Increasing clinical trial readiness in Dravet syndrome- Creation and pilot of Dravet-specific clinical outcome measures

Abbott grant

Megan Abbott, MD – University of Colorado Denver
Increasing clinical trial readiness in Dravet syndrome- Creation and pilot of Dravet-specific clinical outcome measures
Clinician-Researcher Award – $75,000, 1 year

Grant Summary:

Dravet syndrome (DS) is the most common genetic cause of Developmental and Epileptic Encephalopathy (DEE), yet there are no FDA approved disease modifying treatments. Current precision therapeutic clinical trials for DS utilize seizure frequency as the primary outcome measure. Utilizing seizure frequency alone to assess outcome does not capture the full array of challenges associated with DS. This project will focus on refining a set of clinician and caregiver-reported outcome measures previously created for CDKL5-deficiency disorder and piloting them in patients with DS. The overarching objective is to test the hypothesis that valid and feasible outcome measures can be designed specifically for DS that represent the full range of the phenotype beyond seizure frequency. The creation of the Dravet Syndrome clinical severity assessment-clinician and caregiver (DS-CSA) will be a crucial step towards disease modifying clinical trial readiness in DS. 

About the Investigator:

Megan Abbott, MD is a pediatric neurologist and current epilepsy fellow at Children’s Hospital Colorado with a clinical and research interest in neurogenetic conditions within epilepsy. She earned her bachelor’s degree from Texas A&M University and her medical degree from Baylor College of Medicine before coming to Colorado for residency. Her research focus is specifically looking at outcome measures that can be generalized across many DEEs including SCN1A. 

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