Clinical Trials /

A Study of DSP-5336 in Relapsed/Refractory AML/ ALL With or Without MLL Rearrangement or NPM1 Mutation



A Phase 1/2 study of DSP 5336 in patients with relapsed or refractory AML.

Related Conditions:
  • Acute Lymphoblastic Leukemia
  • Acute Myeloid Leukemia
Recruiting Status:

Not yet recruiting


Phase 1/Phase 2

Trial Eligibility



  • Brief Title: A Study of DSP-5336 in Relapsed/Refractory AML/ ALL With or Without MLL Rearrangement or NPM1 Mutation
  • Official 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

Clinical Trial IDs

  • ORG STUDY ID: DSP-5336-101
  • NCT ID: NCT04988555


  • Leukemia, Myeloid, Acute
  • Leukemia, Lymphocytic, Acute


DSP-5336Arm A without Antifungals
AntifungalsPosaconazole, variconazole, fluconazole - investigator's choiceArm B with Antifungals


A Phase 1/2 study of DSP 5336 in patients with relapsed or refractory AML.

Detailed Description

      A Phase 1 dose escalation FIH study in relapsed or refractory AML/ALL to determine the
      maximum tolerated dose (MTD) and/or a recommended Phase 2 dose (RP2D) of DSP 5336, followed
      by a Phase 2 dose-expansion portion to further evaluate the safety and clinical activity of
      DSP 5336 in relapsed or refractory AML patients with an MLLr and/or NPM1m

Trial Arms

Arm A without AntifungalsOtherPatients not taking antifungals within 7 days of study entry
  • DSP-5336
Arm B with AntifungalsOtherPatients taking antifungals at study entry
  • DSP-5336
  • Antifungals

Eligibility Criteria

        Inclusion Criteria

          1. Have a diagnosis of relapsed or refractory AML or ALL for Phase I and for Phase 2 have
             a diagnosis of relapsed or refractory AML with MLLr or NPM1

          2. Be > /= 18 years of age or 20 years if required by local regulation

          3. ECOG < /= 2

          4. WBC below 30,000/μL (hydroxyurea allowed)

          5. Glomerular filtration rate (GFR) ≥ 50 ml/min, assessed by the Cockcroft-Gault formula

          6. Total bilirubin ≤1.5 the upper limit of normal (ULN) (or ≤2.0 ULN for patients with
             known Gilbert's syndrome)

          7. Aspartate aminotransferase (AST) ≤3.0 times ULN

          8. Alanine aminotransferase (ALT) ≤3.0 times ULN

        Exclusion Criteria:

          1. Has a left ventricular ejection fraction (LVEF) <45%, as determined by ECHO

          2. Histological diagnosis of acute promyelocytic leukemia

          3. Received systemic calcineurin inhibitors within 4 weeks prior to the first dose of DSP

          4. Has had abnormal ECGs that are clinically significant, such as QT prolongation (QTc
             >450 msec for males and >470 msec for females, with QTc corrected according to
             Fridericia's formula [QTcF])

          5. Has an active, uncontrolled, bacterial, viral, or fungal infection requiring systemic

          6. Receives concurrent sensitive substrates with a narrow safety window or strong
             inhibitors or inducers of CYP3A4/5, including specifically: ketoconazole, itraconazole
             and isavuconazole. Other antifungals that are used as standard of care to prevent or
             treat infections are permitted
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Assess the safety and tolerability of DSP-5336 in relapsed/refractory AML or ALL
Time Frame:Approximately 2 months after first dose
Safety Issue:
Description:Occurrence of DLTs and frequency, duration and severity of TEAEs and SAEs assessed by NCI CTCAE v 5.0

Secondary Outcome Measures

Measure:Preliminary clinical activity of DSP-5336 in adult patients with AML or ALL
Time Frame:Approximately 6 months after first dose
Safety Issue:
Description:Disease response as assessed by ELN 2017 criteria


Phase:Phase 1/Phase 2
Primary Purpose:Interventional
Overall Status:Not yet recruiting
Lead Sponsor:Sumitomo Dainippon Pharma Oncology, Inc

Trial Keywords

  • Relapsed or refractory AML
  • MLLr or NPM1m
  • Menin

Last Updated

August 9, 2021