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

Temporarily Closed to Enrollment. 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

Participating in the Rare CNS Tumors Outcomes and Risk study will help pave the path toward improving our understanding of risk factors associated with the occurrence of rare CNS tumors. The Outcomes and Risk Study is designed for adults diagnosed with a rare CNS tumor. If you take part in this study, we will ask you to complete online surveys, submit a saliva sample that will be used for genetic testing to evaluate risk factors, and you may choose to submit medical records related to your rare CNS tumor. We designed the instructions for participation to be as simple and straightforward as possible. Efforts such as this would not be possible without the selfless contributions of patients like you.

^{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)}

View or download the Outcomes and Risk study flyer

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)}

View or download the Natural History study flyer

Observational Studies

Patients can participate if they are enrolled in the Natural History and Specimen Banking Study. Patients can participate in this study from home. It does not require a visit to the NCI-CONNECT Clinic. It is a research study that measures if an intervention helps improve outcomes.

Virtual Reality Study for Patients with Brain Cancer

This study will determine if using a virtual reality headset to deliver relaxation techniques helps to reduce distress and anxiety people with brain cancer experience ahead of their MRI scans and clinical appointments.

_{Study Tumor Types: Primary Brain Tumors}

View or download the Virtual Reality study flyer

Sleep Observation Study for Patients with Brain Cancer

This study will measure daytime sleepiness and activity patterns using a smart wearable device. This will provide insight into the impact of sleep disturbance on those with brain cancers.

_{Study Tumor Types: Primary Brain Tumors}

View or download the Sleep Observation study flyer

CALM Therapy Intervention Study for Patients with Brain Cancer

This study will use individualized therapy to address negative feelings that may improve a person’s mood and the way they feel. This study will determine if using the CALM intervention helps to reduce depressive symptoms in people with brain cancer.

_{Study Tumor Types: Primary Brain Tumors}

View or download the CALM Therapy Intervention study flyer

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, Ependymoma, Gliomatosis Cerebri, Gliosarcoma or Primary CNS Sarcoma, Histone Mutated Glioma, Medulloblastoma, Meningioma (High Grade), Pineal Region Tumors, Pleomorphic Xanthoastrocytoma (PXA) and Anaplastic Pleomorphic Xanthoastrocytoma (APXA), and tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNET).}

View or download the Nivolumab study flyer

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}

View or download the Nivolumab IDH study flyer

ONC206 for Patients with Rare CNS Neoplasms

The clinical trial is investigating if the drug ONC206 is an effective treatment for people with recurrent primary CNS neoplasms.

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

Immune Monitoring for Patients with Gliosarcoma and Glioblasoma

This clinical trial is looking at the changes in immune cells in the blood of those who take ipilimumab and nivolumab, along with temozolomide, to see if this combination is an effective treatment for people with gliosarcoma and glioblastoma.

_{Study Tumor Types: Gliosarcoma}

View or download the Immune Monitoring study flyer

Carboplatin and Bevacizumab for Patients with Ependymoma

The clinical trial is closed to accrual. The clinical trial investigated if the combination of bevacizumab and carboplatin will help control recurrent ependymoma.

^{Study Tumor Types: Ependymoma}

Sunitinib for Patients with Sarcomas of the CNS

The clinical trial is closed to accrual. This clinical trial investigated if the drug sunitinib was an effective treatment for people with either a gliosarcoma or primary CNS sarcoma.

^{Study Tumor Types: Gliosarcoma}

Marizomib for Patients with Ependymoma

The clinical trial is closed to accrual. This clinical trial investigated if the drug marizomib was an effective treatment for people with recurrent ependymoma that have the tumor marker called RELA-fusion.

^{Study Tumor Types: Ependymoma}

Learn More

• Oral Drug Combination Shows Benefit for Adults with Ependymoma
• New Multi-institutional Study Analyzes Tumor Tissue and Patient Data to Improve Care
• Reliable Patient-Reported Outcomes Data Informs Treatment Decisions

Clinical Trial Resources

• What to Know About Clinical Trials
• What Are Clinical Trials?
• Paying for Clinical Trials
• Patient Safety in Clinical Trials
• Deciding to Take Part in a Clinical Trial
• Questions to Ask about Clinical Trials