Home Monitoring of Metabolic Disorders

代谢紊乱的家庭监测

基本信息

批准号：
10699781
负责人：
Marylaura Lind Thomas
金额：
$ 54.21万
依托单位：
SEQUITUR HEALTH CORP.
依托单位国家：
美国
项目类别：
财政年份：
2023
资助国家：
美国
起止时间：
2023-09-25 至 2026-08-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10699781
关键词：
Acute Age Ammonia Behavior Blinded Blood Caregivers Cessation of life Chronic Clinical Clinical Trials Cognition Consensus Data Data Analyses Databases Development Devices Diet Disease Drops Emergency department visit Ensure Event Fasting Foundations Frequencies Funding Future Genetic Goals Health Health Sciences Hereditary Disease Home Home environment Hyperammonemia Impaired cognition Individual Institutional Review Boards Intellectual functioning disability Intervention Knowledge Laboratories Manuscripts Marketing Measurement Measures Medical Medical Records Medical emergency Metabolic Metabolic Diseases Metabolism Monitor Morbidity - disease rate Neurologic Neurologic Dysfunctions Neurologic Symptoms Oregon Outcome Participant Patient Care Patient Recruitments Patients Performance Persons Pharmaceutical Preparations Physicians Physiology Plasma Population Prevention Preventive measure Recommendation Research Risk Safety Science Signal Transduction Small Business Innovation Research Grant Surveys Symptoms Target Populations Testing Thinking Time United States Food and Drug Administration United States National Institutes of Health Universities Urea cycle disorders Work clinically relevant cohort commercialization disability effective therapy experience fatty acid oxidation health equity high risk improved innovation innovative technologies interest medical attention meetings member meter mortality participant enrollment patient population point of care prevent real time monitoring recruit response time use

项目摘要

PROJECT SUMMARY This proposal is submitted in direct response to NOT-HD-22-009 Notice of Special Interest (NOSI): Innovative Technologies to Improve Assessments, Interventions, and Outcomes for Individuals with Intellectual and Developmental Disabilities [IDD] (R43/R44). Persons of all ages with certain metabolic disorders are at-risk for metabolic crises when a certain metabolite accumulates, while this metabolite can be measured in a clinical laboratory, currently there is no home-based device to monitor the metabolie that causes the metabolic crises. Our team’s long-term goal is to reduce the morbidity and mortality of metabolic crises associated with certain metabolic accumulation. Through previous and ongoing National Science Foundation and National Institutes of Health Small Business Innovation Research funding, we have developed an at-home device to accurately measure this metabolite from a drop of blood and are working towards FDA clearance. The overall objective of this proposal is to investigate the impact of daily home-based monitoring of the target metabolite using the device we developed. This will be the first time that at-home measurement of the metabolite will be measured in the target population at home. Our central hypothesis is that when participants measure at-home, fasting, morning metabolite levels there will be an association between getting an elevated metabolite result and then the patient taking further action to contacting their medical team to further monitor or take steps to reduce the metabolite level. Our specific aims are 1) to perform a clinical trial in 10 participants with disorders resulting in risk of elevated metabolite levels, 2) to analyze the extensive data collected, and 3) to prepare a manuscript of the results. To complete our approach we will recruit patients from the available patient population at Oregon Health & Science University. We will collect extensive data from the participant medical record, observations, and the device daily metabolite measurements. This research is innovative because, for the first time, it provides a straightforward, home-based device for measurement of the target metabolite. At the conclusion of this project, we expect to understand more the impact of home-based metabolite monitoring on patient care.

项目概要本提案是直接响应 NOT-HD-22-009 特别利益通知 (NOSI)：创新而提交的改善智力和智力个人的评估、干预和结果的技术发育障碍 [IDD] (R43/R44) 患有某些代谢紊乱的所有年龄段的人都有患上这种疾病的风险。当某种代谢物积累时就会出现代谢危机，而这种代谢物可以在临床中进行测量实验室中，目前还没有家用设备来监测导致代谢危机的新陈代谢。我们团队的长期目标是降低与代谢危机相关的发病率和死亡率通过先前和正在进行的国家科学基金会和国家科学基金的一定代谢积累。在健康研究所小型企业创新研究资助下，我们开发了一种家用设备从一滴血中准确测量这种代谢物，并正在努力获得 FDA 的总体许可。该提案的目的是调查日常家庭监测目标代谢物的影响使用我们开发的设备，这将是首次在家测量代谢物。在家里的目标人群中进行测量。我们的中心假设是，当参与者在家中测量空腹的早晨代谢水平时代谢物结果升高与患者进一步服用药物之间存在关联采取行动联系他们的医疗团队以进一步监测或采取措施降低代谢水平。我们的具体目标是 1) 对 10 名患有导致以下风险的疾病的参与者进行临床试验代谢物水平升高，2) 分析收集到的大量数据，3) 准备一份手稿为了完成我们的方法，我们将从俄勒冈州现有的患者群体中招募患者。健康与科学大学。我们将从参与者的医疗记录、观察结果中收集大量数据。以及设备每日代谢测量。这项研究具有创新性，因为它首次提供了一种简单的家用设备用于测量目标代谢物在该项目结束时，我们希望了解更多家庭代谢监测对患者护理的影响。