Duchenne Muscular Dystrophy Video Assessment Registry (ARISE)

March 6, 2026 updated by: The Emmes Company, LLC
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).

Study Overview

Status

Completed

Conditions

Detailed Description

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 (Actual)

150

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Maryland
      • Rockville, Maryland, United States, 20850
        • Emmes

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants will be recruited for this study through referrals from collaborators and advocacy groups as well as through direct marketing via social media, email, phone, or other appropriate methods. Advocacy groups will assist with the facilitation of recruitment by making recruitment materials available to the Duchenne community. Participants who provided consent to be contacted by Casimir staff for future studies and shared their contact information with Casimir staff may be contacted. Potential participants may also share their contact information directly with Casimir study staff to learn about the study. Additionally, recruitment for the study may also occur through postings with patient advocacy groups and on social media. Casimir will supply the advocacy groups with language for website postings and group emails.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Duchenne Video Assessment (DVA)
Time Frame: 24 months
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)
Time Frame: 24 months
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)
Time Frame: 24 months
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
Time Frame: 24 months
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The Emmes Company, LLC

Collaborators

United States Department of Defense

Investigators

  • Principal Investigator: Marielle Contesse, PhD, Emmes Endpoint Solutions

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2022

Primary Completion (Actual)

January 11, 2026

Study Completion (Actual)

January 11, 2026

Study Registration Dates

First Submitted

November 30, 2022

First Submitted That Met QC Criteria

February 1, 2023

First Posted (Actual)

February 3, 2023

Study Record Updates

Last Update Posted (Actual)

March 10, 2026

Last Update Submitted That Met QC Criteria

March 6, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAS-CAS005-01

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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