Holter of Movement in Patients With SMA Undergoing Treatment.

Study Overview
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Brief Summary
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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.

Official Title
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Holter of Movement in Patients With Type 2 or 3 Spinal Muscular Atrophy When Initiating Treatment With Spinraza ° or Risdiplam. Acti-SMA Study.

Conditions
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Spinal Muscular Atrophy

Intervention / Treatment
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* Device: Actimyo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Genetically confirmed spinal muscular atrophy. Treatment by Spinraza ° or by risdiplam planned. Over 6 years old. Signed informed consent. Exclusion Criteria: - Patients with excessive cognitive disorders, limiting the understanding of task or with apparent communication difficulties hindering data collection.

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Other
Intervention Model: Single Group Assignment
Interventional Model Description: all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SMA patients<br>Patients with type 2 or 3 spinal muscular atrophy undergoing Spinraza° or risdiplam treatment. | Device: Actimyo<br>* Actimyo° is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 95th centile of stride velocity | 95th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life. (meter per second). | 39 months | 
| 50th centile of stride velocity | 50th centile of stride velocity obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter per second). | 39 months | 
| 95th centile of stride length | 95th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter). | 39 months | 
| 50th centile of stride length | 50th centile of stride length obtained with a magneto-inertial sensor (Actimyo°) in real-life (meter). | 39 months | 
| Upper limb function evolution in patient with SMA undergoing treatment | Upper limb function assessed with a magneto-inertial sensor (Actimyo°) in real-life. | 39 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 6-minute walk test | The Six-Minute Walk Test is a functional walking test in which investigators assess the distance the patient can walk in six minutes (meter). | 39 months | 
| Dynamometric measures | Assessment of upper limb strength with myogrip and myopinch (kg). | 39 months | 
| 10-metre walk test | The 10-metre walk test is a functional walking test in which investigators assess the time the patient take to walk 10 meters (second). | 39 months | 
| rise from floor | The rise from floor is a functional test in which investigators assess the time the patient take to rise from the floor (second). | 39 months | 
| 4-stair climb | The 4-stair climbr is a functional test in which investigators assess the time the patient take to climb a four-step staircase (second). | 39 months | 
| revised upper limb module (RULM) | Revised Upper Limb Module (RULM) is an assessment specifically designed for upper limb function in Spinal Muscular Atrophy. | 39 months | 
| Hamersmith funcitonnal motor scale (HFMS) | HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy. | 39 months | 
| motor function measure (MFM) | Motor Function Measure is a global quantitative scale created to measure the functional motor abilities of a person affected by a neuromuscular disease. | 39 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring