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Clinical Trials in France / NCT04833348
Active, not recruiting Not applicable

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

NCT04833348 · tracked via the Priya Life Science France tracker
Sponsor
Assistance Publique - Hôpitaux de Paris
Phase
Not applicable
Started
2021-03-20
Last updated
2025-09-15

Condition(s) studied

Spinal Muscular Atrophy

Investigational drug(s) / intervention(s)

Motor function measurement using inertial sensors

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

Study 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.

Eligibility

Sex
ALL
Min age
—
Max age
2 Years
Healthy volunteers
No
Inclusion Criteria: * Infants of both sexes * Suffering from spinal muscular atrophy (diagnosis by genetic study "homozygous deletion of SMN1") * Followed up by the Necker Neuromuscular Reference Center (GNMH) * Eligible for innovative therapy (gene therapy or pharmacogenetics) * age of onset of the disease \<1 year * no severe respiratory impairment (dependence on ventilatory support for more than 16 hours per day) or bulbar involvement * decision of treatment by a Multidisciplinary Consultation Meeting national of experts * Benefiting from social security scheme * Informed consent signed by holders of parental authority and the investigator Exclusion Criteria: * Non-consent of one of the holders of parental authority * Respiratory instability (dependence on ventilatory support for more than 16 hours per day) or hemodynamics * Contraindication to innovative therapy * History of another disease impacting motor skills (neonatal suffering, etc.)

Primary outcome measure(s)

  • Change in the 95th percentile of the norm of acceleration — Month 0 to month 24
    95th percentile of the norm of the acceleration of the feet and the arms.

Trial sites (1)

FacilityCityRegionStatus
Hôpital Necker-Enfants Malades Paris France

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Other trials for the same condition

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy Recruiting
French Register of Patients With Spinal Muscular Atrophy Recruiting
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy Recruiting
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Active, not recruiting
Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® Active, not recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04833348 on ClinicalTrials.gov ↗ ← All trials in France