title: The Bayesian basket design for genomic variant-driven phase II trials.
abstract: Basket clinical trials are a new category of early clinical trials in which a treatment is evaluated in a population of patients with tumors of various histologic types and primary sites selected for containing specific genomic abnormalities. The objective of such studies is generally to discover histologic types in which the treatment is active. Basket trials are early discovery trials whose results should be confirmed in expanded histology specific cohorts. In this report, we develop a design for planning, monitoring, and analyzing basket trials. A website for using the new design is available at https://brbnci.shinyapps.io/BasketTrials/ and the software is available at GitHub in the "Basket Trials" repository of account brbnci.
authors.affiliation: Division of Cancer Treatment & Diagnosis, National Cancer Institute, 9609 Medical Center Dr, Rockville, MD 20892-9735, USA. Electronic address: rsimon@nih.gov.
authors.forename: Richard
authors.initials: R
authors.lastname: Simon
authors.affiliation: Department of Pediatrics, University of South Florida, Tampa, FL, USA.
authors.forename: Susan
authors.initials: S
authors.lastname: Geyer
authors.affiliation: Emmes Corporation, Rockville, MD, USA.
authors.forename: Jyothi
authors.initials: J
authors.lastname: Subramanian
authors.affiliation: Department of Internal Medicine, The James Cancer Center, Ohio State University, Columbus, OH, USA.
authors.forename: Sameek
authors.initials: S
authors.lastname: Roychowdhury
mesh_terms: D001499:Bayes Theorem; D035843:Biomedical Research / Q000379:methods*; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003198:Computer Simulation; D014644:Genetic Variation; D023281:Genomics; D006801:Humans; D020407:Internet; D009369:Neoplasms / Q000188:drug therapy* / Q000235:genetics / Q000473:pathology*; D012107:Research Design; D012984:Software
keywords: Actionable mutations; Basket clinical trials; Genomic clinical trials