Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors

NCT07374692 | Recruiting

• 2 other identifiers: 10002539002539-C
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

Background: Primary central nervous system (CNS) tumors grow in the brain and spinal cord. These tumors are rare, but they are difficult to treat and often fatal. SmartMatch is a new technology that tries to help find the best medicines for the particular tumor by testing how small pieces of surgically removed tumor tissue react to different drugs. The study team wants to see if SmartMatch can complete the analysis and generate a report within 21 days from the time of the surgery. Objective: To better understand CNS tumors so doctors can find better ways to treat them in the future. Eligibility: People aged 15 years and older with recurrent CNS tumors. Patient must already be scheduled for a surgery for the tumor at the NIH. Design: The study team will collect a small amount of tumor samples during the planned surgery. They may also use tumor samples from previous procedures. No new or additional procedures will be done for the purpose of this study. The tumor samples will be sent to a lab for SmartMatch analysis. Once completed, the results will be shared with the patient and his/her local doctor. Together they can decide whether to incorporate the results into the treatment plan. It is important to know that the results may or may not be helpful. There will only be one blood test for research. Blood and tissue samples collected may be used for additional analysis. Tumor tissue may be used to grow additional samples for further study. Participants will receive a pathology diagnosis and mutation profile generated by pathologists who specialize in CNS tumors. The study team will seek updates on participant's health approximately every 6 months for 3 years. Tumor samples may also be collected from any additional surgery done at NIH during this time.

Conditions

Recurrent CNS Tumors; Brain Cancer; Gliomas; IDH-wildtype Gliomas; IDH-mutant Gliomas; Rare CNS Tumor

Keywords

CNS Tumor; Brain Tumor; Rare Tumor; SmartMatch; Gliomas; Drug Screen

Outcome Measures

Primary Outcomes (1)

• To determine the proportion of participants with advanced CNS tumors for whom SmartMatch drug screen analysis results are generated within 21 days from the time of tumor tissue acquisition

  Point estimates and 95% exact confidence intervals will be calculated using the Clopper-Pearson method

  21 days from the time of tumor tissue acquisition

Secondary Outcomes (1)

• To perform molecular profiling of advanced CNS tumors at disease progression.

  End of Study

Study Arms (1)

1/SmartMatch testing

OTHER

Turnaround time for SmartMatch platform implementation and molecular profiling of recurrent CNS tumors

Other: Tumor sample collection

Interventions

Tumor sample collection OTHER

Fresh tumor samples will be collected for the study only if available following a planned biopsy or resection performed at NIH.

1/SmartMatch testing

Eligibility Criteria

• Age: 15 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must have advanced CNS tumors confirmed by a documented pathology report, including:
• recurrent isocitrate dehydrogenase (IDH)-wild-type high-grade glioma
• recurrent IDH-mutant gliomas
• other recurrent CNS tumors
• Participants must have been scheduled for a brain tumor biopsy or resection. Note: Scheduled brain tumor biopsy or resection must be at least 6 months after any previous radiation therapy, if applicable. All procedures are planned to take place at NIH.
• Age \>= 15 years.
• Ability of participant, parent/guardian, or Legally Authorized Representative (LAR) to understand and sign a written informed consent document.

You may not qualify if:

• None.

Sponsors & Collaborators

• National Cancer Institute (NCI): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

RECRUITING

Related Publications (3)

• Ostrom QT, Price M, Neff C, Cioffi G, Waite KA, Kruchko C, Barnholtz-Sloan JS. CBTRUS Statistical Report: Primary Brain and Other Central Nervous System Tumors Diagnosed in the United States in 2015-2019. Neuro Oncol. 2022 Oct 5;24(Suppl 5):v1-v95. doi: 10.1093/neuonc/noac202.

  PMID: 36196752 BACKGROUND

• Aldape K, Brindle KM, Chesler L, Chopra R, Gajjar A, Gilbert MR, Gottardo N, Gutmann DH, Hargrave D, Holland EC, Jones DTW, Joyce JA, Kearns P, Kieran MW, Mellinghoff IK, Merchant M, Pfister SM, Pollard SM, Ramaswamy V, Rich JN, Robinson GW, Rowitch DH, Sampson JH, Taylor MD, Workman P, Gilbertson RJ. Challenges to curing primary brain tumours. Nat Rev Clin Oncol. 2019 Aug;16(8):509-520. doi: 10.1038/s41571-019-0177-5.

  PMID: 30733593 BACKGROUND

• Nishida-Aoki N, Bondesson AJ, Gujral TS. Measuring Real-time Drug Response in Organotypic Tumor Tissue Slices. J Vis Exp. 2020 May 2;(159). doi: 10.3791/61036.

  PMID: 32420994 BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Central Nervous System Neoplasms; Brain Neoplasms; Glioma

Condition Hierarchy (Ancestors)

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

Study Officials

• Jing Wu, M.D.

  National Cancer Institute (NCI)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Christine T McGowan

CONTACT

(240) 858-7330; christine.mcgowan@nih.gov

Jing Wu, M.D.

CONTACT

(240) 760-6036; jing.wu3@nih.gov

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2026
• First Posted: January 29, 2026
• Study Start: February 4, 2026
• Primary Completion (Estimated): December 30, 2030
• Study Completion (Estimated): December 30, 2030
• Last Updated: April 1, 2026

Record last verified: 2026-03-25

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data will be made available as soon as possible or at the time of associated publication. Data not published in a manuscript will be shared via public source once the data set completes QC.
• Access Criteria: Clinical data will be made available upon request and with the permission of the study PI. Genomic data are made available via dbGAP through requests to the data custodians.

Locations

US (1)