FDA Funds Development of Digital Measures for Huntington's Disease
核心洞察
The FDA has awarded funding to the University of Rochester Medical Center (URMC) to develop digital measures for Huntington's disease (搜索).
The study will focus on identifying subtle, early signs of Huntington's disease (搜索) using wearable sensors to track gait and chorea.
Researchers aim to establish reliable and valid digital endpoints for clinical trials, accelerating the development of new therapies.
The Food and Drug Administration (FDA) is funding a University of Rochester Medical Center (URMC)-led initiative to develop new digital measures for Huntington's disease (搜索), with the goal of accelerating the development of new therapies. The research will focus on identifying subtle early signs of the disease through the use of wearable sensors.
The study, led by URMC neurologist Jamie Adams, MD, principal investigator, aims to address the limitations of current Huntington's disease (搜索) assessments, which are often subjective and not sensitive to early disease progression. "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," said Adams.
The FDA funding, provided under the Digital Health Technologies for Drug Development program, will enable URMC and its collaborators to evaluate the reliability, validity, and meaningfulness of digital measures of daily living mobility (gait) and chorea, a hallmark symptom of Huntington's disease (搜索) characterized by involuntary muscle movements. Data will be collected remotely and continuously using wrist- and trunk-worn digital sensors in individuals with early-stage Huntington's disease and control participants. The study will also incorporate qualitative research using symptom mapping to ensure the measures are meaningful to people with Huntington's disease.
"We believe that gait and chorea are meaningful measures in Huntington's disease (搜索), and this study will help confirm that. They're both significant features of the disease that worsen over time and contribute to functional decline, and are present, we think, early in the disease in a subtle way," Adams explained.
The researchers hope that these digital measures will serve as crucial endpoints for clinical trial outcomes. With the emergence of genetic and disease-modifying interventions for neurological disorders like Huntington's, there is a growing need for early and accurate assessment of motor impairments. The ultimate goal is to inform larger-scale validation efforts and clinical endpoint studies in Huntington's disease (搜索). The research may also have implications for other neurological diseases with involuntary movements, such as Parkinson's disease (搜索).
The research team includes Lori Quinn, PhD, with Columbia University, Jeffrey Hausdorff, PhD, with Tel Aviv University, and Jennifer Mammen, PhD, with the University of Massachusetts. Enrollment is expected to begin later this year.