A new registry for Duchenne muscular dystrophy (DMD) video assessment is now recruiting participants. The prospective and longitudinal, observational, non-randomized study aims to create a database of functional motor abilities in DMD patients to support validation efforts of the Duchenne Video Assessment tool.

The study aims to enroll up to 180 participants with the disease who will participate in remote Duchenne video assessments twice at baseline and at 6, 12, 18, and 24 months with the North Star Ambulatory Assessment and/or Performance of Upper Limb 2.0 assessment at baseline and at 12 and 24 months, depending on ambulation status. 

Patients will also be asked to complete electronic questionnaires about their personal information and treatment status.

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The primary endpoint of the study is to create a database of Duchenne Video Assessment data. The secondary endpoints are to capture the North Star Ambulatory Assessment and the Performance of Upper Limb 2.0 to validate the video assessment and establish the measure’s sensitivity to change.

DMD patients ages 2 and above who have been medically diagnosed with DMD and who are able to perform the remote video capture tasks are eligible to participate in the trial, which started on September 1, 2022, and is estimated to be completed on September 1, 2027. 

Read more about DMD diagnosis

The study will take place at Casimir in Plymouth, Massachusetts, but all patient tasks will be performed at the patient’s home. It is sponsored by The Emmes Company, the US Department of Defense Duchenne Muscular Dystrophy Research Program, Parent Project Muscular Dystrophy, and Ultragenyx Pharmaceutical. 

The Duchenne Video Assessment assesses the quality of movement of a patient as an indication of disease severity. Caregivers will record patients while they are performing movement tasks, which will then be rated by physiotherapists using scorecards.

Reference

Duchenne muscular dystrophy video assessment registry (ARISE). US National Library of Medicine. Last updated February 3, 2023. Accessed February 9, 2023.

Cardiologists Gastroenterologists Neurologists Parent Project Muscular Dystrophy Physicians Pulmonologists Researchers The Emmes Company the US Department of Defense Duchenne Muscular Dystrophy Research Program Ultragenyx Pharmaceutical