Analysis of Mobility in the Bath for Infants Affected With Spinal Muscular Atrophy

Status: Recruiting
Location: See location...
Intervention Type: Other
Study Type: Interventional
Study Phase: Not Applicable
SUMMARY

Spinal muscular atrophy (SMA) is a genetic neurodegenerative disease impacting spinal cord motor neurons, leading to motor and respiratory issues and, ultimately, death. With emerging therapies, a need arises to enhance motor function assessment in severely hypotonic infants (SMA type 1) as traditional scales on examination tables lack completeness due to gravity's influence. The study team has developed a bath test to observe infants' motor skills in water, eliminating gravity's effects. This test aims to detect subtle movements using inertial sensors, potentially revealing more active motor neurons in aquatic conditions. It aids in identifying infants with motor improvement potential, even if they show limited mobility outside water, and tracks disease progression and therapy responses. Presently, pediatric neurologists in France use parent-provided bathing videos for evaluations, but these lack standardization and precision. The study aims to establish a standardized evaluation protocol with quantifiable data. The study's key objective is to evaluate severely hypotonic SMA infants using inertial sensors, including accelerometers, gyrometers, and magnetometers. The study will conduct dry and water assessments using a specially designed bathtub. This method's goal is to quantify water-based movements accurately. Simultaneously, the study seeks to establish semi-quantitative evaluation criteria to create a clinical assessment scale for infant motor function in bathtubs. This scale will aid doctors in therapeutic decisions. The study will not influence the treatment or therapeutic decisions made for the children being tested. Collected data from dry and water conditions will be statistically analyzed and compared to reference motor assessment scales (e.g., CHOP INTEND and HINE) and electromyography (CMAP-EMG) results, commonly used in diagnosis and monitoring. Blurred video recordings will assist in protocol monitoring and sensor data analysis.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 1
Healthy Volunteers: f
View:

• Infants with muscle weakness (commonly referred to as floppy babies):

‣ Confirmed diagnosis of SMA through gene mutation or deletion (SMN1 gene).

⁃ Receiving standard care.

⁃ Age less than 12 years and a height of less than 90 cm at the time of inclusion.

• Patients enrolled in a social security scheme.

• Informed consent signed by both parents of the child and the investigator.

Locations
Other Locations
France
Infantile neuromuscular unity - Department of paediatric neurological care and intentive care unity, Raymond Poincaré Hospital - APHP
RECRUITING
Garches
Contact Information
Primary
Susana Quijano-Roy, MD, PhD
susana.quijano-roy@aphp.fr
+ 33 1 47 10 78 90
Backup
Nour Maalouf, Psychomotor Therapist DE
nour.maalouf@outlook.com
Time Frame
Start Date: 2025-06-26
Estimated Completion Date: 2026-06
Participants
Target number of participants: 15
Treatments
Experimental: Mobility Assessment
This study involves a single arm, without a comparator group. The comparison will be made between the data for movements in conditions outside the water and inside the water.
Sponsors
Leads: Assistance Publique - Hôpitaux de Paris

This content was sourced from clinicaltrials.gov