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Leukemia, Myeloid, AcutePhase 2NCT07686107

Trial of Revumenib Plus FLA Chemotherapy for Children With Relapsed or Refractory NUP98-rearranged AML."

A Phase 2 study testing Revumenib, Fludarabine and Cytarabine (Ara-C) in people with Leukemia, Myeloid, Acute, run by Therapeutic Advances in Childhood Leukemia Consortium.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial summary

The goal of this clinical trial is to learn if Revumenib in combination with Fludarabine and Cytarabine can treat children with NUP98-rearranged relapsed or refractory Acute Myeloid Leukemia. Read more about this study

Who is it for?
Ages 1 year to 22 years
Conditions the study is for
Leukemia, Myeloid, Acute
What is being tested?
Revumenib, Fludarabine and Cytarabine (Ara-C)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
No sites listed yet
Study dates
31 Aug 2026to 31 Dec 2030
Phase
Phase 2
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 1 year to 22 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Age Patients must be > 1 year and < 22 years of age at time of enrollment.
  • Diagnosis Patients must have NUP98-r AML (according to WHO Classification of Hematolymphoid Tumors (5th Edition)) that is either refractory to or is in first relapse following initial therapy. Documentation of prior known NUP98-r AML is sufficient.
  • Definition of relapsed disease:
  • Recurrent disease with ≥ 5% leukemic blasts by morphology in the bone marrow after having achieved morphologic remission (morph-CR). Patients who have received any other re-induction therapy following relapse will not be eligible.
  • Morph-CR is defined as attainment of an M1 bone marrow (< 5% morphologic leukemic blasts) no evidence of circulating leukemic blasts or extramedullary disease and with recovery of peripheral blood counts (ANC ≥ 500/µL and platelet count ≥ 50,000/µL).
  • Definition of primary refractory disease:
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  • Refractory disease with ≥ 5% leukemic blasts by morphology in the bone marrow after one attempt at remission induction, which may consist of up to two different therapy courses (e.g., COG AAML1831 de novo therapy including Induction I and Induction II). Patients must not have received more than one remission induction remission attempt.
  • White Blood Cell Count:
  • White blood cell count must be <25,000 cells/uL at the time of enrollment on the study. Cytoreduction with leukapheresis, hydroxyurea, and/or cytarabine (up to a total dose of 500 mg/m^2) prior to enrollment is allowed.
  • Performance Level Patients must have a performance status corresponding to ECOG scores of 0, 1 or 2 (> 50 Lansky or Karnofsky score). Use Karnofsky for patients > 16 years of age and Lansky for patients ≤ 16 years of age (Appendix I). 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.
  • CNS Disease Patients may have status of CNS1, CNS2, or asymptomatic CNS3 disease. Patients with symptomatic CNS 3 disease are ineligible.
  • HIV-Infection HIV-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial, provided antiretroviral therapy does not have clinically significant drug-drug interactions with revumenib.
  • Prior Therapy:
  • Patients must have recovered from all prior treatment-related toxicities to grade ≤1 or the patient's baseline and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment.
  • Cytotoxic Chemotherapy: Must not have received within 14 days of entry onto this study, except for hydroxyurea or cytarabine
  • NOTE: Cytoreduction with hydroxyurea and cytarabine (up to a total dose of 500 mg/m2) must be discontinued prior to the start of protocol therapy.
  • Antibodies: ≥ 21 days must have elapsed from infusion of last dose of an antibody-drug conjugate. For unmodified antibodies or T cell engaging antibodies, 2 half-lives must have elapsed before enrollment. Any toxicity related to prior antibody therapy must be recovered to Grade ≤ 1.
  • Interleukins, Interferons and Cytokines (other than Hematopoietic Growth Factors): ≥ 21 days after the completion of interleukins, interferon or cytokines (other than Hematopoietic Growth Factors).
  • Hematopoietic Growth Factors: ≥ 14 days after the last dose of a long-acting growth factor (e.g., pegfilgrastim) or ≥ 7 days for short acting growth factor.
  • Radiation Therapy (RT):
  • 14 days have elapsed for local palliative RT (small port);
  • ≥ 84 days must have elapsed if prior craniospinal RT or if 50% radiation of pelvis;
  • ≥ 42 days must have elapsed if other substantial BM radiation.
  • Stem Cell Infusions:
  • ≥ 84 days since allogeneic (non-autologous) bone marrow or stem cell transplant (with or without TBI) or boost infusion (any stem cell product; not including DLI);
  • No evidence of graft versus host disease (GVHD).
  • Cellular Therapy: ≥ 28 days after the completion of DLI (donor lymphocyte infusion) or any type of cellular therapy (e.g. modified T cells, NK cells, dendritic cells, etc.).
  • NOTE: Potential subjects who have received an intervention intended to treat their primary malignancy which does not fall into one of the above categories will require a minimum 30 day washout from that intervention and the patient must be discussed with the Study Chair or designee who may determine a longer washout period as necessary.
  • NOTE: Intrathecal chemotherapy: No waiting period is required for patients having received any combination of intrathecal therapy (cytarabine, methotrexate, and/or corticosteroids).
  • Organ Function Requirements
  • Adequate Renal Function Defined as:
  • Patient must have a calculated creatinine clearance or radioisotope GFR ≥ 60ml/min/1.73m2.
  • Adequate Liver Function Defined as:
  • Total bilirubin (sum of conjugated + unconjugated) ≤ 1.5x institutional upper limit of normal for age (unless attributable to leukemic involvement), and
  • SGPT (ALT) and SGOT (ALT) must be ≤3x institutional upper limit of normal. Patients with ALT and AST ≥ 3x ULN attributable to leukemic involvement of the liver are eligible as long as both are ≤5x ULN.
  • Adequate Cardiac Function at Screening, Defined as:
  • Ejection fraction (EF) of ≥ 50% or if EF unavailable, shortening fraction (SF) ≥ 28% by echocardiogram. AND
  • Corrected QT (using Fridericia's correction [QTcF]) interval < 450 msecs. See Appendix II for directions on calculating QTcF.
  • NOTE: There are no specific electrolyte parameters for eligibility. However, it should be noted that, to limit QTc prolongation risk, patients must maintain adequate potassium and magnesium levels to initiate and continue revumenib on protocol therapy. See section 4.1.2.1 for electrolyte monitoring.
  • .
  • Reproductive Function
  • Female patients of childbearing potential must have a negative urine or serum pregnancy test confirmed within 72 hours prior to enrollment.
  • Female patients with infants must agree not to breastfeed their infants while on this study and for one week after the last revumenib dose.
  • Male and female patients of child-bearing potential must agree to use an effective method of contraception approved by the investigator during the study and for up to 4 months (16 weeks) after the last dose of revumenib.

Who cannot join

  • Isolated Extramedullary Disease: Patients with isolated extramedullary disease are ineligible.
  • Prior diagnosis of acute lymphoblastic leukemia: Patients experiencing lineage switch to AML will not be eligible for this study.
  • CNS3 Disease with clinical signs or neurologic symptoms suggestive of CNS leukemia at time of relapse, such as facial nerve palsy, brain/eye involvement or hypothalamic syndrome.
  • Infection: Patients with documented active, uncontrolled infection at the time of study enrollment.
  • Patients are excluded if they have:
  • Positive blood culture within 48 hours of study enrollment;
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  • Fever above 38.2⁰C within 48 hours of study enrollment with clinical signs of infection. NOTE: Fever that is determined to be due to tumor burden does NOT exclude patients if there are documented negative blood cultures for at least 48 hours prior to enrollment and no concurrent signs or symptoms of active infection or hemodynamic instability.
  • A positive fungal culture within 30 days of study enrollment.
  • Active fungal, viral, bacterial, or protozoal infection requiring IV treatment. Chronic prophylaxis therapy to prevent infections does not exclude patients.
  • Cardiac: Patients with a history of congenital prolonged QT syndrome, congestive heart failure, or uncontrolled arrhythmia in the past 6 months prior to study enrollment.
  • Gastrointestinal issues: Patients with gastrointestinal issues of the upper gastrointestinal tract that might affect oral drug absorption.
  • Prior menin inhibitor therapy
  • Patients with active GVHD are ineligible to enroll. Patients who are receiving systemic cyclosporine, tacrolimus, or other agents to prevent or treat either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible to enroll on the trial. Patients must be off calcineurin inhibitors for at least 4 weeks to be eligible.
  • Concomitant Medications CYP3A4 Inhibitors/Inducers: Systemically administered moderate or strong CYP3A4 inducers or strong CYP3A4 inhibitors should be discontinued for at least 5 half-lives or 7 days prior to enrollment (whichever is later) with the following exceptions: itraconazole, ketoconazole, posaconazole, or voriconazole.
  • Corticosteroids: Patients who are receiving systemic corticosteroids are not eligible except when given as physiologic dosing (prednisone equivalent of ≤ 10 mg/day if ≥18 years and ≤ 10 mg/m2/day for patients < 18 years is allowed).
  • Investigational Drugs: Patients who are receiving another investigational drug (for a time frame less than the 5 half-life periods of the respective investigational drug) are not eligible.
  • QTc Prolonging Agents: Medications with a known risk of Torsades de Pointes (TdP) are prohibited, with the exception of drugs that are used as standard supportive therapies (listed below). Medications with a possible or conditional risk of TdP are allowed. Consult CredibleMeds® at https://crediblemeds.org/ for risk classifications.
  • Medications with a known risk of TdP used as standard supportive therapies that are allowed for concomitant use: azithromycin, ciprofloxacin, fluconazole, levofloxacin, methadone (allowed if chronic, scheduled use > 2 weeks prior to revumenib initiation; do not initiate methadone during protocol treatment), ondansetron, pentamidine, and propofol.
  • See Appendix II for Cardiac Monitoring Guidelines Patients with a contraindication or unable to tolerate at least one of the following medications utilized for antifungal prophylaxis: itraconazole, ketoconazole, posaconazole, or voriconazole.
  • Patients will be excluded if they have a known allergy to any of the drugs used in the study.
  • Patients will be excluded if they have significant concurrent disease, illness, psychiatric disorder or social issue that would compromise patient safety or compliance with the protocol treatment or procedures, interfere with consent, study participation, follow up, or interpretation of study results.
  • Patients with DNA fragility syndromes (such as Fanconi anemia, Bloom syndrome) are excluded.
  • Patients with cirrhosis based on PELD and Child-Pugh scoring systems with moderate to severe hepatotoxicity are excluded.

About this study

Official title: T2023-004 Phase 2 Trial of Revumenib (SNDX-5613) in Combination With FLA Chemotherapy for Children With Relapsed or Refractory NUP98-rearranged Acute Myeloid Leukemia

The goal of this clinical trial is to learn if Revumenib in combination with Fludarabine and Cytarabine can treat children with NUP98-rearranged relapsed or refractory Acute Myeloid Leukemia. The main question it aims to answer is:

What is the Best Overall Response Rate by morphologic response criteria after up to two cycles of revumenib in combination with fludarabine and cytarabine (FLA) chemotherapy?

Participants will receive up to two cycles of Revumenib in combination with fludarabine and cytarabine with intrathecal triple therapy. Participant may also receive a stem cell transplant followed by Revumenib monotherapy if it is necessary.

Up to 27 children and young adults will be enrolled.

Being tested
Revumenib, Fludarabine, Cytarabine (Ara-C), Methotrexate (IT), Hydrocortisone (intrathecal)
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.

  • RevumenibDrug

    Patients under 40 kg, and those unable to swallow pills, must receive oral suspension (40 mg/mL):

    • < 40 kg: 95 mg/m2 (max 160 mg/dose) orally twice daily
    • ≥ 40 kg (flat-dose): 160 mg orally twice daily
    • Round to the nearest markings on the syringe, within 10% of the calculated dose

    Patients ≥ 40 kg that are able to swallow pills:

    • 160 mg orally twice daily

  • FludarabineDrug

    • 5 mg for patients with BSA 0.25-0.29
    • 6.25 mg for patients with BSA 0.3-0.34
    • 8.75 mg for patients with BSA 0.35-0.39
    • 10 mg for patients with BSA 0.4-0.44
    • 12.5 mg for patients with BSA 0.45-0.49
    • 15 mg for patients with BSA 0.5-0.54
    • 16.25 mg for patients with BSA 0.55-0.59
    • 30 mg/m2/dose for patients with BSA > 0.6

  • Cytarabine (Ara-C)Drug

    • 300 mg for patients with BSA 0.25-0.29
    • 420 mg for patients with BSA 0.3-0.34
    • 560 mg for patients with BSA 0.35-0.39
    • 680 mg for patients with BSA 0.4-0.44
    • 820 mg for patients with BSA 0.45-0.49
    • 960 mg for patients with BSA 0.5-0.54
    • 1100 mg for patients with BSA 0.55-0.59
    • 2000 mg/m2/dose for patients with BSA > 0.6

  • Methotrexate (IT)Drug

    Given intrathecally

    • 6 mg for patients > 30 days - < 1 year
    • 8 mg for patients > 1 - < 2 years
    • 10 mg for patients > 2 - < 3 years
    • 12 mg for patients > 3 years

  • Hydrocortisone (intrathecal)Drug

    Given intrathecally

    • 12 mg for patients > 30 days - < 1 year
    • 16 mg for patients > 1 - < 2 years
    • 20 mg for patients > 2 - < 3 years
    • 24 mg for patients > 3 years

  • Cytarabine (intrathecal)Drug

    Given intrathecally

    • 18 mg for patients > 30 days - < 1 year
    • 24 mg for patients > 1 - < 2 years
    • 30 mg for patients > 2 - < 3 years
    • 36 mg for patients > 3 years
Study groups in NCT07686107
Study groupRoleWhat they receive
Part 1ExperimentalRevumenib -Days 1-28 Fludarabine -Days 1-5 Cytarabine -Days 1-5 IT Therapy Triples (Methotrexate, Hydrocortisone, Cytarabine) -Day 0. May receive with marrow done at the end of the previous course. For CNS2 and CNS3 patients, in Cycle 1 continue weekly for 3 to 6 total doses or until 3 negative CSF samples, whichever comes first.
Receives: Drug: Revumenib; Drug: Fludarabine; Drug: Cytarabine (Ara-C); Drug: Methotrexate (IT); Drug: Hydrocortisone (intrathecal); Drug: Cytarabine (intrathecal)

What is being measured

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

  1. Best Overall Response Rate (BOR) by morphologic response criteria

    The best Overall Response rate according to morphologic response (Morph-CR/CRi/CRp) after up to 2 cycles

    Measured: At the end of Cycle 1 and Cycle 2 (each cycle is 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 posted7 Jul 2026
  2. Study start31 Aug 2026 (estimated)
  3. Primary completion31 Dec 2029 (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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07686107
Sponsor's protocol ID
T2023-004
Status last verified
Jul 2026
First submitted
25 Jun 2026
Last updated
22 Jul 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 2 study testing Revumenib, Fludarabine and Cytarabine (Ara-C) in people with Leukemia, Myeloid, Acute, run by Therapeutic Advances in Childhood Leukemia Consortium.

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 44 inclusion and 23 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: opening soon, as of the sponsor's last update on 22 Jul 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

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?

Best Overall Response Rate (BOR) by morphologic response criteria (time frame: At the end of Cycle 1 and Cycle 2 (each cycle is 28 days)). 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 NCT07686107.

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 22 Jul 2026.

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