Denis Sukhodolsky and Kevin Pelphrey seek to develop more precise treatments for children with autism spectrum disorder (ASD), guided by their distinct neural profiles. Informed by their recent identification of a neuropredictive biomarker for ASD¹, Sukhodolsky and Pelphrey will conduct a double-blind, randomized, placebo-controlled trial of intranasal oxytocin (OXT) as an enhancer of response to Pivotal Response Treatment (PRT) in young children with ASD**.

Specifically, they plan to assess whether children with lower levels of activity in, and functional connectivity among, key social brain regions will benefit more from the administration of OXT versus placebo. This hypothesis is based on the known effects of OXT as a social cognitive neural circuit enhancer.

The researchers will perform a 16-week trial of PRT in young children (5 – 9 years of age), using state-of-the art brain imaging and eye-tracking paradigms as well as key behavioral outcome measures. Functional magnetic resonance imaging analyses will include a well-validated, sensitive and reliable paradigm involving coherent biological motion versus scrambled biological motion that Sukhodolsky and Pelphrey have previously used to identify a biological marker of ASD¹. Eye-tracking will involve a set of novel, highly practical paradigms that are aligned specifically with behavioral outcomes targeted by PRT. Clinical outcomes will be assessed by the Social Responsiveness Scale-2 (SRS-2) Total Score (primary outcome) and the Vineland Adaptive Behavior Scale (secondary outcome).

This study will advance personalized medicine in ASD and serve as a proof-of-concept analysis of the use of pharmacological compounds to enhance evidence-based behavioral treatments for ASD.

Reference:

1. Yang D. et al. Transl. Psychiatry 6, e948 (2016) PubMed

** Note: This study was originally awarded to Pamela Ventola and Kevin Pelphrey in 2017 (award #: 514534). The current project reflects a change of PI.