A Phase 2 Randomized, Placebo-Controlled Trial of Intranasal Oxytocin in Adults with Autism Spectrum Disorder

Poster Presentation
Friday, May 3, 2019: 5:30 PM-7:00 PM
Room: 710 (Palais des congres de Montreal)
E. Anagnostou1, J. A. Brian1, J. Goldberg2, S. Prat3, L. Capano4, A. Iaboni1, A. Solish1, D. Frisch5, R. Hastie Adams5, L. Genore6, I. Patriciu7, F. Tran2,8 and M. Woodbury-Smith9, (1)Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada, (2)Psychiatry, McMaster University, Hamilton, ON, Canada, (3)McMaster University, Hamilton, ON, Canada, (4)Autism Research Centre, Holland Bloorview Kids Rehabilitation Hospital, Toronto, ON, Canada, (5)Holland Bloorview Kids Rehab Hospital, toronto, ON, Canada, (6)Holland Bloorview Kids Rehab Hospital - ARC, Toronto, ON, Canada, (7)McMaster University, toronto, ON, Canada, (8)Neurology, Sunnybrook Hospital, Toronto, ON, Canada, (9)Newcastle University, Institute of Neuroscience, Newcastle upon Tyne, United Kingdom of Great Britain and Northern Ireland

There are currently no medications with evidence supporting their use to treat the core symptoms of autism spectrum disorder (ASD). Convergent evidence, however, suggests a role for oxytocin in social function. Still, the data regarding potentially therapeutic potential of this compound remains mixed. Methodological limitations of previous studies include small sample sizes (median n = 18), variability in assay sensitivity of outcome measures used, and variability in dosing among others.


To evaluate the efficacy of intranasal oxytocin vs. placebo for the treatment of social deficits in adults with ASD, using a phase II Randomized controlled trial design

Methods: This is a phase II randomized, placebo controlled, multisite trial. Participants were recruited across 2 sites and were randomized to drug or placebo in 1:1 fashion. Safety was evaluated using the SMURF, safety blood work and ECG. Efficacy was evaluated using the CGI as a primary measure. Secondary measures included the ABC, SRS, WHOQOL, Vineland, and SCL-90 anxiety.


70 participants were randomized across 2 sites. Intranasal oxytocin was well tolerated with no related serious adverse events reported. Improvements were noted in self reported SRS (p=0.0006), WHOQOL psychological scale (p=0.08), anxiety (p=0.04) and Vineland total score (p=0.05)

Conclusions: Intranasal oxytocin was well tolerated in adults with ASD. Efficacy data is supportive of therapeutic potential for this mechanism.