A Study of Balovaptan in Adults With Autism Spectrum Disorder With a 2-Year Open-Label Extension

Summary

This study will evaluate the efficacy, safety, and pharmacokinetics of 10 mg of oral administration balovaptan once a day (QD) compared with matching placebo in adults (18 years and older) with autism spectrum disorder (ASD).

Timeline

Start

2018-08-08

Primary completion

2020-03-04

Completion

2020-07-01

Outcome

Missed primary endpoint

Stopped: “A futility analysis assessed that the study is highly unlikely to meet the pre-defined primary objective of the study. No new safety concerns were identified.”

paper The interim futility analysis showed no improvement for balovaptan versus placebo in terms of Vineland-II 2DC score at week 24 compared with baseline PMID 35151410 ↗

paper Balovaptan did not improve social communication in autistic adults. PMID 35151410 ↗

Publications

• Aponte EA, Tillmann J, Gleissl T, Del Valle Rubido M, Murtagh L, Sanders K, Chatham CH, Wiese T, Suter EE. Anxiety, repetitive and restricted behaviors and interests, and social communication in autistic adults: an exploratory analysis of a phase 3, randomized clinical trial. Sci Rep. 2025 Nov 6;15(1):38912. doi: 10.1038/s41598-025-22659-y.
• Jacob S, Veenstra-VanderWeele J, Murphy D, McCracken J, Smith J, Sanders K, Meyenberg C, Wiese T, Deol-Bhullar G, Wandel C, Ashford E, Anagnostou E. Efficacy and safety of balovaptan for socialisation and communication difficulties in autistic adults in North America and Europe: a phase 3, randomised, placebo-controlled trial. Lancet Psychiatry. 2022 Mar;9(3):199-210. doi: 10.1016/S2215-0366(21)00429-6. Epub 2022 Feb 10.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Balovaptan	Small molecule	10 mg	Oral

Indications