title: An adaptive group sequential phase II design to compare treatments for survival endpoints in rare patient entities.
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.
authors.affiliation: Department of Biostatistics, German Cancer Research Center, Heidelberg, Germany. c.wunder@dkfz.de
authors.forename: Christina
authors.initials: C
authors.lastname: Wunder
authors.forename: Annette
authors.initials: A
authors.lastname: Kopp-Schneider
authors.forename: Lutz
authors.initials: L
authors.lastname: Edler
mesh_terms: D000465:Algorithms; D000970:Antineoplastic Agents / Q000627:therapeutic use; D002292:Carcinoma, Renal Cell / Q000188:drug therapy / Q000401:mortality; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data*; D003198:Computer Simulation; D003627:Data Interpretation, Statistical; D006801:Humans; D007211:Indoles / Q000627:therapeutic use; D007680:Kidney Neoplasms / Q000188:drug therapy / Q000401:mortality; D016016:Proportional Hazards Models; D011758:Pyrroles / Q000627:therapeutic use; D035583:Rare Diseases / Q000188:drug therapy* / Q000401:mortality*; D012107:Research Design / Q000706:statistics & numerical data*; D018401:Sample Size; D020123:Sirolimus / Q000031:analogs & derivatives / Q000627:therapeutic use; D000077210:Sunitinib; D013534:Survival
chemical_list: D000970:Antineoplastic Agents; D007211:Indoles; D011758:Pyrroles; C401859:temsirolimus; D000077210:Sunitinib; D020123:Sirolimus