优化早期眼科临床试验:家庭OCT和建模可以将样本大小减少20%至40%
Jacques Hermes1,2,3, Bernhard Steiert1
1Roche Pharma Research and Early Development, Pharmaceutical Sciences, Roche Innovation Center Basel, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Translational vision science & technology
|September 2, 2025
概括
将家庭光学连贯断层扫描 (OCT) 与药物动力学/药物动力学 (PK/PD) 建模相结合,可显著减少临床试验中的视网膜疾病样本大小. 这种方法提高了试验效率和患者的便利性,同时保持了统计能力.
科学领域:
- 眼科 眼科
- 临床试验设计
- 生物统计学
背景情况:
- 对视网膜疾病的临床试验面临挑战,包括患者的变化和长时间的随访.
- 检测治疗效应往往受到终点的高变化所阻碍.
研究的目的:
- 调查家庭光学一致性断层扫描 (OCT) 与药物动力学/药物动力学 (PK/PD) 建模相结合是否可以减少早期视网膜疾病临床试验中的样本大小要求.
- 评估将新型监测技术与已建立的建模技术相结合的效率.
主要方法:
- 开发了一个人口PK/PD模型,利用来自国内的OCT监测的纵向中心子场厚度数据.
- 采用蒙特卡洛模拟和启动测试来评估各种监测策略下的样本规模需求.
- 模拟了大约50μm中央子场厚度的效应大小.
主要成果:
- 在家庭监测中,每组需要33-35名患者,而传统的两周内监测需要41-54名患者.
- 这代表了检测模拟处理效应所需的样本大小减少了20%至40%.
- 该PK/PD模型有效地利用家庭数据来确定试验参数.
结论:
- 家庭监测与PK/PD建模相结合,为视网膜疾病的临床试验提供了更有效的方法.
- 这种策略可以提高试验效率,提高患者的便利性,并加快新的视网膜治疗方法的开发.
- 减少样本大小的要求保持了统计能力,可能改善患者获得新疗法的机会.
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