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

Official title

Observational Study of Intranasal Insulin Delivery in Autism Spectrum Disorder Ages 4 to 21 Years (INS-AUT)

Overview

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.

Detailed description

This is a prospective, single arm observational cohort study designed to evaluate the real world use of intranasal insulin in children and young adults diagnosed with autism spectrum disorder (ASD).

Intranasal insulin represents an off label route of administration of an FDA approved medication. In this study, insulin is prescribed and managed independently by participants' treating healthcare providers as part of routine clinical care. The study does not assign, provide, or direct treatment, dosing, or route of administration. All clinical decisions are made outside of the study.

The purpose of this study is to collect real world evidence (RWE) on the safety, tolerability, and potential clinical associations of intranasal insulin use in individuals with ASD. Insulin signaling in the central nervous system has been associated with neurodevelopment, synaptic plasticity, and cognitive function. Intranasal delivery may facilitate transport to the brain via olfactory and trigeminal pathways, and early studies suggest possible associations with changes in social communication and behavioral outcomes.

Approximately 12 participants ages 4 to 21 years who are receiving or planning to receive intranasal insulin as part of their clinical care will be enrolled and followed prospectively for approximately 6 months. Data will be collected at baseline, during ongoing use, and at follow-up intervals.

Outcome measures will include caregiver reported assessments of social communication, behavior, adaptive functioning, and overall developmental progress. Safety monitoring will include documentation of adverse events, tolerability, and any clinically relevant observations reported by caregivers or healthcare providers.

This study is observational in nature and does not involve the prospective assignment of any intervention. The study team does not provide insulin or any delivery device. Participation in this study does not influence clinical care.

The goal of this study is to generate real world data to better understand the safety profile and potential clinical associations of intranasal insulin use in ASD and to inform future clinical research.

Primary outcome measures

• Change in Social Responsiveness Scale, Second Edition (SRS-2) Total Score [Time frame: Baseline to 6 months]

Secondary outcome measures (4)

• Caregiver Global Impression of Change (CGI-C) [Time frame: Up to 6 months]
• Change in Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) [Time frame: Baseline to 6 months]
• Incidence of Adverse Events and Tolerability [Time frame: Baseline to 6 months]
• Change in Repetitive Behaviors and Social Communication (Caregiver Reported) [Time frame: Baseline to 6 months]

Eligibility criteria

Inclusion criteria

• Age 4 to 21 years
• Clinical diagnosis of autism spectrum disorder (ASD) documented by a qualified healthcare provider
• Currently using or planning to use intranasal insulin as prescribed by a treating healthcare provider as part of routine clinical care
• Stable medical and behavioral therapy regimen for at least 4 weeks prior to baseline (if applicable)
• Caregiver or parent/guardian able to provide informed consent and complete study related assessments
• Willingness to participate in observational data collection over approximately 6 months

Exclusion criteria

• Known diagnosis of diabetes mellitus requiring insulin for glycemic control
• History of recurrent hypoglycemia or clinically significant risk of hypoglycemia
• Known hypersensitivity or adverse reaction to insulin
• Significant uncontrolled medical conditions that, in the opinion of the investigator, would interfere with participation or data interpretation
• Use of investigational drugs or participation in an interventional clinical trial within 30 days prior to enrollment
• Any condition that would prevent reliable participation in study assessments, including inability of caregiver to complete questionnaires

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

United States · 1 center

• Healing Hope International — Spring

Publications

• Rose D, Moreno RJ, Osman H, Rowland ME, Silverman J, Ciernia A, Ashwood P. Sex specific effects of adoptive Tregs transfer on the brain and periphery in maternal immune activation offspring rescuing immune dysregulation. J Neuroinflammation. 2026 Mar 12;23(1):133. doi: 10.1186/s12974-026-03739-w. PMID 41821031
• Zwanenburg RJ, Bocca G, Ruiter SA, Dillingh JH, Flapper BC, van den Heuvel ER, van Ravenswaaij-Arts CM. Is there an effect of intranasal insulin on development and behaviour in Phelan-McDermid syndrome? A randomized, double-blind, placebo-controlled trial. Eur J Hum Genet. 2016 Dec;24(12):1696-1701. doi: 10.1038/ejhg.2016.109. Epub 2016 Aug 31. PMID 27577546
• Gao B, Wang S, Wang Y, Shen D, Xue S, Chen C, Cui H, Song C. Low diversity, activity, and density of transposable elements in five avian genomes. Funct Integr Genomics. 2017 Jul;17(4):427-439. doi: 10.1007/s10142-017-0545-0. Epub 2017 Feb 11. PMID 28190211
• Schmidt H, Kern W, Giese R, Hallschmid M, Enders A. Intranasal insulin to improve developmental delay in children with 22q13 deletion syndrome: an exploratory clinical trial. J Med Genet. 2009 Apr;46(4):217-22. doi: 10.1136/jmg.2008.062141. Epub 2008 Oct 23. PMID 18948358

Identifiers

NCT: NCT07521371 · HHI-INS-AUT-OBS-2026-001

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗