OncoMatch/Clinical Trials/NCT04067336
First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia
Is NCT04067336 recruiting? Yes, currently enrolling (May 2026). This Phase 1/2 trial studies multiple treatments including Ziftomenib and Midazolam for advanced malignant neoplasm.
Treatment: Ziftomenib · Midazolam · Itraconazole — In this trial, ziftomenib, a menin-MLL(KMT2A) inhibitor, will be tested in patients for the first time. The trial includes a Main Study and four sub-studies. In the Main Study (including Phase 1a, Phase 1b, and Phase 2 portions), ziftomenib will be evaluated in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML). The main study has completed enrollment. In Sub-studies 1 and 2, the effects of taking ziftomenib and other common drugs at the same time will be investigated in AML patients. In Sub-study 3, ziftomenib will be evaluated in patients with R/R acute lymphoblastic leukemia (ALL). In Sub-study 4, ziftomenib will be evaluated in patients with R/R AML with certain genetic mutations.
Check if I qualifyExtracted eligibility criteria
Cancer type
Tumor Agnostic
Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
Biomarker criteria
Required: KMT2A (MLL) rearrangement
documented lysine[K]-specific methyltransferase 2-rearrangement (KMT2A-r)
Required: NPM1 mutation
documented nucleophosmin 1 mutation (NPM1-m)
Performance status
ECOG 0–2(Ambulatory, capable of self-care)
Prior therapy
Must have received: standard of care therapies, including HSCT
failed or are ineligible for any approved standard of care therapies, including HSCT
Cannot have received: donor lymphocyte infusion
Donor lymphocyte infusion < 30 days prior to study entry
Cannot have received: immunosuppressive therapy post HSCT
Receiving immunosuppressive therapy post HSCT within 2 weeks of Cycle 1 Day 1
Cannot have received: chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational
Received chemotherapy immunotherapy, radiotherapy, or any ancillary therapy that is considered to be investigational (i.e., used for non-approved indications(s) and in the context of a research investigation) < 14 days prior to the first dose of ziftomenib or within 5 drug half-lives prior to the first dose of study drug
Cannot have received: major surgery
Major surgery within 4 weeks prior to the first dose of study treatment
Lab requirements
Blood counts
Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Patients may receive hydroxyurea to control and maintain white blood cell count prior to enrollment.
Kidney function
Adequate kidney function according to protocol requirements
Liver function
Adequate liver function according to protocol requirements
Cardiac function
Mean QTcF >480 ms on triplicate ECG [excluded]; significant cardiovascular disease including unstable angina pectoris, uncontrolled hypertension or arrhythmia, history of cerebrovascular accident including transient ischemic attack within the past 6 months, congestive heart failure (NYHA Class III or IV) related to primary cardiac disease, ischemic or severe valvular heart disease, or a myocardial infarction within 6 months prior to the first dose of study treatment [excluded]
Adequate liver and kidney function according to protocol requirements. Peripheral white blood cell (WBC) counts ≤ 30,000/μL. Mean QTcF >480 ms on triplicate ECG [excluded]; significant cardiovascular disease [excluded]
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- Banner MD Anderson Cancer Center · Gilbert, Arizona
- Mayo Clinic · Phoenix, Arizona
- University of Southern California · Los Angeles, California
- UCLA Ronald Reagan Medical Center · Los Angeles, California
- Mayo Clinic · Jacksonville, Florida
Showing up to 5 US sites. See all sites on ClinicalTrials.gov →
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