Clinical Studies

Clinical studies are research studies that test or observe how well medical approaches work in people. Some clinical studies, also called clinical trials, test new treatments, such as new drugs or other therapies. These studies help researchers learn if a new treatment is effective or less harmful than standard treatments.

Other clinical studies, called observational studies, do not involve testing new treatments. They are designed to gather information about a disease, understand it better, and improve patient outcomes. They may include medical exams, tests, and questionnaires. 

Patients can consider participating in a clinical trial or observational study through the NCI-CONNECT Clinic at any point, from the time of diagnosis through recurrence. The NCI-CONNECT Clinic is located at the NIH Clinical Center in Bethesda, Maryland and led by the NCI Center for Cancer Research’s Neuro-Oncology Branch (NOB). Patients at the NIH Clinical Center consent to participate in research studies (protocols) and are treated without charge. NIH does not bill for any services. Patients can also receive support for travel expenses, food, lodging and outpatient treatment. 

If you are interested in participating in any of our clinical studies for adults with rare central nervous system (CNS) cancers, have your doctor contact us to discuss the best options for your care. Please understand that we are not accepting self-referrals or international patients at this time, but your care remains our top priority.

Treatment Trials

Patients can participate in the treatment trials listed below by visiting the NCI-CONNECT Clinic at NIH in Bethesda, Maryland, for an evaluation and to determine if they are eligible. All treatments are administered at NIH or participating partner sites.

Phase I/II Trial of PLX038 in Primary Central Nervous System Tumors containing MYC or MYCN Amplifications

This clinical trial seeks to find the optimal dose of the investigational drug PLX038 in adults with primary CNS tumors and provide information on how this study drug affects their CNS tumors containing changes (amplifications) in the MYC or MYCN gene. 

_{Study Tumor Types: Recurrent or Progressive Primary CNS Tumors, Primary CNS Tumors with MYC or MYCN amplifications} 

View the PLX038 in CNS Tumors Study Flyer 

Study of Zotiraciclib for Recurrent High-Grade Gliomas With Isocitrate Dehydrogenase 1 or 2 (IDH1 or IDH2) Mutations

This phase 1/2 clinical trial is studying whether the drug zotiraciclib is effective in people with recurrent diffuse gliomas that have IDH1 or IDH2 mutations.

^{Study Tumor Types: Recurrent Diffuse Glioma with IDH1 or IDH2 Mutation} 

Immune Checkpoint Inhibitor Nivolumab for Patients with Rare CNS Cancers

This phase 2 clinical trial is investigating if the immunotherapy drug nivolumab is an effective treatment for people with rare CNS tumors. 

^{Study Tumor Types (Recurrent): Atypical Teratoid Rhabdoid Tumor (ATRT); Choroid Plexus Tumors; Ependymoma; Gliomatosis Cerebri; Gliosarcoma or Primary CNS Sarcoma; Diffuse Midline Glioma; Medulloblastoma, Meningioma (high grade); Parenchymal Pineal Region Tumors; Pleomorphic Xanthoastrocytoma (PXA) and other BRAF-Altered Tumors; tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNET), including Embryonal Tumor with Multilayered Rosettes, Medulloepithelioma, CNS Neuroblastoma, CNS Ganglioneuroblastoma, and CNS Embryonal Tumor NOS; and tumor entities emerging from methylation profiling of CNS-PNETs, including CNS Neuroblastoma with FOXR2 Activation, CNS Ewing Sarcoma Family Tumor with CIC Alteration, CNS High-Grade Neuroepithelial Tumor with MN1 Alterations, and CNS High-Grade Neuroepithelial Tumor with BCOR Alteration}

View the Immune Checkpoint Inhibitor Nivolumab for Patients with Rare CNS Cancers flyer >

Nivolumab for Patients with IDH-Mutant Gliomas

This phase 2 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: IDH-Mutated Gliomas}

View the Nivolumab for Patients with IDH-Mutant Gliomas flyer > 

ONC206 for Patients with Rare CNS Neoplasms

This phase 1 clinical trial is investigating if the drug ONC206 is an effective treatment for people with recurrent primary CNS neoplasms.

_{Study Tumor Types (Recurrent): Atypical Teratoid Rhabdoid Tumor (ATRT); Choroid Plexus Tumors; Ependymoma; Gliomatosis Cerebri; Gliosarcoma; Diffuse Midline Glioma; Medulloblastoma; Meningioma (high grade); Oligodendroglioma and other IDH-Mutated Tumors; Pineal Region Tumors; Pleomorphic Xanthoastrocytoma (PXA) and Other BRAF-Altered Tumors; and tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNETs)}

Immune Monitoring for Patients with Gliosarcoma and Glioblasoma

This phase 2 clinical trial for patients with newly diagnosed tumors is looking at changes in immune cells in the blood of those who take the immunotherapies ipilimumab and nivolumab, along with temozolomide, to see if this combination is an effective treatment for people with gliosarcoma and glioblastoma. Immune response is monitored in the blood during the course of treatment.

_{Study Tumor Types: Newly Diagnosed Gliosarcoma}

View the Immune Monitoring Study for Patients with Glioblastoma or Gliosarcoma flyer >

Clinical Assessment

Temporarily Closed to Enrollment to New Patients. 

Patients can participate in this study 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 Natural History and Specimen Banking

This study aims to better understand brain and spinal cord tumors, as well as 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: Primary CNS Tumors}

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. 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 Glioma; Ependymoma; Gliomatosis Cerebri; Gliosarcoma; Medulloblastoma; High-Grade Meningioma; Oligodendroglioma and other IDH-Mutated Tumors; Pineal Region Tumors; Pleomorphic Xanthoastrocytoma (PXA) and other BRAF-Altered Tumors; and tumors formerly known as Primitive Neuro-Ectodermal Tumors (PNETs)}​

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

To connect with other patients and caregivers, join our closed Facebook group, NCI-CONNECT Community.