Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease
- Conditions
- Neuromuscular Diseases
- Interventions
- Device: Volume-targeted non-invasive ventilation
- Registration Number
- NCT06339580
- Lead Sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Brief Summary
Assessment of safety and efficacy of volume-targeted ventilation in patients with neuromuscular disease.
- Detailed Description
Patients with neuromuscular diseases (NMD) can suffer from breathing difficulties requiring treatment with a breathing device known as non-invasive ventilation (NIV). NIV aims to support the lungs in removing the waste gas, carbon dioxide (CO2). This is important because patients with high CO2 levels tend to have worse clinical outcomes.
NIV delivers different pressures to the airway when the patient breathes in and when they breathe out. Usually, the pressures it delivers are fixed; i.e. they do not change breath-to-breath. Newer technology allows the machine to independently change the pressures, depending on various patient factors it can measure. There are a small number of studies that suggested that these 'auto-titrating' machines may improve control of carbon dioxide but further work is needed. One of these modes allows us to set a target volume that should be delivered to the patient each breath, and the machine changes settings to deliver this target volume, in response to changing patient parameters. We aim to investigate the safety and efficacy of this volume-targeted NIV (VT-NIV), in order to generate data to design a randomised controlled trial to compare VT-NIV with fixed-NIV.
Patients with NMD who use fixed-NIV will be admitted for a two-night stay to our centre. On the first night, their CO2 control will be assessed on their current ventilator. On the second night, they will be switched to the VT-NIV mode. They will be discharged and asked to use the new mode for three months. Individuals with well-controlled CO2 with their usual mode will allow us to assess the safety of VT-NIV, and individuals with poorly-controlled CO2 will allow us to assess its efficacy. At three months, they will attend an outpatient visit, where use of the new mode will be assessed through data download from the machine and completion of questionnaires.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 30
- Slowly progressive neuromuscular disease
- Established on fixed bi-level ventilation
- Documented clinical respiratory stability by supervising clinician (no hospitalizations, respiratory infections or change to ventilator settings in preceding 6 weeks)
- Rapidly progressive neuromuscular disease
- Decompensated respiratory failure (pH < 7.35)
- Pregnancy
- Aged <18, >80
- Poor adherence to NIV (<4hrs per night)
- Significant physical or psychiatric co-morbidity that would prevent compliance with trial protocol
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VT-NMD arm Volume-targeted non-invasive ventilation all patients will be trialed on volume targeted non invasive ventilation
- Primary Outcome Measures
Name Time Method Mean overnight transcutaneous carbon dioxide 2 night Average carbon dioxide level overnight
- Secondary Outcome Measures
Name Time Method Health-related quality of life 3 months - Quality of Life Measure for people with slowly progressive and genetic neuromuscular disease questionnaire. Minimum score (worst QoL) 0, Maximum score (best QoL) 100.
Maximum overnight transcutaneous carbon dioxide 2 night Maximum carbon dioxide level reached overnight
Overnight desaturation index 2 night The number of desaturation episodes per night during sleep
Visual analogue scale of sleep comfort 2 night Assessment of sleep comfort on each ventilator on 100mm scale. Min score 0mm (worse sleep comfort), Maximum score 100mm (best sleep comfort)
Adherence to ventilation 3 months average hours per night the patient uses the ventilator,
Trial Locations
- Locations (2)
Guy's & St Thomas' NHS Foundation Trust
🇬🇧London, United Kingdom
Guy's and St. Thomas NHS Foundation Trust
🇬🇧London, United Kingdom