Intranasal Oxytocin Treatment for Social Deficits in Children With Autism
Condition: Autism
MeSH terms: Autistic Disorder
Study type: Interventional
Phase: Phase 2

Summary: 
      Autism is a pervasive developmental disorder characterized by core deficits in social
      behavior and communication, and the presence of repetitive or stereotyped behaviors. It is
      one of three recognized disorders in the autism spectrum which affects an estimated 1 in 88
      children in the United States. At present, pharmacotherapies target only associated features
      of autism, with no effective drug treatments for the social impairments. Several lines of
      evidence now suggest that the neuropeptide oxytocin (OT) may be an effective treatment for
      the core social deficits in autism. Here we will test the effects of twice daily intranasal
      OT (24 IU) over a 4-week period for enhancing social deficits in male and female children
      aged 6-12 years with autism. This research has high potential to lead to the development of
      more effective treatments and earlier interventions for children with autism.
    

Detailed description: 
      In recent years, the neuropeptide oxytocin (OT) has been implicated in a wide range of social
      behaviors including attachment bonds, emotion recognition, eye gaze to social cues, and
      memory for social information. Social impairments represent one of the most intractable
      features of autism, and evidence now suggests that OT biology is dysregulated in individuals
      with this disorder. The central aim of the research outlined here is to test whether OT
      administration to children with autism increases their quality and quantity of social
      interactions and enhances their ability to process emotional and social information. Findings
      from initial single-dose OT administration studies in teenaged and adult males with autism
      have shown improvement in some aspects of social functioning, but replication and extension
      to well-controlled treatment trials with younger male and female subjects is necessary to
      evaluate effectiveness. We therefore aim to investigate the effect of intranasal OT on social
      cognition and behavior immediately following a single-dose (24IU) and following a 4-week
      period of OT (24IU BID) administration in a sample of 50 subjects with autism aged 6 to 12
      years. The primary outcome for this study is change in social behavior, as determined by
      parent ratings on the Social Responsiveness Scale (SRS) after the 4-week treatment period.
      Secondary outcomes are changes in functioning on laboratory-based measures of social behavior
      and cognition following single-dose and 4-week OT administration. Research in a small study
      sample (N=13) also identified treatment responders and non-responders to a single-dose of OT.
      Thus, we also aim to identify biological and cognitive and behavioral variables (i.e.,
      pretreatment levels of social functioning and pretreatment plasma hormone levels) that may
      influence treatment response efficacy in our larger study sample. On completion of the 4-week
      treatment period all subjects will have the option of participating in another 4-week
      double-blind trial in which they will be switched to the alternate nasal spray to that which
      they previously received. They will then undergo a fourth and final assessment time-point
      using the same testing procedures as outlined above on completion of the 4-week dosing. By
      providing subjects with the option of participating in a second 4-week treatment trial, all
      subjects will have an opportunity to receive the active oxytocin nasal spray. We also will be
      able to examine any ongoing effects of oxytocin treatment in the group receiving placebo
      during the second 4-week administration period. Subjects not willing to take part in the
      second trial will exit the study and will be referred to their treating physician.
    

Intervention name(s): Oxytocin nasal spray; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): 24IU BID (3 x 0.1 mL [4IU] sprays per nostril twice daily for 4-weeks.; 3 x 0.1 mL sprays per nostril twice daily for 4-weeks.
Other intervention name(s): Syntocinon® Nasal Spray
Intervention MeSH terms: Oxytocin
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment