Rare CNS TumorSUPERNOVA-RBTNCT07851636
Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors
A study testing Personalized Treatment Recommendation in people with Rare CNS Tumor and FOXR2-activated, run by Adriana Fonseca at 1 site in United States.
Trial summary
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… Read more about this study
- Who is it for?
- Ages Up to 45 years
- Conditions the study is for
- Rare CNS Tumor, FOXR2-activated, BCOR ITD Sarcoma
- What is being tested?
- Personalized Treatment Recommendation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Oct 2026to Oct 2032
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 45 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
About this study
Official title: 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.
Read the detailed description
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.
- Conditions
- Rare CNS Tumor, FOXR2-activated, BCOR ITD Sarcoma, CNS Sarcoma, PLAG-altered Tumors, Mesenchymal Tumor
- Being tested
- Personalized Treatment Recommendation
- Keywords
- Brain Tumor, CNS Sarcoma, BCOR altered tumor, Mesenchymal tumor, MN1 fused tumor, PATZ1 fused tumor, MN1 altered tumor, Astroblastoma, Drug Screening, Personalized Treatment
- Medical subject headings
- Brain Neoplasms, Neoplasms, Neuroepithelial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Personalized Treatment RecommendationOther
Also called Individualized treatment plan, Specialized treatment plan
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.
| Study group | Role | What they receive |
|---|---|---|
| Personalized Treatment Recommendation | Experimental | 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. Receives: Other: Personalized Treatment Recommendation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 28 calendar days
Secondary outcomes (2)
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.
Measured: Up to 1 year
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.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2026
- Study startOct 2026 (estimated)
- Primary completionJan 2032 (estimated)
- Study completionOct 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rare CNS TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Adriana Fonseca
- Sponsor type
- University, hospital or other
- Collaborators
- Children's National Research Institute
- Responsible party
- Sponsor-investigator, Adriana Fonseca, Children's National Research Institute
- Investigators
- Adriana Fonseca, MD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07851636
- Sponsor's protocol ID
- STUDY00001868
- Acronym
- SUPERNOVA-RBT
- Status last verified
- Oct 2026
- First submitted
- 31 Aug 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No