NCT04833348

Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies

Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies, IMUSMA Project

• Status: Active Not Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.

Detailed description

Infantile spinal muscular atrophy is a common disease (the second most common fatal autosomal recessive disease after cystic fibrosis), neurodegenerative disorders of childhood causing severe motor impairment and a risk to life through respiratory failure in the most severe forms. Innovative therapies (gene therapy or pharmacogenetics) have recently proven their effectiveness on survival criteria. Nevertheless, the motor benefit of these therapies must be evaluated more precisely. Currently, the reference methods for motor development assessment are fairly robust semi-quantitative motor scales that lack sensitivity and do not reflect function (CHOPINTEND, HINE, BAYLEY SCALE, MFM and CGI-scale). Advances in recent techniques have enabled the emergence of non-invasive, secure, easy-to-use inertial sensors in routine clinical practice that allow quantification of infant movements. The aim of the study is to propose a method for quantifying motor function in infants with spinal muscular atrophy treated with innovative therapies using inertial sensors.

Conditions

• Spinal Muscular Atrophy

Interventions

Type	Name	Description
OTHER	Motor function measurement using inertial sensors	Measurement of motor skills at M0: start of the administration of the innovative therapy and then 1 month, 3 months, 6 months, 1 year and then 2 years later: * Free motor skills in the supine position * Motricity in the supine position stimulated by a play frame * Measurement of the proximal and distal activity of the upper limbs in motor skills stimulated by the play gantry * Measurement of activity in a supported sitting position Longitudinal study, the subject is his own control

Timeline

• Start date: 2021-03-20
• Primary completion: 2025-03-17
• Completion: 2027-03-01
• First posted: 2021-04-06
• Last updated: 2025-09-15

Locations

1 site across 1 country: France