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

Not Applicable

Completed

• Conditions: Spinal Muscular Atrophy; Non Invasive Ventilation; Respiratory Bacterial Infection
• Interventions: Other: non invasive ventilation samples; Other: patient samples; Other: parent questionnaire

• Registration Number: NCT05712330
• Lead Sponsor: Centre Hospitalier Universitaire, Amiens

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2023-01-24
• Study First Submitted: 2023-01-26
• Study First Submitted QC: 2023-01-26
• Study First Posted: 2023-02-03
• Primary Completion: 2024-08-24
• Status Verified: 2025-05
• Study Completion: 2025-05-22
• Last Update Submitted: 2025-05-22
• Last Update Posted: 2025-05-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

• Diagnosis of AMS (previous diagnosis or new diagnosis during the inclusion period)
• Age from 0 to 12 years old
• Nusinersen treatment
• Benefiting from a NIV
• Current monitoring at the University Hospital of Amiens

Exclusion Criteria

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
non invasive ventilation	non invasive ventilation samples	SMA patients with NIV
non invasive ventilation	patient samples	SMA patients with NIV
non invasive ventilation	parent questionnaire	SMA patients with NIV

Primary Outcome Measures

Name	Time	Method
number of hospitalization days for respiratory exacerbations with germs identical to protocol samples	one year

Trial Locations

Locations (1)

Centre Hospitalier Universitaire d'Amiens; 🇫🇷 Amiens, Picardie, France