Choice of designs and doses for early phase trials.
This paper reviews state-of-art statistical designs for dose-escalation procedures in first-into-man studies. The main focus will be on studies in oncology, as most statistical procedures for phase I trials have been proposed in this context. Extensions to situations such as the observation of bivariate outcomes and healthy volunteer studies are also discussed. The number of dose levels and cohort sizes used in early phase trials are considered. Finally, this paper raises some practical issues for dose-escalation procedures.
Zhou
Yinghui
Y
Medical and Pharmaceutical Statistics Research Unit, The University of Reading, PO Box 240, Earley Gate, Reading RG6 6FN, Berkshire, UK.
D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects; D001499:Bayes Theorem; D017321:Clinical Trials, Phase I as Topic / Q000379:methods*; D004305:Dose-Response Relationship, Drug; D064420:Drug-Related Side Effects and Adverse Reactions; D006801:Humans; D020714:Maximum Tolerated Dose; D009369:Neoplasms / Q000188:drug therapy; D004364:Pharmaceutical Preparations / Q000008:administration & dosage*; D012107:Research Design; D018401:Sample Size
D000970:Antineoplastic Agents; D004364:Pharmaceutical Preparations