Duchenne Muscular Dystrophy Video Assessment Registry (ARISE)

The EMMES Corporation

Status: Active, not recruiting

Conditions: Duchenne Muscular Dystrophy

Study type: Observational

Funder types: Industry; Other

Identifiers

NCT05712447

CAS-CAS005-01

Details and patient eligibility

About

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).

Full 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.

Enrollment: 180 estimated patients

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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.