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

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 a ssignment, randomized trial (snSMART) has been proposed to efficiently identify effective treatments for rare diseases.

Source: Contemporary Clinical Trials - March 17, 2020 Category: Radiology Authors: Yan-Cheng Chao, Howard Trachtman, Debbie S. Gipson, Cathie Spino, Thomas M. Braun, Kelley M. Kidwell Source Type: research