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Menin-Inhibitor Targeted Maintenance in AML

Phase 2 Randomized Controlled Study of Revumenib as Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation in Patients With KMT2Ar, NPM1m, or NUP98r Acute Myeloid Leukemia (AML)

Revumenib is a first in class oral menin inhibitor that targets a central oncogenic dependency shared across KMT2Ar, NPM1m, and NUP98r AML. In addition to suppressing leukemogenic transcriptional programs and promoting leukemic differentiation, menin inhibition has been shown to modulate epigenetic states linked to antigen presentation and immune recognition. These properties provide a strong biological rationale for evaluating revumenib as maintenance therapy following alloHCT, with the goal of suppressing residual leukemic clones while preserving or enhancing GVL activity during immune reconstitution.

Studieoversigt

Detaljeret beskrivelse

Menin is a critical cofactor for oncogenic transcriptional programs in AML subsets driven by KMT2A rearrangements, NPM1 mutations, and NUP98 rearrangements. The interaction between menin and KMT2A promotes aberrant expression of HOX and MEIS genes, maintaining leukemic self-renewal and blocking differentiation. Revumenib is a potent, selective, oral small-molecule inhibitor of the menin-KMT2A interaction that has demonstrated clinical activity in relapsed or refractory AML. Beyond its direct anti-leukemic effects, emerging preclinical data indicate that menin inhibition may favorably modulate leukemia-immune interactions in the post-transplant environment. Menin inhibition has been shown to induce myeloid differentiation and increase expression of antigen presentation machinery, including MHC class II, in KMT2Ar and NPM1m AML. This effect is mediated through activation of interferon-related signaling pathways and results in enhanced recognition of leukemia cells by donor T cells. In parallel, menin inhibition has been shown to augment donor T-cell effector function and reduce T-cell exhaustion, collectively strengthening the GVL response without directly increasing alloreactivity against normal tissues.

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

146

Fase

  • Fase 2

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiekontakt

  • Navn: Paul Guo
  • Telefonnummer: 4583 7634064583
  • E-mail: pguo@nmdp.org

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

  • Voksen
  • Ældre voksen

Tager imod sunde frivillige

Ingen

Beskrivelse

Inclusion Criteria:

Inclusion Criteria:

  1. Aged ≥18 years at the time of signing informed consent
  2. Able to provide written informed consent personally or via a legally authorized representative in accordance with applicable regulatory and institutional requirements
  3. Willing and able to comply with all study procedures and available for the duration of the study
  4. Diagnosis of acute myeloid leukemia (AML) in complete morphologic remission with one of the following molecular abnormalities:

1. KMT2A-rearranged (KMT2Ar) AML (Excluding KMT2A partial tandem duplication (KMT2A-PTD) 2. NPM1-mutated (NPM1m) AML (Including FLT3-ITD or TKD co-mutation) 3. NUP98-rearranged (NUP98r) AML 5. Planned first allogeneic hematopoietic cell transplantation (allo-HCT) for AML.

6. Transplant Characteristics

  1. Planned allo-HCT using bone marrow or peripheral blood stem cell graft source.
  2. Planned reduced-intensity/non-myeloablative conditioning (RIC/NMA) or myeloablative conditioning (MAC), using a conditioning regimen permitted- by the protocol and consistent with standard clinical practice, meeting CIBMTR criteria for conditioning intensity

7. Planned donor:

  1. HLA-matched related donor (5/6 or 6/6)
  2. Matched unrelated donor (8/8)
  3. Mismatched unrelated donor (7/8)
  4. Haploidentical donor meeting institutional requirements

8. Performance Status:

1. Karnofsky Performance Status ≥70%. 9. Cardiac Function: left ventricular ejection fraction (LVEF) by transthoracic echocardiogram (TTE) or multigated acquisition (MUGA) with no clinical evidence of heart failure: RIC/NMA: ≥50% MAC: ≥5 10. Pulmonary function meeting the following criteria, without supplemental oxygen other than CPAP:

  1. RIC/NMA: DLCO (corrected for hemoglobin) and FEV1 ≥40% predicted
  2. MAC: DLCO and FEV1 ≥50% predicted

11. Renal Function: estimated creatinine clearance (CrCl) ≥45mL/min calculated using the Cockcroft-Gault formula or 24-hour urine collection, consistent with standard eligibility criteria for allogeneic HCT recipients.

12. Liver function acceptable per local institutional guidelines for allo-HCT eligibility.

13. Reproductive Status: Willingness to use contraception in accordance with local regulations from first study intervention through the required contraceptive period Willingness to use contraception in accordance with local regulations from first study intervention through the required contraceptive period

Exclusion Criteria:

  1. Disease Status:

    a. Evidence of active AML prior to HCT, assessed within 42 days before transplant, defined as any of the following:

    • ≥5% bone marrow blasts
    • Circulating blasts within 14 days before conditioning
    • CNS or other extramedullary disease
  2. Other active malignancy that, in the investigator's judgment, could interfere with safety or efficacy assessment
  3. Treatment with non-protocol antileukemic therapy (donor lymphocyte infusion for relapse prophylaxis or treatment will be considered an EFS event)
  4. Cardiac / QT Risk

    1. Requirement for concomitant medications known to prolong QT/QTc interval, except low-risk agents used as standard supportive care
    2. Diagnosis or suspicion of Long QT syndrome, or a family history of Long QT syndrome
    3. Fridericia's corrected QT interval (QTcF) >450 msec.
    4. History within 6 months of study entry of:

    i. Myocardial infarction ii. Unstable angina iii. Congestive heart failure (NYHA Class ≥ II) iv. Life-threatening or uncontrolled arrhythmia v. Cerebrovascular accident or transient ischemic attack

  5. Chronic respiratory disease requiring continuous supplemental oxygen, or other significant organ dysfunction that would adversely affect study participation.
  6. Active, uncontrolled infection, including any of the following:

    1. Active, uncontrolled systemic fungal, bacterial, or viral infection within 14 days prior to the start of conditioning
    2. Any other documented active, uncontrolled infection at the start of conditioning
  7. Chronic viral infections with evidence of active disease, including:

    HIV: detectable viral load within 6 months prior to screening

    Hepatitis B:

    • HBsAg-positive and/or anti-HBc-positive with detectable HBV DNA
    • Anti-HBc-positive alone Hepatitis C: positive HCV antibody with detectable HCV RNA
  8. Planned HCT using cord blood, ex vivo T cell depletion, engineered grafts, or experimental graft sources
  9. Malabsorption syndrome or GI condition that precludes oral administration, including:

    1. Inability to swallow oral medications
    2. Prior gastric bypass or severe gastroparesis
    3. Cirrhosis with Child-Pugh Class B or C
  10. Pregnant or breastfeeding
  11. Prior intolerance to menin inhibitor therapy resulting in ≥ Grade 3 treatment-related adverse events
  12. Any condition, therapy, laboratory abnormality, or allergy to excipients that, in the investigator's judgment, could confound study results, interfere with the participant's ability to comply with study procedures or complete the study, or make participation not in the participant's best interest.

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Støttende pleje
  • Tildeling: Randomiseret
  • Interventionel model: Parallel tildeling
  • Maskning: Tredobbelt

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: Revumenib BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
oral tablets
Andre navne:
  • REVUFORJ
Placebo komparator: Placebo BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
orale tabletter

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Relapse free survival (RFS) in KMT2Ar, NPM1m, and NUP98r AML in the Intent-to-Treat (ITT) population with a minimum of 1 year of follow-up post-randomization.
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
RFS is defined as the time from randomization to the date of relapse or the date of death from any cause, whichever comes first.
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
RFS in the modified ITT (mITT) population
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
Relapse free survival rate in modified intent to treat population
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
Rate of overall survival (OS) in the ITT population
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Overall survival rate in the intent-to-treat population
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Incidence of relapse in the ITT population
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Incidence of relapses in intent-to-treat participant population
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Rate of event-free survival (EFS) in the ITT population
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
Events such as relapse/progression, death from any cause, graft failure, use of donor lymphocyte infusion which have occurred from time from the date of randomization to the date of event occurrence.
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization
Rate of non-relapse mortality (NRM) in the ITT population
Tidsramme: From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Death in participants in the absence of disease progression or relapse
From randomization to the date of event occurrence, assessed for a minimum of 1 year post-randomization.
Frequency, duration, and severity of Treatment-Emergent Adverse Events (TEAEs), Treatment-Related Adverse Events (TRAEs), Adverse Events of Special Interest (AESIs), and Serious Adverse Events (SAEs) in the Safety Analysis population
Tidsramme: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization
Documentation of number of treatment related of adverse events; their frequencies, duration, and severity
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization
Change from baseline in other observations related to safety for electrocardiograms (ECGs) measuring QT intervals.
Tidsramme: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
Documentation and comparison of abnormal ECGs from baseline measurements in relationship to safety
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
Change from baseline in other observations related to safety for vital signs.
Tidsramme: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
Documentation and comparison of abnormal vital signs from baseline measurements in relationship to safety
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
Change from baseline in other observations related to safety for performance status in the Safety Analysis population
Tidsramme: From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.
Documentation and comparison of abnormal performance from baseline measurements in relationship to safety
From date of randomization to end of treatment, assessed for a minimum of 1 year post-randomization.

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Efterforskere

  • Studiestol: Steven Devine, M.D, NMDP

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Anslået)

1. december 2026

Primær færdiggørelse (Anslået)

1. juni 2031

Studieafslutning (Anslået)

1. juni 2031

Datoer for studieregistrering

Først indsendt

15. april 2026

Først indsendt, der opfyldte QC-kriterier

28. april 2026

Først opslået (Faktiske)

1. maj 2026

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

22. juni 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

16. juni 2026

Sidst verificeret

1. juni 2026

Mere information

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