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GBM AGILE (Glioblastoma Adaptive Global Innovative Learning Environment) is the world’s first “adaptive clinical trial platform.”


Adaptive platform trials offer a unique system for the discovery and testing of various experimental drugs and create a more efficient and cost-effective mechanism for accelerating treatments to patients. The “platform,” or “master protocol,” aspect of the trial allows for testing of more than one investigational drug, and GBM AGILE will simultaneously evaluate multiple therapies for newly diagnosed and recurrent glioblastoma (GBM) patients and identify effective new treatments for subtypes of this tumor based on patients’ biological characteristics in a rapid manner.

GBM AGILE is led by top-tier key opinion leaders in the GBM field and has the collective support of an international group of more than 130 clinicians, researchers, biostatisticians, imagers, pathologists, leaders from government, and industry, and patient advocates. As an adaptive study, the number of patients recruited for GBM AGILE arms, and their allocation within the study, will be continuously adjusted in light of emerging results. This is considered a more efficient approach to drug development, and one that has received significant interest and support from clinicians, industry, and regulatory agencies. NBTS believes this design will allow new potential treatments being tested to move the trial process much faster and require significantly fewer patients, especially for the control arm. We also believe these benefits will be attractive to the companies that make drugs, encouraging even more of them to take shots at developing drugs for brain tumors, creating a multiplying effect. 

We strongly believe that new approaches are desperately needed to advance more, safe and effective treatments for glioblastoma patients. We are planning for GBM AGILE to be a critical vehicle for adapting, learning and ultimately succeeding at changing the trajectory of glioblastoma and improve patient lives.

Attribution: David Arons, CEO, National Brain Tumor Society

The GBM AGILE Clinical Trial

GBM AGILE ( identifier: NCT03970447) is sponsored by the Global Coalition for Adaptive Research (GCAR) a nonprofit organization comprised of some of the world’s foremost clinical, translational, and basic science researchers.

More information is available here

To find a listing of cancer centers and hospitals serving as trial sites for GBM AGILE visit here.

40+ Sites

that are now offering access to the GBM AGILE clinical trial, with five treatment arms operating and three actively enrolling patients

3 Regions

are represented in this clinical trial (Canada, Europe, and the U.S.)

National Brain Tumor Society’s Support of GBM AGILE

National Brain Tumor Society, along with the National Foundation for Cancer Research (NFCR) and the Asian Fund for Cancer Research, is a key strategic partner for GCAR and the GBM AGILE platform, providing philanthropic and advocacy support.

NBTS was there when the concept for this type of adaptive trial was first born in a meeting with leaders from across the neuro-oncology field, when the trial was announced with fanfare (including a cameo from then Vice President, and now, President-elect Joe Biden) in Washington, D.C., and made two substantial investments in the trial’s operations in 2019, totaling more than $1.25 million. We continue to support GBM AGILE as a major, revolutionary approach to clinical trials for patients with a brain tumor. We will keep the community updated when we learn important news about the trial.

“NBTS has long supported GBM AGILE as a scientifically cutting-edge and more patient-friendly way to develop and evaluate emerging new treatments for patients,” says David Arons, chief executive officer, National Brain Tumor Society. “As a patient advocacy partner to the GBM AGILE program, we truly believe that this dynamic, nimble platform offers more opportunities for patients with glioblastoma to access promising therapies in development and hope for new treatment approvals for a patient population that currently lacks adequate options to fight this devastating disease. With the urgency of now, GBM AGILE is exactly the kind of innovation we need to transform brain tumor clinical research to change the unacceptable status quo facing patients and their families with glioblastoma.”

NBTS thanks the Global Coalition for Adaptive Research, the patients involved in the study, the trial’s investigators and participating biopharmaceutical companies, and other partners, including NFCR and StacheStrong, together with support from the Uncle Kory Foundation, for their collaboration on this important trial.

For questions regarding philanthropic support for GBM AGILE, please contact the NBTS Development Office at or 617-237-1731.

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