Recurrent CNS TumorsNCT07374692
Observational Study of Responses to Treatments in Advanced Central Nervous System (CNS) Tumors
An observational study of Recurrent CNS Tumors and Brain Cancer, run by National Cancer Institute (NCI) at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 15 to 120 years
- Conditions the study is for
- Recurrent CNS Tumors, Brain Cancer, Gliomas
- What is being tested?
- SmartMatch testing
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Feb 2026to 10 Sep 2026
- Phase
- Observational
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 15 to 120 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study aims to collect blood and tumor samples for molecular profiling from individuals with advanced CNS tumors.
Who may be able to join
- 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.
Who cannot join
- None.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
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.
Read more
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....
Read the detailed description
Background:
- Primary central nervous system (CNS) tumors are uncommon and are mostly classified as rare diseases. Despite their low incidence, CNS cancers are associated with significant morbidity and mortality across all age groups.
- Treatment options for CNS tumors are limited, especially upon disease progression, largely due to the incomplete understanding of disease biology and the challenges in conducting clinical studies, given the rarity of these diseases.
- The traditional pathway for oncology drug development, involving several phases of clinical trials, can take many years to a decade and comes with a tremendously high financial cost. To overcome this challenge and speed up improving oncology patient care, drug repurposing has emerged as a useful approach.
- Real-time drug screening for rare brain tumors is advancing precision medicine by generating clinical trial concepts aimed at overcoming key challenges in brain tumor management. However, delayed turnaround times remain a significant limitation to their clinical utility.
- The Gujral lab at the Fred Hutchinson Cancer Center has developed SmartMatch, a cutting-edge artificial intelligence (AI)-driven system pharmacology-based platform to address the challenges in precision medicine. The platform allows the generation of drug response data using real-time tissue-based screening using freshly resected tumor or biopsy from a patient. The data collected is used to train a machine learning model, allowing precise prediction to a large panel of Food and Drug Administration (FDA)-approved drugs. This is a test that can be ordered by any physician in the US, and results are available upon request.
Objective:
-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
Eligibility:
- Participants >=15 years with advanced CNS tumors.
- Participants must have been scheduled for a brain tumor biopsy or resection planned to take place at NIH.
Design:
- Tumor and blood samples will be collected for multi-omic analysis.
- Fresh tumor samples will be used for drug screen analysis by SmartMatch.
- After initial sample collection, participants will be followed remotely every 6 (+/-3) months for 3 years.
- Conditions
- Recurrent CNS Tumors, Brain Cancer, Gliomas, IDH-wildtype Gliomas, IDH-mutant Gliomas, Rare CNS Tumor
- Being tested
- SmartMatch testing
- Keywords
- CNS Tumor, Brain Tumor, Rare Tumor, SmartMatch, Gliomas, Drug Screen
- Medical subject headings
- Central Nervous System Neoplasms, Brain Neoplasms, Glioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SmartMatch testingOther
Fresh tumor samples will be collected for the study only if available following a planned biopsy or resection performed at NIH.
| Study group | Role | What they receive |
|---|---|---|
| 1/ IDH-wild-type | Participants with recurrent IDH-wild type high-grade glioma Receives: Other: SmartMatch testing | |
| 2/ IDH-mutant | Participants with recurrent IDH-mutant gliomas Receives: Other: SmartMatch testing | |
| 3/ Other | Other recurrent CNS tumors Receives: Other: SmartMatch testing |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 21 days from the time of tumor tissue acquisition
Secondary outcomes (1)
To perform molecular profiling of advanced CNS tumors at disease progression.
Descriptive statistics will be used to summarize the molecular profiling results
Measured: End of Study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jan 2026
- Study start4 Feb 2026
- Primary completion10 Sep 2026
- Study completion10 Sep 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Recurrent CNS TumorsStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Jing Wu, M.D., Principal investigator, National Cancer Institute (NCI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07374692
- Sponsor's protocol ID
- 10002539
- Other IDs
- 002539-C
- Status last verified
- 25 Sep 2026
- First submitted
- 28 Jan 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This study will comply with the NIH Data Management and Sharing (DMS) Policy, which applies to all new and ongoing NIH-funded research in the IRP, as of January 25, 2023, that is associated with a ZIA, with a clinical protocol that undergoes scientific review and/or will involve genomic data sharing.
Publications
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.
PubMed 36196752Aldape 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.
PubMed 30733593Nishida-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.
PubMed 32420994