参与者,现场人员和赞助商对Courage-ALS:一个随机临床试验中分散试验特征的观点
Stacy A Rudnicki1, Paulos Gebrehiwet1, Stuart Kupfer1
1Cytokinetics Incorporated, South San Francisco, CA, USA.
概括
在ALS中进行分散的临床试验 (DCT) 是可行的,但像强迫生命能力 (FVC) 这样的远程评估存在挑战. 参与者和现场工作人员发现,尽管存在技术问题,但远程访问是有利的,并且错过了FVC.
科学领域:
- 神经学 神经学
- 临床试验 临床试验
- 医疗技术 医疗技术 医学技术
背景情况:
- 肌缩侧面硬化症 (ALS) 研究需要创新的临床试验设计.
- 分散临床试验 (DCT) 提供了潜在的好处,但也带来了独特的运营挑战.
- 了解参与者和现场人员的观点对于优化DCT实施至关重要.
研究的目的:
- 评估参与者,现场人员 (SP) 和赞助商对ALS进行混合分散临床试验 (DCT) 的经验.
- 确定在ALS临床试验环境中实施DCT特征的促进者和障碍.
主要方法:
- COURAGE-ALS试验是一个为期48周的随机,双盲,III期混合DCT,评估ALS的常见动机.
- 与参与者进行了半结构化访谈,并与SP进行了在线调查,以收集关于DCT方面的反.
- 分析了远程诊所 (RV) 和临床内诊 (ICV) 完成率的数据.
主要成果:
- 远程访问 (RVs) 被参与者和SP普遍认为是有利的.
- 远程强迫生命能力 (FVC) 评估对40%的参与者来说是一个问题,与临床内诊 (96%的完成率) 相比,远程强迫生命能力 (FVC) 评估更常被错过 (82%的完成率).
- 现场工作人员报告了常见的技术问题 (65%),但与传统设计相比 (38%),他们也认为时间/劳动较少.
结论:
- 混合DCT对于ALS干预试验是可行的,正如Courage-ALS所证明的那样.
- 解决技术挑战和优化远程评估程序,特别是对于FVC,对于未来的DCT设计至关重要.
- 参与者和现场人员的反为提高神经学研究中DCT的效率和有效性提供了宝贵的见解.
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