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

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.

摘要 疾病修饰是帕金森病（PD）治疗中主要未满足的需求。目前临床 有希望的候选神经保护剂的试验受到重复的低效率和不精确性的限制， 志愿参加这些研究的帕金森氏症患者所需的个人临床评估。 新兴的远程医疗和基于智能手机的遥感评估提供了帮助的机会 克服这些障碍，改善今后审判成功的前景。演示的效用 这些用于测量PD进展的相对便宜的可访问远程健康平台将 还建立基础设施，以便在干预研究完成后对参与者进行长期跟踪。 STEADY-PD 3和SURE-PD 3是NINDS资助的3期临床试验， PD的干预措施。这两项研究均包含约600例早期PD受试者，包括生物标志物子研究，收集 DNA和系列血浆样本以及一个子集中的系列多巴胺转运蛋白（DAT）神经成像数据， 成为帕金森病生物标志物计划（PDBP）的一部分。这项建议的目的是 利用现代技术开发、试点和实施100%虚拟模式，以便长期跟踪 利用远程医疗和智能手机平台对临床医生和患者报告的 结果。具体目标（SA）1是建立III期试验纵向远程随访的基础设施 队列; a）从基于研究中心的协调中心过渡到集中协调中心，以管理所有受试者活动，B） 进行年度远程医疗研究访问（远程访问）计划，以及c）实施智能手机数据 为临床试验队列量身定制的收集平台。SA 2旨在比较智能手机（患者驱动）与远程访视 （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 多年的变化，以及B）比较他们的能力，i）测量PD进展; ii）区分快速与缓慢 基于基线运动量表评分、DAT缺陷或血清尿酸盐水平的进展者;和iii）证明 伊拉地平或肌苷在各自试验中的任何作用的持续性。SA 3是探索新的智能手机- 基于PD残疾及其进展的现实生活中的移动性生物标志物，这些生物标志物通常无法使用 传统的办公室。被动收集的智能手机数据（关于手机和位置）能够 使身体活动和社会化的评估，有意义地整合运动和非运动 功能将被调查。该项目利用了NIH的主要3期试验投资和研究 受试者通过延长随访时间，以确定远程健康指标在跟踪PD进展中的效用， 临床队列。预计其结果将加速对更多有希望的 疾病修饰的候选人。其结果将直接影响到合理选择 智能手机与远程访问监测在设计下一代PD试验，并可能有助于识别和 证实新的数字以及PD的分子生物标志物。

项目成果

Design of a virtual longitudinal observational study in Parkinson's disease (AT-HOME PD).

• DOI: 10.1002/acn3.51236
• 发表时间: 2021-03
• 期刊: Annals of clinical and translational neurology
• 影响因子: 5.3
• 作者: Schneider RB;Omberg L;Macklin EA;Daeschler M;Bataille L;Anthwal S;Myers TL;Baloga E;Duquette S;Snyder P;Amodeo K;Tarolli CG;Adams JL;Callahan KF;Gottesman J;Kopil CM;Lungu C;Ascherio A;Beck JC;Biglan K;Espay AJ;Tanner C;Oakes D;Shoulson I;Novak D;Kayson E;Ray Dorsey E;Mangravite L;Schwarzschild MA;Simuni T;Parkinson Study Group AT-HOME PD Investigators
• 通讯作者: Parkinson Study Group AT-HOME PD Investigators

Recruitment for Remote Decentralized Studies in Parkinson's Disease.

• DOI: 10.3233/jpd-212935
• 发表时间: 2022
• 期刊: Journal of Parkinson's disease
• 作者: Myers TL;Augustine EF;Baloga E;Daeschler M;Cannon P;Rowbotham H;Chanoff E;23andMe Research Team;Jensen-Roberts S;Soto J;Holloway RG;Marras C;Tanner CM;Dorsey ER;Schneider RB
• 通讯作者: Schneider RB