Statistical Inferences of Two-Stage Phase II Cancer Clinical Trials with Two Co-primary Endpoints
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Authors
Sun, Yiming
Date
Type
thesis
Language
eng
Keyword
Point estimate , P-value , Confidence region , phase two cancer clinical trials , likelihood ratio test
Alternative Title
Abstract
In cancer research, two-stage designs are usually used to assess the effect of a new
agent in phase II clinical trials. The optimal two-stage designs with two co-primary
endpoints have been proposed to assess the effects of new cancer treatments, such
as cytostatic or molecularly targeted agents (MTAs), based on both response rate
and early progression rate. Statistical inference procedures, such as, point estima
tion, p-value, and confidence region, for the true response rate and early progression
rate based on the data from the phase II trials conducted according to the optimal
two-stage designs would be very useful for further testing of the agents in phase III
trials but have not been addressed in the literature. In this thesis, I first provide a
review of the optimal two-stage design for phase II clinical trials with one endpoint
and statistical inference procedures developed for this design. Then I propose some
new statistical inference procedures for the optimal two-stage design of phase II clin
ical trials with two co-primary endpoints, which include naive maximum likelihood
estimate (MLE), bias-corrected estimates, and uniformly minimum variance unbiased
estimate (UMVUE) for the point estimation, naive p-value and likelihood ratio test
(LRT) based p-value for the hypothesis testing, and LRT based confidence region.
Simulation studies were performed to evaluate the performance of these procedures.
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Queen's University's Thesis/Dissertation Non-Exclusive License for Deposit to QSpace and Library and Archives Canada
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Intellectual Property Guidelines at Queen's University
Copying and Preserving Your Thesis
This publication is made available by the authority of the copyright owner solely for the purpose of private study and research and may not be copied or reproduced except as permitted by the copyright laws without written authority from the copyright owner.
ProQuest PhD and Master's Theses International Dissemination Agreement
Intellectual Property Guidelines at Queen's University
Copying and Preserving Your Thesis
This publication is made available by the authority of the copyright owner solely for the purpose of private study and research and may not be copied or reproduced except as permitted by the copyright laws without written authority from the copyright owner.