Clinical trial NCT05712447: Duchenne Muscular Dystrophy Video Assessment Registry
Official title: Duchenne Muscular Dystrophy Video Assessment Registry
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne
DMD
Remote video assessment
Lead sponsor: The Emmes Company, LLC
Sponsor class: INDUSTRY
Collaborators: name: Department of Defense Duchenne Muscular Dystrophy Research Program
First posted: 2023-02-03
Enrollment: 180
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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).

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Duchenne Video Assessment (DVA)
timeFrame: 24 months
Secondary outcomes:
measure: North Star Ambulatory Assessment (NSAA)
timeFrame: 24 months
measure: Performance of Upper Limb 2.0 (PUL 2.0)
timeFrame: 24 months
measure: Caregiver Task-Specific Impression of Change
timeFrame: 24 months
Other outcomes:
Locations:
city: Rockville
country: United States
facility: Emmes
state: Maryland
Study officials:
affiliation: Emmes Endpoint Solutions
name: Marielle Contesse, PhD
role: PRINCIPAL_INVESTIGATOR
References: