Duchenne Muscular Dystrophy Video Assessment Registry

Duchenne Muscular Dystrophy Clinical Trial

— ARISE  

Official title:

Duchenne Muscular Dystrophy Video Assessment Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05712447
• Other study ID #: CAS-CAS005-01
• Status: Recruiting
• Start date: September 1, 2022
• Est. completion date: September 1, 2027

Study information

• Verified date: December 2023
• Source: The Emmes Company, LLC
• Contact: Katherine Pleasant
• Phone: 774-433-9313
• Email: kpleasant[@]emmes.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 180
• Est. completion date: September 1, 2027
• Est. primary completion date: September 1, 2025
• Gender: All
• Age group: 2 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
United States	Emmes	Rockville	Maryland

Sponsors (4)

Lead Sponsor	Collaborator
The Emmes Company, LLC	Department of Defense Duchenne Muscular Dystrophy Research Program, Parent Project Muscular Dystrophy, Ultragenyx Pharmaceutical Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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
Secondary	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
Secondary	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