Ivy Foundation Early Phase Clinical Trials Consortium

Ivy Foundation Early Phase Clinical Trials Consortium

Award: $2,911,801 over 4 years

Principal Investigator: Michael Prados, MD

Consortium Members:
Susan Chang, MD; University of California, San Francisco
Timothy Cloughesy, MD; University of California Los Angeles
Mark Gilbert, MD and WKA Yung, MD; MD Anderson Cancer Center
Ingo Mellinghoff, MD; Memorial Sloan Kettering Cancer Center
Patrick Wen, MD and Tracey Batchelor, MD, MPH; Dana-Farber /Harvard Cancer Center

Background: Despite years of clinical trials research involving thousands of patients and hundreds of trials, agents tested rarely produce clinically meaningful benefit to patients or reach the threshold for FDA approval. Large clinical trials are typically used to reduce false-positive or false-negative results, which possibly (although rarely) may reach statistical success but still not be clinically meaningful in terms of actual duration of progression-free or overall survival and may “miss” important subsets of patients because of patient heterogeneity. As a result, trials are expensive and inefficient, typically based upon preclinical models that have not been predictive of success in humans, involve irrelevant patient groups, and include drugs that may not even enter into brain tumor targets.

Hypothesis: Treatment of specific patient subgroups, enriched for targetable pathways will improve trial efficiency and increase the rate of positive trials. Testing of this hypothesis in the setting of drug exposure prior to surgical resection will allow early rejection of agents with no chance of success, ensure rapid selection of agents of true potential benefit, and will be achieved in small sample size protocols.

Primary Objective: To significantly increase the efficiency of therapeutic trials, increase the likelihood of success, and minimize exposure of drugs to patients with little chance of success

Specific Aims:
1. Create a clinical trials consortium of 5 major institutions across the United States who will cooperate to conduct clinical trials in pre-selected surgical patients at the time of relapse
2. Conduct small sample size, enriched patient clinical trials and identify if this strategy results in an efficient strategy to screen promising molecularly targeted agents
3. Create a consortium virtual tissue bank, profiling up to 250 newly diagnosed patients per year to prospectively identify patient subgroups who will be candidates for clinical trials done at the time of relapse
4. Create an ongoing clinical trials group beyond year 4 with support from biopharmaceutical groups to sustain the infrastructure developed in years 1-4