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A study of Enzomenib in adult patients with relapsed or refractory acute leukemia, including those with MLL rearrangement or NPM1 mutation.

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on adults with Acute Leukemia, which is a type of cancer that affects the blood and bone marrow. The research specifically looks at patients with certain genetic changes, such as Mixed Lineage Leukemia or an MLL rearrangement, or those with an NPM1 mutation. These terms refer to specific changes or abnormalities in the DNA of the cancer cells that can influence how the disease behaves. The study also includes patients with Acute Myeloid Leukemia and Acute Lymphoblastic Leukemia that have returned after previous treatment or have not responded to it.

The purpose of the study is to evaluate the safety and effectiveness of the drug enzomenib, also known as DSP 5336. This medication is an oral tablet that is being tested as a single therapy. During the first part of the study, different doses of the drug will be given to determine the safest and most effective amount for patients. In the second part, the drug will be studied further in specific groups of patients to see how well it works against their particular type of cancer.

The research process

The trial runs in 2 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Participation in phase 1

    The first stage focuses on evaluating the safety and how well the body handles the enzomenib tablet.

    The goal is to find the correct dosage, which refers to the amount of medicine taken, by testing different levels to see which provides the best effect without causing too many side effects.

    During this stage, safety is monitored through checks on vital signs, which are basic body functions like heart rate, blood tests to check cell counts and chemistry, urine tests, and heart scans called electrocardiograms and echocardiograms.

  2. Step 2

    Participation in phase 2

    The second stage evaluates how effective the enzomenib tablet is in treating acute leukemia, which is a type of fast-growing blood cancer.

    This part of the study is specifically for those with certain genetic markers, such as mll rearrangement or npm1 mutation.

    Effectiveness is measured by looking for a complete remission, which means a state where signs of the cancer are no longer detectable, or a partial response, where the cancer has decreased in size or amount.

Who can join the trial?

17 criteria

  • You must be at least 18 years of age.
  • You must have a confirmed diagnosis of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), or acute leukemia of ambiguous lineage (a type of blood cancer where the specific cell type is not clearly defined).
  • Your cancer must be relapsed (it has returned after a period of improvement) or refractory (it has not responded to treatment).
  • The cancer must have continued to grow or worsen even after receiving standard treatments that are usually effective.
  • You must have a documented KMT2A (MLL) fusion or NPM1 mutation (specific genetic changes in the cancer cells).
  • For those in the second phase of the study, you must have at least 5% blasts (immature, cancerous white blood cells) in your bone marrow.
  • You must not have previously taken a type of medicine called a menin inhibitor.
  • You must have enough bone marrow or blood available for genomic analysis (testing the DNA of the cancer cells to look for specific genetic markers).
  • Your ECOG performance status must be 2 or less, which is a scale used to measure how well you can perform daily activities and your overall level of functioning.
  • Your white blood cell (WBC) count must be less than 30,000 per microliter at the start of the study.
  • Any side effects or toxicities from previous treatments must have improved to a low level, except for mild alopecia (hair loss) or neuropathy (nerve damage or tingling).
  • You must have adequate renal function (healthy kidney function) and adequate hepatic function (healthy liver function) as measured by specific blood tests.
  • Your doctor must estimate that you have a life expectancy of at least 3 months.
  • Women who are able to become pregnant must have a negative pregnancy test.
  • All participants of child-bearing age must agree to use highly effective contraception (birth control) or other pregnancy prevention methods during the study and for 6 months after the last dose.
  • If you are a candidate for a stem cell transplant (a procedure to replace damaged bone marrow), you must have been offered this option.
  • You must be willing to attend all required study visits as outlined in the research plan.

Who cannot join the trial?

21 criteria

  • You cannot participate if you have been diagnosed with acute promyelocytic leukemia, which is a specific type of blood cancer.
  • You cannot participate if you have cognitive, psychological, or psychosocial issues that prevent you from understanding the study, following the treatment plan, or attending required visits.
  • You cannot participate if you have had interstitial lung disease (inflammation or scarring of the lung tissue) or pneumonitis (inflammation of the lung tissue) caused by drugs that was moderate to severe within the last 6 months.
  • You cannot participate if you have had a hematopoietic stem cell transplant (HSCT), CAR-T therapy, or other modified T-cell therapies (specialized immune system treatments) within the last 60 days.
  • You cannot participate if you are taking certain medications that affect the CYP3A4/5 enzymes (proteins in the liver that help break down drugs) or certain types of antifungals (medicines used to treat fungal infections).
  • You cannot participate if you have an active infection of HIV (human immunodeficiency virus), Hepatitis C, or Hepatitis B.
  • You cannot participate if your doctor believes you have other medical conditions that could make the study unsafe, such as non-healing wounds, congestive heart failure (a condition where the heart cannot pump blood effectively), unstable angina (chest pain that is unpredictable), cardiac arrhythmia (an irregular heartbeat), a myocardial infarction (heart attack) within the last 6 months, or acute coronary syndrome (a sudden reduction in blood flow to the heart) within the last 6 months.
  • You cannot participate if you have significant pulmonary disease (serious lung issues like severe shortness of breath), uncontrolled high blood pressure, or diabetes that caused ketoacidosis (a dangerous buildup of acids in the blood) within the last 6 months.
  • You cannot participate if you have a history of Torsades de Pointes, which is a specific type of dangerous irregular heart rhythm.
  • You cannot participate if your ECG (an electrical test of the heart) shows a QTc interval greater than 480, which is a measurement of the time it takes for your heart muscle to recharge between beats.
  • You cannot participate if you are pregnant, breastfeeding, or planning to become pregnant.
  • You cannot participate if you received a donor lymphocyte infusion within the last 28 days or are receiving immunosuppressive therapy (medicines that lower the immune system) after a transplant.
  • You cannot participate if you have active graft-versus-host disease (GVHD) (a condition where transplanted immune cells attack the body) that requires medical treatment.
  • You cannot participate if you have taken antineoplastic agents (cancer-fighting drugs) or other experimental treatments within the last 14 days.
  • You cannot participate if you have used systemic calcineurin inhibitors (medicines that suppress the immune system) within the last 2 weeks or 42 weeks, depending on the specific protocol requirements.
  • You cannot participate if you have had major surgery within the last 28 days.
  • You cannot participate if you have leukemia in the central nervous system (cancer that has spread to the brain or spinal cord).
  • You cannot participate if you have a known allergy or hypersensitivity to any of the ingredients used in the study medication.
  • You cannot participate if your left ventricular ejection fraction (LVEF) is less than 50%, which is a measurement of how much blood your heart pumps out with each beat.
  • You cannot participate if you have an active and uncontrolled infection (bacterial, viral, or fungal) that requires parenteral therapy (medicine given through an injection or IV rather than by mouth).
  • You cannot participate if you have conditions that make it hard to swallow or absorb medicine, such as dysphagia (difficulty swallowing), short-gut syndrome, or gastroparesis (a condition where the stomach takes too long to empty its contents).
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Investigated drugs

Enzomenib is an experimental oral medication being tested to see if it is safe and effective for treating adults with certain types of blood cancers, such as acute leukemia. The study aims to find the best dose to treat these cancers by observing how patients respond to the medicine.

What is already known about the treatment

Enzomenib - This medication is taken by mouth in the form of a tablet and is currently being studied in clinical trials to evaluate its safety and effectiveness. It is classified as a targeted therapy designed to treat certain types of blood cancers, specifically acute leukemia with specific genetic changes like MLL rearrangement or NPM1 mutations. At the molecular level, it works by interfering with specific proteins that help cancer cells grow and survive, thereby slowing the progression of the disease.

Investigated diseases

Acute Leukemia with Mixed Lineage Leukemia (MLL)-rearrangement or Nucleophosmin 1 (NPM1) Mutation - This is a type of cancer that affects the blood and bone marrow. It involves the rapid and uncontrolled production of abnormal white blood cells. The disease is characterized by specific genetic changes, such as rearrangements in the MLL gene or mutations in the NPM1 gene. These genetic alterations cause the blood cells to grow improperly and interfere with the production of healthy blood cells. As the condition progresses, the abnormal cells accumulate in the bone marrow and blood.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2022-502741-10-00Protocol code​​DSP-5336-101​Estimated enrolment498 patientsSponsorSumitomo Pharma America Inc.

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