Test of Aquatic Mobility of SMA Infants

NCT06152302 | Recruiting

• 2 other identifiers: APHP2312932022-A01419-34
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Infantile Spinal Muscular Atrophy

Keywords

SMA; infantile; bath test; water mobility test; inertial sensors; floppy infant; Garches

Outcome Measures

Primary Outcomes (1)

• Delta of 95th percentile of free acceleration norm (in m/s²)

  Difference of free acceleration norm (in m/s²) for each measurement point respectively (elbows; wrists; knees; ankles), between "dry" and "water" condition.

  through study completion, an average of 15 months

Study Arms (1)

Mobility Assessment

EXPERIMENTAL

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.

Other: Mobility study in dry conditions; Other: Mobility study in water conditions

Interventions

Mobility study in dry conditions OTHER

Record the movements of the baby's extremities using inertial sensors while the baby moves outside of the water.

Mobility Assessment

Mobility study in water conditions OTHER

Record the movements of the baby's extremities using inertial sensors while the baby moves inside the water.

Also known as: Garches Bath Test (GBT)

Mobility Assessment

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Infants with muscle weakness (commonly referred to as floppy babies):
• Confirmed diagnosis of SMA through gene mutation or deletion (SMN1 gene).
• Receiving standard care.
• Patients enrolled in a social security scheme.
• Informed consent signed by both parents of the child and the investigator.

You may not qualify if:

• Patients presenting with lesions in the spinal cord or conditions affecting the cardiorespiratory system.
• Patients covered under the French Aid in Health for Foreign People program (AME).

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Infantile neuromuscular unity - Department of paediatric neurological care and intentive care unity, Raymond Poincaré Hospital - APHP

Garches, 92380, France

RECRUITING

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood; Muscle Hypotonia

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuromuscular Manifestations; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Susana Quijano-Roy, MD, PhD

  Paediatric neurological and intentive care unity, Raymond Poincaré Hospital - APHP

  PRINCIPAL INVESTIGATOR

• Nour Maalouf, Psychomotor Therapist DE

  Paediatric neurological and intentive care unity, Raymond Poincaré Hospital - APHP

  STUDY DIRECTOR

Central Study Contacts

Susana Quijano-Roy, MD, PhD

CONTACT

+ 33 1 47 10 78 90; susana.quijano-roy@aphp.fr

Nour Maalouf, Psychomotor Therapist DE

CONTACT

nour.maalouf@outlook.com

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: There is one arm in this study, without comparator group. The comparison will be between data of movements in the outside water condition and data of inside water condition.

Study Record Dates

• First Submitted: October 30, 2023
• First Posted: November 30, 2023
• Study Start: June 26, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: November 21, 2025

Record last verified: 2025-09

Locations

FR (1)