Status:
ACTIVE_NOT_RECRUITING
Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Collaborating Sponsors:
URC-CIC Paris Descartes Necker Cochin
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 2 years
Phase:
NA
Brief 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 im...
Eligibility Criteria
Inclusion
- 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
- 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.)
Key Trial Info
Start Date :
March 20 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2027
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT04833348
Start Date
March 20 2021
End Date
March 1 2027
Last Update
September 15 2025
Active Locations (1)
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1
Hôpital Necker-Enfants Malades
Paris, France, 75015