Clinical Trials

We believe ending cancer will require a weapon as adaptable and multiform as the disease itself.
We Believe

We believe ending cancer will require a weapon as adaptable and multiform as the disease itself.

Our team at The Preston Robert Tisch Brain Tumor Center understands the need to cross new thresholds when it comes to discovering the cure for brain cancer. Perhaps our most advanced methods of scientific exploration come in the form of clinical trials. While patients who enroll in clinical trials may feel like they are navigating uncharted territories, they are giving scientists an opportunity to unravel maps that can lead to new discoveries, future treatment options, and maybe even a cure.

What Are Clinical Trials?

A clinical trial is a research study involving human participants that aims to understand the effects of a certain drug, device, or treatment. Almost all treatment methods currently available are the product of clinical trials. Patients can enroll in clinical trials only after preclinical studies have been conducted. All clinical trials have a principal investigator (usually a medical doctor) who is in charge of the research staff. The principal investigator also makes sure that the protocol—or the set of rules the clinical trial is based on, such as the length of the trial, eligibility, etc.—is being followed. Through meticulous predesign, clinical trials are set up in a way to answer questions regarding safety, side effects, and benefits of the treatment, as well as whether some people will benefit more than others. There are usually four phases of a clinical trial.

What We Offer

In the past, clinical trials were often perceived as a last resort for brain tumor patients. But here at Duke, we have proven time and again that experimental therapy is often the best option for many of our patients, and could be much more effective than the standard of care treatment. We offer interventional studies for the newly diagnosed and recurrent patients, as well as supportive care studies that explore how to increase the comfort and quality of life for individuals with brain cancer. Below is a list of clinical trials being offered at the Center. Your team at Duke will discuss the best options for you or your loved one.

  • ADULT TRIALS

    Newly Diagnosed 

    Study:  EGFRvIII CAR T Cells for newly diagnosed GBM (ExCel)                                                         
    PI:  David Ashley, MBBS, FRACP, PhD, and Daniel Landi, MD
    Phase:  I
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02664363
    IRB number: Pro00069444 
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301 

    Study: A phase I trial for patients with newly diagnosed high-grade glioma treated with concurrent radiation therapy, Temozolomide, and BMX-001
    PI:
    Katherine Peters, MD, PhD
    Phase: I
    Category: Adult Neuro-Oncology
    Purpose: For detailed information, please view this trial at NCT02655601
    IRB number: 00062660
    Contact: For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301

    Study: Peptide targets for glioblastoma against novel cytomegalovirus antigens (PERFORMANCE) for newly diagnosed GBM
    PI:
    Dina Randazzo, DO
    Phase: II
    Category: Adult Neuro-Oncology
    Purpose: For detailed information, please view this trial at ClinicalTrials.gov at NCT02864368
    IRB number: 00034208
    Contact: For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301

     

  • Recurrent 

    Study:  RESIST: Patients with IDH-positive recurrent grade II glioma enrolled in a phase I safety and immunogenicity study of tumor-specific peptide vaccine
    PI:
      Katherine Peters, MD, PhD
    Phase:  I 
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov 
    IRB number:  Pro00054746 
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301 

    Study:  A phase II study of Sym004 for adult patients with recurrent glioblastoma
    PI:
      Annick Desjardins, MD, FRCPC 
    Phase: II 
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02540161 
    IRB number:  Pro00063483 
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301 

    Study:  A randomized phase 2 study of oncolytic polio/rhinovirus recombinant (PVSRIPO) alone or in combination with lomustine in recurrent WHO grade IV malignant glioma patients
    PI:
    David Ashley, MBBS, FRACP, PhD
    Phase:  II
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02986178
    IRB number:  Pro00077024
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301  

    Study:  A phase I single-center, dose escalation study of D2C7-IT administered intratumorally via convection-enhanced delivery for adult patients with recurrent malignant glioma
    PI:
      Dina Randazzo, DO
    Phase:  I
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at  NCT02303678
    IRB number:  Pro00053325 
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301 

    Study: A randomized phase 3 open-label study to evaluate the efficacy and safety of Eflornithine with Lomustine compared to Lomustine alone in patients with AA that progress/recur after irradiation and adjuvant Temozolomide chemotherapy (STELLAR)
    PI:  Annick Desjardins, MD, FRCPC
    Phase:  III
    Category:  Adult Neuro-Oncology
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02796261
    IRB number:  00076758
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301

    Study: MDNA55-05: An open-label non-randomized, multi-center phase-2 study of convection-enhanced delivery (CED) of MDNA55 in adults with glioblastoma at first recurrence or progression
    PI:  Dina Randazzo, DO
    Phase:  II
    Category:  Adult Neuro-Oncology
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02858895
    IRB number: Pro00076523
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301

  • Supportive Care 

    In addition to therapeutic clinical trials, we are also designing clinical trials that focus on the quality of life and supportive care for brain tumor patients. Currently, we have several projects that are actively recruiting participants. 

    In addition to traditional outcomes such as overall survival and progression-free survival, the measurement of patient-reported outcomes of quality of life, physical function, and neurocognitive function is becoming increasingly important in oncology clinical trials.

    Brain tumor patients have a unique set of challenges because they not only have to deal with the consequences of a cancer diagnosis, which requires surgery, radiation treatments, and chemotherapy treatments but also a neurological diagnosis that leads to neurological dysfunction.

    This neurological dysfunction can, in turn, lead to physical limitations and neurocognitive limitations. Therefore, we believe that our patients have immediate oncologic and neurological concerns that make it more challenging to achieve a positive and productive quality of life.

    Our treatments for brain tumors can also limit the quality of life in our patients.  The limitations can include nausea/vomiting, fatigue, problems with thinking, problems with physical function, problems with blood counts, and blood clots in the limbs and the lungs. We are actively recruiting and designing studies to tackle these issues and improve the lives of our patients and their loved ones. 

    Study: Phase II randomized study to evaluate efficacy, patient satisfaction, and compliance of the oral combination of Rolapitant (Varubi®) plus ondansetron vs. ondansetron monotherapy in malignant glioma patients receiving radiotherapy (RT) and concomitant temozolomide
    PI: 
    Mary Lou Affronti, DNP, RN, MHSc, ANP
    Phase: II
    Category:  Adult Neuro-Oncology 
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT0299146
    IRB number:  Pro00076418
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301                 

    Study: ECOG-ACRIN EAF151: Change in Relative Cerebral Blood Volume as a Biomarker for Early Response to Bevacizumab in Patients with Recurrent Glioblastoma
    PI: 
    Jeffrey Crawford, MD
    Category: Radiology
    Purpose: The purpose of the study is to test whether the advanced perfusion MRI done prior to starting Bevacizumab and at two weeks after starting Bevacizumab can determine response to the treatment better than the standard MRI performed at typical time points. 
    IRB number: Pro00084094
    Contact:  For more information, please contact The Preston Robert Tisch Brain Tumor Center at 919-684-5301    

    Study: Assessing the Impact of Glioblastoma Multiforme on the Quality of Life of Patients and Caregivers (The GlioQoL Observational Study) (AbbVie H15-ONC-22) 
    PI: 
    Katherine Peters, MD, PhD
    Category: Quality of life
    Purpose: This is an observational study to assess the impact of GBM on patients and caregivers.
    IRB number:  Pro00085581
    Contact: The Preston Robert Tisch Brain Tumor Center at 919-684-5301                                                                                                                                                                                                                                                                                                

  • PEDIATRIC TRIALS

    Study:  Developing evidence-based criteria for initiating treatment for neurofibromatosis type I-associated optic pathway glioma
    PI:  David Van Mater, MD, PhD 
    Phase:  N/A (Observational) 
    Category:  Pediatric Neuro-Oncology 
    Purpose:  To understand the natural history of vision in NF1 patients with an optic pathway glioma and to determine predictors of future vision loss or change in tumor size. For detailed information, please view this trial at ClinicalTrials.gov 
    IRB number:  Pro00066558 
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

    Study:  The collection of blood samples from patients with NF1 for research purposes
    PI:  
    David Van Mater, MD, PhD 
    Phase:  N/A (Biorepository) 
    Category:  Pediatric Neuro-Oncology 
    Purpose:  To augment an existing biobank resource for neurofibromatosis type 1 (NF1) research. For detailed information, please view this trial at ClinicalTrials.gov 
    IRB number:  Pro00068686 
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

    Study:  A clinical and molecular risk-directed therapy for newly diagnosed medulloblastoma (SJMB12)
    PI:  
    David Ashley, PhD
    Category:  Pediatric Neuro-Oncology 
    Purpose:  The purposes of this study are to find out if patients with low-risk,  wingless + int-1 (WNT) tumors can be treated with a lower dose of radiation to the brain and spine and a lower dose of the chemotherapy drug cyclophosphamide, while still achieving the same survival rate as past St. Jude studies with fewer side effects; to find out if adding targeted chemotherapy after standard chemotherapy will benefit participants with sonic Hedgehog (SHH)-positive tumors; and to find out if adding new chemotherapy agents to the standard chemotherapy will improve the outcome for intermediate and high risk non-WNT, non-SHH tumors. For detailed information, please view this trial at ClinicalTrials.gov at NCT01878617
    IRB number: Pro00047285  
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288

  • Recurrent 

    Study:  PBTC-042: Phase I study of CDK 4-6 inhibitor PD-0332991 in children with recurrent, progressive or refractory central nervous system tumors
    PI:  David Van Mater, MD, PhD
    Phase:  I
    Category:  Pediatric Neuro-Oncology
    Purpose:  For detailed information, please view this trial at ClinicalTrials.gov at NCT02255461
    IRB number:  Pro00054303
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288
     
    Study: Phase 1b Study PVSRIPO for Recurrent Malignant Glioma in Children
    PI:  Eric Thompson, MD
    Phase: IB
    Category: Pediatric Neuro-Oncology
    Purpose: The purpose of the study is to confirm the safety of the selected dose and potential toxicity of oncolytic poliovirus (PV) immunotherapy with PVSRIPO for pediatric patients with recurrent WHO grade III or IV malignant glioma. For more information, please visit this trial at ClinicalTrials.gov at  NCT03043391
    IRB number:  Pro00071228
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288
     
    Study: PEP-CMV in Recurrent Medulloblastoma/Malignant Glioma (PRiME)
    PI:  Gary Archer, PhD
    Phase: Not yet enrolling
    Category: Pediatric Neuro-Oncology
    Purpose: The primary goal of this prospective clinical trial is to evaluate the safety of PEP-CMV in patients with recurrent medulloblastoma and malignant glioma. Patients with histologically-proven medulloblastoma or malignant glioma who had received definitive chemo-radiotherapy and subsequently had tumor recurrence/progression may be enrolled any time after recurrence/progression regardless of prior adjuvant therapy. PEP-CMV is a vaccine mixture of 2 peptides comprised in two components (referred to as Component A and Component B). Component A is a synthetic long peptide (SLP) of 26 amino acid residues from human pp65. Component B consists of a neutralizing antibody epitope from human CMV glycoprotein B (gB) conjugated to KLH. For more information, please visit this trial at ClinicalTrials.gov at NCT03299309
    Contact:  For more information, please contact Pediatric Neuro-Oncology at 919-684-2288