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Author Notes:

Correspondence: Dr D Umbricht, Roche Innovation Center, Basel, F. Hoffmann-La Roche Ltd, Grenzacherstrasse 124, Basel 4070, Switzerland, Tel: +41 61 688 3043, Fax: +41 61 687 9848, E-mail: daniel.umbricht@roche.com

See publication for full list of author contributions.

EH has acted as a consultant for Roche, and has received research grants from Roche, Curemark, Coronado Biosciences, Forest, Simons Foundation, Foundation for Prader Willi Research, Orphan Products Division of the Food and Drug Administration, and has intellectual property relating to oxytocin and autism.

JTM has served as a consultant for Roche and Dart Neuroscience, has received research grants from Roche, and has received study drug from Shire and AstraZeneca. FS has received research funding from Roche and Janssen Pharmaceuticals.

LSc has served as a consultant to Bracket, MedAdvante, Roche, Neuren and Coronado.

DU, MVR, JN, LB, OK, LSq, CG, HK, and PF are full-time employees of F. Hoffmann-La Roche.

DU, MVR, JN, LB, OK, LSq, CG, HK, and PF are full-time employees of F. Hoffmann-La Roche.


Research Funding:

This study (ClinicalTrials.gov number: NCT01474278) was funded by F. Hoffmann-La Roche.


  • Science & Technology
  • Life Sciences & Biomedicine
  • Neurosciences
  • Pharmacology & Pharmacy
  • Psychiatry
  • Neurosciences & Neurology
  • AVPR1A

A Single Dose, Randomized, Controlled Proof-Of-Mechanism Study of a Novel Vasopressin 1a Receptor Antagonist (RG7713) in High-Functioning Adults with Autism Spectrum Disorder

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Journal Title:



Volume 42, Number 9


, Pages 1914-1923

Type of Work:

Article | Final Publisher PDF


The core symptoms of autism spectrum disorder (ASD) include impaired social communication, repetitive behaviors, and restricted interests. No effective pharmacotherapy for these core deficits exists. Within the domain of social communication, the vasopressin system is implicated in social cognition and social signaling deficits of ASD, and represents a potential therapeutic target. We assessed the effects of a single 20 mg intravenous dose of the arginine vasopressin receptor 1A (V1a) antagonist, RG7713, on exploratory biomarkers (eye tracking), behavioral and clinical measures of social cognition and communication (affective speech recognition (ASR), reading the mind in the eyes, olfactory identification, scripted interaction), and safety and tolerability in a multicenter, randomized, double-blind, placebo-controlled, cross-over study of 19 high-functioning adult male subjects with DSM-IV Autistic Disorder (age 18-45 years; full scale IQ > 70; ABC-Irritability subscale â 1/213). Eye-tracking showed an increase in biological motion orienting preference with RG7713 (ES=0.8, p=0.047) and a non-significant improvement in the composite score (ES=0.2, p=0.29). RG7713 reduced ability to detect lust (ES=-0.8, p=0.03) and fear (ES=-0.7, p=0.07) in ASR. However, when all eight individual emotion subscales were combined into an overall ASR performance score, the reduction was non-significant (ES=-0.1, p=0.59). Thirteen adverse events were reported in 10 subjects; all were of mild (11/13) or moderate (2/13) severity. Although interpretation should be cautious due to multiple comparisons and small sample size, these results provide preliminary evidence from experimental and behavioral biomarkers, that blockade of the V1a receptor may improve social communication in adults with high-functioning ASD.

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© 2017 The Author(s).

This is an Open Access work distributed under the terms of the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (http://creativecommons.org/licenses/by-nc-nd/4.0/).

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