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

12 de octubre de 2026 actualizado por: 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.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

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 estudio

Intervencionista

Inscripción (Estimado)

20

Fase

  • No aplica

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

Ubicaciones de estudio

    • District of Columbia
      • Washington D.C., District of Columbia, Estados Unidos, 20010
        • Reclutamiento
        • Children's National Hospital

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

No mayor que 45 años (Niño, Adulto)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

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.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Otro
  • Asignación: N / A
  • Modelo Intervencionista: Asignación Secuencial
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
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.

Otros nombres:
  • Individualized treatment plan
  • Specialized treatment plan

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
Periodo de tiempo: 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 resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Proportion of participants with Adverse events
Periodo de tiempo: 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
Periodo de tiempo: 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

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Colaboradores

Investigadores

  • Investigador principal: Adriana Fonseca, MD, Children's National Research Institute

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de octubre de 2026

Finalización primaria (Estimado)

1 de enero de 2032

Finalización del estudio (Estimado)

1 de octubre de 2032

Fechas de registro del estudio

Enviado por primera vez

31 de agosto de 2026

Primero enviado que cumplió con los criterios de control de calidad

25 de septiembre de 2026

Publicado por primera vez (Actual)

1 de octubre de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

13 de octubre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

12 de octubre de 2026

Última verificación

1 de octubre de 2026

Más información

Términos relacionados con este estudio

Otros números de identificación del estudio

  • STUDY00001868

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .