Ziftomenib for Acute Myeloid Leukemia (AML)
This study is investigating ziftomenib, an experimental drug, in combination with standard treatments for acute myeloid leukemia (AML). Ziftomenib will be given with venetoclax/azacitidine, venetoclax alone, or a combination of daunorubicin and cytarabine (7+3), sometimes with quizartinib. The study aims to understand the safety and side effects of these combinations, as well as how well they work in patients with AML who have specific genetic changes, like an NPM1 mutation or KMT2A rearrangement. Both newly diagnosed and those whose AML has returned or not responded to previous treatment can join. The study will enroll about 420 participants.
- Study design
- This is an interventional study with three separate arms, meaning participants will receive different combinations of treatments. It plans to enroll about 420 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- You will be followed for side effects from the start of treatment up to 28 days after 36 months of treatment. Your complete remission status will be monitored until relapse, new anti-cancer therapy, death, or up to 36 months of treatment.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05735184
A Study to Investigate the Safety and Tolerability of Ziftomenib in Combination With Venetoclax/Azacitidine, Venetoclax, 7+3, or 7+3+Quizartinib in Patients With AML
Recruiting
PHASE1Ages 18+InterventionalTreatmentKura Oncology, Inc.
~420 participants
Updated 2026-03-13 on ClinicalTrials.gov
What's tested:ZiftomenibVenetoclaxAzacitidineDaunorubicinCytarabineQuizartinib
At a glance
Recruiting sites
44 of 44 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Rate of dose limiting toxicities (DLTs) per dose level (Part 1a only)
Measured over During the first 28 days of ziftomenib in combination with SOC backbone treatment (1 cycle)
+2 more outcomes measured
Conditions
Where it's being run
44 sites across 24 statesNew York5
California4
Ohio3
Texas3
Colorado2
Georgia2
Illinois2
Kentucky2
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Patients must have a documented NPM1 mutation or KMT2A rearrangement and have either newly diagnosed or relapsed/refractory AML
Those intending treatment with intensive chemotherapy in Arm C should be NPM1-m and FLT3-ITD+ with an allelic ratio ≥0.05 and eligible for FLT3-targeted treatment
Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
Adequate liver, renal, and cardiac function according to protocol defined criteria
A female of childbearing potential must agree to use adequate contraception as well as a double barrier method from the time of screening through 180 days following the last dose of study intervention. A male of childbearing potential must agree to use abstinence or use a double barrier method of contraception from the time of screening through 180 days following the last dose of study intervention
Female patients of childbearing potential who receive quizartinib in Arm C should use a highly effective method of contraception during quizartinib treatment and for 7 months after the last dose
Exclusion
Diagnosis of either acute promyelocytic leukemia or blast phase chronic myeloid leukemia
Known history of BCR-ABL alteration
Advanced malignant hepatic tumor
Administration of live attenuated vaccines within 14 days prior to, during, or after treatment until B-cell recovery
Active central nervous system (CNS) involvement by AML.
Clinical signs/symptoms of leukostasis or WBC \> 25,000 / microliter. Hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine if used per institutional SOC for control of leukocytosis are permitted to meet this criterion
Not recovered to Grade ≤1 (NCI-CTCAE v5.0) from all nonhematological toxicities except for alopecia
Known clinically active human immunodeficiency virus, active hepatitis B or active hepatitis C infection
For newly diagnosed cohorts: received prior chemotherapy for leukemia, except hydroxyurea and/or leukapheresis and/or up to 2 doses of cytarabine per institutional standards to control leukocytosis, or prior treatment with all-transretinoic acid for initially suspected acute promyelocytic leukemia
For relapsed/refractory cohorts: received chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational \< 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug
Uncontrolled intercurrent illness including, but not limited to, cardiac illness as defined in the protocol
Mean QT interval corrected for heart rate by Fredericia's formula (QTcF)
Arm A and Arm B: \>480 ms on triplicate ECGs
Arm C: \>450 ms on triplicate ECGs
Uncontrolled infection
Women who are pregnant or lactating
An active malignancy and currently receiving chemotherapy for that malignancy or disease that is uncontrolled/progressing
Patients who have active GVHD requiring \>0.5 mg/kg prednisone or any new or increase in immunosuppressants in the prior 2 weeks for GVHD treatment
What this trial measures
- Rate of dose limiting toxicities (DLTs) per dose level (Part 1a only)During the first 28 days of ziftomenib in combination with SOC backbone treatment (1 cycle)
Assessed by the NCI-CTCAE v5.0
- Descriptive statistics of adverse eventsFrom Cycle 1 Day 1 up to and including 28 days following the end of 36 months of treatment
Assessed by the NCI-CTCAE v5.0
- Complete remission (CR) rateUntil relapse, new anti-cancer therapy, death, or up to 36 months of treatment, whichever occurs first
Assessed by the ELN 2022 criteria