Testing a New Oncology Drug Development Paradigm - Section 4

The distinctive features of phase 0 studies are:

• First-in-human, with single or combination drugs
• Molecules from CCR, academia, small biotech
• Small patient numbers (6–10); joint CCR-DCTD clinical trial effort performed in the Clinical Research Center
• Provide PK/PD data to support rapid future dose escalation based on extensive preclinical toxicology and target effect studies
• Initial target assay development and drug/target assessments (primary and surrogate, imaging and molecular expression)
• Preliminary toxicity evaluation in humans
• Inform subsequent broad phase I/II trials
• High throughput of trials, each completed in three to six months
• Preliminary toxicity evaluation in humans

The following provide more information:

• http://dtp.nci.nih.gov/docs/phase0/PharmacoDynamicAssayDeveloment.html
• http://dtp.nci.nih.gov/timeline/noflash/milestones/M19_PKPD.htm
• /MajorInitiatives/02NExT.htm
• Kummar S, Kinders R, Parchment RE, Murgo AJ, Collins J, Pickeral O, Low J, Steinberg SM, Gutierrez M, Yang S, Helman L, Wiltrout R, Tomaszewski JE, Doroshow JH. Compressing Drug Development Timelines in Oncology Using Phase '0' Trials. Nature Reviews Cancer 2007:7;131-139.