NBTS Brain Tumor Clinical Trials Report: Q4 2021

It can be hard to keep track of new clinical trial opportunities opening across the United States, especially as we continue to navigate the public health crisis created by the coronavirus (COVID-19). There are many clinical trials actively enrolling patients, and this report provides a summary of the studies that have recently opened or started for primary brain tumor patients*. To learn more about each trial, and to contact the party responsible for enrolling patients, just follow the hyperlink.**

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• A study to see if memantine protects the brain during radiation therapy treatment for a brain tumor
  • This phase III trial compares the drug memantine to usual treatments for patients with primary brain tumors that are newly diagnosed or have come back (recurrent) to assess its effectiveness in preserving cognitive function (attention, memory, or other thought processes) in children and adolescents receiving radiation therapy.
    
• RRx-001 given with irinotecan and temozolomide for pediatric patients with recurrent or progressive malignant solid and central nervous system tumors (PIRATE)
  • The phase I PIRATE study tests the experimental drug RRx-001 in combination with two chemotherapy drugs commonly used in patients with brain tumors.

• Quantitative assessment of radiation-induced neuroinflammation: A proof of principle study
  • The phase II QARIN 1 trial is a study of a new imaging technique for longitudinal, quantitative assessment of brain neuroinflammation following whole-brain radiation therapy. 
    
• Association of peripheral blood immunologic response to therapeutic response to adjuvant treatment with immune checkpoint inhibition in patients with newly diagnosed glioblastoma or gliosarcoma
  • This phase II trial will study newly diagnosed adult glioblastoma or gliosarcoma patients to look at the changes in immune cells in the blood of those who take ipilimumab and nivolumab along with temozolomide.
    
• A study of RNA-lipid particle (RNA-LP) vaccines for newly diagnosed pediatric high-grade gliomas and adult glioblastoma 
  • This trial is a first in human phase I/II study of RNA-LP vaccines for newly diagnosed adult MGMT unmethylated glioblastoma patients. The phase I portion of the study will involve a dose-escalation study to identify the maximally tolerated dose (MTD). Up to 28 participants may be enrolled.

• Combination therapy for the treatment of diffuse midline gliomas
  • This phase II trial will determine if the combination of ONC201 with different drugs, panobinostat or paxalisib, is effective for treating pediatric patients with diffuse midline gliomas (DMGs).

• The PCSK9i inhibitor evolocumab: A surgical trial of pharmacodynamics and kinetics evaluation
  • This phase 0 surgical window of opportunity trial seeks to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) properties of an FDA-approved drug called Repatha (evolocumab) in patients with primary and recurrent World Health Organization (WHO) grade IV malignant glioma. The primary objective is to evaluate whether evolocumab crosses the blood-brain barrier (BBB) and is measurable in the resected tumor specimens of patients with primary and recurrent high-grade glioma or glioblastoma.

• Trial of niraparib in participants with newly diagnosed glioblastoma and recurrent glioma
  • This trial is an open-label, multi-center phase 0 study of the drug niraparib, with an expansion phase that will enroll up to 24 participants with newly diagnosed glioblastoma and up to 18 recurrent glioma participants with IDH mutation and ATRX loss.

• SurVaxM plus adjuvant temozolomide for newly diagnosed glioblastoma (SURVIVE) 
  • The main purpose of this phase II study is to determine whether adding SurVaxM to standard-of-care temozolomide chemotherapy is better than temozolomide treatment alone for patients with newly diagnosed glioblastoma. This study is designed to compare the length of survival in patients with newly diagnosed glioblastoma who receive temozolomide plus SurVaxM to that of patients treated with standard-of-care temozolomide plus placebo.
    
• Recurrent glioblastoma treated with neurosurgical resection and intraoperative radiation therapy using the Xoft Axxent eBx System and bevacizumab
  • The purpose of this phase II trial is to assess the overall survival of patients treated with the Xoft Axxent eBx System and post-radiation adjuvant Bevacizumab for single-fraction intraoperative radiotherapy following maximal neurosurgical resection of recurrent glioblastoma. A historical comparison will be made to the results of the EBRT + bevacizumab arm of RTOG 1205.
    
• Dendritic cell vaccination with standard postoperative chemoradiation for the treatment of adult glioblastoma
  • This phase I clinical trial assesses the safety of a novel personalized dendritic cell vaccine administered to glioblastoma patients shortly after completing standard-of-care treatments. 

• Flavored, oral irinotecan VAL-413 (Orotecan®) given with temozolomide for the treatment of recurrent pediatric solid tumors
  • A pilot pharmacokinetic trial to determine the safety and efficacy of a flavored, orally administered irinotecan VAL-413 (Orotecan®) given with temozolomide for the treatment of recurrent pediatric solid tumors including but not limited to neuroblastoma, rhabdomyosarcoma, Ewing sarcoma, hepatoblastoma, and medulloblastoma.
    
• Natural progesterone for the treatment of recurrent glioblastoma
  • This early phase I trial identifies the best dose, possible benefits and/or side effects of natural progesterone in treating patients with glioblastoma that has come back (recurrent). Progesterone is a hormone made by the body that plays a role in the menstrual cycle and pregnancy.
    
• Vaccine therapy for the treatment of newly diagnosed glioblastoma (ATTAC-II)
  • The purpose of this phase II study is to determine if an investigational dendritic cell vaccine, called pp65 DC, is effective for glioblastoma (GBM) when given with stronger doses of routine chemotherapy.

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Below is a list of phase III clinical trials that are currently enrolling patients as recorded at the close of 2021. A phase III clinical trial is traditionally the last, pivotal step in the evaluation of a potential new medicine before it is approved for use by all patients with the indicated disease.

Many of these are trials of a “novel/investigational agent” – meaning it is the first time a particular treatment is being tried for brain tumor patients. There are also a number of trials with therapies already in use for some brain tumor patients, for which researchers are evaluating these existing treatments in different combinations, different dosing regimens, and/or specific subgroups of patients.

Phase III Clinical Trials with Novel/Investigational Agents

• A trial to evaluate multiple regimens in newly diagnosed and recurrent glioblastoma (GBM AGILE)
• Study of AG-881 in participants with residual or recurrent grade II glioma with an IDH1 or IDH2 mutation (INDIGO)
• Testing the use of the immunotherapy drugs ipilimumab and nivolumab plus radiation therapy compared to the usual treatment (temozolomide and radiation therapy) for newly diagnosed MGMT unmethylated glioblastoma
• Gleolan for visualization of newly diagnosed or recurrent meningioma (MEN-301)
• Study to evaluate eflornithine + lomustine vs. lomustine in recurrent anaplastic astrocytoma patients (STELLAR STUDY)
• A study of the drugs selumetinib versus carboplatin/vincristine in patients with neurofibromatosis and low-grade glioma
  • A study of the drugs selumetinib vs. carboplatin and vincristine in patients with low-grade glioma
  • A study to compare treatment with the drug selumetinib alone versus selumetinib and vinblastine in patients with recurrent or progressive low-grade glioma
• Study of tozuleristide and the Canvas imaging system in pediatric patients with central nervous system tumors undergoing surgery
• A study to see if memantine protects the brain during radiation therapy treatment for a brain tumor
• A study of DSP-7888 dosing emulsion in combination with bevacizumab in patients with recurrent or progressive glioblastoma following initial therapy

Phase III Clinical Trials with Prior/Known Agents

• A study comparing two carboplatin containing regimens for children and young adults with previously untreated low-grade glioma
• Radiation therapy with concomitant and adjuvant temozolomide versus radiation therapy with adjuvant PCV chemotherapy in patients with anaplastic glioma or low-grade glioma
• Intraoperative radiotherapy in newly diagnosed glioblastoma
• Standard chemotherapy vs. chemotherapy guided by cancer stem cell test in recurrent glioblastoma (CSCRGBM)
• Observation or radiation therapy in treating patients with newly diagnosed grade II meningioma that has been completely removed by surgery
• A trial of enzastaurin plus temozolomide during and following radiation therapy in patients with newly diagnosed glioblastoma with or without the novel genomic biomarker, DGM1

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*For a comprehensive list of ALL open brain tumor clinical trials, visit the NBTS Clinical Trial Finder, where you can search potential opportunities for enrollment based on criteria specific to you.

**For additional navigation support, you can access NBTS’s Personalized Support and Navigation program by emailing patientnavigator@braintumor.org. Our patient navigation team has years of experience working closely with brain tumor patients and families.
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