Phase I Oncology Clinical Trials with Potential Late Toxicities: Experiences with the TITE-CRM Design

Ben E. Leiby, PhD

Associate Professor and Division Head, Biostatistics

Thomas Jefferson University

Treatment for cancer is evolving at a rapid pace. Finding safe and effective doses of new treatments in the most efficient way is important for advancing these therapies into clinical practice quickly. This is particularly important in the setting of advanced disease where, in spite of recent advances, treatment options remain suboptimal for many patients. In this talk, I will discuss Phase I trial designs in the setting of relatively long follow-up periods for observing toxicity. In particular, I focus on the Time to Event Continual Reassessment Method (TITE-CRM) design which we have implemented in a recently-opened trial at Jefferson. I discuss performance of the design as assessed by simulation studies and the challenges of real-world implementation. Benjamin Leiby, PhD, is Associate Professor and Director of the Division of Biostatistics in the Department of Pharmacology and Experimental Therapeutics at Thomas Jefferson University’s Sidney Kimmel Medical College. He also directs the Biostatistics Shared Resource for the Sidney Kimmel Cancer Center. In recent years, Dr. Leiby’s collaborative work has been in the areas of Cancer, Psychiatry, and Rehabilitation Medicine with particular focus in Prostate and Pancreas cancers. He earned his PhD in Biostatistics from the University of Pennsylvania.