Ziftomenib for AML with NPM1 Mutation or KMT2A Rearrangement
This study is testing a drug called ziftomenib for people with acute myeloid leukemia (AML) that has specific genetic changes (NPM1 mutation or KMT2A rearrangement) and who cannot receive standard treatments. AML is a type of blood cancer, and these genetic changes can activate a protein pathway called menin, which helps cancer cells grow. Ziftomenib works by blocking this menin pathway, which may help to stop the cancer cells from growing. The main goal is to see how many participants achieve a complete remission (when signs of cancer disappear) or a response with hematologic improvement after 6 cycles of treatment. This study plans to enroll 70 participants.
- Study design
- This is a Phase 2 interventional study, meaning all participants will receive the study drug ziftomenib. It plans to enroll 70 participants.
- What's involved
- You would undergo blood sample collection, bone marrow biopsy and/or aspiration, and an echocardiography test. You would also receive cytarabine.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint is measured after 6 cycles of treatment (cycle length = 28 days).
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NCT06930352
Ziftomenib for the Treatment of Patients With NPM1 Mutated or KMT2A Rearranged Acute Myeloid Leukemia Not Eligible for Standard Therapy
Recruiting
PHASE2Ages 18+InterventionalTreatmentUma BorateInvestigator-initiated
~70 participants
Updated 2026-08-27 on ClinicalTrials.gov
What's tested:Biospecimen CollectionBone Marrow AspirationBone Marrow BiopsyCytarabineEchocardiography TestHydroxyurea
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Complete remission (CR) plus CR/response with hematologic improvement
Measured over After 6 cycles of treatment (cycle length = 28 days)
Conditions
Where it's being run
1 sites across 1 statesOhio1
Study leadership
- Uma M Borate, MBBS, MD, MSc · PRINCIPAL_INVESTIGATOR · Ohio State University Comprehensive Cancer Center
Who to contact
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Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Signed informed consent must be obtained prior to participation in the study
Morphologically confirmed diagnosis of the following based on 2022 World Health Organization (WHO) Classification:
Treatment-naïve acute myeloid leukemia
KMT2A rearrangement (defined as KMT2A translocations) OR NPM1 mutation (defined as NPM1 mutation resulting in cytoplasmic localization, or NPM1c) OR other mutations that have been shown to exhibit sensitivity to menin inhibition. Mutation status will be known from initial diagnosis using standard of care testing, which can be performed locally
Patients ineligible or unwilling to receive standard of care induction therapy, such as 7+3, hypomethylating agent, venetoclax, or other standard of care (SOC) regimens with ineligibility defined by the following:
≥ 75 years of age with both of the following;
Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 30 mL/min; calculated by the Cockcroft Gault formula or measured by 24-hours urine collection
Subject must have adequate liver function as demonstrated by aspartate aminotransferase (AST) ≤ 3.0 x upper limit of normal (ULN) and alanine aminotransferase (ALT) ≤ 3.0 x ULN (unless considered due to leukemic organ involvement) OR
≥ 18 to 74 years of age with at least one of the following co-morbidities:
Eastern Cooperative Oncology Group (ECOG) performance status of 2 or 3;
Cardiac history of congestive heart failure (CHF) requiring treatment or ejection fraction ≤ 50% or chronic stable angina;
Diffusion capacity of the lung for carbon monoxide (DLCO) ≤ 65% or forced expiratory volume in 1 second (FEV1) ≤ 65%;
Creatinine clearance ≥ 30 mL/min to \< 45 ml/min;
Moderate hepatic impairment with total bilirubin \> 1.5 to ≤ 3.0 x ULN;
Venous thromboembolism benefitting from prolonged anticoagulation or presence of prosthetic heart valve or any indication for therapeutic anticoagulation with a single agent
Prior history of severe infection requiring hospitalization with risk of recurrence with subsequent immunosuppression
Any other comorbidity that the physician judges to be incompatible with standard frontline therapy must be reviewed and approved by the study team before study enrollment
Peripheral white blood cell (WBC) counts ≤ 10,000/uL. Patients may receive hydroxyurea, cytarabine, or leukapheresis to control and maintain white blood cell count until the end of cycle 1
Women of childbearing potential must be willing to use a highly effective method of contraception throughout the study and for at least 180 days after the last dose of study treatment
Non-sterile male patients must agree to use a highly effective method of contraception with partner(s) throughout the study and for at least 90 days after the last dose of study treatment
Exclusion
Diagnosis of acute promyelocytic leukemia
Diagnosis of chronic myelogenous leukemia in blast crisis
Clinically active central nervous system (CNS) leukemia
Prior treatment for AML except for hydroxyurea and/or cytarabine used for control of leukocytosis
Treatment with concomitant drugs that are strong inhibitors or inducers of cytochrome P450-isozyme 3A4 (CYP3A4) with the exception of antibiotics, antifungals, and antivirals that are used as standard of care or to prevent or treat infections and other such drugs that are considered absolutely essential for the care of the patient
Detectable viral load for human immunodeficiency virus, hepatitis C, or hepatitis B surface antigen indicative of active infection. Patients with controlled disease will not be excluded from study enrollment
Pre-existing disorder predisposing the patient to a serious or life-threatening infection (e.g. cystic fibrosis, congenital or acquired immunodeficiency, bleeding disorder, or cytopenias not related to AML)
Active uncontrolled acute or chronic systemic fungal, bacterial, viral, or other infection
Mean Fridericia's formula-corrected QT interval (QTcF) \> 480 ms on triplicate electrocardiogram (ECG)
Any psychiatric illness that prevents patient from informed consent process
Women who are pregnant or lactating. All female patients with reproductive potential must have a negative serum pregnancy test within 72 hours prior to starting treatment
Participants requiring dual antiplatelet therapy
What this trial measures
- Complete remission (CR) plus CR/response with hematologic improvementAfter 6 cycles of treatment (cycle length = 28 days)
Will be assessed after 6 cycles of treatment using the best response achieved in that time. Will be calculated in each arm for the efficacy analysis population and reported along with two-sided 95% exact binomial confidence limits, in a modified intent-to-treat analysis.