Effects of continual oxytocin treatment on core social symptoms in children with autism with and without comorbid intellectual disability: A verification trial with long-term follow-up

Intranasal administration of the neuropeptide oxytocin (OT) is a therapeutic option targeting the impairments in autism spectrum disorder (ASD). While initial proof-of concept studies have shown improved functioning after multiple-dose OT treatment in children with ASD (<12 years), confirmatory studies are needed in larger cohorts and to study long-term effects. Moreover, most of the studies excluded autistic individuals with comorbid intellectual disability (ID), who make up 40% of people with ASD, and who often display more severe impairments. With the current project, we propose a large-scale randomized, double-blind, placebo-controlled trial testing the effects of multiple-dose OT treatment (four weeks of two daily intranasal administrations of 24 IU) in a confirmatory sample of 200 children with ASD (8-12 years) on core autism symptoms (social functioning, repetitive behaviors) and several secondary outcomes (e.g. attachment, stress, anxiety). This study will be the first to include children with and without a comorbid ID. It will also be the first to explore long-term effects of OT in these groups. Thus far, long-term retention of OT effects has only been reported in adult males with ASD. In summary, this confirmatory trial will provide important insights into the possibility of differential treatment effects depending on comorbid ID and other person-dependent factors (e.g. endogenous OT levels) as well as the long-term effects of OT treatment in children with ASD. These insights are essential for the translation and further development of OT-based therapies for ASD.

Keywords:Oxytocin, Autism spectrum disorder, Clinical trial, Intellectual disability, Neuropsychopharmacology

Disciplines:Biological psychiatry, Pharmacotherapy, Clinical trials