Duchenne Muscular Dystrophy Video Assessment Registry

NCT05712447 | Completed

• 1 other identifier: CAS-CAS005-01
• Study Type: observational
• Target: 150
• Locations: 1 country
• Sites: 1

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).

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne; DMD; Remote video assessment

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• North Star Ambulatory Assessment (NSAA)

  24 months

• Performance of Upper Limb 2.0 (PUL 2.0)

  24 months

• Caregiver Task-Specific Impression of Change

  24 months

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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)

You may not qualify if:

• Participants will be excluded if they are unable to use a hand to hold a pen, pick up pennies or drive a powerchair.

Sponsors & Collaborators

• The Emmes Company, LLC: lead
• United States Department of Defense: collaborator

Study Sites (1)

Emmes

Rockville, Maryland, 20850, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Marielle Contesse, PhD

  Emmes Endpoint Solutions

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2022
• First Posted: February 3, 2023
• Study Start: September 1, 2022
• Primary Completion: January 11, 2026
• Study Completion: January 11, 2026
• Last Updated: March 10, 2026

Record last verified: 2026-02

Locations

US (1)