A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease

What this Trial Is About

This research is focused on patients with slowly progressive neuromuscular diseases who experience breathing difficulties requiring support from non-invasive ventilation (NIV). The study aims to assess the safety and effectiveness of a new type of NIV called volume-targeted ventilation (VT-NIV), which adjusts the pressure delivered to the lungs to reach a specific breath volume. This approach is being evaluated to better control carbon dioxide levels, which are linked to clinical outcomes in these patients. Participants currently using fixed bi-level NIV will undergo a two-night hospital stay for assessment. On the first night, their carbon dioxide control will be measured using their usual ventilator. On the second night, they will switch to VT-NIV. After discharge, participants will use VT-NIV at home for three months. The study will observe both patients with well-controlled and poorly controlled carbon dioxide to evaluate safety and potential benefits of VT-NIV. During the study, researchers will collect data from the ventilation devices and patient questionnaires to assess adherence and quality of life. Measurements include overnight transcutaneous carbon dioxide levels, oxygen desaturation, sleep comfort, and health-related quality of life. The follow-up visit at three months will review these outcomes and monitor participant safety. The total participation includes the initial two-night hospital stay and the three-month home use period.

Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease