Selecting Cognitive & Behavioral Endpoint Measures for Trials in Rare Epilepsy and MPS Disorders
Clinical trials in patients with rare forms of epilepsy and mucopolysaccharidoses (MPS) disorders present considerable challenges, as patients with these conditions are heterogeneous and many are quite impaired.
In this free webinar, Dr. Pamela Ventola will describe the critical factors to consider when selecting cognitive and behavioral endpoints for these trials, including perspectives from regulatory agencies.
Several case examples of current trials will be shared, with a focus on differential outcome measures and rationale for their use.
Webinar Type: Recorded
Pamela Ventola, Senior Science Director, Pediatrics and Rare Disease, Cogstate
Dr. Pamela Ventola leads Cogstate’s pediatric and rare diseases portfolio, providing Cogstate customers with strategic oversight and expert guidance throughout all stages of their study planning and execution — from endpoint selection, rater training and strategic monitoring to final statistical analysis. Additionally, she serves as an assistant professor at the Yale Child Study Center.
As a licensed clinical psychologist, Dr. Ventola’s primary clinical expertise is in pediatric neuropsychology and specifically developmental and genetic disorders. Her research interests involve developmental disabilities and among her recent research is a study on brain-based mechanisms of treatment response in autism spectrum disorder (ASD). She is also utilizing eye-tracking paradigms as a novel outcome of clinical trials in ASD and is involved in studies on novel pharmacological therapeutics in individuals with ASD.
Dr. Ventola received her Doctor of Philosophy in Clinical Psychology from the University of Connecticut and completed her clinical training and Postdoctoral Fellowship at the Yale University School of Medicine, Child Study Center. She serves on the editorial review board of multiple academic journals. Specifically, she serves as a reviewer for the Journal of Autism and Developmental Disorders, Journal of the American Academy of Child and Adolescent Psychiatry, Clinical Psychology Review and the Autism International Journal of Research and Practice. Dr. Ventola has authored numerous peer-reviewed manuscripts, book chapters and scientific presentations.