Research Project 3 will develop novel, objective, and portable measures of Parkinson disease features

摘要 目前的PD测量在很大程度上局限于临床，并基于间歇性的、依赖于评分者的、绝对的 比例。其结果是，临床试验，即使是早期试验，需要大量的样本，持续时间长， 而且成本很高。此外，这些措施容易在第二阶段试验中产生疗效的错误信号。 在阶段3中没有发现，并且可能会产生从未被检测到的遗漏的疗效信号。 21世纪出现了新的工具，可以对帕金森病进行客观、频繁、敏感的评估 在真实世界的环境中。这些超级计算设备，或智能手机，越来越无处不在， 很强大。2015年3月，苹果发布了ResearchKit，这是一个用于创建智能手机的开源平台 研究性研究。与此同时，五项智能手机研究，包括一项针对PD(MPower)的研究， 也推出了。在7个月的时间里，全国有超过7万人参加了这些研究。 通过P20和相关的努力，我们已经证明了对声音、步态、平衡和 在智能手机上进行的运动迟缓可以区分帕金森病患者和非帕金森病患者。 使用传统的PD评估，检测对左旋多巴的反应，并可以在新型移动PD中进行量化 与MDS-UPDRS相关的分数，但几乎任何人都可以随时随地进行管理。 在研究项目3中，我们将评估下一代智能手机研究应用程序(mPower 2.0) 对照目前PD的黄金标准临床测量，评估其产生新的评估的能力 基于被动收集的数据进行社会化，细化移动PD得分，提高可复制性和可重复性 使用先进的信号处理算法和神经网络实现分数的重复性 在大规模推出后的短短两年内，制药公司和学术研究人员 已经将这些设备整合到他们的临床试验中。这项研究项目将有助于加速这些 努力使用智能手机生成客观、被动和主动的、频繁的现实世界评估 帕金森病患者的运动和非运动功能。

SUMMARY Current PD measures are largely limited to the clinic and based on episodic, rater-dependent, categorical scales. The result is that the clinical trials, even early stage ones, require large sample sizes, long durations, and high costs. Moreover, these measures are prone to generating false signals of efficacy in phase 2 trials that are not found in phase 3 and likely generate missed signals of efficacy that are never detected. In the 21st century new tools are available that can generate objective, frequent, sensitive assessments of PD in real-world settings. These super computing devices, or smartphones, are increasingly ubiquitous and powerful. In March 2015, Apple released ResearchKit, an open-source platform for creating smartphone research studies. At the same time, five smartphone research studies, including one for PD (mPower), were also launched. In seven months, over 70,000 individuals across the country enrolled in these studies. Through the P20 and related efforts, we have demonstrated that assessments of voice, gait, balance, and bradykinesia conducted on the smartphone can differentiate individuals with PD from those without, correlate with traditional PD assessments, detect response to levodopa, and can be quantified in a novel mobile PD score that correlates with the MDS-UPDRS but can be administered by almost anyone anywhere anytime. In Research Project 3, we will evaluate the next generation smartphone research application (mPower 2.0) against current gold standard clinical measures of PD, assess its ability to generate novel assessments of socialization based on passively collected data, refine the mobile PD score, and improve the replicability and reproducibility of the score using advanced signal processing algorithms and neural networks In just two years after their large scale introduction, pharmaceutical companies and academic investigators are already incorporating these devices into their clinical trials. This Research Project will help accelerate these efforts to use smartphones to generate objective, passive and active, frequent real-world assessments of motor and non-motor function in PD.