Acute Myeloid LeukemiaPhase 1NCT06769490
Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia
A Phase 1 study testing Ziftomenib and Quizartinib in people with Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r ~ 40-45%). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- Ziftomenib and Quizartinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteUnited States
- Study dates
- 10 Jul 2025to 15 Sep 2030
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age . 18 years.
- ECOG performance status of < 2.
- Relapsed or refractory AML or myeloid mixed-phenotype acute leukemia (MPAL) with NPM1mt, or KMT2Ar, or NUP98r.
- WBC must be below 25,000/ ƒÊL at time of enrollment. Patients may receive cytoreduction prior to enrollment.
- Baseline ejection fraction must be > 40%.
- Adequate hepatic function (total bilirubin < 2x upper limit of normal (ULN) unless increase is due leukemic involvement (<2.5 ULN), unless due to ongoing hemolysis or Gilbert's syndrome and AST and/or ALT < 3x ULN unless considered due to leukemic involvement, in which case direct bilirubin or AST and/or ALT < 5x ULN will be considered eligible).
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- Adequate renal function with an estimated glomerular filtration rate . 50 mL/min (using Cockcroft-Gault) unless related to disease.
- Able to swallow pills.
- Patient or parent/guardian is willing and able to provide informed consent.
- In the absence of rapidly proliferative disease, the interval from prior treatment to time of initiation will be at least 14 days for cytotoxic or non-cytotoxic (immunotherapy agent(s), or an interval of 5 half-lives of the prior therapy, whichever is longer. Oral hydroxyurea and/or cytarabine (up to 1 g/m2) for patients with rapidly proliferative disease is allowed before the start of study therapy, as needed, for clinical benefit and after discussion with the PI.
- Concurrent therapy for central nervous system (CNS) prophylaxis or continuation of therapy for controlled CNS disease is permitted.
- Women of childbearing potential must agree to adequate methods of contraception during the study and at least 7 months for females and 4 months for males after the last treatment. Males must be surgically or biologically sterile or agree to use an adequate method of contraception during the study and at least 4 months after the last treatment.
Who cannot join
- Prior treatment with a menin inhibitor.
- The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea for patients with rapidly proliferative disease or for control of counts during differentiation syndrome. (3) use of steroids for treatment of differentiation syndrome.
- Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications.
- Patients with a concurrent active malignancy under treatment.
- Known active hepatitis B (HBV) or Hepatitis C (HCV) or HIV infection.
- Female subjects who are pregnant or breast-feeding.
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- Patient has an active uncontrolled infection.
- Any of the following within the 6 months prior to study entry: myocardial infarction, uncontrolled/unstable angina, congestive heart failure (New York Heart Association Classification Class .II), life-threatening, uncontrolled arrhythmia including ventricular arrythmias or torsades de pointes, cerebrovascular accident, or transient ischemic attack.
- History of sustained bradycardia of less than 50 beats per minute unless the subject has a pacemaker.
- Diagnosis of or suspicion of congenital long QT syndrome (including family history of congenital long QT syndrome).
- Uncontrolled hypertension with a systolic blood pressure .180 mmHg or diastolic blood pressure .110 mmHg, sustained despite optimal medical management.
- QTc >450 msec using the Fridericia Formula.
- History of or any concurrent condition, therapy, or laboratory abnormality that in the Investigator's opinion might confound the results of the study, interfere with the patient's participation for the full duration of the study, or is not in the best interest of the patient to participate. Patients under legal protection measure (guardianship, trusteeship or safeguard of justice) and/or uncontrolled psychiatric comorbidities, ongoing illicit substance abuse, inability, any impairment or unwillingness to comply with the treatments, follow-up, requirements and procedures of this clinical trial.
- Clinically active central nervous system (CNS) leukemia.
- Patients with Grade > 2 active acute GVHD, moderate or severe limited chronic GVHD, or extensive chronic GVHD of any severity.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030
Ghayas Issa, MD, 713-745-6798, gcissa@mdanderson.org
Ghayas Issa, MD
About this study
Official title: Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia
The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r ~ 40-45%).
Read the detailed description
Primary Objective:
Cohort 1A: To determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with quizartinib and assess the safety and the maximum tolerated dose (MTD).
Secondary Objectives:
To assess the preliminary efficacy of the combination of quizartinib and ziftomenib by FLT3 mutational status.
To assess overall survival (OS), relapse-free survival (RFS), event-free survival (EFS) and duration of response (DOR).
To characterize the pharmacokinetic profile of ziftomenib and quizartinib when used in combination.
To assess minimal residual disease negativity by flow cytometry.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- Ziftomenib, Quizartinib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZiftomenibDrug
Participants will receive treatment in tablet form
QuizartinibDrug
Participants will receive treatment in tablet form
| Study group | Role | What they receive |
|---|---|---|
| Ziftoenib + Quizartinib Q4W | Experimental | Participants will be randomized to study Receives: Drug: Ziftomenib; Drug: Quizartinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Adverse Events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2025
- Study start10 Jul 2025
- Primary completion15 Sep 2028 (estimated)
- Study completion15 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 44 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Ghayas Issa, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06769490
- Sponsor's protocol ID
- 2024-1193
- Other IDs
- NCI-2024-10248, NCI-2025-00080
- Status last verified
- Sep 2026
- First submitted
- 31 Dec 2024
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1 Dose Escalation and Expansion of Ziftomenib in Combination With Quizartinib in Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT06769490
- Acronym / sponsor ID
- 2024-1193
- Conditions
- Acute Myeloid Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 44 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 10 Jul 2025 (actual)
- Primary completion
- 15 Sep 2028 (estimated)
- Study completion
- 15 Sep 2030 (estimated)
- First posted
- 10 Jan 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this all-oral combination is to deliver safe and effective therapy for the largest portion of AML subtypes (NPM1mt, KMT2Ar, NUP98r ~ 40-45%).
Detailed description
Primary Objective:
Cohort 1A: To determine the recommended phase 2 dose (RP2D) of ziftomenib in combination with quizartinib and assess the safety and the maximum tolerated dose (MTD).
Secondary Objectives:
To assess the preliminary efficacy of the combination of quizartinib and ziftomenib by FLT3 mutational status.
To assess overall survival (OS), relapse-free survival (RFS), event-free survival (EFS) and duration of response (DOR).
To characterize the pharmacokinetic profile of ziftomenib and quizartinib when used in combination.
To assess minimal residual disease negativity by flow cytometry.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ziftoenib + Quizartinib Q4W | Experimental | Drug: Ziftomenib, Drug: QuizartinibParticipants will be randomized to study |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Age . 18 years.
- ECOG performance status of < 2.
- Relapsed or refractory AML or myeloid mixed-phenotype acute leukemia (MPAL) with NPM1mt, or KMT2Ar, or NUP98r.
- WBC must be below 25,000/ ƒÊL at time of enrollment. Patients may receive cytoreduction prior to enrollment.
- Baseline ejection fraction must be > 40%.
Key exclusion criteria (15 listed)
- Prior treatment with a menin inhibitor.
- The use of other chemotherapeutic agents or anti-leukemic agents is not permitted during study with the following exceptions (1) intrathecal chemotherapy for prophylactic use or for controlled CNS leukemia. (2) use of hydroxyurea for patients with rapidly proliferative disease or for control of counts during differentiation syndrome. (3) use of steroids for treatment of differentiation syndrome.
- Patients with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications.
- Patients with a concurrent active malignancy under treatment.
- Known active hepatitis B (HBV) or Hepatitis C (HCV) or HIV infection.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZiftomenibParticipants will receive treatment in tablet form | Drug | Ziftoenib + Quizartinib Q4W |
| QuizartinibParticipants will receive treatment in tablet form | Drug | Ziftoenib + Quizartinib Q4W |
Primary outcome
Safety and Adverse Events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ghayas Issa, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Ziftomenib and Quizartinib in people with Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ghayas Issa, MD ((713) 745-6798, gcissa@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Adverse Events (AEs) (time frame: Through study completion; an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.