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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 oktober 2026 bijgewerkt door: 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.

Studie Overzicht

Gedetailleerde beschrijving

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.

Studietype

Ingrijpend

Inschrijving (Geschat)

20

Fase

  • Niet toepasbaar

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studiecontact

Studie Contact Back-up

Studie Locaties

    • District of Columbia
      • Washington D.C., District of Columbia, Verenigde Staten, 20010
        • Werving
        • Children's National Hospital

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

Niet ouder dan 45 jaar (Kind, Volwassen)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

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.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Ander
  • Toewijzing: NVT
  • Interventioneel model: Sequentiële toewijzing
  • Masker: Geen (open label)

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: 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.

Andere namen:
  • Individualized treatment plan
  • Specialized treatment plan

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Number of participants for whom a treatment recommendations are completed within 28 calendar days from tissue collection.
Tijdsspanne: 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

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Proportion of participants with Adverse events
Tijdsspanne: 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
Tijdsspanne: 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

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Onderzoekers

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

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Geschat)

1 oktober 2026

Primaire voltooiing (Geschat)

1 januari 2032

Studie voltooiing (Geschat)

1 oktober 2032

Studieregistratiedata

Eerst ingediend

31 augustus 2026

Eerst ingediend dat voldeed aan de QC-criteria

25 september 2026

Eerst geplaatst (Werkelijk)

1 oktober 2026

Updates van studierecords

Laatste update geplaatst (Werkelijk)

13 oktober 2026

Laatste update ingediend die voldeed aan QC-criteria

12 oktober 2026

Laatst geverifieerd

1 oktober 2026

Meer informatie

Termen gerelateerd aan deze studie

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Nee

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

product vervaardigd in en geëxporteerd uit de V.S.

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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