Tele-Health Extension for Phase 3 Parkinson's Disease Trial Cohorts

帕金森病第三阶段试验队列的远程医疗扩展

• 批准号: 10467996
• 负责人: Earl Ray Dorsey
• 金额: $ 77.42万
• 依托单位: MASSACHUSETTS GENERAL HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2018
• 资助国家: 美国
• 起止时间: 2018-07-01 至 2024-04-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10467996
• 关键词: Biological; Biological Markers; Cellular Phone; Clinic; Clinical; Clinical Data; Clinical Trials; Clinical assessments; Collection; DNA; Data; Data Collection; Development; Disease; Disease Progression; Dropout; Enrollment; Funding; Future; Goals; Home visitation; Informed Consent; Infrastructure; Inosine; Intervention; Intervention Studies; Intervention Trial; Investments; Isradipine; Life; Location; Longterm Follow-up; Measurement; Measures; Modernization; Modification; Monitor; Motor; Movement Disorder Society Unified Parkinson' s Disease Rating Scale; National Institute of Neurological Disorders and Stroke; Neuroprotective Agents; Outcome; Parkinson Disease; Participant; Patient Outcomes Assessments; Patients; Persons; Phase; Physical activity; Physical assessment; Placebos; Plasma; Randomized; Research; Research Personnel; Resources; Sampling; Schedule; Serum; Site; Socialization; Study Subject; System; Technology; Telemedicine; Testing; Therapeutic; United States National Institutes of Health; Urate; Visit; base; biomarker development; cohort; cost; design; digital; disability; dopamine transporter; feasibility testing; follow-up; improved; molecular marker; neuroimaging; new technology; next generation; novel; novel strategies; phase 3 study; phase III trial; prevent; programs; recruit; remote sensor; retention rate; smartphone based assessment; success; telehealth; televisit; virtual; virtual model; volunteer; Biological; Biological Markers; Cellular Phone; Clinic; Clinical; Clinical Data; Clinical Trials; Clinical assessments; Collection; DNA; Data; Data Collection; Development; Disease; Disease Progression; Dropout; Enrollment; Funding; Future; Goals; Home visitation; Informed Consent; Infrastructure; Inosine; Intervention; Intervention Studies; Intervention Trial; Investments; Isradipine; Life; Location; Longterm Follow-up; Measurement; Measures; Modernization; Modification; Monitor; Motor; Movement Disorder Society Unified Parkinson' s Disease Rating Scale; National Institute of Neurological Disorders and Stroke; Neuroprotective Agents; Outcome; Parkinson Disease; Participant; Patient Outcomes Assessments; Patients; Persons; Phase; Physical activity; Physical assessment; Placebos; Plasma; Randomized; Research; Research Personnel; Resources; Sampling; Schedule; Serum; Site; Socialization; Study Subject; System; Technology; Telemedicine; Testing; Therapeutic; United States National Institutes of Health; Urate; Visit; base; biomarker development; cohort; cost; design; digital; disability; dopamine transporter; feasibility testing; follow-up; improved; molecular marker; neuroimaging; new technology; next generation; novel; novel strategies; phase 3 study; phase III trial; prevent; programs; recruit; remote sensor; retention rate; smartphone based assessment; success; telehealth; televisit; virtual; virtual model; volunteer

Abstract Disease modification is the primary unmet need in Parkinson's disease (PD) therapeutics. Currently clinical trials of promising candidate neuroprotectants are limited by the inefficiency and imprecision of repeated in- person clinical assessments required of people with Parkinson's who volunteer to participate in these studies. Emerging telemedicine and smartphone-based remote sensor assessments provide opportunities to help overcome these obstacles and improve prospects for the success of future trials. Demonstrating the utility of these relatively inexpensive accessible tele-health platforms for the measurement of PD progression would also establish infrastructure for long-term follow up of participants after completion of interventional studies. STEADY-PD3 and SURE-PD3 are active NINDS-funded phase 3 trials of potential disease-modifying interventions in PD. Both studies, comprising ~600 early PD subjects, include biomarker sub-studies collecting DNA and serial plasma samples and in a subset serial dopamine transporter (DAT) neuroimaging data that will become part of the Parkinson's Disease Biomarker Program (PDBP). The objective of this proposal is to leverage modern technology to develop, pilot and implement a 100% virtual model for long-term follow up utilizing telemedicine and smartphone platforms for quantitative monitoring of clinician- and patient-reported outcomes. Specific Aim (SA) 1 is to establish infrastructure for longitudinal remote follow up of phase 3 trial cohorts; a) transitioning from site-based to a centralized coordination center to manage all subject activity, b) conducting an annual telemedicine research visit (tele-visit) program, and c) implementing a smartphone data collection platform tailored to a clinical trial cohort. SA2 is to compare smartphone (patient-driven) vs tele-visit (clinician-driven) outcomes by a) correlating these platforms' component and composite features, and their changes over years, and b) comparing their abilities to i) measure PD progression; ii) distinguish rapid vs slow progressors based on baseline motor scale scores, DAT deficit or serum urate levels; and iii) demonstrate persistence of any effects of isradipine or inosine in their respective trials. SA3 is to explore novel smartphone- based real-life mobility biomarkers of PD disability and its progression that are normally inaccessible with traditional office measures. The ability of passively collected smartphone data, on ambulation and location, to enable assessments of physical activity and socialization that meaningfully integrate motor and non-motor functions will be investigated. This project leverages the major phase 3 trial investments of NIH and study subjects by extending their follow up to establish the utility of tele-health metrics for tracking PD progression in clinical cohorts. Its results are expected to accelerate effective testing of a greater number of promising candidates for disease modification. The results will have an immediate impact on the rational selection of smartphone versus tele-visit monitoring in designing the next generation of PD trials, and may help identify and substantiate novel digital as well as molecular biomarkers of PD.

抽象的 疾病的修饰是帕金森氏病（PD）治疗中的主要未满足需求。目前临床 有前途的候选神经保护剂的试验受到反复反复的效率低下和不准确的限制 帕金森氏症患者自愿参加这些研究所需的人临床评估。 新兴远程医疗和基于智能手机的遥控传感器评估提供了帮助 克服这些障碍并改善未来试验成功的前景。证明了实用性 这些相对便宜的可访问的电信平台用于测量PD的进展 还要在完成介入研究后建立基础设施，以对参与者进行长期随访。 Steady-PD3和Sure-PD3是潜在疾病改良的活跃Ninds资助的3期试验 PD干预措施。这两项研究包括约600名早期PD受试者，包括收集生物标志物子研究 DNA和串行等离子体样品以及子集串行多巴胺转运蛋白（DAT）神经成像数据 成为帕金森氏病生物标志物计划（PDBP）的一部分。该提议的目的是 利用现代技术来开发，试行和实施100％虚拟模型，以进行长期跟进 利用远程医疗和智能手机平台来定量监测临床医生和患者报告 结果。特定目标（SA）1是为第三阶段试验的纵向远程跟进建立基础设施 队列； a）从基于站点的集中协调中心过渡以管理所有主题活动，b） 进行年度远程医疗研究访问（Tele-Visit）计划，c）实施智能手机数据 针对临床试验队列量身定制的收集平台。 SA2将比较智能手机（患者驱动）与电视访问 （临床医生驱动的）结果通过a）将这些平台的组件和复合特征及其相关联。 多年来的变化，b）将其能力与i）测量PD进展； ii）区分快速与慢速 基于基线运动量表得分，DAT赤字或血清尿酸尿酸盐水平的进度者；和iii）演示 在其各自的试验中，以radipine或肌苷的任何影响的持续存在。 SA3将探索新颖的智能手机 - 基于现实生活中的PD残疾生物标志物及其进展通常是无法访问的 传统的办公措施。被动收集的智能手机数据，在行动和位置上的能力， 启用对体育锻炼和社会化的评估，这有意义地整合了电动机和非运动器 功能将进行研究。该项目利用NIH的主要3阶段试用投资和研究 通过扩展其后续行动以建立电信指标的实用性来跟踪PD进展 临床队列。预计其结果将加速对更多有希望的有效测试 疾病修饰的候选人。结果将对合理选择的立即影响 在设计下一代PD试验时，智能手机与电视监视监视，可能有助于识别和识别和 证实PD的新型数字和分子生物标志物。