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

NCT07851636 | Not Yet Recruiting DMC

• 1 other identifier: STUDY00001868
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief 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 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.

Conditions

Rare CNS Tumor; FOXR2-activated; BCOR ITD Sarcoma; CNS Sarcoma; PLAG-altered Tumors; Mesenchymal Tumor

Keywords

Brain Tumor; CNS Sarcoma; BCOR altered tumor; Mesenchymal tumor; MN1 fused tumor; PATZ1 fused tumor; MN1 altered tumor; Astroblastoma; Drug Screening; Personalized Treatment

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 28 calendar days

Secondary Outcomes (2)

• Proportion of participants with Adverse events

  Up to 1 year

• The preliminary clinical activity of tumor board-recommended treatment

  Up to 2 years

Study Arms (1)

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.

Other: Personalized Treatment Recommendation

Interventions

Personalized Treatment Recommendation OTHER

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.

Also known as: Individualized treatment plan, Specialized treatment plan

Personalized Treatment Recommendation

Eligibility Criteria

• Age: Up to 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Adriana Fonseca: lead
• Children's National Research Institute: collaborator

Study Sites (1)

Children's National Hospital

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Brain Neoplasms; Neoplasms, Neuroepithelial

Condition Hierarchy (Ancestors)

Central Nervous System Neoplasms; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Adriana Fonseca, MD

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rachel Reed, MS, CCRP

CONTACT

202-476-5111; rreed@childrensnational.org

Hope Lovell

CONTACT

202-476-3898; hlovell@childrensnational.org

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Director of the Rare Brain Tumor Program; Attending Neuro-Oncologist

Study Record Dates

• First Submitted: August 31, 2026
• First Posted: October 1, 2026
• Study Start: October 1, 2026
• Primary Completion (Estimated): January 1, 2032
• Study Completion (Estimated): October 1, 2032
• Last Updated: October 1, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)