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

NCT ID: NCT05712330

Last Updated: 2025-05-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-01-24
• Study Completion Date: 2025-05-22

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

non invasive ventilation

SMA patients with NIV

Group Type: EXPERIMENTAL

non invasive ventilation samples

Intervention Type: OTHER

machine samples for bacterial culture will be performed

patient samples

Intervention Type: OTHER

Patient samples for sputum culture

parent questionnaire

Intervention Type: OTHER

parent questionnaire

Interventions

non invasive ventilation samples

machine samples for bacterial culture will be performed

Intervention Type: OTHER

patient samples

Patient samples for sputum culture

Intervention Type: OTHER

parent questionnaire

parent questionnaire

Intervention Type: OTHER

Eligibility Criteria

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

• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Centre Hospitalier Universitaire, Amiens

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Centre Hospitalier Universitaire d'Amiens

Amiens, Picardie, France

Countries

France

Other Identifiers

PI2022_843_0095

Identifier Type: -

Identifier Source: org_study_id