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Two-stage design for phase II cancer clinical trials with multiple endpoints

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TitleInfo
Title
Two-stage design for phase II cancer clinical trials with multiple endpoints
Name (type = personal)
NamePart (type = family)
Gu
NamePart (type = given)
Hui
NamePart (type = date)
1984-
DisplayForm
Hui Gu
Role
RoleTerm (authority = RULIB)
author
Name (type = personal)
NamePart (type = family)
LIN
NamePart (type = given)
YONG
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YONG LIN
Affiliation
Advisory Committee
Role
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chair
Name (type = personal)
NamePart (type = family)
Shih
NamePart (type = given)
Weichung Joe
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Weichung Joe Shih
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Advisory Committee
Role
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internal member
Name (type = personal)
NamePart (type = family)
Wang
NamePart (type = given)
Yaqun
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Yaqun Wang
Affiliation
Advisory Committee
Role
RoleTerm (authority = RULIB)
internal member
Name (type = personal)
NamePart (type = family)
Liu
NamePart (type = given)
Kejian
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Kejian Liu
Affiliation
Advisory Committee
Role
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outside member
Name (type = corporate)
NamePart
Rutgers University
Role
RoleTerm (authority = RULIB)
degree grantor
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NamePart
School of Graduate Studies
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school
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Text
Genre (authority = marcgt)
theses
OriginInfo
DateCreated (qualifier = exact)
2017
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2017-10
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2017
Place
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xx
Language
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eng
Abstract (type = abstract)
The main purpose of a single-arm phase II cancer trial of a new regimen is to determine whether it has sufficient anti-tumor activity against a specific type of tumor to warrant its further clinical development. Such a research question can be answered under the frame- work of hypothesis testing. With the advent of targeted therapies that prolong disease stabilization, cancer patients typically experience stable disease (SD) rather than tumor shrinkage. It has been shown that patients with SD also achieve clinical benefits. There- fore, when evaluating the anti-tumor activity of a new treatment, clinicians are interested not only in overall response rate (complete or partial response(s)), but also in other types of measurements indicating clinical benefit. Taking two primary efficacy endpoints as an example, if the new treatment can improve on either endpoint(s), it may be promising for further evaluation. Therefore, "OR" logical relationship between the two primary efficacy endpoints is used when specifying the alternative hypothesis. In phase II cancer clinical trials, two-stage designs rather than single-stage ones are widely used for its possibility of early termination for futility to protect cancer patients. Motivated by two real cancer clinical trials, we propose a single-arm two-stage phase II cancer clinical trial design with two dichotomous alternative primary efficacy endpoints. Because of unknown correlation between two endpoints at the design stage, minimax rule is used to determine the optimal design, which minimizes the maximum of the expected sample size among all possible correlations, subject to the type I and II error constraints. Optimal designs for a variety of design parameters as well as the corresponding operating characteristics are provided. In addition, the statistical inferences of the design are studied. The MLE point estimators as well as confidence regions for the true event rates for the two efficacy endpoints are derived. Three types of confidence regions are obtained by inverting likelihood based test statistics: Wald, Score, and Likelihood ratio statistics. Among the three, the likelihood ratio-type confidence region performs the best in terms of good coverage probability and comparable expected area, and thus is recommended for this two-endpoint two-stage design.
Subject (authority = RUETD)
Topic
Public Health
Subject (authority = ETD-LCSH)
Topic
Clinical trials
RelatedItem (type = host)
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Title
Rutgers University Electronic Theses and Dissertations
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ETD
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ETD_8464
PhysicalDescription
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electronic resource
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application/pdf
InternetMediaType
text/xml
Extent
1 online resource (x, 110 p. : ill.)
Note (type = degree)
Ph.D.
Note (type = bibliography)
Includes bibliographical references
Note (type = statement of responsibility)
by Hui Gu
RelatedItem (type = host)
TitleInfo
Title
School of Graduate Studies Electronic Theses and Dissertations
Identifier (type = local)
rucore10001600001
Location
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NjNbRU
Identifier (type = doi)
doi:10.7282/T31R6TQM
Genre (authority = ExL-Esploro)
ETD doctoral
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Rights

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The author owns the copyright to this work.
RightsHolder (type = personal)
Name
FamilyName
Gu
GivenName
Hui
Role
Copyright Holder
RightsEvent
Type
Permission or license
DateTime (encoding = w3cdtf); (qualifier = exact); (point = start)
2017-09-30 01:09:31
AssociatedEntity
Name
Hui Gu
Role
Copyright holder
Affiliation
Rutgers University. School of Graduate Studies
AssociatedObject
Type
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Name
Author Agreement License
Detail
I hereby grant to the Rutgers University Libraries and to my school the non-exclusive right to archive, reproduce and distribute my thesis or dissertation, in whole or in part, and/or my abstract, in whole or in part, in and from an electronic format, subject to the release date subsequently stipulated in this submittal form and approved by my school. I represent and stipulate that the thesis or dissertation and its abstract are my original work, that they do not infringe or violate any rights of others, and that I make these grants as the sole owner of the rights to my thesis or dissertation and its abstract. I represent that I have obtained written permissions, when necessary, from the owner(s) of each third party copyrighted matter to be included in my thesis or dissertation and will supply copies of such upon request by my school. I acknowledge that RU ETD and my school will not distribute my thesis or dissertation or its abstract if, in their reasonable judgment, they believe all such rights have not been secured. I acknowledge that I retain ownership rights to the copyright of my work. I also retain the right to use all or part of this thesis or dissertation in future works, such as articles or books.
RightsEvent
DateTime (encoding = w3cdtf); (qualifier = exact); (point = start)
2017-10-31
DateTime (encoding = w3cdtf); (qualifier = exact); (point = end)
2018-10-31
Type
Embargo
Detail
Access to this PDF has been restricted at the author's request. It will be publicly available after October 31st, 2018.
Copyright
Status
Copyright protected
Availability
Status
Open
Reason
Permission or license
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