Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- 95th Centile of Stride Velocity (SV95C)
研究概览
简要总结
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or metabolic diseases affecting movement. Conducted at the Centre de Référence Liégeois des Maladies Neuromusculaires in Liège, Belgium, the study will enroll 300 ambulant patients, including individuals with neuromuscular disorders and obesity. Using the Syde® wearable device, the study aims to continuously monitor motor function in real-life settings over a period of up to two years. The primary objective is to evaluate the utility of digital mobility outcomes, such as the 95th centile of stride velocity (SV95C), as reliable and objective endpoints for future clinical trials.
详细描述
Following the qualification of the 95th centile of stride velocity (SV95C) as a primary digital endpoint in Duchenne muscular dystrophy (DMD), there is growing interest in extending such digital assessments to other neurological and metabolic diseases that impair movement. Traditional in-clinic functional tests (e.g., 6-minute walk test, 10-meter walk/run) provide only limited snapshots of motor ability and are influenced by external factors such as motivation and fatigue. Similarly, motor function scales and biomarkers, while useful, often lack objectivity or established clinical relevance.
The ActiLiège-Adult study aims to address these limitations by leveraging the Syde® device, a wearable magneto-inertial sensor worn on the wrist and/or ankle, to continuously monitor motor activity in daily life. This device has been previously validated in DMD and other neuromuscular conditions and has been used in both interventional and natural history studies.
This academic study will enroll 300 ambulant adult patients, including 220 with neurological diseases and 20 with obesity, with a minimum of 20 patients per disease type. Ambulation is defined as the ability to walk 10 meters unaided. Patients who lose ambulation during the study will continue to be followed with adapted assessments.
Participants will be monitored for up to two years (six months for diseases affecting the neuromuscular junction). Standardized clinical assessments-including timed tests, motor function evaluations, and strength measurements-will be conducted at baseline and every six months. These will be compared with continuous data collected by the Syde® device. Additionally, patients will complete a Patient Global Impression of Change (PGI-C) questionnaire every six months.
The study's goal is to generate robust, real-world data to support the development of objective, sensitive, and clinically meaningful digital endpoints for use in future therapeutic trials targeting movement disorders.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ambulant patients (i.e. able to walk 10 meters without assistance)
- •Confirmed diagnosis by the investigator based on current gold standard in his/her disease (genetic testing, clinical criteria, etc.)
- •Myotonic dystrophy type 1 (DM1) and Charcot-Marie-Tooth (CMT) patients should present sensitive of motor signs on physical examination.
- •Myasthenic patients should be seropositive, and Myasthenia Gravis Foundation of America (MGFA) class II to IV.
- •Patient with morbid obesity (Body Mass Index> or = 35 at inclusion visit).
- •Signed informed consent form by patient him/herself and patient willing and able to comply with all study procedures.
排除标准
- •Non-ambulant 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
- •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
- •Pregnant or breastfeeding women
研究组 & 干预措施
Neuromuscular condition - Neuropathy
干预措施: Syde (Device)
Neuromuscular condition - Neuropathy
干预措施: Dynamometric measurements of muscle strength (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: 9-Hole Peg Test (9-HPT) (Diagnostic Test)
Neuromuscular condition - Neuropathy
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Neuromuscular condition - Neuropathy
干预措施: The Charcot-Marie-Tooth examination score (CMTES) (Diagnostic Test)
Ataxia
干预措施: Syde (Device)
Ataxia
干预措施: 9-Hole Peg Test (9-HPT) (Diagnostic Test)
Ataxia
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Ataxia
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Ataxia
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Ataxia
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Ataxia
干预措施: The Scale for the Assessment and Rating of Ataxia (SARA) (Diagnostic Test)
Ataxia
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Huntington disease (HD) and HD-like
干预措施: Syde (Device)
Huntington disease (HD) and HD-like
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Huntington disease (HD) and HD-like
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Huntington disease (HD) and HD-like
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Huntington disease (HD) and HD-like
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Huntington disease (HD) and HD-like
干预措施: The Unified Huntington's Disease Rating Scale (UHDRS) (Diagnostic Test)
Huntington disease (HD) and HD-like
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: Syde (Device)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The Timed up and go test (TUG) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The Progressive Supranuclear Palsy Rating Scale (PSP-RS) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: The Montreal Cognitive Assessment (MOCA) (Diagnostic Test)
Progressive supranuclear palsy (PSP) and PSP-like
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: Syde (Device)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Spastic paraplesia and conditions where spasticity predominates, incl. stroke
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Obese patients
干预措施: Syde (Device)
Obese patients
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Obese patients
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Obese patients
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Obese patients
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Obese patients
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Neuromuscular condition - Neuromuscular Junction
干预措施: Syde (Device)
Neuromuscular condition - Neuromuscular Junction
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: Quantitative Myasthenia Gravis (QMG) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: The Myasthenia Gravis Activities of Daily Living Scale (MG-ADL) (Diagnostic Test)
Neuromuscular condition - Neuromuscular Junction
干预措施: Patient's Global Impression of Change (PGIC) (Other)
Neuromuscular condition - Muscle disease
干预措施: Syde (Device)
Neuromuscular condition - Muscle disease
干预措施: The 6-minute walk test (6MWT) (Diagnostic Test)
Neuromuscular condition - Muscle disease
干预措施: The 10-meter test (10MWT) (Diagnostic Test)
Neuromuscular condition - Muscle disease
干预措施: The 4 stair-climbing test (4SC) (Diagnostic Test)
Neuromuscular condition - Muscle disease
干预措施: Test of rising from the floor (TRF) (Diagnostic Test)
Neuromuscular condition - Muscle disease
干预措施: Patient's Global Impression of Change (PGIC) (Other)
结局指标
主要结局
95th Centile of Stride Velocity (SV95C)
时间窗: Every 6 months for up to 2 years
Change in SV95C as measured by the Syde® wearable device, used to assess ambulatory function in real-life settings.
次要结局
- Device Acceptability and Adherence(Over 2 years)
- Correlation between Syde® and 6MWT(Every 6 months for up to 2 years)
- Correlation between Syde® and 10MWT(Every 6 months for up to 2 years)
- Correlation between Syde® and 4SC(Every 6 months for up to 2 years)
- Correlation between Syde® and TRF(Every 6 months for up to 2 years)
研究者
Laurent Servais
Principal Investigator, Pediatrician
Centre Hospitalier Universitaire de Liege