Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 8
- 主要终点
- Stride velocity
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Basic Science
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •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 DM
- •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.
排除标准
- •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.
研究组 & 干预措施
Patients with DMD/FSHD/CMT/DM1/CNM/FKRPmutation or control subjects
Patients and control subjects will be included over a 3-years 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 and then every 6 to 12 months (depending on age).
From February 2024, controls subjects can be remotely recruited and enrolled. They won't be evaluated on site, but data, such as age, sex, weight, and height, will be collected by phone or visio-conference at inclusion and every 6 months for 3 years.
Patients will be asked to wear the device during 1 to 3 months at baseline (depending on disease group) and then for 1 month every 3-12months (depending on age and disease group).
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).
干预措施: ActiMyo/Syde (Device)
结局指标
主要结局
Stride velocity
时间窗: through study completion (3 year)
Stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
Stride length
时间窗: through study completion (3 year)
Stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Stairs height
时间窗: through study completion (3 year)
Height of the strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life
Stairs speed
时间窗: through study completion (3 year)
Vertical speed during strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life
Stairs number
时间窗: through study completion (3 year)
Total number of strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life
次要结局
未报告次要终点
研究者
Laurent Servais
Professor Laurent Servais
Centre Hospitalier Universitaire de Liege