One- and two-stage designs for stratified phase II clinical trials.
A global one-sample test for response rates for stratified phase II clinical trials is proposed. Such a test is analogous to that of a stratified log-rank test for time-to-event data. Both one- and two-stage tests are developed, and conditional and unconditional approaches are introduced in each case, where the conditional approach involves conditioning on the observed samples sizes within the strata. The methodology generates samples sizes and stopping boundaries that provide designs with the desired power and type I error probability. These methods are useful for designing stratified phase II clinical trials. An application to a Children's Oncology Group phase II clinical trial in relapsed neuroblastoma patients is presented.
London
Wendy B
WB
Children's Oncology Group (COG), Department of Statistics, University of Florida, Gainesville, FL 32601, USA. wendy@cog.ufl.edu
Chang
Myron N
MN
D000970:Antineoplastic Agents / Q000627:therapeutic use; D000971:Antineoplastic Combined Chemotherapy Protocols; D002648:Child; D002675:Child, Preschool; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003520:Cyclophosphamide / Q000627:therapeutic use; D003627:Data Interpretation, Statistical; D006801:Humans; D007223:Infant; D009364:Neoplasm Recurrence, Local / Q000517:prevention & control; D009447:Neuroblastoma / Q000188:drug therapy; D012107:Research Design; D019772:Topotecan / Q000627:therapeutic use
D000970:Antineoplastic Agents; D019772:Topotecan; D003520:Cyclophosphamide