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  • A Phase 1/2A First in Human Study of the Menin-MLL (KMT2A) Inhibitor KO-539 in Patients with Relapsed or Refractory Acute Myeloid Leukemia

    Cancer Categories
    • Hematologic (Blood Cancers)
    Karmanos Trial ID
    • 2022-014
    NCT ID
    • NCT04067336
    Age Group
    • Adult
    • National
    PhaseClick for Clinical Trial Phase DefinitionPhase I
    Includes initial studies to determine the metabolism and pharmacologic actions of drugs in humans, the side effects associated with increasing doses, and to gain early evidence of effectiveness; may include healthy participants and/or patients.
    • Phase I/II
    Principal Investigator
    • Suresh
      Balasubramanian, M.D.

      Oncology - Hematology, Oncology - Medical View Profile


    Part 1a: Dose-Escalation

    Primary Objective: To determine the maximal tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) of KO-539 in patients with relapsed or refractory acute myeloid leukemia (AML).

    • Key Secondary Objective 1: To investigate the safety and tolerability of KO-539 according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
    • Key Secondary Objective 2: To characterize the pharmacokinetics (PK) of KO-539 after single oral (po) dose administration and after multiple po dose administrations.
    • Key Secondary Objective 3: Explore early evidence of anti-leukemic activity (ALA) according to the criteria proposed by the 2017 European Leukemia Network (ELN) Recommendations for AML (Döhner et al. 2017) and the US Food and Drug Administration (FDA) Guidance for Industry for AML (US FDA 2020):
      • Composite of complete remission (CR) plus CR with partial hematologic recovery (CRh)
      • CR with and without minimal residual disease (MRD)
      • Duration of remission (DOR)

    Part 1b: Dose-Validation/ Cohort Expansion

    Primary Objective: Determine the safety, tolerability and minimal biologically effective dose of KO-539 in dosing cohorts which have demonstrated early biological activity and have been determined to be safe as part of the dose-escalation phase.

    Secondary Objective: Explore early evidence of ALA according to the criteria proposed by the 2017 ELN Recommendations for AML (Döhner et al. 2017) and the US FDA Guidance for Industry for AML (US FDA 2020).

    • Key Secondary:
      • Composite of CR plus CRh
      • CR with and without MRD
      • DOR
    • Other Secondary:
      • Transfusion Independence (TI)
      • Relapse-free survival (RFS)
      • Overall Survival (OS)
  • Locations


    Karmanos Cancer Institute - Detroit Headquarters

    4100 John R
    Detroit, MI 48201
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    Phone: 800-527-6266

    Karmanos Cancer Institute at Weisberg Cancer Center - Farmington Hills

    31995 Northwestern Hwy
    Farmington Hills, MI 48334
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    Phone: 800-527-6266