Clinical Research Directory

Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism

The goal of this study is to conduct a randomized, placebo-controlled trial (RCT) of ganaxolone, a neuroactive steroid (NAS), in autistic children and adolescents aged 5 to 17 years old. Ganaxolone is approved and effective for treating seizures in children as young as 2 years old who have CDKL5 deficiency disorder (CDD), a neurogenetic condition associated with developmental delays, seizure disorder, hypotonia, visual impairments, and autistic features. The primary outcome of interest for this trial is irritability on the Aberrant Behavior Checklist (ABC) because it is a common symptom of emotion dysregulation in ASD that impacts quality of life, including mental health, independence, educational opportunities, and integration into the community. The secondary domains of interest for this trial are restricted and repetitive behaviors (RRB), specifically insistence on sameness (IS), a subdomain of RRB characterized by inflexibility and a strong preference for predictable routines and familiar environments. Secondary outcome measures include the IS subscale from the Dimensional Assessment of Repetitive Behaviors (DARB) and subscales of the Clinical Global Impressions Scale for irritability (CGI-IR) and IS (CGI-IS). For participants living within 150 miles of Stanford University, we require participants to attend site visits and attempt EEG and MRI procedures before and after the trial, though we are recruiting nationally and the study can be completed without site vists.

Intranasal Insulin for Autism Spectrum Disorder in Children and Young Adults Aged 4 to 21 Years

This observational study evaluates the real-world use of intranasal insulin in children and young adults with autism spectrum disorder (ASD) utilizing the ViaNase™ device developed by Kurve Therapeutics. Intranasal insulin represents an off label use of an FDA approved medication and is prescribed by participants' treating healthcare providers as part of routine clinical care. Insulin is a hormone involved in cerebral energy metabolism and may play a role in cognitive processes such as learning, memory, and behavior. Emerging research suggests that intranasal delivery using specialized delivery systems such as ViaNase™ may facilitate transport along olfactory and trigeminal pathways, potentially allowing insulin to reach central nervous system targets. This delivery approach has been associated in early studies with changes in social communication and functional outcomes in individuals with neurodevelopmental conditions. This study will follow approximately 12 participants between the ages of 4 and 21 years who are already receiving, or planning to receive, intranasal insulin as part of their standard clinical care using the ViaNase™ device. This is a non-interventional observational study; no treatment is assigned or provided by the study team. Participants will be monitored over an approximate 6-month period for changes in autism-related symptoms, including social interaction, communication, repetitive behaviors, and overall functional development. In addition, safety data will be collected, including tolerability and any reported adverse events. The primary objective of this study is to generate real-world evidence to better characterize the safety profile and potential functional effects of intranasal insulin delivered via ViaNase™ in individuals with ASD, and to inform the design of future controlled clinical investigations.