Clinical Trials

To find out if your child or patient is eligible to enroll in a PNOC clinical trial, contact the closest participating site or contact us at For a detailed description of the protocol, click on the trial name below.

Current Trials

This trial is testing  the drug TAK-580 in children and adolescents (< 18 years) with radiographically recurrent or radiographically progressive non-hematologic malignancies (CNS or solid tumors) associated with activation of the RAS/RAF/MEK/ERK pathway. TAK-580 is an oral, highly selective small-molecule pan-RAF-kinase inhibitor that demonstrates potent inhibition of the oncogenic BRAFv600e mutant enzyme as well as a small subset of kinases including Abl, Arg, FRK, Lck, EphA2, EphA8, DDR2 and SPAK2a. Funding is provided by A Kids Brain Tumor Foundation (aka The PLGA Foundation) and Team Jack Foundation.

Irinotecan is an important anti-cancer drug, but cannot distribute well into the brain or brainstem because of the blood brain barrier.  For this study, nanoliposomal irinotecan is injected directly into the tumor using small, flexible catheters inserted into the tumor.  The drug is slowly injected throughout the tumor over several hours.  This technique is called convection-enhanced delivery or "CED."  Nanoliposomal irinotecan means the irinotecan has been "packaged" into a nanoparticle that remains active in the tumor for weeks.  When the drug is given by CED, it allows the drug to bypass the blood brain barrier and remain in the tumor.  After each CED treatment with nanoliposomal irinotecan, the insertion catheters are removed and can be replaced every 4 to 6 weeks as needed for repeat treatments. Funding is provided by The V Foundation.

An oncolytic virus derived from a modified measles virus targets the protein CD46, which is highly overexpressed by medulloblastoma and AT/RT cells. When the oncolytic virus recognizes CD46 it causes cell destruction and tumor regression. This first-of-its-kind therapy is a new, targeted treatment option for patients who cannot be cured with standard care for these tumors. Funding is provided by No More Kids with Cancer.

This is a study of an immunotherapy vaccine for pediatric glioma, including diffuse intrinsic pontine glioma (DIPG). Recent genetic studies have revealed that malignant gliomas in children often show recurrent missense mutations in H3F3A, which encodes the replication-independent histone 3 variant H3.3. This study will evaluate safety and immunological activity of a vaccine using a specific synthetic peptide for the H3.3K27M epitope in HLA-A2+ children with newly diagnosed DIPG or other gliomas who are positive for the H3.3K27M mutation. Funding is provided by The V Foundation.

This is an open label trial to assess the safety and tolerability of the adult-tolerated dose of hyperpolarized pyruvate for metabolic imaging in children with brain tumors who do not require sedation for their MR imaging. Patients will receive a single MR imaging examination that includes the acquisition of hyperpolarized 13C metabolic data in combination with anatomic, diffusion, perfusion and lactate edited 1H spectroscopic imaging data. The data will be processed using custom designed software to estimate changes in levels of lactate/pyruvate and to relate them to abnormalities observed in the data from other MR modalities.

Recent advances in gene expression and sequencing technologies allow oncologists now for the first time to use these technologies in real time for clinical decision making and to determine an individualized treatment plan based on each child’s tumor characteristics. This trial will test the feasibility using a precision based medicine approach in collaboration with the Translation Genomic Research Institute (TGEN) for children with newly diagnosed diffuse intrinsic pontine glioma (DIPG). Based on the molecular characteristics of each child’s tumor a specialized tumor board will recommend a treatment plan based on the results of the molecular profiling. All current FDA approved drugs will be considered for the treatment plan. This will be the first trial in the US to test such an approach for children with DIPG. Funding provided by the Pediatric Brain Tumor Foundation and the V Foundation.

This study is testing the oral drug everolimus to determine whether it can block an abnormal cell-signaling pathway (PI3K/Akt/mTOR) involved in the growth of low-grade gliomas. Funding provided by A Kids' Brain Tumor Cure Foundation (aka The PLGA Foundation). 

This trial is testing the drug vemurafenib (also called PLX4032) in children with pediatric astrocytomas that have the BRAFV600E mutation. Vemurafenib works by blocking the activity of BRAF, a key protein in the RAS/RAF/MAPK pathway that is overactive in these tumors.