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NCT04833348Active, not recruiting

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

Source: ClinicalTrials.gov · Last updated 15 Sept 2025

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Study type
Interventional
Enrollment
35 (actual)
First submitted
23 Mar 2021
Start date
20 Mar 2021
Primary completion date
17 Mar 2025
Lead sponsor
Assistance Publique - Hôpitaux de Paris
NCT number
NCT04833348

What you commit to

  • Study type: Interventional
  • Participants enrolled: 35
  • From 20 Mar 2021 to the primary completion date, 17 Mar 2025
  • 1 site in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Prior treatment

  • Contraindication to innovative therapy

Other illness

  • History of another disease impacting motor skills (neonatal suffering, etc.)

Other

  • 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

Who the study is looking for

  • 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

Age in the registry: - to 2 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Hopital Necker Enfants Malades
Coordinator of this site? Confirm its status
ParisFranceUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.