Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study

Study Overview
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Brief Summary
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The objective of ActiLiège Next Study is to collect natural history data in Duchenne muscular dystrophy (DMD) (ambulatory and non-ambulatory patients), and in affected patients with Facio-Scapulo-Humeral dystrophy (FSHD), a disease in which results are currently lacking. It consists of obtaining longitudinal data for the patients, as well as normative data for the control subjects, with a particular emphasis on pediatric subjects using the device on both ankles (for walking) in order to qualify the ascent rate of staircase.

Official Title
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Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study

Conditions
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Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy

Intervention / Treatment
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* Device: ActiMyo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Genetically confirmed diagnosis of DMD or FSHD. Between 5 and 20 years of age for patients with DMD, or between 5 and 80 years of age for patients with FSHD. Ambulant (as defined by having the ability to walk without assistance for 10 meters) or non-ambulant DMD patients. Ambulant FSHD patients Non-ambulant DMD patients must be able to remain seated in an arm- or a wheel-chair for at least one hour. Signed informed consent form by patient himself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians. Exclusion Criteria: Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed. Patients treated with corticosteroids for less than 6 months. Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs (for ambulant patients). A concomitant chronic or acute neurological, endocrine, infectious, allergic, or inflammatory pathology within the 3-week period immediately prior to inclusion. Patients who are participating in an interventional clinical trial.

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Other: Patients with DMD/FSHD or control subjects<br>Patients and control subjects will be included over a one-year study period. Patients will be examined by a neuropaediatrician or neurologist and perform standardized assessments (timed tests, motor function tests, and strength tests) at baseline, 6 and 12 months. Patients will be asked to wear the device during one year. Control subjects will be examined by a physician and perform the same tests than those for ambulant patients at baseline and 12 months. Control subjects will be asked to wear the device for two months (one month at inclusion, one month 11 months after inclusion). | Device: ActiMyo<br>* The two watches can be worn as wristwatch or placed near the ankle and on the wheelchair. Patients will wear the ActiMyo° during one year in order to collect natural history data in DMD and FSHD. Control subjects will wear the ActiMyo° during one month after inclusion and during one other month 11 months after inclusion.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stride velocity | Stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second). | through study completion (3 year) | 
| Stride length | Stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter). | through study completion (3 year) | 
| Stairs number | Total number of strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life | through study completion (3 year) | 
| Stairs speed | Vertical speed during strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life | through study completion (3 year) | 
| Stairs height | Height of the strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life | through study completion (3 year) |