安吉尔曼综合征中的 gabaxadol:一个双盲,并行组,随机的安慰剂控制的第三期研究
Christopher Keary1, Lynne M Bird2, Marie-Claire de Wit3
1Massachusetts General Hospital, Boston, MA, USA.
概括
在治疗12周后儿童的安吉尔曼综合征 (AS) 时,GABOXADOL与安慰剂相比没有显著改善. 药物耐受性很好,但在这个第三阶段试验中没有达到疗效终点.
科学领域:
- 神经科学是一个神经科学.
- 儿科神经学 儿科神经学
- 临床药理学 临床药理学
背景情况:
- 安吉尔曼综合征 (AS) 是一种罕见的遗传疾病,影响神经发育.
- 目前对AS症状的治疗方法有限,需要对新型治疗剂进行研究.
- 加博沙多尔是一种选择性GABAA受体激动剂,已被研究其潜在的神经学的益处.
研究的目的:
- 评估在被诊断为安吉尔曼综合征的儿科患者中加博的疗效和安全性.
- 为了确定加博克萨多尔是否与安慰剂相比,在患有AS的儿童中提供了显著的临床改善.
主要方法:
- 这是一项国际双盲第三期临床试验,涉及患有安吉尔曼综合征的4-12岁儿童.
- 参与者每天接受基于体重的加博沙多尔剂量或与之匹配的安慰剂,持续12周.
- 主要疗效终点是第12周临床全球印象改善-AS (CGI-I-AS) 评分.
主要成果:
- 招募了104名参与者;47名接受了加博沙多尔,50名接受了安慰剂.
- 在第12周,在加博沙多和安慰剂组之间没有观察到平均CGI-I-AS得分的统计学上显著差异.
- 在所有接受治疗的儿科参与者中,GABOXADOL显示出良好的耐受性和安全性.
结论:
- 与安慰剂相比,GABOXADOL在改善儿童安吉尔曼综合征症状方面没有表现出更高的疗效.
- 该研究得出结论,在12周治疗后,加博克萨多尔和安慰剂之间临床改善没有显著差异.
- 可能需要进一步的研究来探索安吉尔曼综合征的替代治疗策略.
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