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Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors (SUPERNOVA-RBT)

2026年10月12日 更新者:Adriana Fonseca

A Pilot Trial of Screening and Ultra-Rapid Profiling to Enable Real-Time Neuro-Oncology Validation and Actionable Treatment Selection for Patients With Relapsed or Recurrent Rare Brain Tumors

The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in of performing real-time drug screening on tissue taken during surgery in patients with relapsed ultra rare brain tumors (BCOR -altered, MN1-altered, CNS Sarcomas, other rare brain tumors) and of having a specialized tumor board assign a treatment plan based on the results of the drug screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed ultra rare brain tumors to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.

研究概览

详细说明

Following clinically indicated biopsy or resection, fresh tissue will be submitted for real-time high-throughput drug screening and tumor/paired-normal molecular profiling will be initiated. A specialized tumor board will issue a documented management recommendation within 28 days . Recommendations will integrate successful drug screening, all molecular/pathology data available at the time of review, prior therapy, and clinical context. Later molecular results may prompt an addendum. The treating physician retains responsibility for final treatment decisions.

研究类型

介入性

注册 (估计的)

20

阶段

  • 不适用

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

研究联系人备份

学习地点

    • District of Columbia
      • Washington D.C.、District of Columbia、美国、20010
        • 招聘中
        • Children's National 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

不超过 45年 (孩子、成人)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria:

  1. Patients must have a relapse or recurrence of one of the following rare brain tumors:

    • CNS sarcoma (e.g. EWS, CIC, DICER1 altered)
    • BCOR altered tumors (e.g. BCOR ITD; BCOR, BCORL1, EP300, fused)
    • Mesenchymal tumors (e.g. FET::CREB altered tumors)
    • MN1::PATZ1 fused tumors
    • PLAG- altered tumors
    • Astroblastomas/MN-1 altered (e.g. EWSR2BEND2 altered)
    • Unclassifiable tumors
    • Other rare brain tumors: (e.g. recently described, poorly characterized, and/or ambiguous entities)
  2. Participant must be a candidate for surgical resection or biopsy.

    • Participants must have surgically accessible disease.

  3. Prior therapy

    • The participant must have a relapsed or recurrent rare brain tumor following at least one prior therapy for initial diagnosis or previous recurrence prior to study registration:

    • Surgery followed by observation.
    • Multi-modality therapy: surgery, radiation, and/or chemotherapy
  4. Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.

    • Biologic agent: Participant must have recovered from any toxicity potentially related to the agent.
    • Last radiation at least 7 days prior to therapy start
  5. Karnofsky 50 for participants > 16 years of age and Lansky 50 for participants <16 years of age

  6. Corticosteroids: Subjects who are receiving dexamethasone or equivalent must be on a stable or decreasing dose for at least 1 week prior to therapy start.

  7. The effects of the agents used in this study on the developing human fetus are unknown. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation and 4 months after completion of therapy administration.

  8. Adequate neurologic function defined as:

    • Participants with seizure disorders may be enrolled if seizures are well controlled. Participants on non-enzyme inducing anticonvulsants may be excluded pending interaction(s) with study drug.

  9. A legal parent or guardian must be able to understand and agree to sign a written informed consent and assent document, as appropriate.

Exclusion Criteria:

  1. Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.
  2. Women of childbearing potential must not be pregnant or breast-feeding. A negative serum or urine pregnancy test is required prior to start of therapy.

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:其他
  • 分配:不适用
  • 介入模型:顺序分配
  • 屏蔽:无(打开标签)

武器和干预

参与者组/臂
干预/治疗
实验性的:Personalized Treatment Recommendation

Participants will be provided a treatment recommendation by the tumor board within 28 days from tissue acquisition. Based on the results of the real-time, High-throuput drug screeningRNA sequencing (RNAseq), whole genome sequencing (WGS), and clinical details of each subject's tumor.

Specialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by Rare Brain Tumor specialized tumor board. Recommendations will be provided and to primary physician.

其他名称:
  • Individualized treatment plan
  • Specialized treatment plan

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
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
大体时间:Up to 28 calendar days

Time from tissue collection to generation of tumor board recommendations will be used to determine the feasibility of using the results of real-time high-throughput screening, WGS and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent ultra-rare brain tumors. Participants are expected to receive treatment plan within 28 calendar days.

Up to 28 calendar days

次要结果测量

结果测量
措施说明
大体时间
Proportion of participants with Adverse events
大体时间:Up to 1 year

Proportion of participants with grade 3 or higher toxicities as per the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 6.0. Serious adverse events (SAEs), unexpected adverse reactions, and adverse events of special interest.

Adverse events will be summarized by maximum grade and relationship to the drugs recommended by the tumor board.

Up to 1 year
The preliminary clinical activity of tumor board-recommended treatment
大体时间:Up to 2 years

Best overall radiographic response (CR , PR, SD, PD) as per RAPNO criteria in patients receiving tumor board-recommended treatment.

Up to 2 years

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。

调查人员

  • 首席研究员:Adriana Fonseca, MD、Children's National Research Institute

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10月1日

初级完成 (估计的)

2032年1月1日

研究完成 (估计的)

2032年10月1日

研究注册日期

首次提交

2026年8月31日

首先提交符合 QC 标准的

2026年9月25日

首次发布 (实际的)

2026年10月1日

研究记录更新

最后更新发布 (实际的)

2026年10月13日

上次提交的符合 QC 标准的更新

2026年10月12日

最后验证

2026年10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

此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.

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