What is a Phase 0 Clinical Trial?

Moving Beyond the Status Quo

For most brain tumor patients, enrolling in conventional clinical trials involves a leap of faith. After being prescribed a new agent, you struggle through significant side-effects and the agonizing wait for brain imaging several months later. Under ideal circumstances, the experimental therapies arrests disease progression and, in these cases, the time and effort invested by you and your family were worthwhile. Unfortunately, a more common result is that the new therapy is ineffective, costing you precious time and energy as you, once again, face another set of imperfect treatment options.

The Ivy Brain Tumor Center does not subscribe to such conventions. Our clinical trials are precision medicine studies, which means that each patient’s tumor is first defined by its distinct signature of genetic abnormalities and each experimental treatment regimen is then selected to attack that specific code. More importantly, all of our studies integrate a ‘Phase 0’ clinical trial design, which enables us to test dozens of new therapies quickly in brain tumor patients – not just in the laboratory – and get patients the new regimen that fits their exact circumstances.

Understanding the Phase 0 Clinical Trial Paradigm

The Ivy Brain Tumor Center is home to the largest Phase 0 clinical trials program for brain tumor patients in the world. Our studies select patients with specific genetic signatures to receive a single dose of a matched experimental regimen a day prior to their previously-planned brain tumor operation. Instead of placing all our hopes on a single drug, our trials emphasize combined-agent approaches that recognize the inherent complexities, and resistance mechanisms, of brain tumors. At the time of surgery, tissue samples are rapidly collected and then tested in our laboratories using an array of highly-sensitive molecular and pharmacological technologies. Within days of surgery, our Phase 0 team identifies which investigational therapies have successfully penetrated the patient’s tumor and whether these agents have also effectively modulated the tumor’s biology. Once the experimental regimen proves its ability to reach its target and undermine the tumor’s core programming, then the patient is ‘graduated’ to this chosen regimen at maximum therapeutic doses. Now, the tumor is finally under siege.

Phase 0 patients who are graduated to therapeutic dosing will have the confidence of knowing that their tailored treatment regimen is based upon direct evidence of their individual tumor’s responsiveness. Conversely, Phase 0 studies also identify which specific strategies will not work for individual patients and tumors. This critical information spares patients from investing their precious time, effort, and energy in futile therapies that will not meet their needs. Because of the unprecedented speed with which our analysis is completed, our Phase 0 trial patients do not lose any opportunity to pursue other avenues for treatment at any center, including any other clinical trial, after surgery. We understand that time is the most important commodity for brain tumor patients and our clinical trials stand apart from conventional studies that routinely take months to inform patients that a therapy is ineffective.

Phase 0 studies are not synonymous with first-in-human studies. While some of our experimental agents are being tested in patients for the first time ever, many more of our clinical trials consist of new agents and new combinations of agents that are proven safe in patients, but have never been trialed as a therapy for brain tumors. Without exception, the Ivy Brain Tumor Center and its investigators have no financial or proprietary interest in any agent in our clinical trials. 

Trial Screening Requests

For brain tumor patients facing the fight of their lives, no stone should be left unturned. Our clinical investigators treat more brain tumor patients than any hospital in the nation and the Ivy Brain Tumor Center’s portfolio of early-phase clinical trials serves as a resource for the world’s brain tumor community. Within 36 hours of a trial screening request, our team assesses a patient’s suitability for our clinical trials program, as well as provides additional support to the physicians managing these cases. Patients enrolled in our studies are not required to transfer their care to our center, as our philosophy is to partner with, not replace, their treatment team. 

Trial Screening Requests are provided at no cost and, for patients we determine to be eligible for a clinical trial, the Ivy Brain Tumor Center covers all costs associated with study participation.

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