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Fourth Ventricular Administration of Immune Checkpoint Inhibitor (Nivolumab) and Methotrexate or 5-Azacytidine for Recurrent Medulloblastoma, Ependymoma, and Other CNS Malignancies
CA-4948-101: Open-Label, Dose Escalation and Expansion Trial of Emavusertib (CA-4948) in Relapsed or Refractory Primary Central Nervous System Lymphoma (R/R PCNSL)
Pharmacogenomic Contributions to Trihexyphenidyl Biotransformation and Response in Children With Dystonic Cerebral Palsy
Customized Neuro-Imaging Referenced Symptom Video for the Reduction of Patient and Caregiver Anxiety Around Radiation Treatment for Brain Tumors
Testing the Addition of an Anti-cancer Drug, Selinexor, to the Usual Chemotherapy Treatment (Temozolomide) for Brain Tumors That Have Returned After Previous Treatment
What is brain cancer, and what kinds of brain tumors are there?
Brain tumors are classified by the cell they start from and by grade, from 1 (slow growing) to 4 (fastest). Meningiomas grow from the membranes covering the brain, are usually benign, and are often watched or removed surgically; about 40 percent of all primary brain tumors. Gliomas grow from the brain's supporting cells and range from grade 2 astrocytomas and oligodendrogliomas, which often carry an IDH mutation and affect younger adults, to grade 4 glioblastoma. Brain metastases are cancers that started elsewhere, most often in the lung, breast, skin, or kidney, and are far more common than primary brain cancer. Pediatric tumors, including medulloblastoma and diffuse midline glioma, are the most common solid cancers in children and are treated and studied separately. Primary CNS lymphoma, pituitary tumors, and schwannomas round out the list.
Symptoms depend on location rather than type: headaches that are new or worse in the morning, seizures, weakness or numbness on one side, vision or speech problems, personality change, or balance trouble. Diagnosis is by MRI and usually surgery, which both removes tumor and provides tissue for the molecular tests that now guide treatment. Treatment ranges from observation for small meningiomas to surgery, radiation, and chemotherapy, with two notable recent advances outside glioblastoma. In August 2024 the FDA approved vorasidenib, the first targeted pill for grade 2 IDH mutant gliomas, which delayed progression substantially in its trial and may let young adults postpone radiation and chemotherapy for years. And in 2025 the FDA granted accelerated approval to dordaviprone, the first treatment for H3 K27M mutant diffuse midline glioma, a childhood tumor that previously had none. For brain metastases, focused radiation, targeted drugs that cross into the brain, and new sequencing of surgery and radiosurgery are the main research fronts. Our guide explains how clinical trials for cancer work.
Types of brain tumor clinical trials
Brain metastases trials. Radiosurgery given before rather than after surgery, drugs that reach the brain in HER2 positive breast cancer and other cancers, and ways to protect memory during whole brain radiation.
Low grade glioma trials. IDH inhibitors, when to start treatment, and quality of life studies in young adults living with a slow growing tumor for decades.
Meningioma trials. Medicines for tumors that recur after surgery and radiation, where no drug is yet approved, and studies of when to treat versus watch.
Pediatric brain tumor trials. CAR T cells and targeted drugs for relapsed childhood tumors, and studies of how to assess and protect neurological function during treatment.
Surgical and imaging studies. Fluorescence guided surgery, intraoperative tissue monitoring, and advanced MRI to tell tumor from treatment effect.
Supportive care trials. Exercise, cognitive rehabilitation, seizure control, and fatigue management for people living with a brain tumor.
Glioblastoma, the most common malignant brain tumor in adults, has its own glioblastoma clinical trials page.
Who can participate in brain tumor clinical trials?
Trials are organized by tumor type and molecular features, so the pathology report is the key document. Low grade glioma trials require IDH status; metastasis trials specify the primary cancer, the number and size of brain lesions, and whether they have been treated; meningioma trials usually require recurrence after surgery or radiation; pediatric trials have age bands from 1 to 25 and require a specific diagnosis. Most require that you can manage daily activities and are on stable or low steroid doses, and many exclude people with uncontrolled seizures. Supportive care and surgical studies often enroll anyone with the diagnosis. Eligibility always varies by study; our guide covers eligibility criteria.
Are there brain tumor clinical trials for children?
Yes. Pediatric trials test CAR T cells and targeted drugs for relapsed tumors, and in 2025 the first treatment for H3 K27M diffuse midline glioma received accelerated approval. Children's hospitals share trials nationally, and many accept patients up to age 25.
How brain tumors are graded, and why it matters for trials
Brain tumors are graded rather than staged, because they rarely spread outside the nervous system. Grade 1 and 2 tumors grow slowly; many meningiomas and IDH mutant gliomas fall here, and trials focus on timing of treatment and targeted pills. Grade 3 tumors grow faster and are treated with surgery, radiation, and chemotherapy. Grade 4 includes glioblastoma and some pediatric gliomas, covered on their own pages. Metastases are described by the primary cancer and by the number of lesions. Since 2021, molecular markers such as IDH, 1p/19q, and H3 K27M are part of the diagnosis itself, and study titles name them. Your grade, your molecular results, and, for metastases, your primary cancer tell you which studies fit.
How to enroll in a brain tumor clinical trial
Identify your trial. Use the filters and search your tumor type by name: "meningioma," "astrocytoma," "brain metastases," or the childhood diagnosis. Glioblastoma studies are on their own page.
Select your preferred location. Enter your city or state, then widen the radius; brain tumor programs concentrate at academic centers, and many help with travel.
Explore study details. Click "Learn More" for molecular requirements, prior treatment rules, steroid limits, and whether surgery is part of the study.
Complete the health profile. Click "Get started" to begin the 5-step application. Have three things ready: your pathology report with grade and molecular results, your treatment history, and, for metastases, your primary cancer and its treatments. A family member can complete the application on your behalf.
Submit the application. A clinical trial coordinator reviews it and contacts you. Nothing is decided until informed consent, and participation is voluntary at every step.