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Clinical Studies

Clinical studies are research studies that test or observe how well medical approaches work in people. 

Some clinical studies test new treatments such as a new drug or medical therapy. Treatment studies help researchers learn if a new treatment is effective or less harmful than standard treatments.

Other clinical studies do not involve testing new treatments. They are designed to gather information about a disease. They may include medical exams, tests, and questionnaires. The aim is to help researchers better understand the disease to improve patient outcomes and develop new therapies. 

Studies can be considered at any point, from the time of diagnosis through recurrence. Patients should talk to their doctor about their options.

Learn about our research studies for adult patients with rare central nervous system (CNS) cancers below. Contact us to schedule an appointment at NIH. Patients will meet with our neuro-oncology team for a clinical assessment to determine if they are eligible for an NCI-CONNECT clinical study.

Online Study

Patients can participate in this study from home. It does not require a visit to the NCI-CONNECT Clinic. It is not a treatment trial and we do not provide medical advice. It is a research study that collects information to help researchers better understand rare CNS cancers.

Outcomes and Risk Project for Patients with Rare CNS Cancers

The project aims to improve our understanding of outcomes and risk factors related to the occurrence of rare CNS cancers. Research shows that certain risk factors may increase a person’s chances of developing cancer. Factors can include exposure to chemicals or other substances, and personal and family medical history. If you take part in this study, you will complete a web-based survey and submit a saliva sample that will be used for genetic testing.

Study Tumor Types: Atypical Teratoid Rhabdoid Tumor (ATRT), Choroid Plexus Tumors, Diffuse Midline Gliomas, Ependymoma, Gliomatosis Cerebri, Gliosarcoma, Medulloblastoma, High Grade Meningioma, Oligodendroglioma, Pineal Region Tumors, Pleomorphic Xanthoastrocytoma (PXA) and Anaplastic Pleomorphic Xanthoastrocytoma (APXA), and tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNET)

Clinical Assessment

Patients participate by visiting the NCI-CONNECT Clinic at NIH in Bethesda, Maryland, for a baseline evaluation. The visit may include other exams and testing. The number of times participants visit NIH for the study may depend on the treatment plan and the wishes of the participants and their local doctors.

Evaluation of the Natural History and Specimen Banking for Patients with CNS Cancers

The study objective is to better understand brain and spinal cord tumors and uncover areas for further research. It also aims to connect patients with doctors who can help them manage their illness and give them new treatment options. 

Study Tumor Types: Atypical Teratoid Rhabdoid Tumor (ATRT), Choroid Plexus Tumors, Diffuse Midline Gliomas, Ependymoma, Gliomatosis Cerebri, Gliosarcoma, Medulloblastoma, High Grade Meningioma, Oligodendroglioma, Pineal Region Tumors, Pleomorphic Xanthoastrocytoma (PXA) and Anaplastic Pleomorphic Xanthoastrocytoma (APXA), and tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNET)

Treatment Trials

Patients participate by visiting the NCI-CONNECT Clinic at NIH in Bethesda, Maryland, for an evaluation and to determine if they are eligible for a clinical trial. All clinical trial treatments are administered at NIH.

Immune Checkpoint Inhibitor Nivolumab for Patients with Rare CNS Cancers

The clinical trial is investigating if the immunotherapy drug nivolumab is an effective treatment for people with rare CNS tumors. 

Study Tumor Types: Atypical Teratoid Rhabdoid Tumor (ATRT), Choroid Plexus Tumors, Diffuse Midline Gliomas, Ependymoma, Gliomatosis Cerebri, Gliosarcoma, Medulloblastoma, High Grade Meningioma, Pineal Region Tumors

Marizomib for Patients with Ependymoma

This clinical trial is investigating if the drug marizomib is an effective treatment for people with recurrent ependymoma that have the tumor marker called RELA-fusion.

Study Tumor Types: Ependymoma

Nivolumab for Patients with IDH-Mutant Gliomas

This clinical trial is investigating if the immunotherapy drug nivolumab is an effective treatment for people with recurrent IDH-mutant gliomas with and without hypermutator phenotype.

Study Tumor Types: Gliomatosis Cerebri, Oligodendroglioma

Sunitinib for Patients with Sarcomas of the CNS

This clinical trial is investigating if the drug sunitinib is an effective treatment for people with either a gliosarcoma or primary CNS sarcoma.

Study Tumor Types: Gliosarcoma

Carboplatin and Bevacizumab for Patients with Ependymoma

The clinical trial is investigating if the combination of bevacizumab and carboplatin will help control recurrent ependymoma.

Study Tumor Types: Ependymoma

Clinical Trial Resources