Clinical trial NCT05982119: Assessments in Patients With Muscular Pathology and in Control Subjects : The ActiLiège Next Study
Official title: Gait Analysis Parameter, Stair Climbing and Upper Limb Evaluation in Patients With Muscular Pathology and in Control Subjects: The ActiLiège Next Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Duchenne Muscular Dystrophy
Fascioscapulohumeral Muscular Dystrophy
Myotonic Dystrophy 1
Charcot-Marie-Tooth
Centronuclear Myopathy
Congenital Muscular Dystrophy
Lead sponsor: Centre Hospitalier Universitaire de Liege
Sponsor class: OTHER
Collaborators: name: SYSNAV
First posted: 2023-08-08
Enrollment: 300
Sex: ALL
Minimum age (years): 1
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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.


Eligibility criteria: Inclusion criteria

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Stride velocity
timeFrame: through study completion (3 year)
measure: Stride length
timeFrame: through study completion (3 year)
measure: Stairs number
timeFrame: through study completion (3 year)
measure: Stairs speed
timeFrame: through study completion (3 year)
measure: Stairs height
timeFrame: through study completion (3 year)
Secondary outcomes:
Other outcomes:
Locations:
city: Liège
country: Belgium
facility: CHR de la Citadelle
city: Prague
country: Czechia
facility: Fakultni nemocnice v Motole
city: Cairo
country: Egypt
facility: Galaa Military Medical Complex
city: Budapest
country: Hungary
facility: Semmelweis University 2nd Department of Paediatrics
city: Warsaw
country: Poland
facility: Warsaw Medical University Hospital, Department of Neurology
city: Bucarest
country: Romania
facility: National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"
city: Bucharest
country: Romania
facility: Pediatric Neurology Clinic, Clinical Hospital of Psychiatry "Prof. Dr. Al. Obregia"
city: Ljubljana
country: Slovenia
facility: University Children's Hospital, Department for Pediatric Neurology
Study officials:
References: