NCT04988555 · Sumitomo Pharma America, Inc.
A Study of DSP-5336 in Relapsed/Refractory AML/ ALL With or Without MLL Rearrangement or NPM1 Mutation
What this study is about
A phase 1/2 gradually increasing doses / dose expansion study of Enzomenib (DSP-5336) in adult patients with acute leukemia.
View original scientific description
A phase 1/2 dose escalation / dose expansion study of Enzomenib (DSP-5336) in adult patients with acute leukemia.
Interventions
DRUG
Enzomenib
DSP-5336 orally
DRUG
azoles
Posaconazole, Voriconazole, or Fluconazole
DRUG
Venetoclax
Venetoclax orally
DRUG
Gilteritinib
Gilteritinib orally
DRUG
Azacitidine (AZA)
Azacitidine orally
DRUG
Intensive chemotherapy with 7 + 3
chemotherapy
Primary outcome measures
Number of patients with adverse events and serious adverse events in Phase 1
Time frame: 30 days from last dose
Assessment of safety of DSP-5336 administered in participants with advanced hematologic malignancies by reporting of adverse events and serious adverse events in Phase 1
Determination of Recommended Phase 2 Dose (RP2D)
Time frame: Within 4 months from first dose
The RP2D is based on adverse events, pharmacokinetics, and clinical response
Determination of Recommended Phase 2 Dose (RP2D) for patients with relapse and refractory AML who are enrolled into the combination venetoclax and azacitidine arm
Time frame: Within 4 months from first dose
The RP2D is based on adverse events, pharmacokinetics, and clinical response
Determination of Recommended Phase 2 Dose (RP2D) for patients with relapse and refractory AML who are enrolled into the gilteritinib arm
Time frame: Within 4 months from first dose
The RP2D is based on assessment of Dose Limiting Toxicities
Optimal dose of DSP-5336 (RP2D) for patients newly diagnosed with AML enrolled into the combination venetoclax and azacitidine arm
Time frame: Within 4 months from the first dose
The RP2D is based on adverse events, pharmacokinetics and clinical response
Determination of Recommended Phase 2 Dose (RP2D) for patients enrolled into the 7 + 3 arm
Time frame: Within 4 months from first dose
The RP2D is based on assessment of Dose Limiting Toxicities
Number of patients achieving complete response (CR) and complete response with partial hematologic recovery (CRh) in Phase 2
Time frame: Approximately 6 months after first dose
Disease response defined by the FDA guidance and ELN2017
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Patients in the Phase 1 dose-escalation portion must have a diagnosis of relapsed or refractory AML, ALL or acute leukemia of ambiguous lineage according to World Health Organization (WHO) 2022 classification, or, in the US only, a diagnosis of MDS or MM as determined by pathology review at the treating institution, and whose disease has progressed after available standard therapies known to be active for their AML, ALL, or acute leukemia of ambiguous lineage or, in the US, for MM or MDS. For patients with MDS (US only): 1. Patients with MDS must have IPSS-R risk categorization of "high" or "very high" at initial diagnosis or at study entry and have bone marrow blasts ≥ 5% (which is the definition of high-risk MDS in this study) 2. Patients with MDS must have relapsed or refractory disease and have exhausted available standard therapies including at least 2 cycles of treatment with HMA For patients with MM (US only): 1. Have a confirmed diagnosis of multiple my
Where
- Newport Beach, California
- Palo Alto, California
- Denver, Colorado
- Washington D.C., District of Columbia
- Miami, Florida
- Tampa, Florida
- Chicago, Illinois
- Baltimore, Maryland
- Boston, Massachusetts
- Morristown, New Jersey
- New Brunswick, New Jersey
- Buffalo, New York
And 15 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Mar 24, 2026 · Source of record for eligibility and locations