ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the Duchenne Video Assessment (DVA).
ARISE is a longitudinal, observational study of individuals with Duchenne muscular dystrophy (DMD). The Duchenne Video Assessment (DVA) is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients doing specific movement activities at home using a secure mobile application. In this study, participants will remotely complete the DVA in addition to existing DMD functional assessments (North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL 2.0)) to evaluate the longitudinal measurement properties of the DVA.
Study Type
OBSERVATIONAL
Enrollment
150
Emmes
Rockville, Maryland, United States
Duchenne Video Assessment (DVA)
The DVA is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA includes 18 standardized movement tasks that are assigned to participants based on functional subgroup. The DVA directs and trains caregivers to video record patients doing specific movement tasks at home using a secure mobile application. DVA videos are scored by DVA-certified physical therapists using validated scorecards with prespecified compensatory movement criteria.
Time frame: 24 months
North Star Ambulatory Assessment (NSAA)
The NSAA is a 17-item tool used to measure functional motor abilities in ambulatory individuals with DMD. Physical therapists with experience administering the NSAA will remotely administer and record the live NSAA sessions via a secure video conference platform. Participants will receive the appropriate standardized testing equipment.
Time frame: 24 months
Performance of Upper Limb 2.0 (PUL 2.0)
The PUL 2.0 is a 22-item rating scale used to measure upper limb abilities in individuals with DMD. The test includes an entry test item to define starting functional level and 21 test items subdivided into shoulder level, elbow level, and distal level. Physical therapists with experience administering the PUL 2.0 will remotely administer and record the live PUL 2.0 sessions via secure video conference platform. Participants will receive the appropriate standardized testing equipment.
Time frame: 24 months
Caregiver Task-Specific Impression of Change
At each follow-up assessment timepoint, parents/legal guardians or recording partners will complete an electronic questionnaire to rate their impression of change in the participant's ability to perform each DVA movement task since the prior timepoint.
Time frame: 24 months
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