TY - JOUR
T1 - Randomized phase II trials
T2 - Time for a new era in clinical trial design
AU - Mandrekar, Sumithra J.
AU - Sargent, Daniel J.
N1 - Funding Information:
Supported in part by the National Cancer Institute Grants: Mayo Clinic Cancer Center (CA-15083) and the North Central Cancer Treatment Group (CA-25224).
PY - 2010/7
Y1 - 2010/7
N2 - The classic single-arm oncology phase II trial designs for evaluating an experimental regimen/agent are limited by multiple sources of bias arising from the inability to separate trial effects (such as patient selection, trial eligibility, imaging techniques and assessment schedule, and treatment locations) from treatment effect on clinical outcomes. Changes in patient population based on biologic subsetting, newer imaging technologies, the use of alternative end points, constrained resources, and the multitude of promising therapies for a given disease make randomized phase II designs, with a concurrent control arm where necessary, attractive. In this brief report, we discuss the salient features of the randomized designs for phase II trials, which when properly applied under the constraints of their underlying inference framework can assure optimal use of limited phase III financial and patient resources.
AB - The classic single-arm oncology phase II trial designs for evaluating an experimental regimen/agent are limited by multiple sources of bias arising from the inability to separate trial effects (such as patient selection, trial eligibility, imaging techniques and assessment schedule, and treatment locations) from treatment effect on clinical outcomes. Changes in patient population based on biologic subsetting, newer imaging technologies, the use of alternative end points, constrained resources, and the multitude of promising therapies for a given disease make randomized phase II designs, with a concurrent control arm where necessary, attractive. In this brief report, we discuss the salient features of the randomized designs for phase II trials, which when properly applied under the constraints of their underlying inference framework can assure optimal use of limited phase III financial and patient resources.
KW - False-negative
KW - False-positive
KW - Phase II
KW - Randomized
KW - Screening
KW - Selection
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U2 - 10.1097/JTO.0b013e3181e2eadf
DO - 10.1097/JTO.0b013e3181e2eadf
M3 - Article
C2 - 20581575
AN - SCOPUS:77954420205
SN - 1556-0864
VL - 5
SP - 932
EP - 934
JO - Journal of Thoracic Oncology
JF - Journal of Thoracic Oncology
IS - 7
ER -