This $650,000 Project Grant awarded by the Food and Drug Administration (FDA) Research program (CFDA 93.103) supports a study titled "PHASE 2A/2B STUDY EMAPALUMAB: A WINDOW OF OPPORTUNITY IN PEDIATRIC APLASTIC ANEMIA". The primary objective is to evaluate the effect of an early upfront emapalumab prophase on response-adapted stratification of newly diagnosed patients with aplastic anemia. The study also aims to prospectively validate the presence or absence of specific clonal hematopoiesis as predictive biomarkers for response to immune suppression therapy.
Through sub-awards, researchers at Virginia Commonwealth University, Children's Hospital of Philadelphia, and Cincinnati Children's Hospital Medical Center will provide services such as patient enrollment, sample/data collection, study protocol oversight, data management, and data analysis. This multi-institutional collaboration seeks to develop an algorithm to identify pediatric aplastic anemia patients most likely to be cured by immune suppression therapy. The award period runs from September 2024 through June 2028.
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