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

Официальное название

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

Обзор

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.

Вмешательства

• Устройство ActiMyo/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 mont

Первичные конечные точки

• Stride velocity [Срок оценки: through study completion (3 year)]
• Stride length [Срок оценки: through study completion (3 year)]
• Stairs number [Срок оценки: through study completion (3 year)]
• Stairs speed [Срок оценки: through study completion (3 year)]
• Stairs height [Срок оценки: through study completion (3 year)]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

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

Здоровые добровольцы: Да

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Фундаментальное исследование

Центры проведения

Румыния · 2 центра

• Pediatric Neurology Clinic, Clinical Hospital of Psychiatry "Prof. Dr. Al. Obregia" — Bucharest
• National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu" — Bucharest

Бельгия · 1 центр

• CHR de la Citadelle — Liège

Чехия · 1 центр

• Fakultni nemocnice v Motole — Prague

Египет · 1 центр

• Galaa Military Medical Complex — Cairo

Венгрия · 1 центр

• Semmelweis University 2nd Department of Paediatrics — Budapest

Польша · 1 центр

• Warsaw Medical University Hospital, Department of Neurology — Warsaw

Словения · 1 центр

• University Children's Hospital, Department for Pediatric Neurology — Ljubljana

Идентификаторы

NCT: NCT05982119 · ActiLiege Next

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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