Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology

Обзор

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.

Подробное описание

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.

Вмешательства

• Устройство Syde
  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.
• Диагностический тест Dynamometric measurements of muscle strength
  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.
• Диагностический тест The 6-minute walk test (6MWT)
  This test consists of covering the greatest possible distance within 6 minutes, walking back and forth along a 25-meter hallway. The subject may stop if they need to. The evaluator will record the elapsed time after each half-lap, and the final distance that was covered at the end of the test. No help will be allowed during the test.
• Диагностический тест The 10-meter test (10MWT)
  During this test, the subject must cover a distance of 10 meters as rapidly as possible. This test will be performed 3 times. The participant will be allowed to rest for one minute after the first and second instances of the test. No help will be allowed during the test.
• Диагностический тест The 4 stair-climbing test (4SC)
  This is a standardized test during which the subject must climb the 4 stair steps as rapidly as possible. This test will be performed 3 times. The participant will be allowed to rest for one minute after the first and second instances of the test, or more if needed.
• Диагностический тест Test of rising from the floor (TRF)
  This is a standardized test during which the subject must rise from the floor as rapidly as possible. The test starts with the participant lying on their back. This test will be performed 3 times. The participant will be allowed to rest for one minute after the first and second instances of the test.
• Диагностический тест The Timed up and go test (TUG)
  During this standardized test, the subject is observed and timed while he/she rises from a chair, walks 3 meters, performs a 180° turn, walk back toward the chair and sits down.
• Диагностический тест The Progressive Supranuclear Palsy Rating Scale (PSP-RS)
  This 28-item clinician-rated scale assesses motor and non-motor symptom severity in patients with PSP, with a score ranging from 0 (normal) to 100.
• Диагностический тест The Montreal Cognitive Assessment (MOCA)
  The MOCA was designed as a rapid screening instrument for mild cognitive dysfunction. It assesses different cognitive domains: attention and concentration, executive function, memory, language, visuoconstructional skills, conceptual thinking, calculations, and orientation. Scores on the MOCA range from 0 to 30, with higher score being better outcome.
• Диагностический тест The Charcot-Marie-Tooth examination score (CMTES)
  The CMTES is a 28-point composite score that rates the patient's symptoms and signs. It is part of the Charcot-Marie-Tooth (CMT) neuropathy score and has been validated as a reproducible measure of disability in CMT. Higher scores indicate more severe neuropathy.

Первичные конечные точки

• 95th Centile of Stride Velocity (SV95C) [Срок оценки: Every 6 months for up to 2 years]

Вторичные конечные точки (5)

• Device Acceptability and Adherence [Срок оценки: Over 2 years]
• Correlation between Syde® and 6MWT [Срок оценки: Every 6 months for up to 2 years]
• Correlation between Syde® and 10MWT [Срок оценки: Every 6 months for up to 2 years]
• Correlation between Syde® and 4SC [Срок оценки: Every 6 months for up to 2 years]
• Correlation between Syde® and TRF [Срок оценки: Every 6 months for up to 2 years]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Нерандомизированное

Модель

Параллельные группы

Маскирование

Открытое

Основная цель

Диагностика

Центры проведения

Бельгия · 1 центр

• Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citade — Liège

Идентификаторы

NCT: NCT07136844 · ActiLiège Adult

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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