Phase 3 Clinical Trial for Sickle Cell Disease: Betty Pace, MD, received a $178K, 2-year award from Mast Therapeutics, Inc. for “MST-188-01: Evaluation of Purified Poloxamer 188 in Children in Crisis (EPIC): A Phase 3 Randomized, Double blind, Placebo-Controlled Multicenter Trial of MST-188.”
The EPIC study will test an experimental compound called MST-188, which is designed to restore microvascular function that is compromised in a number of diseases, including sickle cell disease (SCD). Specifically, the EPIC study will evaluate whether MST-188 can reduce 1) the duration of vaso-occlusive crisis (VOC) in subjects with SCD, and 2) the frequency of re-hospitalization due to a recurrence of VOC. EPIC will also investigate the development of acute chest syndrome during VOC in subjects who receive MST-188. The study officially opened at GRU on November 1, 2013 and will enroll nearly 400 individuals aged 8-17 with sickle cell anemia from participating academic centers across the United States through December 2015.