Digital Monitoring of Upper Limb Function in Non-Ambulant DMD (Acti-nRoll)

Feasibility, Reliability, Clinical Validity and Sensitivity of Digital Outcomes to Monitor Upper Limb Function in Non-ambulant Patients With Genetically Confirmed Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration. Symptoms typically begin in early childhood and result in loss of ambulation by early adolescence, followed by cardiorespiratory complications. Although early treatment, including corticosteroids and emerging therapies, can slow disease progression, sensitive tools to monitor functional decline-particularly in non-ambulant patients-remain limited.

Current assessments rely primarily on clinical scales and hospital-based evaluations, which may not detect subtle changes or reflect real-life function. Digital outcome measures derived from wearable sensors offer a promising approach for continuous, objective monitoring in daily life. This study aims to evaluate the feasibility, reliability, clinical validity, and sensitivity of digital measures to assess upper limb function in non-ambulant patients with genetically confirmed DMD. The Syde device, previously validated in ambulant DMD patients, will be investigated for its applicability in this population.

Study Overview

• Status: Not yet recruiting
• Conditions: Duchenne Muscular Dystrophy (DMD)
• Intervention / Treatment: Device: Syde; Diagnostic test: Dynamometric measurements of muscle strength; Diagnostic test: Performance of the Upper Limb (PUL) Test; Diagnostic test: Brook test; Diagnostic test: Forced Vital Capacity (FVC) test; Diagnostic test: Clinical Global Impression - Severity (CGI-S); Diagnostic test: Clinical Global Impression - Improvement (CGI-C); Other: Patient Global Impression of severity (PGI-S)

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Dounia Chelha; Phone Number: +3243215498; Email: Khadidja.Chelha@citadelle.be

Study Locations

• Belgium
  • Liège, Belgium, 4000
    • Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patient with genetically confirmed Duchenne Muscular Dystrophy (DMD).
• Non-ambulant at the time of inclusion (not able to walk 10m without external aid).
• A legal guardian willing and able to provide written informed consent for participation in the study if < 18 years old.

Exclusion Criteria:

• Any acute or chronic condition that, in the opinion of the investigator, may significantly interfere with the assessments and/or motor function progression.
• Participation in an interventional clinical trial.
• No access to internet connection or alternatively no capacity to come on-site to bring the Syde every 6 months after the recording periods for data retrieval by Liège team
• Scoliosis surgery within the previous 6 months or planned within the next year

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Diagnostic
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Device: Syde; Syde is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle.; Diagnostic test: Dynamometric measurements of muscle strength; Dynamometric measurements of the maximum force of the following functions will be taken with the MyoTools: palmar grip (MyoGrip) and thumb-index pinch (MyoPinch). Test will be realized on the dominant side. Patients will be encouraged during the test. They will be given three trials and the best score will be entered.; Diagnostic test: Performance of the Upper Limb (PUL) Test; This test consists of a set of small manoeuvres (lifting a box, writing, etc.) designed to evaluate the upper-limb functionality of non-ambulatory patients. It was developed specifically for use in cases of Duchenne muscular dystrophy.; Diagnostic test: Brook test; The Brooke Upper Extremity Functional Rating Scale will be used to assess the global functional level of upper limb involvement. This 6-point ordinal scale classifies patients according to their highest achievable upper limb function, from full arm abduction to absence of useful hand function.; Diagnostic test: Forced Vital Capacity (FVC) test; Forced Vital Capacity (FVC) will be assessed using standardized spirometry procedures. The primary parameter will be FVC expressed as percentage of predicted values. At least three acceptable and reproducible maneuvers will be performed according to standard guidelines.; Diagnostic test: Clinical Global Impression - Severity (CGI-S); The CGI-S is a clinician-rated scale used at baseline to assess the overall severity of the participant's condition, based on all available clinical information. The rating reflects the clinician's judgment without standardized scoring rules and must be performed by a clinician experienced in Duchenne Muscular Dystrophy. The CGI-S evaluates three domains: physical motor function, respiratory function, and bulbar function. It is completed after all other study assessments (excluding patient-reported outcomes) to ensure a comprehensive evaluation.; Diagnostic test: Clinical Global Impression - Improvement (CGI-C); The CGI-C is used at each follow-up visit to assess changes in the participant's condition relative to baseline. It provides a clinician-determined measure of improvement or worsening, based on overall clinical judgment rather than fixed criteria. Like the CGI-S, it covers physical motor, respiratory, and bulbar domains and is performed after all other study assessments (excluding patient-reported outcomes). The CGI-C is expected to track consistently with prior CGI-S evaluations, reflecting changes in disease status over time.; Other: Patient Global Impression of severity (PGI-S); The PGI-S (Patient Global Impression of Severity) is a simple, validated, single-item self-administered scale used to assess the current severity of a patient's condition. Widely used in clinical trials, it allows patients to rate their condition, on a 4- to 6-point scale (from 'Normal' to 'Very severe').

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Device usage (recording time)	Total recording time per recording period (hours)	Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Patient compliance (Min)	Percentage of participants achieving minimal threshold (expected 50h) of recording time per period	Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Patient compliance (Max)	Percentage of participants achieving optimal threshold (expected 180h) of recording time per period	Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reliability	Intraclass Correlation Coefficient (ICC) for two distinct consecutive half-periods	Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs Brooke	Correlation between digital outcome and Brooke Upper Extremity Rating Scale (Brooke score)	Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs PUL	Correlation between digital outcome and Performance of Upper Limb (PUL) score	Baseline, Month 6, Month 12, Month 18, Month 24
Sensitivity of Digital Upper Limb Outcome Measures	Sensitivity will be evaluated by assessing the ability of digital outcome measures to detect change over time combining mean change from baseline, slope of change over time, and standardized response mean (SRM).	Baseline, Month 6, Month 12, Month 18, Month 24

Collaborators and Investigators

• Sponsor: Centre Hospitalier Universitaire de Liege
• Collaborators: Centre Hospitalier Régional de la Citadelle; SYSNAV
• Investigators: Principal Investigator: Laurent Servais, MD, Centre Hospitalier Universitaire de Liege

Study record dates

Study Major Dates

• Study Start (Estimated): July 30, 2026
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): April 30, 2030

Study Registration Dates

• First Submitted: June 15, 2026
• First Submitted That Met QC Criteria: June 18, 2026
• First Posted (Actual): June 23, 2026

Study Record Updates

• Last Update Posted (Actual): June 23, 2026
• Last Update Submitted That Met QC Criteria: June 18, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: upper limb; Non-ambulant; digital endpoint
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Muscular Disorders, Atrophic; Muscular Dystrophies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Muscular Dystrophy, Duchenne; Diagnostic Techniques and Procedures; Diagnosis; Circulatory and Respiratory Physiological Phenomena; Diagnostic Techniques, Respiratory System; Respiratory Physiological Phenomena; Respiratory Function Tests; Total Lung Capacity; Lung Volume Measurements; Vital Capacity
• Other Study ID Numbers: Acti-nRoll

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No