Digital Monitoring of Upper Limb Function in Non-Ambulant DMD

NCT07664124 | Not Yet Recruiting

• 1 other identifier: Acti-nRoll
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

Non-ambulant; digital endpoint; upper limb

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (4)

• Reliability

  Baseline, Month 6, Month 12, Month 18, Month 24

• Clinical validity of digital outcome vs Brooke

  Baseline, Month 6, Month 12, Month 18, Month 24

• Clinical validity of digital outcome vs PUL

  Baseline, Month 6, Month 12, Month 18, Month 24

• Sensitivity of Digital Upper Limb Outcome Measures

  Baseline, Month 6, Month 12, Month 18, Month 24

Study Arms (1)

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

EXPERIMENTAL

Diagnostic Test: Performance of the Upper Limb (PUL) Test; Diagnostic Test: Brook test; Diagnostic Test: Dynamometric measurements of muscle strength; 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); Device: Syde

Interventions

Performance of the Upper Limb (PUL) Test DIAGNOSTIC_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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Brook test DIAGNOSTIC_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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Dynamometric measurements of muscle strength DIAGNOSTIC_TEST

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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Forced Vital Capacity (FVC) test DIAGNOSTIC_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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Syde DEVICE

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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Clinical Global Impression - Severity (CGI-S) DIAGNOSTIC_TEST

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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Clinical Global Impression - Improvement (CGI-C) DIAGNOSTIC_TEST

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.

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Patient Global Impression of severity (PGI-S) OTHER

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').

Non-ambulant participants with Duchenne Muscular Dystrophy (DMD)

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient with genetically confirmed Duchenne Muscular Dystrophy (DMD).
• A legal guardian willing and able to provide written informed consent for participation in the study if \< 18 years old.

You may not qualify if:

• 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

Sponsors & Collaborators

• SYSNAV: collaborator
• Centre Hospitalier Universitaire de Liege: lead
• Centre Hospitalier Régional de la Citadelle: collaborator

Study Sites (1)

Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle

Liège, 4000, Belgium

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Vital Capacity

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Total Lung Capacity; Lung Volume Measurements; Respiratory Function Tests; Diagnostic Techniques, Respiratory System; Diagnostic Techniques and Procedures; Diagnosis; Respiratory Physiological Phenomena; Circulatory and Respiratory Physiological Phenomena

Study Officials

• Laurent Servais, MD

  Centre Hospitalier Universitaire de Liege

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Dounia Chelha

CONTACT

+3243215498; Khadidja.Chelha@citadelle.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator, Pediatrician

Study Record Dates

• First Submitted: June 15, 2026
• First Posted: June 23, 2026
• Study Start: July 30, 2026
• Primary Completion (Estimated): June 30, 2029
• Study Completion (Estimated): April 30, 2030
• Last Updated: June 23, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1)