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

项目摘要 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）的进展和对治疗的反应。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，在此期间获得评估， 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给定患者的下降速率的估计- 临床试验的主要结果--具有很大的不确定性。此外，访问这些中心是一个主要的 患者和护理人员的负担;许多患者必须退出，因为他们变得越来越虚弱 在研究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。的一种方法 改善我们的评估，增加便利性，鼓励更广泛的入学， 在家中更频繁地进行生物标志物评估。随着互联网Skype®的出现， 和无线技术，因此护理人员或患者对在家中收集数据产生了兴趣。而 仍需要进行安全性评估、药物核对和抽血，这些可以通过以下方式进行： 当地经过培训的卫生专业人员（如护士和医疗助理），并可获得同意 电子化，使下一代ALS和其他神经肌肉疾病的临床试验成为可能 在很大程度上，远程发生的疾病。此外，这些程序一旦实施， 除了临床试验之外，还可以帮助患者的常规管理，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 开发和完善电阻抗肌电描记术（EIM）技术用于临床。基础上 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 建议设计和开发一种用于家庭医疗监测的设备。我们的重点是肌萎缩侧索硬化症，但这个设备 也可以很容易地用于其他疾病，包括肌营养不良症、肌炎和失调。我们 这项拟议的2B期研究的具体目标包括：1.开发一种用户友好的家用设备（ mSelfTM）用于高度可靠的临床试验质量肌肉数据收集; 2.开发专用软件， 后端信息技术（IT）基础设施，以确保易用性，持续向用户提供反馈，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 很好地定位于寻求FDA批准，并将寻求建立这个系统作为ALS和其他疾病的生物标志物。 神经肌肉疾病

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.