Large multicenter randomized trials in autism: key insights gained from the balovaptan clinical development program.
Suma Jacob, Evdokia Anagnostou, Eric Hollander and 14 others
PMID 35690870WHAT IT FOUND
Balovaptan failed to improve social communication in autism across three large trials.
The null result was driven by high placebo response, which varied by site type, rater experience, and participant baseline severity, complicating future drug development.
Key findings
01Balovaptan showed no significant difference versus placebo on primary endpoints for social communication in adults or children.
02Placebo response was higher in private research sites and sites with less specific trial experience compared to academic or experienced sites.
03Participants with lower baseline adaptive skills showed greater improvement on outcome measures in both treatment and placebo groups.
STILL TO COME
How it was doneWhat they found
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What it does not show
This is a review of completed trials rather than a new primary experiment, so it relies on data from previous publications. The reasons for the lack of balovaptan efficacy are hypothesized based on trial data; direct causal links for placebo response drivers are not definitively proven. The trials excluded individuals with IQ < 70, limiting generalizability to those with intellectual disability. The high placebo response may be specific to the autism population and the outcome measures used, limiting applicability to other conditions.
Declared interests
The study was funded by F. Hoffmann-La Roche Ltd, the manufacturer of balovaptan. The authors are affiliated with or supported by the sponsor.
The easy way to misread this
Do not interpret the improvements seen in the placebo groups as evidence that behavioral or environmental factors alone can resolve core social communication deficits. These gains reflect a high placebo response in autism trials, which can mask true treatment effects or lack thereof.