ARISE: Duchenne Muscular Dystrophy Video Assessment Registry
Study Details
Study Description
Brief Summary
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).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Detailed Description
This is a longitudinal, observational, non-randomized study enrolling up to 180 participants with DMD. Participants will be asked to participate in remote DVA at baseline, baseline retest, and Months 6, 12, 18 and 24 and/or the NSAA and/or PUL 2.0 at baseline, and Months 12 and 24 based on their ambulatory status. Participants will also complete electronic questionnaires on demographic information and report on treatment status and disorder progression (i.e., ambulation status, changes in medication). Participants will not be asked to stop or change any of their current treatments, and they are not prohibited from initiating treatments during this observational study.
This study will be remotely conducted (all visits and captures performed at home).
Study Design
Outcome Measures
Primary Outcome Measures
- DVA Database [24 months]
To create a database of DVA data including test-retest and longitudinal captures that can be used to further develop and validate the measure.
Secondary Outcome Measures
- North Star Ambulatory Assessment [24 months]
To capture the North Star ambulatory Assessment (NSAA), a DMD assessments of physical function for the purposes of validating the DVA and establishing the measure's sensitivity to change. The NSAA is a 17-item rating scale, where activities are graded on an ordinal scale from 0-2, yielding 24 as the maximum which indicates fully-independent function. Scoring values range from 2: "Normal"- no obvious modification of activity; 1: Modified method but achieves goal independent of physical assistance from another; 0: Unable to achieve independently.
- Performance of Upper Limb [24 months]
To capture the Performance of Upper Limb 2.0 (PUL 2.0) for the purposes of validating the DVA and establishing the measure's sensitivity to change. The PUL 2.0 is 22-items graded on an ordinal scale from 0-2, in three dimensions (shoulder, mid-, distal), yielding the max global score of 42. The maximum score for the shoulder level is 12, the maximum score for the mid-level is 17, for the distal level is 13. Scoring values range from 0: unable to 2: complete the activity with noted degrees of compensations.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Participant is 2 years or older at time of consent
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Participant is willing and able to participate and provide informed consent; if between the ages of 7 to the day before the age of majority in the participant's location is willing and able to provide assent and has a parent or legal guardian who is willing and able to provide consent
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Participant and recording partner must speak, read, and understand English proficiently
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Provide confirmation and documentation of the participant's clinical medical diagnosis of Duchenne muscular dystrophy (i.e., genetic report, clinic note)
Exclusion Criteria:
- The participant is unable to perform the remote video capture tasks specified by the Duchenne Video Assessment, North Star Ambulatory Assessment or Performance of Upper Limb 2.0, as specified by the branching questionnaire
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Casimir | Plymouth | Massachusetts | United States | 02360 |
Sponsors and Collaborators
- The Emmes Company, LLC
- Department of Defense Duchenne Muscular Dystrophy Research Program
- Parent Project Muscular Dystrophy
- Ultragenyx Pharmaceutical Inc
Investigators
- Principal Investigator: Alyssa Burns, Casimir
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CAS-CAS005-01