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Sci Transl Med. 2019 May 08;11(491). doi: 10.1126/scitranslmed.aau7356. Epub 2019 May 01.

A randomized placebo-controlled pilot trial shows that intranasal vasopressin improves social deficits in children with autism.

Science translational medicine

Karen J Parker, Ozge Oztan, Robin A Libove, Noreen Mohsin, Debra S Karhson, Raena D Sumiyoshi, Jacqueline E Summers, Kyle E Hinman, Kara S Motonaga, Jennifer M Phillips, Dean S Carson, Lawrence K Fung, Joseph P Garner, Antonio Y Hardan

Affiliations

  1. Department of Psychiatry and Behavioral Sciences, Stanford University, Stanford, CA 94305, USA. [email protected].
  2. Department of Psychiatry and Behavioral Sciences, Stanford University, Stanford, CA 94305, USA.
  3. Department of Pediatrics, Stanford University, Stanford, CA 94305, USA.
  4. Department of Comparative Medicine, Stanford University, Stanford, CA 94305, USA.

PMID: 31043522 PMCID: PMC6716148 DOI: 10.1126/scitranslmed.aau7356

Abstract

The social impairments of autism spectrum disorder (ASD) have a major impact on quality of life, yet there are no medications that effectively treat these core social behavior deficits. Preclinical research suggests that arginine vasopressin (AVP), a neuropeptide involved in promoting mammalian social behaviors, may be a possible treatment for ASD. Using a double-blind, randomized, placebo-controlled, parallel study design, we tested the efficacy and tolerability of a 4-week intranasal AVP daily treatment in 30 children with ASD. AVP-treated participants aged 6 to 9.5 years received the maximum daily target dose of 24 International Units (IU); participants aged 9.6 to 12.9 years received the maximum daily target dose of 32 IU. Intranasal AVP treatment compared to placebo enhanced social abilities as assessed by change from baseline in this phase 2 trial's primary outcome measure, the Social Responsiveness Scale, 2nd Edition total score (SRS-2

Copyright © 2019 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works.

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