A small n sequential multiple assignment randomized trial design for use in rare disease research

Abstract

<div class="section"> <a class="named-anchor" id="S1"> <!-- named anchor --> </a> <h5 class="section-title" id="d8572329e204">Background</h5> <p id="P3">Clinical trials in rare diseases are difficult to conduct due to the limited number of patients available with each disorder. We developed a Phase 2 trial which is a small n sequential multiple assignment randomized trial (snSMART) design to test several treatments for a rare disease for which no standard therapy exists. </p> </div><div class="section"> <a class="named-anchor" id="S2"> <!-- named anchor --> </a> <h5 class="section-title" id="d8572329e209">Purpose</h5> <p id="P4">This paper illustrate the design, sample size estimation and operating characteristics of an snSMART. </p> </div><div class="section"> <a class="named-anchor" id="S3"> <!-- named anchor --> </a> <h5 class="section-title" id="d8572329e214">Methods</h5> <p id="P5">We investigate the performance of a class of weighted Z statistics via computer simulations.</p> </div><div class="section"> <a class="named-anchor" id="S4"> <!-- named anchor --> </a> <h5 class="section-title" id="d8572329e219">Results</h5> <p id="P6">We demonstrate the increase in power over traditional single stage designs, and indicate how the power changes as a function of the weight given to each stage. </p> </div><div class="section"> <a class="named-anchor" id="S5"> <!-- named anchor --> </a> <h5 class="section-title" id="d8572329e224">Conclusion</h5> <p id="P7">The snSMART design is promising in a rare diseases setting where several alternative treatments are under consideration and small sample sizes are necessary. </p> </div>