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SNDX-5613 的扩展访问程序

2025年8月28日更新者：Syndax Pharmaceuticals

SNDX-5613 在患有与 HOXA 过度表达相关的基因改变的复发/难治性急性白血病患者中的扩大使用计划

This expanded access program will provide an investigational treatment option in a controlled clinical setting for participants who are not otherwise eligible to participate in other Syndax-sponsored clinical studies and have no approved treatment options.

研究概览

地位

不再可用

条件

复发/难治性急性白血病

干预/治疗

药品：Revumenib

研究类型

扩展访问

扩展访问类型

治疗 IND/方案

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。

学习地点

美国
- Alabama
  - Birmingham、Alabama、美国、35233
    - Alabama Center for Childhood Cancer And Blood Disorders, Children's of Alabama
- Arizona
  - Phoenix、Arizona、美国、85016
    - Phoenix Children's Hospital
- California
  - Duarte、California、美国、91010
    - City of Hope
  - Irvine、California、美国、92618
    - City of Hope at Orange County Lennar Foundation Cancer Center
  - Los Angeles、California、美国、90095
    - UCLA, UCLA RRMC, Drug Information Center, Department of Pharmaceutical Services Drug Supply Shipment
  - Palo Alto、California、美国、94304
    - Lucile Packard Children's Hospital-Stanford
  - San Diego、California、美国、92123
    - Rady Children's Hospital
  - San Francisco、California、美国、94158
    - University of California, San Francisco
- Colorado
  - Aurora、Colorado、美国、80045
    - Center for Cancer and Blood Disorders, Colorado Children's Hospital
  - Aurora、Colorado、美国、80045
    - HCTU, Division of Hematology, University of Colorado, Anschutz Medical Center
- Florida
  - Orlando、Florida、美国、32804
    - Advent Health Orlando
- Georgia
  - Atlanta、Georgia、美国、30322
    - Children's Healthcare of Atlanta
  - Atlanta、Georgia、美国、30322
    - Winship Cancer Institute at Emory University
- Illinois
  - Chicago、Illinois、美国、60611
    - Lurie Children's Hospital of Chicago
  - Chicago、Illinois、美国、60637
    - University of Chicago Medical Center
- Iowa
  - Iowa City、Iowa、美国、52242
    - University of Iowa Hospitals and Clinics
- Louisiana
  - New Orleans、Louisiana、美国、70118
    - Children's Hospital
- Massachusetts
  - Boston、Massachusetts、美国、02215
    - Dana-Farber Cancer Institute, Boston Children's Cancer and Blood Disorders Center
- Missouri
  - Kansas City、Missouri、美国、64108
    - Children's Mercy Hospital-Kansas City
  - St Louis、Missouri、美国、63110
    - Siteman Cancer Center - Washington University
- New Jersey
  - Hackensack、New Jersey、美国、07601
    - Hackensack University Medical Center
- New York
  - Long Island City、New York、美国、10065
    - Memorial Sloan Kettering Cancer Center
  - The Bronx、New York、美国、10467
    - Montefiore Medical Center
- North Carolina
  - Durham、North Carolina、美国、27705
    - Duke University Medical Center
- Ohio
  - Cincinnati、Ohio、美国、45229
    - Cincinnati Children's Hospital Medical Center
  - Cincinnati、Ohio、美国、45267
    - Division of Hematology and Oncology, Division of Pulmonary, Critical Care and Sleep Medicine, Vontz Center for Molecular Studies
  - Columbus、Ohio、美国、43210
    - OSU Medical Center
- Oregon
  - Portland、Oregon、美国、97239
    - Doernbecher Children's Hospital, Oregon Health & Science University
- Pennsylvania
  - Philadelphia、Pennsylvania、美国、19104
    - Children's Hospital of Philadelphia
- Tennessee
  - Memphis、Tennessee、美国、38105
    - St. Jude Children's Research Hospital
- Texas
  - Houston、Texas、美国、77030
    - MD Anderson Cancer Center
  - Houston、Texas、美国、77030
    - Texas Children's Hospital
  - Houston、Texas、美国、77030
    - Memorial Hermann-Texas Medical Center
- Utah
  - Salt Lake City、Utah、美国、84112
    - Division of Hematology and Hematologic Malignancies, University of Utah-Huntsman Cancer Hospital
- Washington
  - Seattle、Washington、美国、98105
    - Seattle Children's Research Institute, Seattle Childrens Hospital

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

6个月及以上 (孩子、成人、年长者)

接受健康志愿者

不适用

有资格学习的性别

全部

描述

Key Inclusion Criteria:

Male or female participant aged ≥6 months.
Not eligible for participation in an ongoing clinical study and have no approved treatment options.
Participant or participant's health care proxy is able and willing to provide written informed consent and able to follow study instructions.
Relapsed or refractory (R/R) acute leukemia, as defined by standardized criteria, after standard of care therapy, including but not limited to 1 or 2 cycles of intensive chemotherapy, or venetoclax combinations:
1. R/R leukemia is defined by the presence of ≥5% blasts in the bone marrow and/or persistence or reappearance of peripheral blasts.
2. Participants with persistent leukemia after initial therapy or recurrence of leukemia at any time after achieving a response during or after the course of treatment (including allogeneic hematopoietic stem cell transplant [HSCT]) are eligible.
3. Participants with isolated extra-medullary disease are allowed if extramedullary disease was confirmed with biopsy.
4. Participants previously treated on a revumenib clinical study who are entering the expanded access program for post-transplant maintenance because they are not eligible to receive revumenib on study or because the study is closed are not required to meet the R/R status.
5. Participants who underwent HSCT and are eligible to resume treatment with revumenib will be dosed with the last revumenib tolerated dose before transplant.
Acute leukemia harboring a lysine (K) methyltransferase 2A gene rearrangement (KMT2Ar), nucleoporin 98 rearrangement, nucleophosmin 1 mutation (or mutated) or any other genetic alteration with overexpression of HOXA genes predicted to potentially respond to menin inhibitors.
- Note: As revumenib is now approved in the United States, only participants with a KMT2Ar who are not included in the United States prescribing information indication or cannot be accurately dosed (within a 20% margin) with commercial supply and require use of oral solution will be allowed into the study.
Adequate liver, renal, and cardiac function.
Use of highly effective methods of contraception are required for females and males of childbearing potential from the time of enrollment through 120 days following the last study intervention dose.

For participants currently being treated with revumenib in a Syndax-sponsored clinical study or Syndax investigator-sponsored trial, the following criteria must be met:

In the opinion of the Investigator, participant demonstrated acceptable benefit from and tolerability of the study intervention.
Participant is considered compliant with study intervention and procedures.
Participant does not meet any criteria for study intervention discontinuation.
Investigator and participant agree to continue study intervention treatment.
Participant continues to experience clinical benefit.

Key Exclusion Criteria:

Evidence of uncontrolled infection.
Pregnant or nursing women.
Cardiac or gastrointestinal disease.
Graft-Versus-Host Disease (GVHD): Signs or symptoms of acute or chronic GVHD >Grade 1 within 4 weeks of enrollment. All transplant participants must have been off all systemic immunosuppressive therapy and calcineurin inhibitors for at least 1 week before enrollment, with the exception of steroids.
History of or any concurrent condition, therapy, laboratory abnormality, or allergy to excipients that, in the Investigator's opinion, either may interfere with the participant's participation or results in the conclusion that it is not in the best interest of the participant to participate.
Participants receiving other antileukemic therapy within 14 days of start of study drug and who have not recovered from previous adverse reactions.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

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？

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。

赞助

Syndax Pharmaceuticals

调查人员

研究主任：Medical Director、Syndax Pharmaceuticals

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。

研究注册日期

首次提交

2023年6月15日

首先提交符合 QC 标准的

2023年6月15日

首次发布 (实际的)

2023年6月26日

研究记录更新

最后更新发布 (实际的)

2025年9月5日

上次提交的符合 QC 标准的更新

2025年8月28日

最后验证

2025年8月1日