News & Blog

Potential Brain Tumor Treatments Entering the Pivotal Phase of Evaluation – June 2018

A large, randomized, controlled 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. A year ago, the NBTS staff conducted and published a review of all the Phase III clinical trials for glioblastoma patients that have been registered with regulators. This became our most read blog of 2017, and to this day continues to be a top destination for visitors to this blog. All told, more than 15,000 unique individuals have viewed the post. So, we’ve committed to publishing this feature twice-yearly moving forward – once at the half-way point of the year and once at the end of the year – and expanding the feature beyond glioblastoma to all brain tumor types.

So, below is a list of Phase III clinical trials that are currently enrolling patients to evaluate potential new brain tumor treatments as recorded at the halfway point of 2018.

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 – meaning researchers are evaluating these already-in-use treatments in different combinations, different dosing regimens, or subpopulations of patients.

What follows is a breakdown/overview of the current Phase III brain tumor clinical trial landscape, followed by the full list of trials with hyperlinks for more information.

Phase III Brain Tumor Clinical Trials Overview

  • There are 14 Phase III clinical trials currently enrolling for brain tumor patients
    • Eleven (11) are for adult (18+) patients
    • Three (3) are for pediatric and young adult (up to age 21) patients
    • Eleven (11) have measures of extending survival as their primary outcome measure (endpoint)
    • Two (2) have measures of “quality of life” or patient-centric measures as their primary endpoint
    • One has “extent of resection” as its primary endpoint
  • Seven (7) trials are of “novel” or “investigational” treatments (two of these trials, however, are of the same novel treatment, just in different combinations)
    • One novel treatment (being evaluated in two separate trials), is an immunotherapy known as nivolumab (Opdivo), which is already being successfully used to treat some lung, skin, kidney, and bladder cancer patients. It’s known as a “checkpoint inhibitor” and works by “releasing the breaks” on the body’s immune system – blocking a signal that would have prevented activated T cells from attacking the cancer, thus allowing the immune system to clear the cancer.
    • One is of a gene therapy known as Toca 511 and Toca FC
      • We’ve covered this treatment – which NBTS helped fund – on multiple occasions, check out these posts (here and here)
    • One (ABT-414) is of a “targeted therapy,” or treatments designed to attack a specific abnormality in the tumor (this is the concept of “precision” or “personalized” medicine), in this case, a common mutation found glioblastoma tumor cells known as “EGFRvIII.”
    • Two (2) are of drugs (TSC and Eflornithine) that might be able to sensitize tumors better to chemotherapy and radiation
    • One is for a drug (Armodafinil) that might help reduce fatigue in patients with high-grade glioma
  • Seven (7) trials are registered with known/already approved brain tumor therapies/treatment types. These trials include different variations of chemotherapy, radiation, surgery, or combinations thereof.

Phase III Clinical Trials with Novel/Investigational Agents

Phase 3 Clinical Trials with Prior/Known Agents

*To support GBM research and help us get to even more potential new glioblastoma treatments faster, please visit here.

*To check out more clinical trial options, please visit the NBTS Clinical Trial Finder at and check-out yesterday’s post on all the new clinical trials – of any phase – that have opened in recent months, here

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