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

Study Status

Open to Enrollment

Study Description

The purpose of this clinical trial is to study the safety and efficacy of an experimental drug called PLX3397 in patients with relapsed or refractory Flt3-positive Acute Myeloid Leukemia. PLX3397 selectively targets a number of cell types that are involved in various aspects of tumor growth and metastasis.

This is a two-part study. The study physician will tell you which part and dose group you will be participating in prior to enrollment.

  • Part 1 (Dose Escalation Phase) will look at the safety of increasing dose levels of PLX3397. Each dose group will include between 3-6 patients. The study physician and sponsor will review the safety data of each dose level of PLX3397 following 15 days of dosing, before the patients in the next dose level are dose. The highest level (maximum tolerated dose or MTD) tolerated by patients in Part 1 will be used as the dose in Part 2 of this study.
  • Part 2 (Cohort Expansion Phase): The dose will be the highest tolerated dose from Part 1. Part 2 will look at how PLX3397 affects your cancer and your body.



Key Eligibility

  • Men and women age 18 and older
  • Relapsed or refractory Acute Myeloid Leukemia (AML)
  • Positive for Flt3-ITD activating mutation during Screening
  • Detailed eligibility reviewed when you contact the study team

Principal Investigator

Gail Roboz, MD


Healthy Volunteers


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Contact Us

For general inquiries, or if you need assistance finding a study, please contact:

Robert Hagerty
Subject Recruitment Manager
Tel: (646) 962-9340
[email protected]

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