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Investigating Ziftomenib with Other Treatments for Acute Myeloid Leukemia

Phase 1 Study of Venetoclax/Azacitidine or Venetoclax in Combination With Ziftomenib or Standard Induction Cytarabine/Daunorubicin (7+3) Chemotherapy in Combination With Ziftomenib for the Treatment of Patients With Acute Myeloid Leukemia

Phase 1 Interventional Kura Oncology, Inc. · NCT05735184

This study is testing if a new drug called ziftomenib, when combined with other treatments, can help people with acute myeloid leukemia who have certain genetic mutations feel better and stay safe during treatment.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment420 (estimated)
Ages18 Years and up
SexAll
SponsorKura Oncology, Inc. Industry-sponsored
Drugs / interventionsquizartinib, chemotherapy, immunotherapy, prednisone
Locations44 sites (Phoenix, Arizona and 43 other locations)
Trial IDNCT05735184 on ClinicalTrials.gov

What this trial studies

This Phase 1 study aims to evaluate the safety and tolerability of ziftomenib when combined with venetoclax and azacitidine, venetoclax alone, or the 7+3 regimen in patients with acute myeloid leukemia (AML) who have specific genetic mutations. The study will focus on two molecularly-defined groups: those with NPM1 mutations and those with KMT2A rearrangements. Participants will be monitored for preliminary antileukemic activity and overall safety of the treatment combinations. The study is designed for patients who are newly diagnosed or have relapsed/refractory AML.

Who should consider this trial

Good fit: Ideal candidates include patients with newly diagnosed or relapsed/refractory AML who have documented NPM1 mutations or KMT2A rearrangements.

Not a fit: Patients with acute promyelocytic leukemia or those with a known history of BCR-ABL alteration may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new effective treatment option for patients with specific genetic profiles of AML.

How similar studies have performed: Other studies have shown promise in targeting specific genetic mutations in AML, suggesting that this approach may be beneficial.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

* 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

Key Exclusion Criteria:

* 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

Where this trial is running

Phoenix, Arizona and 43 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Acute Myeloid LeukemiaMixed Lineage Leukemia Gene MutationRefractory AMLAML With Mutated NPM1Acute Myeloid Leukemia RecurrentAcute Myeloid Leukemia, in RelapseNPM1 MutationKMT2Ar
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.