The U.S. Food and Drug Administration (FDA) is supporting financially a study into how well wearable sensors measure two early signs of Huntington’s disease, which could be of use to clinical trials of potential treatments.
The funding comes from the agency’s Digital Health Technologies for Drug Development program, and the study will be led by researchers at the University of Rochester Medical Center (URMC)’s Center for Health and Technology, who plan to begin enrolling participants later this year.
Researchers will focus on validating the reliability and accuracy of two digital measures in tracking Huntington’s progression at early stages, when disease symptoms are not yet fully manifest, relative to measures in a control group of people without this disease. The measures to be assessed are walking patterns or gait, and chorea, or the jerky, uncontrolled movements that are a hallmark of Huntington’s.
Motor symptoms like walking difficulties may be subtle in early disease stages
“Huntington’s is a genetic disease with a long pre-symptomatic stage,” Jamie Adams, MD, the study’s principal investigator, and a neurologist and an associate professor at URMC, said in a university news release. “This study employs a new staging system and focuses on the early stages, which are the likely target populations of disease-modifying clinical trials.”
Huntington’s symptoms usually begin to manifest between ages 30 and 50 — earlier in juvenile Huntington’s — and gradually worsen over time. These typically include motor symptoms such as chorea and difficulty walking, as well as nonmotor symptoms such as difficulty concentrating and mood swings.
“Gait and chorea are meaningful measures in Huntington’s disease, and this study will help confirm that,” Adams said. “They’re both significant features of the disease that … contribute to functional decline, and are present, we think, early in the disease in a subtle way.”
Scientists at the Rochester center have experience in using digital health tools, such as smartphones and smartwatches, to measure early changes in Parkinson’s disease, another neurodegenerative disorder marked by motor and nonmotor symptoms.
“Similar to Parkinson’s, our traditional measures of Huntington’s disease are subjective, episodic, and have not reliably been sensitive to progression, especially in early disease,” Adams said.
Portable digital health tools are transforming how motor symptoms can be monitored. Worn by a patient or placed in their home or environment, they allow doctors to collect objective data remotely in real time, without requiring frequent clinic visits. Some measures could help in identifying someone at risk of developing a disease before overt symptoms appear.
In the planned study, people with early-stage Huntington’s and healthy individuals will wear sensors around their wrists and trunk that collect data on gait and chorea. The researchers will analyze findings to make sure data collected are meaningful to those with the disease, using an innovative symptom mapping approach they also intend to evaluate, and look at how movement symptoms affect daily life.
An ultimate goal is to determine if the digital measures can serve as objective measures of efficacy for experimental treatments in clinical trials in Huntington’s patients.
The amount of FDA financing given for this work was not disclosed.
