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

Not Applicable

Recruiting

• Conditions: Neuromuscular Diseases; Obesity (Disorder); Myotonic Dystrophy 1; Myasthenic Syndrome; Charcot Marie Tooth Disease (CMT); Glycogen Storage Disease Type II Pompe Disease; Facio-Scapulo-Humeral Dystrophy; Myasthenia Gravis; Huntington Disease; Progressive Supranuclear Palsy (PSP)

• Registration Number: NCT07136844
• Lead Sponsor: Centre Hospitalier Universitaire de Liege

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2024-03-29
• Status Verified: 2025-08
• Study First Submitted: 2025-08-07
• Study First Submitted QC: 2025-08-14
• Last Update Submitted: 2025-08-14
• Study First Posted: 2025-08-22
• Last Update Posted: 2025-08-22
• Primary Completion: 2029-12
• Study Completion: 2030-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

• 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.

Exclusion Criteria

• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Primary Outcome Measures

Name	Time	Method
95th Centile of Stride Velocity (SV95C)	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.

Secondary Outcome Measures

Name	Time	Method
Device Acceptability and Adherence	Over 2 years	Measures of user engagement with the Syde® device over time, including total recording time, valid days (≥5 hours), and time to reach 50 and 180 hours.
Correlation between Syde® and 6MWT	Every 6 months for up to 2 years	Correlation between the Syde digital variable and results from the 6-minute walk test (6MWT)
Correlation between Syde® and 10MWT	Every 6 months for up to 2 years	Correlation between the Syde digital variable and results from the 10-meter test (10MWT)
Correlation between Syde® and 4SC	Every 6 months for up to 2 years	Correlation between the Syde digital variable and results from the 4 stair-climbing test (4SC)
Correlation between Syde® and TRF	Every 6 months for up to 2 years	Correlation between the Syde digital variable and results from the test of rising from the floor (TRF)

Trial Locations

Locations (1)

Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle; 🇧🇪 Liège, Belgium