A Statistical Design of Phase II Cancer Trials Using Total and Complete Response Endpoints

• Activity Number: 219
• Type: Contributed
• Date/Time: Tuesday, August 10, 2004 : 10:30 AM to 12:20 PM
• Sponsor: WNAR
• Author(s): Ying Lu*+ and Hua Jin and Kathleen R. Lamborn
• Companies: University of California, San Francisco and University of California, San Francisco and University of California, San Francisco
• Keywords: phase II cancer trial ; optimal ; two-stage design ; trinomial

Abstract

Phase II clinical trials in oncology are used to initially evaluate the therapeutic efficacy of a new treatment regimen. Simon's two stage desig based on total response (TR) rate is commonly used for such trials. Several authors have proposed alternative strategies to jointly consider either the response and toxicity or the responses and early progression. Because TR consists of both partial response (PR) and complete response (CR), and these two types of responses may have a different impact on patient survival, Lin and Chen (2000) proposed a flexible design that is based on a weighted average score of PR and CR rates to take into account the relative importance of a CR vs. PR. Panageas and colleagues (2002), on the other hand, used a trinomial model to develop a rejection region based on the joint frequencies of PRs and CRs . We reformat their hypotheses to assess the treatment based on TR and CR frequencies. A new two-stage optimum phase II trial design based on TR and CR is developed. We provide guides on searching the stopping and rejecting regions and determination of sample size.