= Fully recruited
= Not yet recruiting
= Enrolling by invitation
This decentralised study aims to evaluate whether new video-based electronic clinical outcome assessments (eCOAs) captured with the Atom5TM platform DMDhome are able to detect disease progression from the late ambulatory to transfer stage and early non-ambulatory stage, compared with standard validated clinical assessments.
This decentralised study will be fully conducted at the patient’s home. This study will enrol 28 ambulant and 16 non-ambulant DMD patients, and 15 age-/gender-matched healthy controls that can be siblings, friends or other family members. Participants must be 8 years of age and upwards.
Participants or/and their parents (if they are younger than 16) will self-onboard and provide electronic consent using the DMDhome web portal (htpps://dmdhome.atom5.co.uk). After the e-consent is signed, participants will be able to respond to screening questions and upload a proof of their DMD diagnosis, including genetic information if available. This proof could be a letter from their DMD specialist centre.
(PLEASE NOTE: The eligibility and e-consent process are meant to be accessed ideally from a computer, as it is designed as a web portal. The rest of the study procedures, ie. recording videos and answering PROs, can be done through the app).
Depending on your child’s current health state you will also be asked to provide:
Following registration and screening, if a participant is deemed eligible, the study team will schedule a visit from a trained physiotherapist who will attend the participant’s home to conduct the North Star Ambulatory Assessment, the Performance of the Upper Limb (PUL) 2.0 and the Egenklassification scale version 2.
The participant will also download the DMDhome study app and video record the following electronic clinical outcome assessments (eCOAs) using their smartphone:
Upper limb eCOAs (both ambulant and non-ambulant)
Lower limb eCOAs (ambulant)
Optional tasks that represent activities of daily living, e.g., drink from a glass, washing hands, feeding self, etc.
Participants will perform all assessments every 6 months and up to 24 months (5 time points).
Change from baseline in DMDhome upper limb/lower limb eCOAs compared with physiotherapist assisted NSAA and PUL2.0 total scores at 6, 12, 18 and 24 months.
Evaluate test-retest reliability of new DMDhome eCOAs.
Evaluate the sensitivity to change of new DMDhome eCOAs over 24 months compared with PUL2.0 and NSAA.
Estimate the Minimal Clinically Important Difference of DMDhome scores vs NSAA and PUL 2.0
Correlate new eCOAs to patient-reported outcome measures such as the DMD Upper Limb PROM.
Evaluate the quality of life of patients/carers.
Identification of digital parameters during activities of daily living (ADLs) natural movement via video assessments and computer vision analysis. Participants will be able to choose tasks from four domains: food/nutrition, self-care, household/environment, leisure & communication.
All DMD patients:
Ambulant DMD patients, likely to have declining ambulatory function defined as:
Non ambulant DMD patients
If you want more information about the study please contact email@example.com
If you want to join the study please visit: https://dmdhome.atom5.co.uk/