Validating Community-Based Measures of Activity in Ambulatory Duchenne Muscular Dystrophy
Abstract
Background: Development of novel technologies and therapeutic agents to treat Duchenne muscular dystrophy (DMD) has increased interest by regulatory bodies such as the Food and Drug Administration in the development of “clinically meaningful” study endpoints for clinical trials. There is a need for the development of valid measures of ambulatory ability in very young children while effectively evaluating treatment effects in clinical trials. Objective: This proposal focuses on validating the use of patient-generated step and activity data to facilitate future data collection at the point-of-care to support the development of stride rate patterns as an outcome measure for ambulatory patients with DMD. It is to include step activity monitoring by StepWatch™ and a consumer level step monitoring devices (Garmin VivoActiveHR and Polar M400 activity watches and the Polar StrideSensor activity monitor) that can feed data through Apple HealthKit into the UC Davis [University of California at Davis] instance of the Epic electronic medical record system. Up to 60 ambulatory patients with DMD will be recruited to represent the early, middle, and late stages of the progressive loss of ambulation in DMD. Patients will be sorted into three age groups of (n=20) participants: early (ages 2-6), middle (ages 7-11), and late (ages 12-16). Up to 150 control participants will be recruited and split into similar age ranges. Participants will be assessed using step monitoring devices and traditionally performed clinical outcome measures (6MWT, TFTs, NSAA), followed by community monitoring. StepWatch™ data collected from two previously enrolled cohorts of children with DMD will also be used for longitudinal analyses and determination of responsiveness to steroid treatment. We will develop model plans for data collection, validation, visualization, transfer, archival and lock of raw instrument data, as well as guidelines for integration with existing data platforms including Epic electronic health records and I2B2 NIH [National Institutes of Health] data repository. Applicability: Well-designed community mobility measures can be used in both clinical trials and day-to-day clinical practice. For clinical trials, they provide researchers with the ability to measure day-to-day walking ability across a broad range of ages, including very young children with DMD. Those results can then also be compared to other clinical trial measures such functional evaluations and timed function tests to help teach researchers and regulatory authorities about how “in clinic” tests commonly used in clinical trials relate to a person’s mobility in his/her daily life and whether those tests are “clinically meaningful.” Within 3 years, this project will be able to produce such a tool. Impact and Contributions: Data from this project will provide the basis for development of a “clinical trial-ready” community mobility measure that has been constructed against a background of comprehensive clinical assessments of functional ability across the range of ambulatory ability. This measure will be rapidly usable as a sensitive measure for use in the growing field of DMD clinical trials, and will help to demonstrate “clinically meaningful” results to regulatory agencies in charge of new drug approval.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Oct 29, 2018
- Source ID
- W81XWH1710477
Entities
People
- Craig M. McDonald
Organizations
- United States Army
- University of California, Davis