Dynamic treatment regimens in small n, sequential, multiple assignment, randomized trials: An application in focal segmental glomerulosclerosis.

ABSTRACT: Focal segmental glomerulosclerosis (FSGS) is a rare kidney disease with an annual incidence of 0.2-1.8 cases per 100,000 individuals. Most rare diseases like FSGS lack effective treatments, and it is difficult to implement clinical trials to study rare diseases because of the small sample sizes and difficulty in recruitment. A novel clinical trial design, a small sample, sequential, multiple assignment, randomized trial (snSMART) has been proposed to efficiently identify effective treatments for rare diseases. In this work, we review and expand the snSMART design applied to studying treatments for FSGS. The snSMART is a multistage trial that randomizes participants to one of three active treatments in the first stage and then re-randomizes those who do not respond to the initial treatment

SUBMITTER: Chao YC 

PROVIDER: S-EPMC8173713 | biostudies-literature | 2020 May

REPOSITORIES: biostudies-literature