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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.

研究概览

详细说明

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.

研究类型

介入性

注册 (估计的)

146

阶段

  • 阶段2

联系人和位置

本节提供了进行研究的人员的详细联系信息,以及有关进行该研究的地点的信息。

学习联系方式

  • 姓名:Paul Guo
  • 电话号码:4583 7634064583
  • 邮箱:pguo@nmdp.org

参与标准

研究人员寻找符合特定描述的人,称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

  • 成人
  • 年长者

接受健康志愿者

不

描述

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.

学习计划

本节提供研究计划的详细信息,包括研究的设计方式和研究的衡量标准。

研究是如何设计的?

设计细节

  • 主要用途:支持治疗
  • 分配:随机化
  • 介入模型:并行分配
  • 屏蔽:三倍

武器和干预

参与者组/臂
干预/治疗
实验性的:Revumenib BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
oral tablets
其他名称:
  • Revuforj
安慰剂比较:Placebo BID
For patients not taking strong CYP3A4 inhibitor and patients taking strong CYP3A4 inhibitor
口服片剂

研究衡量的是什么?

主要结果指标

结果测量
措施说明
大体时间
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.
大体时间: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.

次要结果测量

结果测量
措施说明
大体时间
RFS in the modified ITT (mITT) population
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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
大体时间: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.
大体时间: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.
大体时间: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
大体时间: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.

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查,以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (估计的)

2026年12月1日

初级完成 (估计的)

2031年6月1日

研究完成 (估计的)

2031年6月1日

研究注册日期

首次提交

2026年4月15日

首先提交符合 QC 标准的

2026年4月28日

首次发布 (实际的)

2026年5月1日

研究记录更新

最后更新发布 (实际的)

2026年6月22日

上次提交的符合 QC 标准的更新

2026年6月16日

最后验证

2026年6月1日

更多信息

与本研究相关的术语

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)?

不

药物和器械信息、研究文件

研究美国 FDA 监管的药品

不

研究美国 FDA 监管的设备产品

不

在美国制造并从美国出口的产品

不

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