Trial reportPsychiatry and clinical neurosciences2025
AST-001 versus placebo for social communication in children with autism spectrum disorder: A randomized clinical trial.
Trial report in Psychiatry and clinical neurosciences, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 5 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
5 citing papers in PubMed.
- Safety and efficacy of AST-001 in children with autism spectrum disorder: a 52-week multicenter long-term follow-up study.Frontiers in psychiatry · 2026Trial
- Integrated proteomic and single-cell transcriptomic profiling elucidates immunomodulatory effects of L-serine in autism spectrum disorder.Scientific reports · 2026Article
- Linking autism risk genes to morphological and pharmaceutical screening by high-content imaging: Future directions and opinion.Psychiatry and clinical neurosciences · 2025Review
- Correction to "AST-001 versus placebo for social communication in children with autism spectrum disorder: A randomized clinical trial".Psychiatry and clinical neurosciences · 2025Article
- Population pharmacokinetic and pharmacodynamic model guided weight-tiered dose of AST-001 in pediatric patients with autism spectrum disorder.Frontiers in pharmacology · 2024Article
Corrections and comments
- Erratum issued
Authors and funding
11 authors.
Funding
Abstract
aimThis study examined the efficacy of AST-001 for the core symptoms of autism spectrum disorder (ASD) in children.
methodsThis phase 2 clinical trial consisted of a 12-week placebo-controlled main study, a 12-week extension, and a 12-week follow-up in children aged 2 to 11 years with ASD. The participants were randomized in a 1:1:1 ratio to a high-dose, low-dose, or placebo-to-high-dose control group during the main study. The placebo-to-high-dose control group received placebo during the main study and high-dose AST-001 during the extension. The a priori primary outcome was the mean change in the Adaptive Behavior Composite (ABC) score of the Korean Vineland Adaptive Behavior Scales II (K-VABS-II) from baseline to week 12.
resultsAmong 151 enrolled participants, 144 completed the main study, 140 completed the extension, and 135 completed the follow-up. The mean K-VABS-II ABC score at the 12th week compared with baseline was significantly increased in the high-dose group (P = 0.042) compared with the placebo-to-high-dose control group. The mean CGI-S scores were significantly decreased at the 12th week in the high-dose (P = 0.046) and low-dose (P = 0.017) groups compared with the placebo-to-high-dose control group. During the extension, the K-VABS-II ABC and CGI-S scores of the placebo-to-high-dose control group changed rapidly after administration of high-dose AST-001 and caught up with those of the high-dose group at the 24th week. AST-001 was well tolerated with no safety concern. The most common adverse drug reaction was diarrhea.
conclusionsOur results provide preliminary evidence for the efficacy of AST-001 for the core symptoms of ASD.
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