A Phase I/II Study of PLX3397 to Treat Patients with Acute Myeloid Leukemia that Has Returned or Persisted Despite Therapy

Full Title
A Phase 1/2 Safety and Efficacy Study of Orally Administered PLX3397 in Adults With Relapsed or Refractory Flt3-ITD Positive Acute Myeloid Leukemia
Disease Status

PLX3397 is an investigational anticancer drug that works by targeting a family of enzymes called kinases — particularly Kit, FLT3, and Fms kinase — which drive cancer growth. The purpose of this study is to examine the safety and effectiveness of PLX3397 treatment in patients with acute myeloid leukemia (AML) which has abnormal FLT3 and has returned or persisted despite other therapies. PLX3397 is taken orally (by mouth) twice a day.


To be eligible for this study, patients must meet several criteria, including but not limited to the following:

  • Patients must have AML has returned or persisted despite prior treatment.
  • Patients’ AML must contain abnormal FLT3.
  • At least 2 weeks must have passed since completion of prior therapy and entry into the study.
  • Patients must be able to be ambulatory for more than half of their normal waking hours.
  • This study is open to patients age 18 and older.

For more information and to inquire about eligibility for this study, please contact Dr. Eytan Stein at 212-639-3314.

Leukemia: Acute Myelocytic Leukemia
Leukemia: Acute Myelogenous Leukemia