Validating a Scalable, Open Science Framework for Collecting Laboratory-Grade Data Remotely in Specialized Populations

验证可扩展的开放科学框架，用于在特殊人群中远程收集实验室级数据

• 批准号: 10289016
• 负责人: Bridgette Lynne Kelleher
• 金额: $ 23.05万
• 依托单位: PURDUE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-09-01 至 2023-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10289016
• 关键词: Acute; Address; Assessment tool; Attention; Behavioral; Biological Assay; COVID-19 pandemic; Calibration; Caregivers; Child; Clinic; Clinical; Clinical Research; Clinical Sciences; Clinical Trials; Cognitive; Computer software; Data; Data Collection; Development; E-learning; Ensure; Equipment; Failure; Floor; Fragile X Syndrome; Funding; Heart Rate; Human Resources; Individual; Intellectual functioning disability; Laboratories; Measures; Methods; Modeling; Monitor; Multi-Institutional Clinical Trial; Natural History; Outcome; Outcome Measure; Output; Parents; Participant; Patient Monitoring; Patients; Pharmaceutical Preparations; Population; Positioning Attribute; Procedures; Process; Protocols documentation; Psychophysiology; Publishing; Research; Research Personnel; Resources; Science; Signal Transduction; Site; Standardization; Symptoms; Techniques; Testing; Time; Training; Translating; Travel; United States National Institutes of Health; base; clinical outcome measures; cohort; cost; cost effective; data analysis pipeline; data standards; efficacy validation; graphical user interface; high risk population; improved; innovation; network models; open data; patient response; peer; programs; remote delivery; self-directed learning; skills; sustained attention; telehealth; tool; treatment trial; user friendly software; user-friendly; virtual

PROJECT SUMMARY The limited repertoire of clinical outcome measures suitable for children with intellectual and developmental disabilities (IDD) is compromising the promise of clinical trials. Non-standardized laboratory techniques that capture “spectral” signals – such as psychophysiological assays – are increasingly recognized as promising methods for monitoring patient responses over time. However these tools require specialized equipment and personnel to administer and are not easily deployed via telehealth, biasing spectral studies toward patients who are able and willing to travel to clinics. Thus, there is a significant gap in available telehealth-based protocols for collecting laboratory-grade data remotely in IDD populations. The present study addresses this gap by developing a comprehensive training protocol for PANDABox (Parent Assisted Neurodevelopmental Assessment), an open-science, telehealth-based assessment protocol that PI Kelleher developed for remotely collecting high quality, integrated clinical, behavioral, and spectral assays from participants with IDD. Published findings indicate that PANDABox is highly feasible and acceptable to caregivers and generates high quality, integrated, “laboratory-grade” data at low cost. Already, PANDABox is being deployed in a variety of treatment and natural history studies across IDD populations and is being translated to Spanish to promote accessibility. The present study aims to enhance the scalability of PANDABox by accomplishing two specific aims. First, we will develop and validate an open-science training protocol and peer-to-peer reliability network to facilitate standardized, cross-laboratory implementation of the PANDABox protocol in IDD. This protocol will include both a virtual training hub for self-paced training, as well as a reliability network to facilitate cross-site calibration. Second, we will create user-friendly software program to support users to efficiently process the PANDABox attention assay, which has produced promising outcomes in clinical trials but requires computational expertise to analyze, limiting its scalability. Addressing these gaps would shift the status quo of clinical science in IDD by (1) providing a scalable, accessible protocol for collecting laboratory- grade data remotely in IDD populations and (2) producing standardized data outputs necessary for large scale, multi-site, collaborative science.

项目概要 适合智力低下儿童的临床结果测量手段有限 发育障碍（IDD）正在损害非标准化临床试验的前景。 捕获“光谱”信号的实验室技术——例如心理生理学测定—— 越来越多地被认为是随着时间的推移监测患者反应的有前途的方法。 然而，这些工具需要专门的设备和人员来管理，并且不是 通过远程医疗轻松部署，将光谱研究偏向有能力且愿意的患者 因此，可用的基于远程医疗的协议存在很大差距。 本研究解决了 IDD 人群远程收集实验室级数据的问题。 通过为 PANDABox（家长辅助）制定全面的培训协议来弥补差距 神经发育评估），一种开放科学、基于远程医疗的评估协议， PI Kelleher 开发用于远程收集高质量、集成的临床、行为和 对患有 IDD 的参与者进行的光谱分析已发表的研究结果表明 PANDABox 具有高度的敏感性。 可行且为护理人员所接受，并产生高质量、集成的“实验室级” PANDABox 已经被部署在各种治疗和自然领域。 针对 IDD 人群的历史研究，正在被翻译成西班牙语以促进 本研究旨在通过实现可访问性来增强 PANDABox 的可扩展性。 首先，我们将制定并验证开放科学培训协议。 点对点可靠性网络，以促进标准化、跨实验室实施 IDD 中的 PANDABox 协议 该协议将包括一个用于自定进度的虚拟培训中心。 其次，我们将创建一个可靠性网络以促进跨站点校准。 用户友好的软件程序，支持用户高效处理PANDABox注意力 测定，在临床试验中产生了有希望的结果，但需要计算 专业知识来分析，限制其可扩展性，解决这些差距将改变现状。 IDD 的临床科学通过 (1) 提供可扩展、可访问的协议来收集实验室数据 对 IDD 人群中的数据进行远程分级，以及 (2) 生成必要的标准化数据输出 用于大规模、多地点、协作科学。