关于脑瘤I期临床试验的最新情况:对探索性疗效终点的监管科学研究
Shinya Watanabe1,2, Takahiro Nonaka3, Makoto Maeda4
1Department of Neurosurgery, Mito Kyodo General Hospital, Tsukuba University Hospital Mito Area Medical Education Center, 3-2-7 Miyamachi, Mito, 310-0015, Ibaraki, Japan. shinya-watanabey@md.tsukuba.ac.jp.
Therapeutic innovation & regulatory science
|March 26, 2024
概括
针对脑瘤 (BTs) 的I期试验经常使用二次终点来评估疗效. 缺乏标准化的成像标准,这给在BT临床研究中评估治疗有效性带来了挑战.
科学领域:
- 在瘤学瘤学.
- 临床试验 临床试验
- 神经瘤学神经瘤学
背景情况:
- 第一阶段试验对于产生脑瘤 (BT) 治疗的探索性疗效数据至关重要.
- BTs的独特特征需要定制的有效性评估策略.
- 本研究探讨了在I期BT试验中如何评估探索性疗效.
研究的目的:
- 分析用于脑瘤的第一阶段试验中的有效性终点的利用和类型.
- 为了比较专门针对BT的试验和在混合固体瘤中包括BT的试验之间的疗效终点评估.
- 确定在早期临床试验中评估脑瘤的探索性疗效的挑战.
主要方法:
- 在2020-2022年使用CortellisTM.进行的42个I阶段BT干预试验的分析.
- 疗效终点的分类为初级 (PE) 或二级 (SE).
- 仅BT试验 (A组) 和混合固体瘤试验 (B组) 之间的终点使用比较.
主要成果:
- 疗效终点主要是SE (78%) 而不是PE (5%).
- 通常报告的终点包括整体响应率 (ORR),无进展生存期 (PFS) 和整体生存期 (OS).
- 与混合瘤试验相比,仅在BT试验中使用固体瘤响应评估标准 (RECIST) 的频率较低.
结论:
- 最近的I期试验越来越多地使用SE来评估BT的疗效.
- 目前还没有确定的BT成像评估标准.
- 混合瘤试验设计为评估探索性BT疗效提出了独特的挑战.
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