行动:在新诊断的H3 K27M突变扩散质瘤患者中对ONC201 (多达维) 进行随机的第3期研究
Isabel Arrillaga-Romany1, Andrew Lassman2, Susan L McGovern3
1Mass General Cancer Center, Neuro-Oncology, Massachusetts General Hospital, Boston, Massachusetts, USA.
Neuro-oncology
|March 6, 2024
概括
这一第三阶段试验调查了ONC201新诊断的H3 K27M突变结质瘤,这是一个罕见的儿童癌症,缺乏有效治疗方法. 这项研究旨在确定ONC201是否能改善这些患者的整体存活率和无进展存活率.
科学领域:
- 神经瘤学神经瘤学
- 儿科瘤学 儿科瘤学
- 翻译性瘤学是指翻译性瘤学.
背景情况:
- H3 K27M突变扩散质瘤是一种侵袭性脑瘤,主要影响年轻人.
- 目前新诊断的H3 K27M突变质瘤的治疗选择有限,预后不佳.
- 在复发性疾病环境中,ONC201已经显示出先前的疗效.
研究的目的:
- 评估ONC201 (多达维) 在新诊断的H3 K27M突变扩散质瘤患者的疗效和安全性.
- 评估ONC201对整体生存时间 (OS) 和无进展生存时间 (PFS) 的影响.
主要方法:
- 一个随机的,双盲的,安慰剂对照的国际第三期试验 (ACTION;NCT05580562).
- 患者随机分为1:1:1与安慰剂,每周一次的ONC201或前线放射治疗后每周两次的ONC201.
- 主要终点:通过神经瘤学高度质瘤 (RANO-HGG) 标准的响应评估来评估OS和PFS.
主要成果:
- 该研究目前正在国际范围内招募患者.
- 主要疗效终点 (OS和PFS) 尚未确定.
结论:
- 该ACTION试验将提供有关ONC201在新诊断的H3 K27M突变结质瘤中的疗效和安全性的关键数据.
- 预计结果将为未来的治疗策略提供信息,以应对这一具有挑战性的儿科脑瘤.
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