A phase 2 clinical trial of a vasopressin V1a receptor antagonist shows improved adaptive behaviors in men with autism spectrum disorder
Abstract
= 39). Balovaptan treatment was not associated with a change from baseline compared with placebo at 12 weeks in the primary efficacy endpoint (Social Responsiveness Scale, 2nd Edition). However, dose-dependent and clinically meaningful improvements on the Vineland-II Adaptive Behavior Scales composite score were observed for participants treated with balovaptan 4 or 10 mg compared with placebo. This was driven principally by improvements in the Vineland-II socialization and communication scores. Balovaptan was well tolerated across all doses, and no drug-related safety concerns were identified. These results support further study of balovaptan as a potential treatment for the socialization and communication deficits in ASD.
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Based on a subset of preclinical studies demonstrating anxiogenic and agonistic effects of vasopressin, the V1aR antagonist balovaptan has been tested in clinical trials in autism spectrum disorder individuals with mixed effects on social end points.
“Based on a subset of preclinical studies demonstrating anxiogenic and agonistic effects of AVP, the V1aR antagonist balovaptan has been tested in clinical trials in ASD individuals with mixed effects on social end points (164, 165).”
From Oxytocin, Vasopressin, and Social Behavior: From Neural Circuits to Clinical Opportunities (2022) · cites this paper as [165]
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Self-citation
- Authors
- Bolognani, F., del Valle Rubido, M., Squassante, L., Wandel, C., Derks, M., Murtagh, L., Sevigny, J., Khwaja, O., Umbricht, D., & Fontoura, P.
- Affiliations
- F. Hoffmann-La Roche AG (CH)
- Neuroscience, Ophthalmology, and Rare Diseases (NORD), Roche Pharma Research and Early Development, Roche Innovation Center Basel, F. Hoffmann–La Roche Ltd., Basel, Switzerland.
- Roche (United Kingdom) (GB)
- Corresponding author
- Umbricht, Daniel
- Fontoura, Paulo
- Venue
- Science Translational Medicine
- Published
- 2019-05-08
- Volume
- 11
- Issue
- 491
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2942769136
- Cited by
- 128
- References
- 44
- Citations by year
- 2026: 13 · 2025: 8 · 2024: 14 · 2023: 17 · 2022: 25 · 2021: 26 · 2020: 16 · 2019: 9
- FWCI
- 11.34
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Funders
- F. Hoffmann-La Roche Ltd.
- Topics
- Life Sciences › Neuroscience › Cognitive Neuroscience › Autism Spectrum Disorder Research
- Social Sciences › Psychology › Social Psychology › Neuroendocrine regulation and behavior
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Keywords
- autism spectrum disorder, adaptive behavior, socialization, communication difficulties, Social Responsiveness Scale, Vineland-II, drug safety
- MeSH terms
- Autism Spectrum Disorder, Adaptation, Psychological, Adolescent, Adult, Behavior, Benzodiazepines, Dose-Response Relationship, Drug, Humans, Male, Middle Aged, Pyridines, Quality of Life, Triazoles, Treatment Outcome, Receptors, Vasopressin, Young Adult, Antidiuretic Hormone Receptor Antagonists
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