Incorporating Objective Assessments in Clinical Trials Testing Disease Modification in Early-Stage Parkinson s Disease
Abstract
FY19 PRP Focus Areas: (1) Clinical and research application of digital health technology for Parkinson s disease monitoring including early identification; (2) Sleep biology in Parkinson s disease. Objective: This study will test if new brain scans and wearable movement trackers should be added to Parkinson s disease (PD) studies that are focused on new treatments that may slow disease progression. The results of this study could improve many future Parkinson s disease studies and will directly influence an upcoming study of deep brain stimulation (DBS) in people with very early-stage PD. Rationale: Each year 60,000 Americans are newly diagnosed with Parkinson s disease. PD is a relentlessly progressive disease that causes major disability in over 1 million Americans, including 80,000 Veterans. Parkinson s disease causes progressive tremor, muscle stiffness, slowed movement, falls, and eventually dementia. Medications help at first but then slowly lose the ability to control symptoms. Studies to find new treatments that slow, stop, or cure Parkinson s must be conducted. DBS is approved by the Food and Drug Administration (FDA) for people with mid- and advanced-stage Parkinson s disease. The success of DBS for later Parkinson s disease stages raises the question of whether DBS given at a very early stage will provide added benefits to patients over and above standard medications. Researchers at Vanderbilt University completed the only study of DBS in early-stage Parkinson s disease (IDE G050016, NCT 0282152, CRC 1363, IRB 040797). The results of that study provided the first ever Class II evidence of a therapy slowing the progression of a motor feature of Parkinson s disease. That discovery was made possible by washing out study participants from all PD treatment for 7 days, a study design that allows researchers to track the underlying progression of the disease. Since the original study was completed, new methods have been developed to measure Parkinson s disease severity. This study will prepare researchers to launch a multicenter study testing DBS in very early-stage PD by first examining the feasibility of adding brain scans and wearable movement trackers to the 7-day washout. Positron emission tomography (PET) scans are used to detect differences in brain activity in people with Parkinson s disease. Two types of wearable movement trackers will be worn by patients to measure symptoms and side effects of medications. Many people with PD also have problems with sleep, and one of the wearable trackers provides details about sleep which will also be potentially included in the future study. Research Impact: This research project will provide completely new information to the Parkinson s research community, including data from PET scans and wearable movement trackers collected for the first time as part of a 7-day washout of PD treatments. Results from this project can be used in future studies of new treatments that may slow the progression of PD and will directly impact a future FDA-approved multicenter study of DBS in people with early-stage Parkinson s disease (IDE G050016). The results of the future study (positive or negative) will provide valuable information to patients, caregivers, and physicians on whether DBS is an appropriate therapy for people with very early-stage Parkinson s disease. PI Career Goals: After completing this study, Dr. Hacker will have advanced training and hands-on experience collecting PET scans and using wearable movement trackers in people with PD. She will also gain knowledge and skills that will enhance her ability to lead multidisciplinary research teams. Dr. Hacker s career development plan is designed to complement this research study by providing her with focused mentorship and formal training. Completing this study and training plan will help Dr. Hacker achieve her long-term goal of becoming an independent scientist leading studies that test new treatments with the potent
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Mar 10, 2021
- Source ID
- W81XWH2010374
Entities
People
- Mallory Hacker
Organizations
- United States Army
- Vanderbilt University