Phase 0 Key Points

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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.

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Phase 0 Key Points

• 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
• Allows for the assessment of target inhibition as
  a potential primary endpoint in subsequent trials
• Develop assay proceduresfrom tissue acquisition
  to the assay itselfthat actually work in
  patients in the clinic

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Phase 0 Key Points

• 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

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Phase 0 Key Points

• Do you know whom to ask for help?

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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

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Special Thanks to the NCI Phase 0 Team

• 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

• 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

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