Centronuclear Myopathy, Charcot-Marie-Tooth, Congenital Muscular Dystrophy, Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1
Conditions
Brief summary
The objective of the ActiLiège Next study is to collect longitudinal data from patients and control subjects using a wearable magneto-inertial device. By collecting natural history data in various neuromuscular disorders (Duchenne Muscular Dystrophy, Fascioscapulohumeral Muscular Dystrophy, Myotonic Dystrophy 1, Charcot-Marie-Tooth, Centronuclear Myopathy, Congenital Muscular Dystrophy), we aim to validate digital outcome measures to continuously assess motor function in real-life.
Interventions
DEVICEActiMyo/Syde
The two watches can be worn as wristwatch or placed near the ankle and on the wheelchair. * Patients with DMD or FKRP mutation will wear the ActiMyo°/Syde° during 3 months at baseline and then for one month every 3 months. * Patient with FSHD, DM1, CMT, CNM will wear the ActiMyo°/Syde° will wear the ActiMyo/Syde° during 3 months at baseline and then for one month every 6 months. * Control subjects \>4years will wear the ActiMyo°/Syde° during one month after inclusion and during one other month 11 months after inclusion. * Control subjects \<4years will wear the ActiMyo°/Syde° during one month after inclusion and during one other month every 6months after inclusion.
Sponsors
SYSNAV
Centre Hospitalier Universitaire de Liege
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 80 Years
Healthy volunteers
Yes
Inclusion criteria
* For the patients: * Genetically confirmed diagnosis of DMD, FSHD, DM1, CMT or FKRP mutations or confirmed CNM based on muscle biopsy. * FSHD, DM1, CMT and CNM patients should be ambulant or in transition. * DM1 and CMT patients should present sensori-motor signs on physical examination. * Under the age of 20 years for patients with DMD, CNM or between the ages of 5 and 80 years for patients with FSHD, CMT and DM1. * More than 2 years old for patients with FKRP mutations * Non-ambulant DMD patients must be able to remain seated in an arm- or a wheelchair for at least one hour. * Patients with DMD treated with corticosteroids for at least 6 months or initiated corticosteroid at V0 (except for patients under 4). * Signed informed consent form by patient himself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians. * For the control subjects: * Ambulant boys and girls under 20 years old * Signed informed consent form by patient him/herself or, in case of minor patients, signed informed consent form by patient's parents or legal guardians.
Exclusion criteria
* For the patients: * Patients with extreme cognitive disorders that limit their understanding of the exercises to be performed. * 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. * DMD patients in transition who are not on corticosteroids. * For the control subjects: * Patients who have undergone a surgical procedure or who have experienced recent trauma (within fewer than 6 months) affecting the upper or lower limbs. * Elite athletes (at the national level). * A chronic or acute muscular, neurological, infectious, or inflammatory pathology within the 3-week period immediately prior to inclusion. * An orthopedic, neuromuscular, or neurological pathology that affects the quality of the subject's walking gait.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Stride velocity | through study completion (3 year) | Stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second). |
| Stride length | through study completion (3 year) | Stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter). |
| Stairs number | through study completion (3 year) | Total number of strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life |
| Stairs speed | through study completion (3 year) | Vertical speed during strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life |
| Stairs height | through study completion (3 year) | Height of the strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life |
Countries
Belgium, Czechia, Egypt, Hungary, Poland, Romania, Slovenia
Contacts
Primary ContactCharline DUBOIS
Outcome results
None listed