DSP: Phase 1/2 DSP-5336 in Acute Leukemia with or with MLL or NPM1 mutation

What is the Purpose of this Study?

We are doing this study to find out how well an experimental drug called DSP-5336 (the study drug) works in people with AML or ALL.

What is the Condition Being Studied?

Mixed Lineage Leukemia (MLL) Rearrangement or Nucleophosmin 1 (NPM1) Mutation

Who Can Participate in the Study?

Adults ages 18+ who:

  • Are diagnosed with Relapsed or Refractory AML or ALL
  • Have a confirmed KMT2A (MLL)-fusion or NPM1 mutation
  • Have acceptable organ function

For more information about who can join this study, please contact the study team at 919-660-2077.

Age Group

What is Involved?

If you choose to join this study, you will go through a screening period to find out if you are eligible. During this screening period, you will:

  • Have a physical exam
  • Have a heart scan (ECG)
  • Have blood draws

After the screening period, this study is divided into 2 parts:

  • In Part 1, you will be assigned to either:
    • Arm A (not taking a type of antifungal treatment)
    • Arm B (you are taking a type of antifungal treatment).
  • In Part 2, you will be assigned to either:
    • Arm A [R/R AML with Mixed Lineage Leukemia that is rearranged (MLLr AML)]
    • Arm B [R/R leukemia that has the Nucleophosmin 1 gene mutated (NPM1)]

The study drug will be taken by mouth twice daily.

Once you stop taking the study drug, you will enter a follow-up period. The study team will call you approximately every 3 months to see how you are feeling during this period.

Study Details

Full Title
A Phase 1/2, Open-Label, Dose-Escalation, Dose-Expansion Study of DSP-5336 in Adult Acute Leukemia Patients with and without Mixed Lineage Leukemia (MLL)-rearrangement or Nucleophosmin 1 (NPM1) Mutation
Principal Investigator
Professor of Medicine
Protocol Number
IRB: PRO00109316
NCT: NCT04988555
Phase I/II
Enrollment Status