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Acute Myeloid LeukemiaPhase 1NCT06448013

A Phase I Study Investigating the Combination of the Menin Inhibitor Ziftomenib With Venetoclax and Gemtuzumab in Pediatric Patients With Acute Myeloid Leukemia

A Phase 1 study testing Venetoclax, Gemtuzumab and Ziftomenib in people with Acute Myeloid Leukemia, run by M.D. Anderson Cancer Center at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

To find the recommended dose of ziftomenib in combination with gemtuzumab ozogamicin and venetoclax that can be given to pediatric participants who have relapsed or refractory AML or MPAL. Read more about this study

Who is it for?
Ages 3 to 21 years
Conditions the study is for
Acute Myeloid Leukemia
What is being tested?
Venetoclax, Gemtuzumab and Ziftomenib
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
1 siteUnited States
Study dates
7 Mar 2025to 31 Dec 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 3 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age ≥ 3 year to 21 years
  • Karnofsky for children >16yo and Lansky <16yo.
  • Relapsed/refractory AML, or MPAL with a myeloid phenotype.
  • Evidence of leukemia (AML, MPAL with a myeloid phenotype) in the bone marrow as detected by morphology or molecular diagnostics.
  • Presence of KMT2Ar, NUP98r, NPM1c, UBTF-ITD or other HOX pathway mutation.
  • WBC must be below 25,000/ƒÊL at time of enrollment. Participants may receive cytoreduction prior to enrollment.
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  • Baseline ejection fraction must be > 40%.
  • Adequate hepatic function (direct bilirubin < 1.5x upper limit of normal (ULN) unless increase is due to Gilbert's disease or leukemic involvement, 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).
  • Adequate renal function (creatinine clearance ≥ 30 mL/min) unless related to disease.
  • 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. Oral hydroxyurea and/or cytarabine (up to 2 g/m2) for participants 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.
  • 3 month washout prior from bone marrow transplantation.
  • Unless surgically or biologically sterile: Women of childbearing potential must agree to adequate methods of contraception during the study and at least 3 months for males, and 6 months for females, after the last treatment.

Who cannot join

  • Participants who weigh less than 10kg.
  • Participants with any concurrent uncontrolled medical condition, laboratory abnormality, or psychiatric illness which could place the patient at unacceptable risk of study treatment.
  • 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.
  • Participants with any severe gastrointestinal or metabolic condition which could interfere with the absorption of oral study medications.
  • Participants with chronic liver disease.
  • Participants with a concurrent active malignancy under treatment.
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  • Known active hepatitis B (HBV) or Hepatitis C (HCV) infection or known HIV infection.
  • Female participants who are pregnant or breast-feeding.
  • Participants 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, cerebrovascular accident, or transient ischemic attack.
  • Mean corrected QT interval by Fredericia's formula >480 ms on triplicate 12-lead electrocardiograms performed within approximately 5 minutes of each other.
  • 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 participant's participation for the full duration of the study, or is not in the best interest of the patient to participate.
  • Clinically active central nervous system (CNS) leukemia.
  • The use of topical steroids for cutaneous graft-versus-host disease (GVHD) or stable systemic steroid doses less than or equal to 20 mg of prednisone daily are permitted.
  • Participants 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.

Branko Cuglievan, MD contact

(713) 563-1499

bcuglievan@mdanderson.org

United States1 site, 1 recruiting

  • MD Anderson Cancer Center

    Houston, Texas, 77030

    Branko Cuglievan, MD, 713-563-1499, bcuglievan@mdanderson.org

    Branko Cuglievan, MD

    Recruiting

Other trials in United States

About this study

To find the recommended dose of ziftomenib in combination with gemtuzumab ozogamicin and venetoclax that can be given to pediatric participants who have relapsed or refractory AML or MPAL.

Read the detailed description

Primary Objectives:

  • To determine the safety, tolerability, and recommended Phase II dose (RP2D) of ziftomenib in combination with gemtuzumab ozogamicin and venetoclax for pediatric participants with acute myeloid leukemia (AML).

Secondary Objectives:

  • To determine the preliminary assessment of efficacy by overall response (OR), including complete remission (CR), CR with incomplete blood count recovery and partial remission, overall survival (OS), event-free survival (EFS) and duration of response (DOR) of pediatric participants treated with this combination.
  • To determine time to first response and time to best response of pediatric participants treated with this combination.

Exploratory Objective

  • To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.
Being tested
Venetoclax, Gemtuzumab, Ziftomenib
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.

  • VenetoclaxDrug

    Also called ABT-199, GDC-0199

    Given by PO

  • GemtuzumabDrug

    Also called Gemtuzumab ozogamicin, Mylotarg

    Given by IV

  • ZiftomenibDrug

    Given by PO

Study groups in NCT06448013
Study groupRoleWhat they receive
Dose Escalation + Dose ExpansionExperimentalParticipants found to be eligible to take part in this study, you will be assigned to a dose level of ziftomenib based on when you join this study. Up to 4 dose levels of ziftomenib will be tested. Up to 6 participants will be enrolled at each dose level. The first group of participants will receive the lowest dose level. Each new group will receive a higher dose than the group before it, if no intolerable side effects were seen. This will continue until the recommended dose of ziftomenib is found.
Receives: Drug: Venetoclax; Drug: Gemtuzumab; Drug: Ziftomenib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

  1. First posted7 Jun 2024
  2. Study start7 Mar 2025
  3. Primary completion31 Dec 2028 (estimated)
  4. Study completion31 Dec 2030 (estimated)

Resources

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
22 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06448013
Sponsor's protocol ID
2023-0911
Other IDs
NCI-2024-04821
Status last verified
Jun 2026
First submitted
3 Jun 2024
Last updated
12 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Venetoclax, Gemtuzumab and Ziftomenib 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 12 Jun 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 Branko Cuglievan, MD ((713) 563-1499, bcuglievan@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?

On the ClinicalTrials.gov record for NCT06448013.

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 12 Jun 2026.

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