AST-001 versus placebo for social communication in children with autism spectrum disorder: A randomized clinical trial.

IF 5 3区 医学 Q1 CLINICAL NEUROLOGY Psychiatry and Clinical Neurosciences Pub Date : 2024-10-18 DOI:10.1111/pcn.13757
Hyo-Won Kim, Ji-Hoon Kim, Un Sun Chung, Johanna Inhyang Kim, Se-Hoon Shim, Tae Won Park, Moon-Soo Lee, Jun-Won Hwang, Eun-Jin Park, Su-Kyeong Hwang, Yoo-Sook Joung
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Abstract

Aim: This study examined the efficacy of AST-001 for the core symptoms of autism spectrum disorder (ASD) in children.

Methods: This 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.

Results: Among 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.

Conclusions: Our results provide preliminary evidence for the efficacy of AST-001 for the core symptoms of ASD.

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AST-001 与安慰剂治疗自闭症谱系障碍儿童的社交沟通:随机临床试验。
目的:本研究探讨了AST-001对儿童自闭症谱系障碍(ASD)核心症状的疗效:这项2期临床试验包括为期12周的安慰剂对照主要研究、为期12周的扩展研究和为期12周的随访,研究对象为2至11岁的自闭症谱系障碍儿童。在主要研究期间,参与者按1:1:1的比例随机分配到高剂量组、低剂量组或安慰剂与高剂量对照组。安慰剂对高剂量对照组在主要研究期间服用安慰剂,在延长研究期间服用高剂量 AST-001。先验主要结果是韩国维尼兰适应行为量表II(K-VABS-II)适应行为综合(ABC)得分从基线到第12周的平均变化:在 151 名参加者中,144 人完成了主要研究,140 人完成了扩展研究,135 人完成了后续研究。与安慰剂转大剂量对照组相比,大剂量组在第12周的K-VABS-II ABC平均得分与基线相比显著增加(P = 0.042)。与安慰剂对高剂量对照组相比,高剂量组(P = 0.046)和低剂量组(P = 0.017)在第 12 周的 CGI-S 平均得分明显下降。在延长期内,服用大剂量AST-001后,从安慰剂到大剂量对照组的K-VABS-II ABC和CGI-S评分迅速发生变化,并在第24周赶上了大剂量组。AST-001的耐受性良好,没有安全问题。最常见的药物不良反应是腹泻:我们的研究结果为 AST-001 治疗 ASD 核心症状的疗效提供了初步证据。
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来源期刊
CiteScore
7.40
自引率
4.20%
发文量
181
审稿时长
6-12 weeks
期刊介绍: PCN (Psychiatry and Clinical Neurosciences) Publication Frequency: Published 12 online issues a year by JSPN Content Categories: Review Articles Regular Articles Letters to the Editor Peer Review Process: All manuscripts undergo peer review by anonymous reviewers, an Editorial Board Member, and the Editor Publication Criteria: Manuscripts are accepted based on quality, originality, and significance to the readership Authors must confirm that the manuscript has not been published or submitted elsewhere and has been approved by each author
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