A Phase 1 Study of KT-253 in People With Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, and Myelodysplastic Syndrome


Full Title

A Phase 1, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Intravenously Administered KT-253 in Adult Patients with High Grade Myeloid Malignancies and Acute Lymphocytic Leukemia, Lymphoma and Advanced Solid Tumors


The purpose of this study is to find the best dose of KT-2543 to use in people with blood cancers. The people in this study have acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or myelodysplastic syndrome (MDS). In addition, their cancers keep growing even with treatment.

KT-253 targets the MDM2 protein. The MDM2 protein affects the p53 protein, which plays an important role in cell growth and cell death. Cancer cells can make too much MDM2, preventing p53 from causing cell death. By targeting MDM2, KT-253 may help cause cancer cells to die. KT-253 is given intravenously (by vein).

Who Can Join

To join this study, there are a few conditions. You must:

  • Have AML, ALL, or MDS that came back or keeps growing even with treatment.
  • Have recovered from the serious side effects of prior therapies before getting KT-253.
  • Be able to walk and do routine activities for more than half the time you are awake.
  • Be age 18 or older.


For more information and to see if you can join this study, please call Dr. Eytan Stein’s office at 646-608-3749.




Phase I (phase 1)

Disease Status

Relapsed or Refractory



ClinicalTrials.gov ID