Designs for randomized phase II clinical trials with two treatment arms

The most common primary statistical end point of a phase II clinical trial is the categorization of a patient as either a ‘responder’ or ‘nonresponder’. The primary objective of typical randomized phase II anticancer clinical trials is to evaluate experimental treatments that potentially will increa...

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Bibliographic Details
Published in:Statistics in medicine 2013-11, Vol.32 (25), p.4367-4379
Main Authors: Hou, Wei, Chang, Myron N., Jung, Sin-Ho, Li, Yang
Format: Article
Language:English
Subjects:
Antibodies, Monoclonal, Murine-Derived - therapeutic use
Antineoplastic Agents - therapeutic use
average sample size
Bias
Clinical trials
Clinical Trials, Phase II as Topic - methods
Endpoint Determination
Error analysis
Humans
Lymphoma
Lymphoma, Non-Hodgkin - drug therapy
minimizing the maximum sample size
power
Prescription drugs
Randomized Controlled Trials as Topic - methods
Recurrence
Research Design
Rituximab
single-stage design
Thalidomide - analogs & derivatives
Thalidomide - therapeutic use
threshold
Treatment Outcome
two-stage design
type I error rate
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Summary:The most common primary statistical end point of a phase II clinical trial is the categorization of a patient as either a ‘responder’ or ‘nonresponder’. The primary objective of typical randomized phase II anticancer clinical trials is to evaluate experimental treatments that potentially will increase response rate over a historical baseline and select one to consider for further study. We propose single‐stage and two‐stage designs for randomized phase II clinical trials, precisely defining various type I error rates and powers to achieve this objective. We develop a program to compute these error rates and powers exactly, and we provide many design examples to satisfy pre‐fixed requirements on error rates and powers. Finally, we apply our method to a randomized phase II trial in patients with relapsed non‐Hodgkin's disease. Copyright © 2013 John Wiley & Sons, Ltd.
ISSN:0277-6715
1097-0258
DOI:10.1002/sim.5829