pinometostat With Standard Chemotherapy in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia and MLL Gene Rearrangement Active, not recruiting USA 0
NCT03674411 Phase II Busulfan + Filgrastim + Fludarabine + Melphalan + MGTA-456 + Mycophenolate mofetil + Tacrolimus Cyclophosphamide + Filgrastim + Fludarabine + MGTA-456 + Mycophenolate mofetil + Tacrolimus Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy Active, not recruiting USA 0
NCT01338987 Phase II Tacrolimus Busulfan Methotrexate Leuprolide Cyclophosphamide Fludarabine Pilot Study of Leuprolide to Improve Immune Function After Allogeneic Bone Marrow Transplantation Completed USA 0
NCT04326439 Phase II Cytarabine + Etoposide Cytarabine + Etoposide + Sorafenib Asparaginase Erwinia chrysanthemi + Cytarabine + Sorafenib Cytarabine + Daunorubicin + Etoposide + Gemtuzumab ozogamicin + Sorafenib Cytarabine + Mitoxantrone + Sorafenib Cytarabine + Daunorubicin + Etoposide + Gemtuzumab ozogamicin Asparaginase Erwinia chrysanthemi + Cytarabine Cytarabine + Mitoxantrone AflacLL1901 (CHOA-AML) Terminated USA 0