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| Name | Class |
|---|---|
| Centre Hospitalier Régional de la Citadelle | OTHER |
| SYSNAV | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 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. |
| 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 |
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Dounia Chelha | Contact | +3243215498 | Khadidja.Chelha@citadelle.be |
| Name | Affiliation | Role |
|---|---|---|
| Laurent Servais, MD | Centre Hospitalier Universitaire de Liege | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle | Liège | 4000 | Belgium |
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|
| 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. |
|
| 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. |
|
| 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. |
|
| 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. |
|
| 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. |
|
| 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'). |
|
| 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. |
|
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 |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D014797 | Vital Capacity |
| ID | Term |
|---|---|
| D014109 | Total Lung Capacity |
| D008176 | Lung Volume Measurements |
| D012129 | Respiratory Function Tests |
| D003948 | Diagnostic Techniques, Respiratory System |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D012143 | Respiratory Physiological Phenomena |
| D002943 | Circulatory and Respiratory Physiological Phenomena |
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