Holter of Movement in Patients with SMA Undergoing Treatment.
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Device: Actimyo
- Registration Number
- NCT04888702
- Lead Sponsor
- Centre Hospitalier Universitaire de Liege
- Brief Summary
Acti-SMA is a multi-centric academic study. It aims to monitor the progress of patients with spinal muscular atrophy under treatment with Spinraza° or risdiplam. First, we want to quantify improvement of both ambulant and non-ambulant patients under treatment. A secondary objective would also be to identify suitable accelerometric measurements that are sensitive to change but also well correlated to other clinical scores.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 30
- Genetically confirmed spinal muscular atrophy.
- Treatment by Spinraza ° or by risdiplam planned.
- Over 6 years old.
- Signed informed consent.
- Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description SMA patients Actimyo Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment.
- Primary Outcome Measures
Name Time Method 95th centile of stride velocity 39 months 95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life.
(meter per second).50th centile of stride velocity 39 months 50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second).
95th centile of stride length 39 months 95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
Upper limb function evolution in patient with SMA undergoing treatment 39 months Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life.
50th centile of stride length 39 months 50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter).
- Secondary Outcome Measures
Name Time Method 6-minute walk test 39 months The Six-Minute Walk Test is a functional walking test in which investigators assess the distance the patient can walk in six minutes (meter).
Dynamometric measures 39 months Assessment of upper limb strength with myogrip and myopinch (kg).
10-metre walk test 39 months The 10-metre walk test is a functional walking test in which investigators assess the time the patient take to walk 10 meters (second).
revised upper limb module (RULM) 39 months Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy.
4-stair climb 39 months The 4-stair climbr is a functional test in which investigators assess the time the patient take to climb a four-step staircase (second).
Hamersmith funcitonnal motor scale (HFMS) 39 months HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy.
rise from floor 39 months The rise from floor is a functional test in which investigators assess the time the patient take to rise from the floor (second).
motor function measure (MFM) 39 months Motor Function Measure is a global quantitative scale created to measure the functional motor abilities of a person affected by a neuromuscular disease.
Trial Locations
- Locations (3)
Erasme Hospital - Cliniques universitaires de Bruxelles
🇧🇪Bruxelles, Belgium
Centre Hospitalier Régional de la Citadelle
🇧🇪Liège, Belgium
National Clinical Hospital for Children Neurohabilitation "Dr Nicolae Robanescu"
🇷🇴Bucharest, Romania