The phase II/III transition: Toward the proof of efficacy in cancer clinical trials Journal Article
| Authors: | Fazzari, M.; Heller, G.; Scher, H. I. |
| Article Title: | The phase II/III transition: Toward the proof of efficacy in cancer clinical trials |
| Abstract: | Few phase III investigations show a benefit for an experimental treatment when compared to a standard therapy or placebo. This illustrates the need for more reliable estimates of treatment effects from the phase II investigations used to design the more definitive phase III trials. In this manuscript, we examine four aspects of phase II clinical trial designs: (1) selecting endpoints; (2) defining the patient population for evaluation; (3) determining a level of activity that would justify a phase III trial; and (4) estimating sample sizes. In each area, problems with the conventional approaches are discussed and alternatives for the successful transition of phase II results to a phase III setting are suggested. An application of the design for patients with androgen-independent prostate cancer is illustrated. Control Clin Trials 2000;21:360-368 Copyright (C) 2000 Elsevier Science Inc. |
| Keywords: | controlled study; survival analysis; major clinical study; clinical trial; placebo; drug efficacy; patient selection; antineoplastic agents; research design; methodology; controlled clinical trial; phase 2 clinical trial; clinical protocol; prostate cancer; prostatic neoplasms; survival time; statistical analysis; data interpretation, statistical; population; mitomycin c; short survey; phase 3 clinical trial; phase ii; sample size; sample; surrogate endpoint; patient population; clinical trials, phase ii; clinical trials, phase iii; humans; human; male; historical data |
| Journal Title: | Controlled Clinical Trials |
| Volume: | 21 |
| Issue: | 4 |
| ISSN: | 0197-2456 |
| Publisher: | Elsevier Science, Inc. |
| Date Published: | 2000-08-01 |
| Start Page: | 360 |
| End Page: | 368 |
| Language: | English |
| DOI: | 10.1016/s0197-2456(00)00056-8 |
| PUBMED: | 10913810 |
| PROVIDER: | scopus |
| DOI/URL: | |
| Notes: | Export Date: 18 November 2015 -- Source: Scopus |
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