Phase 0 Key Points Are Phase 0 studies feasible? ¬Phase 0 recruitment is feasible but depends on patient altruism and prior physician-patient relationship.

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Presentation transcript:

Phase 0 Key Points Are Phase 0 studies feasible? ¬Phase 0 recruitment is feasible but depends on patient altruism and prior physician-patient relationship. ¬Statistical methodology is more challenging in Phase 0 trials, which involve novel PD endpoints and small number of samples. ¬Require additional up-front investment in imaging or assay development, in addition to that needed for standard PK.

Phase 0 Key Points In what circumstances are Phase 0 studies applicable for early drug development? In what circumstances are Phase 0 studies applicable for early drug development? ¬Phase 0 trials provide the opportunity to develop PD assays measuring target function in tumors in humans u Allows for the assessment of target inhibition as a potential primary endpoint in subsequent trials u Develop assay proceduresfrom tissue acquisition to the assay itselfthat actually work in patients in the clinic

Phase 0 Key Points Does conducting a Phase 0 study save clinical development time? Does conducting a Phase 0 study save clinical development time? ¬Critical question is timewill take years to assess ¬Likely that imaging-driven PD will make a major impact soon

Phase 0 Key Points Do you know whom to ask for help? Do you know whom to ask for help?

Special Thanks to the NCI Phase 0 Team DCTD James H. Doroshow Joseph E. Tomaszewski Jerry Collins Tony Murgo (Jennifer Low) (Oxana Pickeral) Melinda Hollingshead Gurmeet Kaur Sherry Yang Larry Phillips Larry Rubinstein Seth Steinberg Alice Chen CCR Shivaani Kummar Martin Gutierrez Richard Chang Anthony Kim Yvonne Horneffer Lamin Juwara Maryann Yancey Janelle Bingham Yves Pommier William M. Bonner Olga A. Sedelnikova Christophe Redon Abbott Laboratories ABT-888 Team

Special Thanks to the NCI Phase 0 Team SAIC Anne Monks Will Jacob Carrie Bonomi Suzanne Borgel John Carter Raymond Divelbiss Kelly Dougherty Christina Hernandez Les Stotler Sadie Frary Jagada Thillianathan Jessica Zalek Kimberly Hill Eva Majerova Gina Uhlenbrauck FDA Ray Klecker SAIC Ralph Parchment Robert Kinders Jay Ji Sonny Khin Weimin Zhu Yiping Zhang Kate Luyegu Sylvan McDowell Ravithat Putvatana Tiziano DiPaolo Sima Hayavi Kristi Robillard Dorj Dorjsuren Shawn Clopper Will Yutzy Dwight Simmons Melanie Simpson Vali Sevastita

All the present and future patient participants of Phase 0 clinical trials