Duchenne Muscular Dystrophy Video Assessment Registry

Study Overview
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Brief Summary
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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).

Detailed Description
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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.

Official Title
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Duchenne Muscular Dystrophy Video Assessment Registry

Conditions
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Duchenne Muscular Dystrophy

Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: At least 2 years of age at time of consent Written consent (English only) by adult participant or parent/legal guardian of minor participant Written assent (English only) if minor participant is at least 7 years of age Documentation provided for the participant's diagnosis of DMD (i.e., genetic report, clinic note) Exclusion Criteria: Participants will be excluded if they are unable to use a hand to hold a pen, pick up pennies or drive a powerchair.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Study Plan
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How is the study designed?
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What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 24 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 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. | 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. | 24 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Duchenne, DMD, Remote video assessment