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 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.

Clinical Assessment

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 the Natural History and Specimen Banking for Patients with CNS Cancers

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}

View or download the Natural History Study flyer >

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.

Trial Expands Across the Nation to Advance Research

NCI-CONNECT launched an immunotherapy drug trial across its national network of collaborative institutions to reach more people with rare brain and spine tumors.

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

This 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 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 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: IDH-Mutated Gliomas}

View or download the nivolumab IDH study flyer >

Older woman holding and reading pill bottle with a glass of water and younger woman looking at her phone.

First-in-Human Trial for People with Recurrent Rare CNS Tumors

NCI-CONNECT launched a phase 1 clinical trial for people with recurrent rare brain and spine tumors as a first step toward developing a novel drug.

ONC206 for Patients with Rare CNS Neoplasms

This 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 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 or download the immune monitoring study flyer >

Observational Studies

Patients can participate in the observational studies listed below if they are enrolled in the Evaluation of the Natural History and Specimen Banking for Patients with CNS Cancers study. Patients can participate in these studies from home. They do not require a visit to the NCI-CONNECT Clinic. These are studies that measure if an intervention helps improve outcomes.

Girl sitting on couch using a virtual reality headset

Clinical Trial to Reduce Stress and Improve Mood

Researchers have developed a clinical trial using virtual reality to improve the feelings of distress and anxiety that people with brain tumors experience.

Virtual Reality Study for Patients with Brain Cancer

This study is determining if using a virtual reality headset to deliver relaxation techniques helps to reduce the distress and anxiety that 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 is measuring daytime sleepiness and activity patterns using a smart wearable device called a Fitbit. 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 uses an individualized therapy intervention called CALM (Managing Cancer and Living Meaningfully) to address negative feelings and depressive symptoms, with the aim to improve a person’s mood and the way they feel.

_{Study Tumor Types: Primary CNS Tumors}

View or download the CALM Therapy Intervention study flyer >

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.

Online Outcomes and Risk Project for Patients with Rare CNS Cancer

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)}

View or download the Outcomes and Risk Study flyer >

Dr. Armstrong conducts a cognitive assessment with a patient

Natural History Study Enrolls One Thousand Participants

The Natural History Study is improving treatment and quality of life for patients with rare central nervous system cancers.

Learn More

Clinical Trial Resources

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