Objectives: We conducted a Maximum Tolerated Dose (MTD) pilot study of intranasal oxytocin in children and adolescents 10-18 years of age and explored effects on safety, socialperception cognition and function and anxiety.
Methods: 25 children and youth with ASD, with diagnosis confirmed by expert clinician using ADOS, ADI-R and clinical information were enrolled into the study. They were exposed to active medication for 12 weeks and followed for 24 weeks. MTD design involved assignment in the following dose range: 0.2, 0.26. 0.33. 0.4 IU/kg/dose, in 3 patient increments. Safety was measured using the SMURF, vital signs, safety bloodworkand EKG. Potential efficacy was measured by exploring effects on social cognition / perception (LETS FACE IT, Irony and Empathy), the social responsiveness scale, and anxiety (CASI).
Results: Statistically significant improvements were noted within group in aspects of social conition / perception [face recognition (p =0.002 ) as measured by the LFI, and theory of mind (p = 0.06) as measured by the Irony and Empathy task]. Improvements were noted in overall symptoms as measured by the SRS (p= 0.003 ), and anxiety as measured by the CASI (p=0.006 ). Effects were maintain after 3 months of oxytocin discontinuation. Side effects were mild. No serious adverse events were reported at any of the dose categories
Conclusions: Preliminary data supports that manipulation of oxytocin system over a 12 week period is safe, and may produce therapeutic effects in terms of social perception / cognition and also leads to anxiolytic effects. Larger studies are required to further examine such effects