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

NCT05982119 | Recruiting

• 1 other identifier: ActiLiege Next
• Study Type: interventional
• Target: 300
• Locations: 7 countries
• Sites: 8

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy; Fascioscapulohumeral Muscular Dystrophy; Myotonic Dystrophy 1; Charcot-Marie-Tooth; Centronuclear Myopathy; Congenital Muscular Dystrophy

Outcome Measures

Primary Outcomes (5)

• Stride velocity

  Stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).

  through study completion (3 year)

• Stride length

  Stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).

  through study completion (3 year)

• Stairs number

  Total number of strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life

  through study completion (3 year)

• Stairs speed

  Vertical speed during strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life

  through study completion (3 year)

• Stairs height

  Height of the strides in stairs obtained with a magneto-inertial sensor (Actimyo°) in real-life

  through study completion (3 year)

Study Arms (1)

Patients with DMD/FSHD/CMT/DM1/CNM/FKRPmutation or control subjects

OTHER

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).

Device: ActiMyo/Syde

Interventions

ActiMyo/Syde DEVICE

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 month 11 months after inclusion. * Control subjects \<4years will wear the ActiMyo°/Syde° during one month after inclusion and during one other month every 6months after inclusion.

Patients with DMD/FSHD/CMT/DM1/CNM/FKRPmutation or control subjects

Eligibility Criteria

• Age: 1 Year - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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).
• 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).
• An orthopedic, neuromuscular, or neurological pathology that affects the quality of the subject's walking gait.

Sponsors & Collaborators

• Centre Hospitalier Universitaire de Liege: lead
• SYSNAV: collaborator

Study Sites (8)

CHR de la Citadelle

Liège, 4000, Belgium

RECRUITING

Fakultni nemocnice v Motole

Prague, Czechia

ACTIVE NOT RECRUITING

Galaa Military Medical Complex

Cairo, Egypt

ACTIVE NOT RECRUITING

Semmelweis University 2nd Department of Paediatrics

Budapest, Hungary

ACTIVE NOT RECRUITING

Warsaw Medical University Hospital, Department of Neurology

Warsaw, Poland

ACTIVE NOT RECRUITING

Pediatric Neurology Clinic, Clinical Hospital of Psychiatry "Prof. Dr. Al. Obregia"

Bucharest, 041914, Romania

RECRUITING

National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"

Bucharest, Romania

ACTIVE NOT RECRUITING

University Children's Hospital, Department for Pediatric Neurology

Ljubljana, Slovenia

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophy, Facioscapulohumeral; Myotonic Dystrophy; Charcot-Marie-Tooth Disease; Myopathies, Structural, Congenital

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Myotonic Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Hereditary Sensory and Motor Neuropathy; Nervous System Malformations; Polyneuropathies; Peripheral Nervous System Diseases; Congenital Abnormalities

Central Study Contacts

Charline DUBOIS

CONTACT

043215695; charline.dubois@citadelle.be

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor Laurent Servais

Study Record Dates

• First Submitted: July 20, 2021
• First Posted: August 8, 2023
• Study Start: July 10, 2020
• Primary Completion: March 1, 2026
• Study Completion: March 1, 2026
• Last Updated: May 20, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

BE (1); CZ (1); HU (1); PL (1); SL (1); RO (2); EG (1)