Home Monitoring of Metabolic Disorders

项目总结

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.