Neuro-Oncology

No one investigator is going to find solutions for pediatric brain and spinal cord tumors alone. That’s why our team is deeply rooted in global initiatives around the world to provide solutions and advance discovery. If there is a collaborative effort to find new discoveries for pediatric brain and spinal cord tumors, our team is a part of it. 

Young girl that's being treated at Nationwide Children's Hospital smiling for a photo.

Neuro-Oncology Clinical Studies

Nationwide Children’s is a national leader in cancer research and clinical trials. Our research focuses on why brain and spine tumors form and how to better treat them in children and young adults. By partnering with leading research groups including the Pediatric Brain Tumor Consortium (PBTC), Collaborative Network of Neuro-oncology Clinical Trials (CONNECT), Pacific Neuro-oncology Consortium (PNOC) and the Children’s Oncology Group (COG), there are many open clinical trials testing new drugs in difficult-to-treat tumors. Working together, we focus on improving outcomes for children with brain and spinal cord tumors.

Open Neuro-Oncology Clinical Studies

Adamantinomatous Craniopharyngioma (ACP)

Researchers Studying IV Medicine to Treat Pediatric Adamantinomatous Craniopharyngioma (ACP)

Help researchers as they study an IV infusion drug to see if it is effective in treating ACP patients who may or may not have undergone surgery or radiation as it may help shrink this type of brain tumor or slow its growth.

Researchers Studying Oral Drug to Treat Pediatric Adamantinomatous Craniopharyngioma (ACP)

Help researchers learn more about the effects of the oral tablet drug binimetinib in patients with adamantinomatous craniopharyngioma (ACP) who may or may not have undergone surgery or radiation as it may help shrink this type of brain tumor or slow its growth.

Central Nervous System (CNS) Tumors

Researchers Studying Oral Medicine to Treat Medulloblastoma, High-Grade Glioma, DIPG Brain Tumors and Other Brain Tumor Types

Help researchers as they study an oral drug to see if it is an effective treatment for recurrent or refractory primary CNS brain tumors in children.

Phase I/II Study of Lutathera in Pediatric and Young Adult Patients with Recurrent and/or Progressive CNS Tumors which Express Somatostatin Type 2A Receptors and Demonstrate Uptake on DOTATATE PET

In this research study, we want to find out if the study drug Lutathera, an IV infusion drug, is effective in treating different kinds of brain cancer.

Researchers Studying Oral Medicine for Solid Tumors Dependent on KIT or PDGFRA Signaling

The goal of this study is to determine if oral medication Avapritinib is safe and effective in treating pediatric patients with solid tumors that depend on KIT or PDGFRA signaling.

Researchers Studying Oral Medication to See if it Protects the Brain During Radiation Therapy Treatment for Primary Central Nervous System Tumors

The goal of this study is to see if oral medication memantine protects the brain during radiation therapy treatment for primary central nervous system tumors.

Diffuse Intrinsic Pontine Glioma (DIPG)

Researchers Studying Oral Medicine During and After Radiation to Treat Newly Diagnosed DIPG and HGG

The goal of this study is to learn more about the safety of the study drug has when taken during radiation.

Researchers Studying Maximum Tolerated Dose of IV Infusion and Radiation Therapy in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG) (KONQUER)

The goal of this study is to test the safety and determine the maximum tolerated dose of an IV infusion drug in children with newly diagnosed DIPG or DMG.

Researchers Studying Oral Medicine During and After Radiation to Treat Newly Diagnosed DIPG and HGG

Help researchers as they test the safety, side effects, and best dose of an oral medicine given in combination with standard radiation therapy to treat children and young adults with newly diagnosed diffuse intrinsic pontine glioma (DIPG) or high-grade glioma (HGG) with a genetic change called H3 K27M mutation.

Study of PEP-CMV Vaccine for Treating Newly Diagnosed Pediatric Brain Tumors and Recurrent Medulloblastoma

The purpose of this phase 2 study is to see if a cancer vaccine (PEP-CMV) can help patients’ immune system fight against high-grade glioma and medulloblastoma.

Researchers Study if Olutasidenib and Temozolomide (TMZ) Can Prolong the Life of Patients With IDH-mutant HGG

The goal of this study is to determine the efficacy of the study drug olutasidenib to treat newly diagnosed pediatric and young adult patients with a high-grade glioma (HGG) harboring an IDH1 mutation.

Ependymoma

PBTC-059: Phase 1 Trial of HER2-specific CAR T Cells for Children with Hard-to-Treat or Returning Ependymoma

Help researchers evaluate the safety profile of a type of immune therapy called HER2 CAR T cells (short for HER2 chimeric antigen receptor T cells).

Gliomas

Researchers Compare Oral Drug Treatment With IV Drug Treatment in Patients With Neurofibromatosis and Low-Grade Glioma

Help researchers study if the oral drug selumetinib works just as well as the standard treatment with IV carboplatin/vincristine (CV) for patients with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in improving vision in subjects with LGG of the optic pathway (vision nerves).

Researchers Studying Oral Medication Combination After Radiation Therapy to Treat Patients With Newly Diagnosed High-Grade Glioma

Help researchers as they study how well the combination of dabrafenib and trametinib works after radiation therapy in children and young adults with high-grade glioma who have a genetic change called BRAF V600 mutation.

Researchers Studying Two Oral Drugs Given After Radiotherapy to Patients Just Diagnosed With High-Grade Glioma (HGG), including DIPG

The goal of this study is to determine the efficacy of the study drugs ribociclib and everolimus to treat pediatric and young adult patients newly diagnosed with a high-grade glioma (HGG), including DIPG, that have genetic changes in pathways (cell cycle, PI3K/mTOR) that these drugs target.

Researchers Studying Oral Medicine to Treat Newly Diagnosed High-Grade Glioma With NTRK Fusion

Help researchers learn more about the effects of a study drug on children with brain tumors.

Targeted Pediatric High-Grade Glioma Therapy

The study aims to analyze the genes in brain tumors from kids, teens, and young adults who have just been diagnosed with a severe type of brain cancer.

Researchers Study if Olutasidenib and Temozolomide (TMZ) Can Prolong the Life of Patients With IDH-mutant HGG

The goal of this study is to determine the efficacy of the study drug olutasidenib to treat newly diagnosed pediatric and young adult patients with a high-grade glioma (HGG) harboring an IDH1 mutation.

Medulloblastoma

Researchers Studying Reduced Craniospinal Radiation Therapy and Chemotherapy for Patients With Newly Diagnosed WNT-Driven Medulloblastoma

Help researchers study how well reduced doses of radiation therapy to the brain and spine (craniospinal) and chemotherapy work in treating patients with newly diagnosed type of brain tumor called WNT)/Wingless (WNT)-driven medulloblastoma.

Study of PEP-CMV Vaccine for Treating Newly Diagnosed Pediatric Brain Tumors and Recurrent Medulloblastoma

The purpose of this phase 2 study is to see if a cancer vaccine (PEP-CMV) can help patients’ immune system fight against high-grade glioma and medulloblastoma.

Recurrent, Progressive or Refractory Brain Tumors

Early-Phase Study of Targeted Natural Killer Cell Therapy for Children and Young Adults With Recurrent, Progressive or Refractory Brain Tumors

The purpose of this study is to see if it is safe and feasible to treat malignant brain tumors with donor natural killer (NK) cells. The hope is to see if the NK cells are effective against the tumors and improve quality of life.