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Acute Lymphoblastic LeukemiaPhase 1NCT07498465

A Study to Find the Highest Dose of SNDX-5613 (Revumenib) as a Treatment Option After Hematopoietic Stem Cell Transplant in Children With Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Mixed Phenotype Acute Leukemia

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Children's Oncology Group.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

This phase I trial tests the safety, best dose, and effectiveness of revumenib given as maintenance therapy after standard hematopoietic stem cell transplant (HSCT) in patients with acute lymphoblastic leukemia, acute myeloid leukemia, or mixed phenotype acute leukemia. Read more about this study

Who is it for?
Ages 30 days to 22 years
What is being tested?
Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
10 Sep 2026to 12 Nov 2028
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 30 days to 22 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • STEP 0 PRE-TRANSPLANT INCLUSION CRITERIA:
  • Patients must be ≥ 30 days and < 22 years of age
  • PLEASE NOTE: Eligibility criteria to enroll onto Step 1 for the treatment trial is < 22 years of age at the time of Step 1 enrollment. Please plan accordingly to ensure that patients who are screened with Step 0 will be at an eligible age at the time of enrollment onto Step 1. Patients who are 21 at the time of screening who turn 22 at the time of enrollment onto Step 1 will not be eligible to enroll onto the study
  • Patients with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or mixed phenotype acute leukemia (MPAL) with a KMT2a rearrangement, NUP98 rearrangement, or NMP1 mutation confirmed in a College of American Pathologists (CAP)/Clinical Laboratory Improvement Act (CLIA) certified laboratory. Patients with a history of isolated or combined central nervous system (CNS) or extramedullary disease are eligible if they have no evidence of active CNS or extramedullary disease at the time of trial enrollment (Step 0) and treatment enrollment (Step 1). Eligible patients with histories of isolated or combined CNS or extramedullary disease at time of relapse are required to be in complete remission at time of transplant to be eligible for this study
  • AML and MPAL must be in morphologic complete remission confirmed by multiparameter flow (MDF) testing (with or without detectable minimal residual disease [MRD]). ALL must have bone marrow MRD < 0.1%
  • Pre-HSCT bone marrow:
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  • Assessment of disease status (complete response [CR] and minimal residual disease [MRD]) by multiparameter flow cytometry will be performed on bone marrow aspirate samples locally. Disease assessment will be required within 30 days prior to the start date of HSCT to determine CR (as part of the Step 0 screening criteria). Patients with CNS or extramedullary disease within 14 days prior to the start of the HSCT condition regimen are not eligible
  • Human immunodeficiency virus (HIV)-infected patients are eligible for this trial if the following criteria are met:
  • No history of HIV complications with the exception of CD4 count < 200 cells/mm^3
  • No antiretroviral therapy with overlapping toxicity such as myelosuppression
  • CD4 count > 500 cells/mm^3 prior to the diagnosis of newly diagnosed, relapsed, refractory AML
  • HIV viral loads below the limit of detection within 6 months, as long as the patient is NOT receiving anti-retroviral agents that may interact with revumenib
  • No history of highly active antiretroviral therapy (HAART)-resistant HIV
  • Lansky/Karnofsky performance status ≥ 70%
  • Must be receiving an allogeneic hematopoietic stem cell transplant (all graft and donor types will be eligible)
  • Must be receiving myeloablative conditioning as defined by Center for International Blood and Marrow Transplant Research (CIBMTR) criteria
  • STEP 1 INCLUSION CRITERIA:
  • Patients must be ≥ 30 days and < 22 years of age at the time of study enrollment to Step 0 and Step 1
  • Post-HSCT bone marrow:
  • Assessment of disease status (CR and minimal residual disease [MRD]) by multiparameter flow cytometry will be performed on bone marrow aspirate samples locally. Disease assessment will be required within 30 days prior to enrollment onto Step 1 to confirm MRD negativity (as part of the Step 1 Enrollment eligibility criteria). Disease must be in complete remission with marrow minimal residual disease < 0.05% for AML and < 0.01% for ALL by MDF. Patients with CNS or extramedullary disease post-HSCT are not eligible
  • Karnofsky ≥ 50% for patients > 16 years of age and Lansky ≥ 50% for patients ≤ 16 years of age. Patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
  • Patients must have recovered from the acute pre-transplant conditioning regimen related toxicities. If, after 42-100 days post-transplant, the eligibility criteria are met in criteria below the patient is considered to have recovered adequately
  • Pre-transplant exposure to revumenib will be allowed with the exception of patients who experienced a serious toxicity (CTCAE grade 4) attributed to revumenib (probably or definitely related) or those that experienced progression or relapse while receiving revumenib
  • For patients ≤ 17 years old estimated GFR (eGFR) ≥ 60 mL/min/1.73 m^2 "Bedside" Schwartz formula OR for any age group a 24 hour urine creatinine clearance ≥ 60 mL/min/1.73 m2 OR a GFR ≥ 60 mL/min/1.73 m2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) (must be performed within 7 days prior to enrollment)
  • For patients > 17 years old the Cockroft-Gault equation should be utilized to calculate eGFR OR for any age group a 24 hour urine creatinine clearance ≥ 60 mL/min/1.73 m2 OR a GFR ≥ 60 mL/min/1.73 m2. GFR must be performed using direct measurement with a nuclear blood sampling method OR direct small molecule clearance method (iothalamate or other molecule per institutional standard) (must be performed within 7 days prior to enrollment)
  • Bilirubin (sum of conjugated + unconjugated) ≤ 1.5 x upper limit of normal (ULN) for age (must be performed within 7 days prior to enrollment)
  • Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) ≤ 3 x upper limit of normal (ULN) (must be performed within 7 days prior to enrollment)
  • Aspartate aminotransferase (AST) ≤ 3 x upper limit of normal (ULN) (must be performed within 7 days prior to enrollment)
  • Albumin ≥ 2 g/dL (must be performed within 7 days prior to enrollment)
  • Shortening fraction of > 27% by echocardiogram, or ejection fraction of > 50% by gated radionuclide study with no evidence of congestive heart failure
  • Ejection fraction of > 50% by gated radionuclide study with no evidence of congestive heart failure
  • Corrected QT interval (QTc) < 450 ms
  • No evidence of pulmonary disease and without need for continuous supplemental oxygen
  • Potassium > 3.5mEq/L (supplementation allowed) (must be performed within 7 days prior to enrollment)
  • Magnesium ≥ 1.6mg/dL (supplementation allowed) (must be performed within 7 days prior to enrollment)
  • For patients with leukemia: platelet count ≥ 50,000 µL (without requirement for platelet transfusion within the last 7 days) (must be performed within 7 days prior to enrollment)
  • For patients with leukemia: hemoglobin ≥ 8.0 g/dL at baseline (may receive red blood cell [RBC] transfusions) (must be performed within 7 days prior to enrollment)
  • For patients with leukemia: absolute neutrophil count ≥ 1,000 µL with no myeloid growth factor support within the last 3 days (must be performed within 7 days prior to enrollment)

Who cannot join

  • STEP 0 PRE-TRANSPLANT EXCLUSION CRITERIA:
  • Participants who have had a previous hematopoietic stem cell transplantation
  • Participants who previously experienced a serious toxicity (Common Terminology Criteria for Adverse Events [CTCAE] grade 4) attributed to revumenib (probably or definitely related)
  • Participants who previously experienced a relapse while receiving revumenib
  • Patients diagnosed with Down syndrome
  • Patients known to have one of the following syndromes: Bloom syndrome, ataxia-telangiectasia, Fanconi anemia, Kostmann syndrome, Schwachman syndrome, or any other known bone marrow failure syndrome
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  • Patients with a secondary KMT2A-r leukemia that developed after treatment of prior malignancy with cytotoxic chemotherapy
  • Patients with a history of congenital prolonged QT syndrome, congestive heart failure or uncontrolled arrythmia in the past 6 months prior to study enrollment
  • AML with FLT3 ITD or other activating mutation, unless disease has proven unresponsive to TKI therapy or patient has experienced serious TKI related toxicity
  • Estimated glomerular filtration rate (GFR) of < 60 mL/min/1.73 m^2
  • Cardiac ejection fraction < 50% or shortening fraction < 27% (ECHO may be performed 3 months prior)
  • Clinical evidence of pulmonary disease or need for continuous supplemental oxygen for greater than 24 hours
  • Uncontrolled infection
  • Patients who have received another investigational drug within 30 days prior to Step 0 enrollment
  • STEP 1 EXCLUSION CRITERIA:
  • Pregnant or breast-feeding women will not be entered on this study because there is yet no available information regarding human fetal or teratogenic toxicities. Pregnancy tests must be obtained in girls who are post-menarchal. Males or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control, including a medically accepted barrier or contraceptive method (eg, male or female condom) for the duration of the study and for 4 months after the last revumenib dose. Abstinence is an acceptable method of birth control
  • Patients who are currently receiving another investigational drug are not eligible.
  • Patients who are currently receiving other anti-cancer agents are not eligible. Patients receiving intrathecal chemotherapy in the prior 14 days are eligible
  • Moderate or strong CYP3A4 inducers are prohibited during treatment. These agents should be discontinued at least 7 days prior to starting protocol therapy. Concomitant use of strong CYP3A4 inhibitors is permitted with appropriate revumenib dose modification
  • Avoid concomitant use with other drugs with a known potential to prolong QTc interval
  • Patient is not able to start the first cycle of revumenib from day +42 through day +100 post-HSCT
  • Patients with graft loss are not eligible
  • Patients with steroid refractory or dependent acute grade 3-4 graft versus host disease (GVHD) are not eligible
  • Patients who have an uncontrolled viral, bacterial, fungal, or protozoal infection are not eligible
  • Patients who have received a prior solid organ transplantation are not eligible
  • Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible

About this study

Official title: A Phase 1 Trial of the Menin Inhibitor SNDX-5613 (Revumenib) (NSC# 852942) for Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Patients With Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, and Mixed Phenotype Acute Leukemia

This phase I trial tests the safety, best dose, and effectiveness of revumenib given as maintenance therapy after standard hematopoietic stem cell transplant (HSCT) in patients with acute lymphoblastic leukemia, acute myeloid leukemia, or mixed phenotype acute leukemia. Revumenib binds to a protein called menin, which prevents menin from interacting with another protein called MLL. This results in an inhibition of the proliferation of leukemic cells with certain genetic alterations. Revumenib may inhibit the survival, growth, transformation and proliferation of certain kinds of leukemia cells. It is approved for the treatment of patients with certain types of acute leukemia, but it is not approved for maintenance therapy (treatment that aims to prevent cancer from coming back) after HSCT.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To estimate the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of revumenib monotherapy administered as maintenance therapy, orally post-HSCT, on continuous 28-day cycles for patients with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or mixed phenotype acute leukemia (MPAL).

II. To characterize the steady state pharmacokinetics of revumenib administered as post-HSCT maintenance in children with acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), or mixed phenotype acute leukemia (MPAL).

SECONDARY OBJECTIVES:

  1. To preliminarily estimate the 2-year relapse free survival of patients receiving revumenib as post-HSCT maintenance therapy at the MTD/RP2D, within the confines of a phase 1 study.

II. To describe treatment related adverse events in patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy up to 12 cycles.

III. To estimate the proportion of patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy who complete 12 cycles of maintenance therapy.

IV. To estimate the proportion of patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy who discontinue therapy due to treatment related adverse events.

EXPLORATORY OBJECTIVES:

  1. To estimate the cumulative incidence of transplant-related mortality in patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy for up to 12-cycles.

II. To estimate the cumulative incidence of transplant-related relapse in patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy for up to 12-cycles.

III. To preliminarily estimate overall survival and disease-free survival in patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy for up to 12-cycles, within the confines of a phase 1 study.

IV. To estimate the cumulative incidence of acute and chronic graft versus host disease (GVHD) in patients with ALL, AML, or MPAL receiving revumenib post-HSCT as maintenance therapy for up to 12-cycles.

OUTLINE: This is a dose-escalation study of revumenib followed by a dose-expansion study.

Starting 42-100 days after HSCT, patients receive revumenib orally (PO) or via nasogastric (NG)- or gastric (G)-tube every 12 hours on days 1-28 of each cycle. Cycles repeat every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients may receive optional intrathecal therapy (methotrexate intrathecally [IT] or cytarabine IT or methotrexate, hydrocortisone, and cytarabine IT) at the discretion of the physician on study. Patients also undergo bone marrow biopsy/aspiration and collection of blood samples throughout the trial. Patients may undergo echocardiography (ECHO) and radiologic assessment as clinically indicated.

After completion of study treatment, patients are followed for up to 1 year.

Being tested
Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Cytarabine, Echocardiography Test
Medical subject headings
Precursor Cell Lymphoblastic Leukemia-Lymphoma, Leukemia, Myeloid, Acute, Leukemia, Biphenotypic, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Biospecimen CollectionProcedure or surgery

    Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection

    Undergo collection of blood samples

  • Bone Marrow AspirationProcedure or surgery

    Undergo bone marrow aspiration

  • Bone Marrow BiopsyProcedure or surgery

    Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow

    Undergo bone marrow biopsy

  • CytarabineDrug

    Also called .beta.-Cytosine arabinoside, 1-.beta.-D-Arabinofuranosyl-4-amino-2(1H)pyrimidinone, 1-.beta.-D-Arabinofuranosylcytosine, 1-Beta-D-arabinofuranosyl-4-amino-2(1H)pyrimidinone

    Given IT

  • Echocardiography TestProcedure or surgery

    Also called EC, Echocardiography

    Undergo ECHO

  • MethotrexateDrug

    Also called Abitrexate, Alpha-Methopterin, Amethopterin, Brimexate

    Given IT

  • Radiologic ExaminationProcedure or surgery

    Also called Radiologic Evaluation, Radiologic Exam

    Undergo radiologic assessment

  • RevumenibDrug

    Also called Menin-Mixed Lineage Leukemia Protein-Protein Interaction Inhibitor SNDX-5613, Menin-MLL Inhibitor SNDX-5613, Menin-MLL Interaction Inhibitor SNDX-5613, SNDX 5613

    Given PO or via NG- or G-tube

  • Therapeutic HydrocortisoneDrug

    Also called Aeroseb-HC, Barseb HC, Barseb-HC, Cetacort

    Given IT

Study groups in NCT07498465
Study groupRoleWhat they receive
Treatment (revumenib)ExperimentalStarting 42-100 days after HSCT, patients receive revumenib PO or via NG- or G-tube every 12 hours on days 1-28 of each cycle. Cycles repeat every 28 days for up to 12 cycles in the absence of disease progression or unacceptable toxicity. Patients may receive optional intrathecal therapy (methotrexate IT or cytarabine IT or methotrexate, hydrocortisone, and cytarabine IT) at the discretion of the physician on study. Patients also undergo bone marrow biopsy/aspiration and collection of blood samples throughout the trial. Patients may undergo ECHO and radiologic assessment as clinically indicated.
Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Cytarabine; Procedure: Echocardiography Test; Drug: Methotrexate; Procedure: Radiologic Examination; Drug: Revumenib; Drug: Therapeutic Hydrocortisone

What is being measured

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

  1. Maximum tolerated dose (MTD)

    Will use the Bayesian Optimal Interval (BOIN) design to estimate the MTD. The BOIN design with up to three pre-defined dose levels will estimate the MTD.

    Measured: During cycles 1 and 2 (cycle length = 28 days)

  2. Recommended phase 2 dose (RP2D)

    Will use the BOIN design to estimate the RP2D. The BOIN design with up to three pre-defined dose levels will estimate the RP2D.

    Measured: During cycles 1 and 2 (cycle length = 28 days)

  3. Area under the concentration time curve of revumenib

    Median and range of the area under the concentration time curve of revumenib determined by sampling on cycle 1 day 7 at time 0, 0.25, 0.5, 1, 2, 4, and 6 hours post dose by study part and dose level.

    Measured: Up to day 7

  4. Maximum concentration of the time curve of revumenib

    Median and range of the maximum concentration of revumenib determined by sampling on cycle 1 day 7 at time 0, 0.25, 0.5, 1, 2, 4, and 6 hours post dose by study part and dose level.

    Measured: Up to day 7

  5. Half-life of revumenib

    Median and range of the half-life of revumenib determined by sampling on cycle 1 day 7 at time 0, 0.25, 0.5, 1, 2, 4, and 6 hours post dose by study part and dose level.

    Measured: Up to day 7

  6. Clearance of revumenib

    Median and range of the clearance of revumenib determined by sampling on cycle 1 day 7 at time 0, 0.25, 0.5, 1, 2, 4, and 6 hours post dose by study part and dose level.

    Measured: Up to day 7

Secondary outcomes (4)

  1. Relapse free survival

    Median relapse-free survival time will be estimated with 95% confidence intervals. 12-month relapse-free rates will also be estimated with 95 confidence intervals.

    Measured: At 2 years

  2. Incidence of treatment-related adverse events

    Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade. A patient will be counted only once for a given toxicity for the worst grade of that toxicity reported for that patient. Toxicity information recorded will include the type, severity, time of onset, time of resolution, and the probable association with the study regimen.

    Measured: Up to 12 cycles (cycle length = 28 days)

  3. Proportion of patients who complete 12 cycles of maintenance therapy

    The percent of adverse event-evaluable patients who successfully complete 12-cycles of maintenance therapy with revumenib will be estimated with a 95% confidence interval. Evaluable patients who do not complete 12-cycles of maintenance therapy for any reason will be considered a failure, and those who complete 12-cycles will be considered a success.

    Measured: Up to 12 cycles (cycle length = 28 days)

  4. Proportion of patients who discontinue therapy due to treatment-related adverse events

    The percent of adverse event-evaluable patients without discontinuation-related adverse events will be estimated with a 95% confidence interval.

    Measured: Up to 12 cycles (cycle length = 28 days)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted27 Mar 2026
  2. Study start10 Sep 2026 (estimated)
  3. Primary completion12 Nov 2028 (estimated)
  4. Study completion12 Nov 2028 (estimated)

Why it stopped: Other - Protocol moved to Withdrawn

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
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07498465
Sponsor's protocol ID
PEPN2513
Other IDs
NCI-2026-01768, PEPN2513, PEPN2513, UM1CA228823
Status last verified
Apr 2026
First submitted
19 Mar 2026
Last updated
5 May 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Acute Lymphoblastic Leukemia and Acute Myeloid Leukemia, run by Children's Oncology Group.

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 38 inclusion and 26 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 5 May 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?

Maximum tolerated dose (MTD) (time frame: During cycles 1 and 2 (cycle length = 28 days)). The study lists 6 primary outcomes in all. 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 NCT07498465.

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 5 May 2026; AzTrials last refreshed its copy on 7 Nov 2026.

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