Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors (SUPERNOVA-RBT)
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.
調査の概要
状態
状態
条件
条件
介入・治療
介入・治療
詳細な説明
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.
研究の種類
研究の種類
入学 (推定)
入学
段階
段階
- 適用できない
連絡先と場所
研究連絡先
研究連絡先
- 名前:Hope Lovell
- 電話番号:202-476-3898
- メール:hlovell@childrensnational.org
研究連絡先のバックアップ
- 名前:Rachel Reed, MS, CCRP
- 電話番号:202-476-5111
- メール:rreed@childrensnational.org
研究場所
-
-
District of Columbia
-
Washington D.C.、District of Columbia、アメリカ、20010
- 募集
- Children's National Hospital
-
-
参加基準
適格基準
適格基準
就学可能な年齢
健康ボランティアの受け入れ
受講資格のある性別
説明
Inclusion Criteria:
-
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)
-
Participant must be a candidate for surgical resection or biopsy.
• Participants must have surgically accessible disease.
-
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
-
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
-
Karnofsky 50 for participants > 16 years of age and Lansky 50 for participants <16 years of age
-
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.
-
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.
-
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.
-
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:
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection.
- 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.
研究計画
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:他の
- 割り当て:なし
- 介入モデル:順次割り当て
- マスキング:なし(オープンラベル)
アーム数
武器と介入
参加者グループ / アーム参加者グループ / アーム |
介入・治療介入・治療 |
|---|---|
|
実験的: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.
他の名前:
|
この研究は何を測定していますか?
主要な結果の測定
主要な結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
時間枠: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
|
二次結果の測定
二次結果の測定
結果測定 |
メジャーの説明 |
時間枠 |
|---|---|---|
|
Proportion of participants with Adverse events
時間枠: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
時間枠: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
|
協力者と研究者
協力者
協力者
捜査官
捜査官
- 主任研究者:Adriana Fonseca, MD、Children's National Research Institute
研究記録日
主要日程の研究
研究開始 (推定)
研究開始
一次修了 (推定)
一次修了
研究の完了 (推定)
研究の完了
試験登録日
最初に提出
最初に提出
QC基準を満たした最初の提出物
QC基準を満たした最初の提出物
最初の投稿 (実際)
最初の投稿
学習記録の更新
投稿された最後の更新 (実際)
投稿された最後の更新
QC基準を満たした最後の更新が送信されました
QC基準を満たした最後の更新が送信されました
最終確認日
最終確認日
詳しくは
本研究に関する用語
キーワード
追加の関連 MeSH 用語
その他の研究ID番号
その他の研究ID番号
- STUDY00001868
個々の参加者データ (IPD) の計画
個々の参加者データ (IPD) を共有する予定はありますか?
医薬品およびデバイス情報、研究文書
米国FDA規制医薬品の研究
米国FDA規制機器製品の研究
米国で製造され、米国から輸出された製品。
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