Intranasal Oxytocin in the Treatment of Autism
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.
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||Intranasal Oxytocin in the Treatment of Autism|
- Clinical Global Impressions Scale (CGI) [ Time Frame: Baseline visit, week 2, 4, and 6 visits ] [ Designated as safety issue: No ]
- Repetitive Behavior Scale (RBS) [ Time Frame: Baseline visit, week 2, 4, and 6 visits ] [ Designated as safety issue: No ]
- Diagnostic Analysis of Nonverbal Accuracy, Adult Paralanguage Test (DANVA2-AP) [ Time Frame: Baseline ] [ Designated as safety issue: No ]
- Diagnostic Analysis of Nonverbal Accuracy, Adult Paralanguage Test (DANVA2-AP) [ Time Frame: 6 week visit ] [ Designated as safety issue: No ]
- Yale-Brown Obsessive-Compulsive Scale (YBOCS) [ Time Frame: Baseline visit, week 2, 4, and 6 visits ] [ Designated as safety issue: No ]
- Social Responsivity Scale (SRS) [ Time Frame: Baseline visit, week 2, 4, and 6 visits ] [ Designated as safety issue: No ]
|Study Start Date:||June 2006|
|Study Completion Date:||April 2012|
|Primary Completion Date:||September 2011 (Final data collection date for primary outcome measure)|
|Experimental: Intranasal Oxytocin||
Placebo Comparator: Placebo
Drug: Placebo Comparator
Definition: Extended description of the protocol, including information not already contained in other fields, such as comparison(s) studied.
Autism is a developmental disorder characterized by abnormalities in speech and communication, impaired social functioning, and repetitive behaviors and restricted interests (American Psychiatric Association, 2000). A number of researchers have suggested that the neuropeptide oxytocin may be implicated in the etiology of autism (Hollander et al., 2003; Insel et al., 1999; Lim et al., 2005; McCarthy & Altemus, 1997; Modahl et al., 1992; Waterhouse et al., 1996).
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 US and this form may or may not pass the blood-brain barrier. In addition, IV 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 US, we are in the process of receiving an IND 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 (ECR) to index changes in social functioning and affect. ECR 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.
|United States, New York|
|Mount Sinai School of Medicine|
|New York, New York, United States, 10029-6574|
|Principal Investigator:||Evdokia Anagnostou, MD||Mount Sinai School of Medicine|