Phase 1/2 Study of Enzomenib for Acute Leukemia

This study, called Horizen-1, is testing a new oral medication called Enzomenib (DSP-5336) for people with acute leukemia (a type of blood cancer), including those whose disease has returned or not responded to previous treatments. It's also looking at Enzomenib in combination with other standard treatments like Venetoclax, Azacitidine, Cytarabine, and Daunorubicin. The study aims to find the safest and most effective dose of Enzomenib and see how well it works. Researchers will be monitoring for any side effects and how the body processes the medication. You may be eligible if you are at least 12 years old and have certain types of leukemia, myelodysplastic syndromes (MDS), or multiple myeloma (MM). The study is currently enrolling up to 606 participants, but its overall status is unclear.

Study design
This is an open-label, Phase 1/2 study that will gradually increase the dose of Enzomenib to find the best amount. It plans to enroll up to 606 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Researchers will monitor for side effects for 30 days after your last dose. The recommended dose will be determined within 4 months from your first dose.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT04988555

A Phase 1/2 Study of Enzomenib (DSP-5336) in Patients With Acute Leukemia (Horizen-1)

Recruiting
PHASE1Ages 12+InterventionalTreatment
Sumitomo Pharma America, Inc.
~606 participants
Updated 2026-03-24 on ClinicalTrials.gov
What's tested:EnzomenibazolesVenetoclaxGilteritinibAzacitidine (AZA)Intensive chemotherapy with 7 + 3

At a glance

Recruiting sites
80 of 104 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of patients with adverse events and serious adverse events in Phase 1
Measured over 30 days from last dose
+6 more outcomes measured
Leukemia, Myeloid, Acute
Leukemia, Lymphocytic, Acute
Multiple Myeloma
Myelodysplastic Syndromes
104 sites across 36 states
France12
Spain11
United Kingdom10
Italy9
Japan6
Belgium4
South Korea4
Florida3

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  • Number of patients with adverse events and serious adverse events in Phase 130 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)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 armWithin 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 armWithin 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 armWithin 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 armWithin 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 2Approximately 6 months after first dose

    Disease response defined by the FDA guidance and ELN2017