Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Volume-targeted non-invasive ventilation.
- Who it may be relevant to
- Registry conditions: Neuromuscular Diseases. Basic parameters: 18 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease
Overview
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.
Interventions
- Device Volume-targeted non-invasive ventilation
A target tidal volume is set on the ventilation device, and the device will then automatically adjust delivered pressures to reach this target tidal volume.
Primary outcome measures
- Mean overnight transcutaneous carbon dioxide [Time frame: 2 night]
Secondary outcome measures (5)
- Maximum overnight transcutaneous carbon dioxide [Time frame: 2 night]
- Overnight desaturation index [Time frame: 2 night]
- Visual analogue scale of sleep comfort [Time frame: 2 night]
- Health-related quality of life [Time frame: 3 months]
- Adherence to ventilation [Time frame: 3 months]
Eligibility criteria
Inclusion criteria
- 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)
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United Kingdom · 2 centers
- Guy's & St Thomas' NHS Foundation Trust — London
- Guy's and St. Thomas NHS Foundation Trust — London
Identifiers
NCT: NCT06339580 · 324456