Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Spinal Muscular Atrophy Under NIV

NCT05712330 | Completed

• 1 other identifier: PI2022_843_0095
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

SMA (Spinal Muscular Atrophy) is a rare neuromuscular disease characterized by motoneuron damage. Symptoms consist of respiratory involvement with numerous respiratory infections and eventually respiratory failure, for which NIV (Non Invasive Ventilation) is often used. Ventilation machines are in close contact with the respiratory tract of patients. They contain heated water to humidify the circuit. These humid and warm environments are conducive to the development of bacteria such as Pseudomonas aeruginosa. In this context, it is interesting to look for the presence or absence of bacteria, in comparison with the respiratory ecology of the patients. The aim is to highlight the microbiological role of NIV on the occurrence of respiratory bacterial infections or secondary infections in patients with SMA. To do this, samples are taken from the machines, and ECBCs are performed on patients during respiratory physiotherapy sessions.

Conditions

Non Invasive Ventilation; Spinal Muscular Atrophy; Respiratory Bacterial Infection

Keywords

non invasive ventilation; Spinal Muscular Atrophy; Respiratory Bacterial Infection

Outcome Measures

Primary Outcomes (1)

• number of hospitalization days for respiratory exacerbations with germs identical to protocol samples

  one year

Study Arms (1)

non invasive ventilation

EXPERIMENTAL

SMA patients with NIV

Other: non invasive ventilation samples; Other: patient samples; Other: parent questionnaire

Interventions

non invasive ventilation samples OTHER

machine samples for bacterial culture will be performed

non invasive ventilation

patient samples OTHER

Patient samples for sputum culture

non invasive ventilation

parent questionnaire OTHER

parent questionnaire

non invasive ventilation

Eligibility Criteria

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

You may qualify if:

• Age from 0 to 12 years old
• Nusinersen treatment
• Benefiting from a NIV
• Current monitoring at the University Hospital of Amiens

You may not qualify if:

• Invasive ventilation on tracheotomy
• No ventilation
• Other treatment than Nusinersen or no treatment
• Parental refusal to participate in the study

Sponsors & Collaborators

• Centre Hospitalier Universitaire, Amiens: lead

Study Sites (1)

Centre Hospitalier Universitaire d'Amiens

Amiens, Picardie, 80000, France

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Design

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

Study Record Dates

• First Submitted: January 26, 2023
• First Posted: February 3, 2023
• Study Start: January 24, 2023
• Primary Completion: August 24, 2024
• Study Completion: May 22, 2025
• Last Updated: May 28, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)