Sunday, Aug 4: 4:25 PM - 4:45 PM
Invited Paper Session
Oregon Convention Center
Genetic diseases often result from errors in protein or enzyme production which are critical to the functioning of the central nervous system. The consequences of these disorders manifest early in life and have a broad impact, with symptoms include developmental delay, motor impairment, sleep disruption, and seizures. Fortunately, our improved understanding of these diseases' genetic origins has paved the way for promising, targeted treatments such as antisense oligonucleotides, AAV gene therapies, and enzyme replacements.
However, designing clinical trials for these diseases is challenging. They have many manifestations and it may be difficult to choose endpoints which are amenable to treatment when designing a clinical trial. Another issue is identifying treatment related growth, as these children which may demonstrate some natural improvements as they age. Finally, the rarity of these diseases means that clinical trials are necessarily small, often unblinded, and there is limited data available to assist in planning. This talk will share insights into designing clinical trials for neurodevelopmental disorders, highlighting techniques including disease progression modeling and multivariate analyses.