Electrical impedance myography technology for quantitative, at-home muscle assessment in amyotrophic lateral sclerosis and other neuromuscular disorders.

电阻抗肌电描记技术用于肌萎缩侧索硬化症和其他神经肌肉疾病的定量、家庭肌肉评估。

• 批准号: 9769159
• 负责人: Elmer C Lupton
• 金额: $ 63.28万
• 依托单位: MYOLEX, INC.
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-04-01 至 2022-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9769159
• 关键词: Algorithms; Amyotrophic Lateral Sclerosis; Biological Markers; Blood; Caregivers; Clinical; Clinical Research; Clinical Trials; Computer software; Consent; Country; Data; Data Collection; Data Quality; Databases; Development; Device Designs; Devices; Disease; Disease Progression; Dose; Effectiveness; Electrodes; Enrollment; Ensure; Feedback; Frequencies; Funding; Health Insurance Portability and Accountability Act; Health Professional; Home environment; Information Technology; Infrastructure; Internet; Limb structure; Measurement; Measures; Medical; Metals; Modification; Monitor; Morphologic artifacts; Motor; Muscle; Muscular Dystrophies; Myography; Myositis; Neuromuscular Diseases; Neuromuscular conditions; Nurses; Outcome; Output; Participant; Patient Care; Patients; Pharmaceutical Preparations; Phase; Positioning Attribute; Procedures; Research Personnel; Secure; Skin; Small Business Innovation Research Grant; Speech; System; Technology; Therapeutic Effect; Time; Transportation; Uncertainty; Visit; Vital capacity; Wireless Technology; Work; deconditioning; design; electric impedance; fitness; grasp; handheld equipment; health training; improved; individual patient; interest; next generation; novel; pulmonary function; recruit; response; safety assessment; screening; smartphone Application; software development; standard measure; tertiary care; tool; touchscreen; user-friendly

Project Summary To improve the efficiency, convenience and quality of ALS clinical trials, we need better biomarkers for assessing amyotrophic lateral sclerosis (ALS) progression and response to therapy. All recent ALS trials require patients to visit an ALS clinical trials center every 2-3 months at which time assessments are obtained, including limb strength, vital capacity, and the ALS functional rating scale-revised (ALSFRS-R) score. However, such infrequent assessments mean that the estimation of the rate of decline for a given patient--the major outcome of interest in clinical trials--has great uncertainty. Moreover, visits to such centers are a major burden for the patients and caregivers; many patients must withdraw as they become increasingly debilitated during the study. It also means that many potentially eligible people cannot participate because they lack convenient transportation or simply live too far away from a participating tertiary care center. One approach for both improving our assessments and increasing convenience and encouraging broader enrollment is to perform biomarker assessment on a more frequent basis at home. Given the advent of the Internet, Skype®, and wireless technologies, there has been interest in collecting data at home by caregivers or patients. While safety assessments, medication reconciliation, and blood draws will still be needed, these can be performed by local trained health professionals (e.g. nurses and medical assistants) and consent can be obtained electronically, making it possible for the next generation of clinical trials in ALS and other neuromuscular disorders to take place, to a great extent, remotely. Moreover, such procedures, once in place, could extend beyond clinical trials, to assist in the routine management of patients, to monitor therapy effectiveness, including the need for dose adjustment or therapy discontinuation. Myolex, Inc, has as its major focus, the development and refinement of electrical impedance myography (EIM) technology for clinical use. Building on a successful Phase 2 SBIR study that demonstrated the power of EIM to assess ALS progression, here we propose to design and develop a device for at-home medical monitoring. Our focus is on ALS, but this device could be readily used in other disorders, including muscular dystrophy, myositis, and deconditioning. Our specific aims of this proposed Phase 2B study include: 1. To develop a user-friendly at-home device (the mSelfTM) for highly reliable, clinical-trial quality muscle data collection; 2. To develop dedicated software and backend information technology (IT) infrastructure to ensure ease of use, ongoing feedback to the user, and secure data transfer and storage. 3. To complete a clinical study to assess disease progression with the mSelfTM and its ability to track disease versus standard measures. With the completion of this work, we will be well positioned to seek FDA-approval and will seek to establish this system as a biomarker in ALS and other neuromuscular conditions.

项目概要 为了提高 ALS 临床试验的效率、便利性和质量，我们需要更好的生物标志物 评估肌萎缩侧索硬化症 (ALS) 的进展和治疗反应。所有最近的 ALS 试验 要求患者每 2-3 个月访问一次 ALS 临床试验中心，以获得评估结果， 包括肢体力量、肺活量和 ALS 功能评定量表修订版 (ALSFRS-R) 评分。 然而，这种不频繁的评估意味着对特定患者的衰退率的估计—— 临床试验中感兴趣的主要结果具有很大的不确定性。此外，访问这些中心也是一项重要的活动。 患者和护理人员的负担；许多患者因变得越来越虚弱而必须退出 在学习期间。这也意味着许多潜在符合条件的人无法参与，因为他们缺乏 交通便利或只是住得离参与的三级护理中心太远。一种方法 改进我们的评估、增加便利性和鼓励更广泛的注册都是为了 在家更频繁地进行生物标志物评估。鉴于互联网、Skype® 的出现， 和无线技术，人们对护理人员或患者在家中收集数据产生了兴趣。尽管 仍然需要安全评估、药物协调和抽血，这些可以由 当地经过培训的卫生专业人员（例如护士和医疗助理）并可以获得同意 电子化，使 ALS 和其他神经肌肉疾病的下一代临床试验成为可能 在很大程度上，疾病是远程发生的。此外，此类程序一旦到位，可以延长 除了临床试验之外，协助患者的日常管理，监测治疗效果， 包括需要调整剂量或停止治疗。 Myolex, Inc. 的主要重点是 临床应用电阻抗肌动描记（EIM）技术的开发和完善。建立在 一项成功的 2 期 SBIR 研究证明了 EIM 评估 ALS 进展的能力，在这里我们 提议设计和开发一种用于家庭医疗监测的设备。我们的重点是 ALS，但是这个设备 可以很容易地用于其他疾病，包括肌营养不良症、肌炎和功能失调。我们的 这项拟议的 2B 阶段研究的具体目标包括： 1. 开发一种用户友好的家用设备（ mSelfTM）用于高度可靠、临床试验质量的肌肉数据收集； 2. 开发专用软件 后端信息技术 (IT) 基础设施，确保易用性、持续向用户反馈，以及 安全的数据传输和存储。 3. 完成一项临床研究以评估疾病进展 mSelfTM 及其跟踪疾病与标准测量的能力。随着这项工作的完成，我们将 已做好充分准备寻求 FDA 批准，并将寻求将该系统建立为 ALS 和其他疾病的生物标志物 神经肌肉状况。

项目成果

Reducing sample size requirements for future ALS clinical trials with a dedicated electrical impedance myography system.

• DOI: 10.1080/21678421.2018.1510008
• 发表时间: 2018-11
• 期刊: Amyotrophic lateral sclerosis & frontotemporal degeneration
• 影响因子: 2.8
• 作者: Shefner JM;Rutkove SB;Caress JB;Benatar M;David WS;Cartwright MS;Macklin EA;Bohorquez JL
• 通讯作者: Bohorquez JL