An Adaptive Group Sequential Phase II Design to Compare Treatments for Survival Endpoints in Rare Patient Entities |
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Authors: | Christina Wunder Annette Kopp-Schneider Lutz Edler |
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Affiliation: | 1. Department of Biostatistics , German Cancer Research Center , Heidelberg , Germany c.wunder@dkfz.de;3. Department of Biostatistics , German Cancer Research Center , Heidelberg , Germany |
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Abstract: | For rare diseases, standard treatments are often not available and essential study parameters are difficult or impossible to obtain. Therefore, designs of clinical trials for these diseases are often based on little information. Adaptive designs allow such trials to be started and to gain information during the study. Motivated by a trial for a rare subtype of renal-cell carcinoma, we present a two-stage adaptive design for right-censored time-to-event data and a two-sided test. After the first stage, one can stop for futility or continue with reestimated sample size. The properties of such designs are analyzed by simulation studies. |
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Keywords: | Adaptive group sequential design Conditional rejection probability principle Rare tumors Sample size recalculation Survival Two-sided hypothesis |
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