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- Ensaio Clínico NCT07851636
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.
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Descrição detalhada
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.
Tipo de estudo
Inscrição (Estimado)
Estágio
- Não aplicável
Contactos e Locais
Contato de estudo
- Nome: Hope Lovell
- Número de telefone: 202-476-3898
- E-mail: hlovell@childrensnational.org
Estude backup de contato
- Nome: Rachel Reed, MS, CCRP
- Número de telefone: 202-476-5111
- E-mail: rreed@childrensnational.org
Locais de estudo
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District of Columbia
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Washington D.C., District of Columbia, Estados Unidos, 20010
- Recrutamento
- Children's National Hospital
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
Inclusion Criteria:
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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)
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Participant must be a candidate for surgical resection or biopsy.
• Participants must have surgically accessible disease.
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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
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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
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Karnofsky 50 for participants > 16 years of age and Lansky 50 for participants <16 years of age
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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.
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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.
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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.
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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.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Outro
- Alocação: N / D
- Modelo Intervencional: Atribuição sequencial
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: 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.
Outros nomes:
|
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
Prazo: 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
|
Medidas de resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Proportion of participants with Adverse events
Prazo: 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
|
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The preliminary clinical activity of tumor board-recommended treatment
Prazo: 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
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Adriana Fonseca, MD, Children's National Research Institute
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Estimado)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Doenças Cerebrais
- Doenças do Sistema Nervoso Central
- Doenças do Sistema Nervoso
- Neoplasias por local
- Neoplasias
- Neoplasias por Tipo Histológico
- Neoplasias Glandulares e Epiteliais
- Tumores Neuroectodérmicos
- Neoplasias, Células Germinativas e Embrionárias
- Neoplasias, Tecido Nervoso
- Neoplasias do Sistema Nervoso
- Neoplasias do Sistema Nervoso Central
- Neoplasias Cerebrais
- Neoplasias Neuroepiteliais
Outros números de identificação do estudo
- STUDY00001868
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Informações sobre medicamentos e dispositivos, documentos de estudo
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