帕金森病疾病疾病修改试验的结果措施:由EJS ACT-PD多臂多阶段试验倡议的共识文件
Cristina Gonzalez-Robles1, Rimona S Weil1, Daniel van Wamelen2
1University College London, London, UK.
Journal of Parkinson's disease
|August 7, 2023
概括
这项研究对帕金森病 (PD) 临床试验的结果措施 (OM) 进行了清单. 它提供了一个选择最佳OM的框架,用于识别疾病修饰治疗,提高试验效率.
科学领域:
- 神经科学是一个神经科学.
- 临床试验 临床试验
- 生物标志物 生物标志物
背景情况:
- 多臂,多阶段 (MAMS) 平台试验加速帕金森病 (PD) 治疗的确定.
- 对于疾病修饰PD试验的最佳结果 (OM) 缺乏共识.
- 加快帕金森病临床试验 (ACT-PD) 倡议解决了这一差距.
研究的目的:
- 为改进疾病的PD试验创建一个最新的OM清单.
- 建立一个框架,在未来的PD研究中选择合适的OM.
- 纳入患者和公众参与和参与 (PPIE) 的观点.
主要方法:
- 一个包括PPIE代表在内的专家小组审查和评估了PD试验中OM的证据.
- OM的排名基于有效性,对变化的敏感性,参与者负担和多站点的实用性.
- 从经过审查的证据和专家意见中,编制了一个全面的OM清单.
主要成果:
- 创建了一个广泛的OM库存,分类为各种领域 (例如,机动,非机动,数字生物标志物,生活质量).
- 为OM评估和未来的更新制定了一个透明的框架.
- PPIE的投入强调了OM需要反映患者体验和多样化的适用性.
结论:
- 一个分类的OM范围被介绍为指导疾病修饰PD试验的选择过程.
- 提供的框架允许随着新证据的出现而进行持续更新.
- 本资源旨在提高PD临床试验的效率和相关性.
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