Document Detail

Optimal two-stage designs allowing flexibility in number of subjects for phase II clinical trials.
MedLine Citation:
PMID:  20183436     Owner:  NLM     Status:  MEDLINE    
Phase II clinical trials are conducted to test whether a drug has a minimum desired effect and to assess whether further development of the drug is warranted. They are often designed as one-arm trials with response rate as the primary endpoint, and a two-stage design is often used to ensure early termination of the trial for futility. To control the type I error rate and guarantee the specified power of the study, planned sample sizes for both stages must be rigidly followed, but a literature review suggests that actual sample size often differs from that planned. We propose to extend simple two-stage designs to allow more flexible sampling plans in both stages. Our designs are preferable to similar extensions proposed to control type I and II error probabilities. Additionally, our assumptions regarding distribution of the actual sample size at the end of stage 1 are more lenient. A list of optimal designs for typical error rates and the selected null and alternative response rates is presented.
Nobuyuki Masaki; Tatsuki Koyama; Isao Yoshimura; Chikuma Hamada
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Publication Detail:
Type:  Journal Article; Research Support, Non-U.S. Gov't    
Journal Detail:
Title:  Journal of biopharmaceutical statistics     Volume:  19     ISSN:  1520-5711     ISO Abbreviation:  J Biopharm Stat     Publication Date:  2009 Jul 
Date Detail:
Created Date:  2010-02-25     Completed Date:  2010-05-20     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  9200436     Medline TA:  J Biopharm Stat     Country:  England    
Other Details:
Languages:  eng     Pagination:  721-31     Citation Subset:  IM    
Department of Biostatistics, Bristol-Myers K.K, Tokyo, Japan.
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MeSH Terms
Clinical Trials, Phase II as Topic / statistics & numerical data*
Computer Simulation
Data Interpretation, Statistical
Models, Statistical*
Sample Size*
Treatment Outcome

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