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.
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
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.
Sequential
Personalized Treatment Recommendation
Other interventionSpecialized 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.
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
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.
Proportion of participants with Adverse events
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.
The preliminary clinical activity of tumor board-recommended treatment
Best overall radiographic response (CR , PR, SD, PD) as per RAPNO criteria in patients receiving tumor board-recommended treatment.
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Adriana Fonseca
Lead sponsor
Children's National Research Institute
Sponsor institution
Children's National Research Institute
Collaborator
