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 webinar, Dr. Pamela Ventola describes the critical factors to consider when selecting cognitive and behavioral endpoints for these trials, including perspectives from regulatory agencies. Dr. Ventola describes 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 differentiates between the measures so viewers will be better positioned to select outcome assessments based on the specific indication and needs of the trial.
Several case examples of current trials are shared, with a focus on differential outcome measures and rationale for their use.
- Dr. Pamela Ventola, Senior Science Director, Pediatrics and Rare Disease, Cogstate