Intranasal Oxytocin in the Treatment of Autism
Condition: Autism
MeSH terms: Autistic Disorder
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to learn whether or not the drug called oxytocin is helpful in
      improving mood and social functioning in adults with autism.
    

Detailed description: 
      Autism is a developmental disorder characterized by abnormalities in speech and
      communication, impaired social functioning, and repetitive behaviors and restricted
      interests. A number of researchers have suggested that the neuropeptide oxytocin may be
      implicated in the etiology of autism.

      Given the likely possibility of dysregulated oxytocin in autism, the goal of this pilot study
      is to investigate the long-term therapeutic effects of oxytocin in the treatment of autism.
      One practical issue with oxytocin is that it does not exist in a pill form. Only the
      intravenous form is available in the United States and this form may or may not pass the
      blood-brain barrier. In addition, intravenous oxytocin is not practical for treatment
      studies. One alternative is intranasal oxytocin; this form of administration is known to pass
      the blood-brain barrier, and it is easy for participants to self-administer. Although not
      available in the United States, we are in the process of receiving an Investigational New
      Drug exemption for its use and can import it from Europe.

      Thus, this pilot investigation will explore daily intranasal oxytocin in the treatment of
      autism. Also, there are very few, if any, outcome measures to assess social functioning in
      the "real world" in the context of clinical trials; yet, this is a major target for
      intervention, especially in autism. Thus, a final goal of this study will be to explore the
      use of Event Contingent Recording to index changes in social functioning and affect. Event
      Contingent Recording is a methodology developed by personality/social psychologists, which
      allows participants to report on symptoms, affect, and behavior close in time to experience.
      In addition, to enabling more sensitive assessments, this methodology allows for the
      assessment of more diverse (e.g., at home versus work) and more detailed measurements of mood
      and behavior.

      Finally, a portion of this study aims to perform gene expression profiling using fresh whole
      blood to explore the molecular mechanisms underlying oxytocin therapy and oxytocin efficacy
      in adults with high functioning autism or Asperger's syndrome. The systemic effects of
      oxytocin therapy and the molecular basis for a positive treatment response to oxytocin are
      not well understood. An understanding of the former may help predict those persons who may
      suffer side-effects from treatment and the latter may help provide easily accessible
      peripheral biomarkers that could predict treatment response.
    

Intervention name(s): Oxytocin; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Intranasal Oxytocin; Placebo Comparator
Other intervention name(s): Syntocinon
Intervention MeSH terms: Oxytocin
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Autism; Treatment; Oxytocin; Adults; Autism Spectrum Disorder