Selecting Cognitive & Behavioral Endpoint Measures for Trials in Rare Epilepsy and MPS Disorders

Life Sciences, Clinical Trials,
  • Wednesday, November 20, 2019

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. Further, comorbid sensory and motor deficits are common as are disruptive behaviors. Additionally, these diseases are often regressive, making the assessment of cognitive and behavior even more complex.

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. Dr. Ventola will describe common measures such as developmental assessments (e.g., Bayley-4 and Mullen), the Wechsler Scales, Kaufman Assessment Battery for Children-Second Edition and the Vineland Adaptive Behavior Scales-Third Edition. She will differentiate between the measures so attendees will be better positioned to select outcome assessments based on the specific indication and needs of the trial.

Several case examples of current trials will be shared, with a focus on differential outcome measures and rationale for their use.


Pamela Ventola, Cogstate

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 DisordersJournal of the American Academy of Child and Adolescent PsychiatryClinical Psychology Review and the Autism International Journal of Research and Practice. Dr. Ventola has authored numerous peer-reviewed manuscripts, book chapters and scientific presentations.

Message Presenter

Who Should Attend?

Executives, directors, scientists and managers from pharma, biotech and CROs responsible for the clinical development of rare disease therapeutics, including:

  • Research and Development
  • Clinical Operations
  • Clinical Science
  • Outcomes Research
  • Outsourcing and Procurement
  • Project Management
  • Clinical Trial Planning and Optimization
  • Medical Affairs

What You Will Learn

Participants will learn about:

  • Critical factors in selecting cognitive/behavioral endpoints for trials in rare epilepsy and MPS disorders
  • Key features of developmental assessments
  • Differential aspects of common cognitive assessments
  • The range of cognitive and behavioral assessments used in trials involving rare epilepsy and MPS disorders

Xtalks Partner

Cogstate Ltd

Cogstate Ltd (ASX:CGS) is a leading science and technology solutions provider dedicated to optimizing the measurement of cognition in clinical trials, academic research and healthcare. Cogstate provides enabling technologies and professional services for higher quality neuropsychological assessments and is a pioneer in commercializing rapid, reliable and highly sensitive computerized cognitive tests. Cogstate customers include the world’s leading biopharmaceutical companies; elite sporting organizations and military; physicians and patients; renowned academic institutions and public-private partnerships. For more information, please visit

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