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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.

調査の概要

詳細な説明

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.

研究の種類

介入

入学 (推定)

146

段階

  • フェーズ2

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究連絡先

  • 名前:Paul Guo
  • 電話番号:4583 7634064583
  • メール:pguo@nmdp.org

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

  • 大人
  • 高齢者

健康ボランティアの受け入れ

いいえ

説明

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.

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか?

デザインの詳細

  • 主な目的:支持療法
  • 割り当て:ランダム化
  • 介入モデル:並列代入
  • マスキング:トリプル

武器と介入

参加者グループ / アーム
介入・治療
実験的:Revumenib BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
oral tablets
他の名前:
  • Revuforj
プラセボコンパレーター:Placebo BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
経口錠

この研究は何を測定していますか?

主要な結果の測定

結果測定
メジャーの説明
時間枠
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.
時間枠: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.

二次結果の測定

結果測定
メジャーの説明
時間枠
RFS in the modified ITT (mITT) population
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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
時間枠: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.
時間枠: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.
時間枠: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
時間枠: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.

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

捜査官

  • スタディチェア:Steven Devine, M.D、NMDP

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (推定)

2026年12月1日

一次修了 (推定)

2031年6月1日

研究の完了 (推定)

2031年6月1日

試験登録日

最初に提出

2026年4月15日

QC基準を満たした最初の提出物

2026年4月28日

最初の投稿 (実際)

2026年5月1日

学習記録の更新

投稿された最後の更新 (実際)

2026年6月22日

QC基準を満たした最後の更新が送信されました

2026年6月16日

最終確認日

2026年6月1日

詳しくは

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個々の参加者データ (IPD) を共有する予定はありますか?

いいえ

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米国FDA規制機器製品の研究

いいえ

米国で製造され、米国から輸出された製品。

いいえ

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