Trial reportFrontiers in psychiatry2026
Safety and efficacy of AST-001 in children with autism spectrum disorder: a 52-week multicenter long-term follow-up study.
Trial report in Frontiers in psychiatry, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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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.
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Authors and funding
6 authors.
Funding
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Abstract
Background: This study examined the 52-week safety and efficacy of AST-001 syrup for the core symptoms of autism spectrum disorder (ASD) in children aged 2-11 years. Methods: This multicenter long-term follow-up study enrolled children with ASD who were identified as responders in an antecedent phase II randomized controlled trial. A total of 61 participants were given the same dose (high or low) they received during the extension phase of the phase-II trial for 52 weeks. Safety and tolerability were evaluated by reporting adverse events. Efficacy was assessed using the Ohio State University Autism Clinical Global Impression (CGI)-Severity and Improvement scale. Results: The proportion of participants experiencing any treatment-emergent adverse events was 40.98%, most commonly due to COVID-19 (11/61 participants, 18.03%), but none were related to AST-001. Two participants experienced adverse drug reactions (2/61 participants, 3.28%), including decreased appetite and enteritis. The incidence of serious adverse events was 4.92% (3/61 participants), which were dental caries, otitis media, and septic shock, but none were related to medication. The mean CGI-S score decreased slightly from 4.25 ± 0.87 at baseline to 4.10 ± 0.79 at week 52 of the long-term follow-up study. Among the 48 participants who were CGI-I responders at week 24 of phase-II trial, 45 continued to meet the responder criteria at week 52. Conclusions: AST-001 was well tolerated over the 52-week period, and clinical improvement was maintained among participants who had previously responded to treatment, suggesting continued benefit on ASD core symptoms. These findings provide preliminary long-term data on safety, tolerability, and clinical outcomes in a field where pharmacologic treatment options for the core symptoms of ASD remain limited. However, given the uncontrolled long-term follow-up design in prior responders, the results should be interpreted with caution and do not constitute confirmatory evidence of sustained efficacy. Further large-scale, well-controlled studies are warranted. Clinical Trial Registration: https://cris.nih.go.kr/cris/search/detailSearch.do?seq=26492&search_page=L&search_lang=&class_yn=, identifier KCT0007519.
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