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 is a multi-center, target validation study of Fimepinostat, a novel oral, potent dual pan-HDAC and PI3K inhibitor. Fimepinostat, has shown broad antitumor activity in hematologic and solid tumors. This study will include children and young adults (3-39 years of age) with newly diagnosed DIPG, recurrent medulloblastoma, or recurrent HGG who will undergo tissue collection as part of standard of care.  Patients will initiate drug prior to resection/biopsy and continue drug during a maintenance phase after resection/biopsy. Patients will be divided into 3 strata: newly diagnosed DIPG (Stratum A), recurrent medulloblastoma (Stratum B), or recurrent HGG (Stratum C). We hypothesize that drug penetration might vary between these entities given location as well as degree of enhancement.


Funding is provided by Curis & Cannonball Kids Cancer.


This is a Phase I and target validation, multicenter study for adolescent and young adult (AYA) patients with either newly diagnosed IDH1/2-mutant HGG following maximal resection followed by adjuvant radiotherapy with or without concurrent temozolomide (Arm A) or recurrent IDH1/2-mutant LGG or HGG after any treatment, including resection alone in the LGG population (Arm B).

A precision medicine analysis of pediatric brain tumors showed IDH1/2 mutations to be particularly concentrated in the adolescent age group. The PARP inhibitor BGB-290 was selected for this trial, because of high levels of CNS penetration in mouse models relative to other PARP inhibitors and increased levels of PARP trapping (a mechanism correlated with increased synthetic lethality in IDH1/2 mutant cells). BGB-290 will be tested in combination with temozolomide.

Funding is provided by BeiGene & CureSearch for Children’s Cancer.

Patients with Neurofibromatosis 1 (NF1) have an increased risk of developing tumors of the peripheral nervous system, including plexiform neurofibromas (PN), which are benign nerve sheath tumors that are among the most debilitating complications of NF1. There are no standard treatment options for PN other than surgery, which is often difficult due to the extensive growth and invasion of surrounding tissues. This study is testing the oral drug binimetinib to determine whether it can block the MAPK signaling pathway which has been shown to be important in PN growth. Funding is provided by the NF Consortium and Array BioPharma.

Immunotherapy is emerging as new treatment for a variety of malignancies, including pediatric DIPG and HGG. Immunotherapy uses one’s own anti-tumor immune capabilities and has gained recent attention as a complementary approach to traditional therapies like radiation. One protein called programmed cell death-1 (PD-1) can be made by tumor cells as a mechanism to shut off the body's immune response to tumor. PD-1 works by binding to the body’s T-cells and giving them a message to turn off. Preclinical studies looking at blocking PD-1 in brain tumors has shown that PD-1 blockade increases survival and leads to long-term responses in mice. Additionally, PD-1 blockade combined with radiation appears to heighten the local immune response and increase the number of active T-cells surrounding the tumor. Hypofractionated radiation (higher doses given over fewer days) may heighten the effects by further increasing local inflammation and leading to greater numbers and activation of tumor-associated immune cells. In DIPG, hypofractionated radiation may also improve quality of life by shortening radiation periods and decreasing time spent in the hospital. These findings have led to interest that PD-1 blockade combined with radiation, either standard or hypofractionated, could be a new way to treat brain tumors.

REGN2810 is a novel, human antibody directed at the PD-1 receptor that prevents PD-1 activation by blocking interaction with its signal proteins, PD-L1 and –L2. PNOC013 combines REGN2810 with radiation as a new treatment strategy for children and young adults with newly diagnosed DIPG, newly diagnosed, and recurrent HGG, as well as children with recurrent solid tumors. The study is a multi-arm clinical trial that will randomize patients with DIPG or HGG to different radiation schedules (standard or hypofractionated) to be given in combination with REGN2810. REGN2810 will then continue as monotherapy once radiation is complete. PNOC013 will evaluate the safety of this treatment approach, as well as look at the impact on survival and disease responses in patients treated on this study.  Funding is provided by Regeneron Pharmaceuticals.

Many types of cancer are primarily treated with surgery and patient survival is directly related to the extent to which the tumor is able to be removed. It is often difficult for surgeons to distinguish tumor tissue from normal tissue or to detect tumor cells that have spread from the original tumor site, resulting in incomplete removal of the tumor and reduced patient survival. In some sites, such as the brain, it is critical to avoid damage to normal tissue around the tumor to prevent adverse effects of surgery on function. Tozuleristide is a drug that is thought to attach to tumor tissue and then fluoresces (glows) when a special light from the Canvas is shined on it. It is hypothesized that tozuleristide, when imaged with the Canvas, will improve surgical outcomes by allowing surgeons to visualize the edges of the tumor or other ambiguous tissue in real-time as they operate. The purpose of this study is to evaluate how well tozuleristide imaged with Canvas work at helping to distinguish between tumor and normal tissue during surgery in pediatric primary central nervous system tumors. Funding is provided by Gateway for Cancer Research.

Advancements in gene expression and sequencing technologies allow oncologists to utilize 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 using a precision based medicine approach in collaboration with the NantOmics GPS Cancer® platform for children with newly diagnosed High Grade Glioma (HGG). 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. Funding is provided by The V Foundation.

MTX110 is a soluble form of panobinostat, which has been shown to be effective against brain cells in pre-clinical studies. For this study, MTX110 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."  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 MTX110, the insertion catheters are removed and can be replaced every 4 to 8 weeks as needed for repeat treatments. Funding is provided by Cannonball Kids’ Cancer and The Cure Starts Now.

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 8 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 and Solving Kids’ 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 and the Mithil Prasad 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.

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).