Spinal Muscular Atrophy
Conditions
Keywords
Actimyo, Accelerometry, Motor neuron disease, Risdiplam, Nusinersen, Home monitoring
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.
Interventions
DEVICEActimyo
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.
Sponsors
Centre Hospitalier Universitaire de Liege
SYSNAV
Erasme University Hospital
Queen Fabiola Children's University Hospital
Centre Hospitalier Régional de la Citadelle
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Intervention model description
all patients undergo clinical evaluation and wear Actimyo device in daily living to evaluate their improvement under treatment.
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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). |
| 50th centile of stride length | 39 months | 50th 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| 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). |
| 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. |
| Hamersmith funcitonnal motor scale (HFMS) | 39 months | HFMS is a scale is specifically designed outcome measure for people affected by Spinal Muscular Atrophy. |
| 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). |
| 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). |
| 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). |
Countries
Belgium, Romania
Outcome results
None listed