Pediatric Acute LeukemiaPhase 1NCT07052994
A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients
A Phase 1 study testing Revumenib, Cytrabine and Daunorubicin in people with Pediatric Acute Leukemia, run by M.D. Anderson Cancer Center.
Trial summary
The goal of Phase 1a of this clinical research study is to find the highest tolerable dose of revumenib that can be given in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin to patients who have acute leukemia. Read more about this study
- Who is it for?
- Ages 6 months to 21 years
- Conditions the study is for
- Pediatric Acute Leukemia
- What is being tested?
- Revumenib, Cytrabine and Daunorubicin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 21 May 2026to 21 May 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 6 months to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age . 6 months - 21 years of age.
- ECOG performance status of < 2.
- Phase 1a portion of the study: Relapsed/refractory AML, or MPAL per ELN/NCCN with a myeloid phenotype with KMT2Ar, NPM1c, NUP98r and UBTF-ITD.
- Phase 1b portion of the study: Frontline AML, or MPAL per ELN/NCCN with a myeloid phenotype with KMT2Ar, NUP98r and UBTF-ITD.
- WBC must be below 25,000/ ƒÊL at time of enrollment. Patients may receive cytoreduction prior to enrollment.
- Baseline ejection fraction must be > 50%.
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- Adequate hepatic function (direct bilirubin < 2x 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).
- Potassium should be maintained at . 4.0 mEq/L and magnesium at > 2.0 mg/dL. Maintenance PO supplementation should be started for patients who are below these thresholds, and Revumenib dosing instructions as provided in Table 2 should be followed.
- Adequate renal function (creatinine clearance .
- Willing and able to provide informed consent.
- The time between the previous treatment and the start of the new treatment will be at least 14 days for both cytotoxic and non-cytotoxic therapies, including immunotherapies, or it will be equivalent to 5 half-lives of the prior therapy, whichever is shorter.
- Oral hydroxyurea and/or cytarabine (up to 2 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 4 months (120 days) 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 (120 days) after the last treatment.
Who cannot join
- Patients 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.
- 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) infection or known 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, cerebrovascular accident, or transient ischemic attack.
- QTc >450 msec for males and QTc >470 msec for females 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 fs 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.
- Patients on immunosuppressive therapy post-HSCT at the time of screening with R/R leukemia (must be off all systemic immunosuppression therapy for at least 2 weeks and calcineurin inhibitors for at least 4 weeks). The use of topical steroids for cutaneous graft-versus-host disease (GVHD) or stable systemic steroid doses less than or equal to 0.2-0.3mg/kg of prednisone daily are permitted.
- Patients GVHD grade >1 is not allowed.
About this study
The goal of Phase 1a of this clinical research study is to find the highest tolerable dose of revumenib that can be given in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin to patients who have acute leukemia.
The goal of Phase 1b of this clinical research study is to learn if the dose of revumenib in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin found in Phase 1a can help to control the disease.
Read the detailed description
Primary Objective:
To determine the safety, tolerability and recommended phase II dose (RP2D) of Revumenib incombination with cytarabine and daunorubicin and Gemtuzumab ozogamicin (GO) in relapsed refractory and frontline pediatric patients with acute leukemias, with primary endpoint defined as DLT (per Section 7.5)
Secondary Objective:
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 patients treated with this combination.
Exploratory Objective:
To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.
- Conditions
- Pediatric Acute Leukemia
- Being tested
- Revumenib, Cytrabine, Daunorubicin
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RevumenibDrug
Given by IV
CytrabineDrug
Given by IV
DaunorubicinDrug
Given by IV
| Study group | Role | What they receive |
|---|---|---|
| Dose Escalation of Revumenib in combination with cytrabine and Daunorubicin and GO | Experimental | Treatment will be administered on an inpatient basis Receives: Drug: Revumenib; Drug: Cytrabine; Drug: Daunorubicin |
| Dose Expansion of Revumenib in combination with cytrabine and Daunorubicin and GO | Experimental | Treatment will be administered on an inpatient basis Receives: Drug: Revumenib; Drug: Cytrabine; Drug: Daunorubicin |
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 posted8 Jul 2025
- Study start21 May 2026 (estimated)
- Primary completion21 May 2026 (estimated)
- Study completion21 May 2026 (estimated)
Why it stopped: PI requested
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pediatric Acute 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Branko Cuglievan, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07052994
- Sponsor's protocol ID
- 2024-1396
- Other IDs
- NCI-2025-04739
- Status last verified
- Sep 2026
- First submitted
- 30 Jun 2025
- Last updated
- 23 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 23 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients
- ClinicalTrials.gov ID
- NCT07052994
- Acronym / sponsor ID
- 2024-1396
- Conditions
- Pediatric Acute Leukemia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI requested
- Enrolment
- 0 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 21 May 2026 (estimated)
- Primary completion
- 21 May 2026 (estimated)
- Study completion
- 21 May 2026 (estimated)
- First posted
- 8 Jul 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 Phase 1a of this clinical research study is to find the highest tolerable dose of revumenib that can be given in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin to patients who have acute leukemia.
The goal of Phase 1b of this clinical research study is to learn if the dose of revumenib in combination with cytarabine, daunorubicin, and gemtuzumab ozogamicin found in Phase 1a can help to control the disease.
Detailed description
Primary Objective:
To determine the safety, tolerability and recommended phase II dose (RP2D) of Revumenib incombination with cytarabine and daunorubicin and Gemtuzumab ozogamicin (GO) in relapsed refractory and frontline pediatric patients with acute leukemias, with primary endpoint defined as DLT (per Section 7.5)
Secondary Objective:
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 patients treated with this combination.
Exploratory Objective:
To evaluate molecular and cellular markers that may be predictive of antitumor activity and/or resistance.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dose Escalation of Revumenib in combination with cytrabine and Daunorubicin and GO | Experimental | Drug: Revumenib, Drug: Cytrabine, Drug: DaunorubicinTreatment will be administered on an inpatient basis |
| Dose Expansion of Revumenib in combination with cytrabine and Daunorubicin and GO | Experimental | Drug: Revumenib, Drug: Cytrabine, Drug: DaunorubicinTreatment will be administered on an inpatient basis |
Who may qualify
- Ages
- 6 months to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Age . 6 months - 21 years of age.
- ECOG performance status of < 2.
- Phase 1a portion of the study: Relapsed/refractory AML, or MPAL per ELN/NCCN with a myeloid phenotype with KMT2Ar, NPM1c, NUP98r and UBTF-ITD.
- Phase 1b portion of the study: Frontline AML, or MPAL per ELN/NCCN with a myeloid phenotype with KMT2Ar, NUP98r and UBTF-ITD.
- WBC must be below 25,000/ ƒÊL at time of enrollment. Patients may receive cytoreduction prior to enrollment.
Key exclusion criteria (13 listed)
- Patients 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.
- 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) infection or known 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 |
|---|---|---|
| RevumenibGiven by IV | Drug | Dose Escalation of Revumenib in combination with cytrabine and Daunorubicin and GO, Dose Expansion of Revumenib in combination with cytrabine and Daunorubicin and GO |
| CytrabineGiven by IV | Drug | Dose Escalation of Revumenib in combination with cytrabine and Daunorubicin and GO, Dose Expansion of Revumenib in combination with cytrabine and Daunorubicin and GO |
| DaunorubicinGiven by IV | Drug | Dose Escalation of Revumenib in combination with cytrabine and Daunorubicin and GO, Dose Expansion of Revumenib in combination with cytrabine and Daunorubicin and GO |
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
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 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. This study is not recruiting at the moment.
Branko Cuglievan, MD principal investigator
M.D. Anderson Cancer Center
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Revumenib, Cytrabine and Daunorubicin in people with Pediatric Acute Leukemia, run by M.D. Anderson Cancer Center.
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 13 inclusion and 13 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: withdrawn, as of the sponsor's last update on 23 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. 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 23 Sep 2026.