The Bayesian basket design for genomic variant-driven phase II trials

Richard Simon; Susan Geyer; Jyothi Subramanian; Sameek Roychowdhury

doi:10.1053/j.seminoncol.2016.01.002

The Bayesian basket design for genomic variant-driven phase II trials

Semin Oncol. 2016 Feb;43(1):13-18. doi: 10.1053/j.seminoncol.2016.01.002. Epub 2016 Jan 18.

Authors

Richard Simon¹, Susan Geyer², Jyothi Subramanian³, Sameek Roychowdhury⁴

Affiliations

¹ Division of Cancer Treatment & Diagnosis, National Cancer Institute, 9609 Medical Center Dr, Rockville, MD 20892-9735, USA. Electronic address: rsimon@nih.gov.
² Department of Pediatrics, University of South Florida, Tampa, FL, USA.
³ Emmes Corporation, Rockville, MD, USA.
⁴ Department of Internal Medicine, The James Cancer Center, Ohio State University, Columbus, OH, USA.

PMID: 26970120
DOI: 10.1053/j.seminoncol.2016.01.002

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.

Keywords: Actionable mutations; Basket clinical trials; Genomic clinical trials.

MeSH terms

Bayes Theorem*
Biomedical Research / methods*
Clinical Trials, Phase II as Topic / methods*
Computer Simulation
Genetic Variation*
Genomics
Humans
Internet
Neoplasms / drug therapy*
Neoplasms / genetics
Neoplasms / pathology*
Research Design
Software