A Sequential Multiple Assignment Randomized Phase 2 Trial for Rare Diseases
*Roy Tamura, University of South Florida Keywords: Clinical research in rare diseases is difficult for a number of reasons, including limited number of patients, and patient attitudes toward active and placebo therapy. Oftentimes, a number of available drugs are used for a rare disease with limited information about the efficacy of any of the drugs. In such situations, it is logical to first design a trial which would determine if any of the drugs shows promising efficacy. In cooperation with the Vasculitis Clinical Research Consortium under the NIH, we have recently developed a sequential multiple assignment randomized trial (SMART) for rare vasculitis diseases. The goal of the design is to determine the best out of a number of potential drugs and to compare the chosen drug to the best of the remaining drugs. In this talk, I will present the design, analysis, and operating characteristics of the SMART for three drugs when the number of subjects is limited.
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Key Dates
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June 3, 2014 - September 7, 2015
Online Registration -
June 3, 2015 - August 15, 2015
Housing -
July 31 - August 17, 2015
Invited Abstract Editing -
August 10, 2015
Short Course materials due from Instructors -
August 26, 2015
Advanced Registration Deadline -
September 7, 2015
Cancellation Deadline -
September 16 - 18, 2015
Marriott Wardman Park, Washington, DC