Research ArticleAutism Spectrum Disorder

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

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Science Translational Medicine  08 May 2019:
Vol. 11, Issue 491, eaau7356
DOI: 10.1126/scitranslmed.aau7356

Modulating vasopressin in ASD

The neuropeptide vasopressin has been implicated in the regulation of social behaviors in animals and humans. The VANILLA clinical trial evaluated balovaptan, an oral selective vasopressin V1a receptor antagonist, in 223 men with autism spectrum disorder (ASD). This 12-week phase 2 trial showed no improvement on the primary endpoint (SRS-2 score) but did show improvement on the secondary outcome measure of adaptive behaviors assessed by the Vineland-II scale (Bolognani et al.). In a related clinical study involving a 4-week intranasal administration of vasopressin to children with ASD, improvements were observed on the SRS-2 primary outcome measure (Parker et al.). Both drugs were well tolerated and had an acceptable safety profile, suggesting that modulating the vasopressin pathway may be a useful therapeutic strategy for ASD.

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