RecruitingPHASE2INTERVENTIONAL

Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease

This study is for people with muscle-weakening conditions (neuromuscular diseases) who already use a breathing device called non-invasive ventilation (NIV). NIV helps your lungs remove a waste gas called carbon dioxide. Normally, NIV devices deliver a set amount of air, but a new type, called volume-targeted NIV (VT-NIV), can adjust itself. This study will check if this new VT-NIV is safe and effective in controlling carbon dioxide levels. We will ask participants to stay in our centre for two nights, trying both their usual device and the new one. Then, they’ll use the new device at home for three months before a final check-up. The information we gather will help us plan larger studies to see if VT-NIV is better than current treatments.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Guy's and St Thomas' NHS Foundation Trust

Enrolment target

Start

09 Apr 2024

Estimated completion

30 Sep 2026

Neuromuscular Diseases

What is this study about?

If you have a neuromuscular disease, your breathing muscles might not work as well as they should. This can lead to a build-up of a waste gas called carbon dioxide (CO2) in your body. When CO2 levels are too high, it can make you feel unwell and potentially lead to more serious health problems. To help with this, many patients use a special breathing machine called non-invasive ventilation (NIV) at home. NIV helps by providing support to your lungs.

Usually, the NIV machine provides a set amount of air pressure when you breathe in and out, and this pressure doesn't change much. However, newer technology allows the machine to adjust how much air it gives you, depending on your breathing. One of these newer methods is called volume-targeted non-invasive ventilation (VT-NIV). With VT-NIV, the machine is set to deliver a specific amount of air with each breath, and it automatically changes its settings to make sure you get that amount, even if your breathing patterns change a little.

There have been a few smaller studies that suggest these 'smart' machines might be better at controlling carbon dioxide levels, but we need more research to be sure. This study aims to carefully check how safe and effective this VT-NIV is. The information we gather will be really important for us to plan a bigger study in the future. That larger study would compare VT-NIV directly with the standard, fixed-pressure NIV, to see which one works best for patients in the long run.

Key takeaways

This study tests a new 'smart' breathing machine setting for muscle conditions.
It aims to see if it safely and effectively controls carbon dioxide levels.
It involves a two-night stay, then three months of home use, followed by a check-up.
You must already use a breathing machine for your condition.
The findings will help plan future studies to improve breathing support.

Who may be eligible?

This study is looking for volunteers who have a muscle-weakening condition that is getting worse slowly, not quickly. You should already be using a specific type of breathing machine called fixed bi-level ventilation for your breathing support. It's important that your breathing has been stable recently, meaning you haven't been in hospital or had changes to your breathing machine settings due to lung problems in the last six weeks. If your breathing isn't currently stable, or if your condition is getting worse very quickly, this study isn't suitable for you right now.

There are also some age requirements; participants need to be between 18 and 80 years old, so if you're younger or older than that, you won't be able to join. We also ask that you are already using your current breathing machine for at least 4 hours every night. If you're pregnant, or have other serious health conditions that might make it difficult for you to follow the study plan, you wouldn't be able to take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do you have a neuromuscular disease that is slowly getting worse?
Are you currently using a fixed-pressure non-invasive breathing machine?
Have you been stable with your breathing for the last 6 weeks (no hospital stays or ventilator changes)?
Are you between 18 and 80 years old?
Do you use your breathing machine for at least 4 hours every night?
Are you not pregnant and don't have other serious health issues that would affect your participation?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you'll first be admitted to our centre for a two-night stay. On the first night, we'll monitor your carbon dioxide levels while you use your usual breathing machine. On the second night, we will switch your machine to the new volume-targeted setting, and we will monitor your carbon dioxide levels again.

After these two nights, you'll be discharged and asked to use this new volume-targeted setting on your breathing machine at home for three months. During this time, you'll use your machine as usual, but with the new settings. At the end of the three months, you'll come back to our clinic for an outpatient visit. At this visit, we'll download information from your machine to see how it's been working and ask you to fill in some questionnaires about your experience and how you're feeling. There are no study medications involved.

Potential risks and benefits

Participating in this study might offer you the benefit of potentially better control of your carbon dioxide levels with the new breathing machine settings. For individuals whose CO2 levels are currently well-controlled, this study will help us confirm the safety of the new approach. Any risks would mostly relate to the inconvenience of the two-night hospital stay and getting used to the new machine settings for three months. As with all research, you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (2)

Guy's & St Thomas' NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting
Guy's and St. Thomas NHS Foundation Trust
Verified postcode
London, United Kingdom· Recruiting

Common questions

What is 'non-invasive ventilation'?

It's a breathing machine that helps support your lungs without needing a tube placed directly into your windpipe. It uses a mask over your nose or nose and mouth.

What are neuromuscular diseases?

These are conditions that affect your nerves and muscles, which can lead to muscle weakness, including weakness in the muscles you use to breathe.

Why is controlling carbon dioxide (CO2) important?

High levels of CO2 in your body can make you feel tired, have headaches, and can be bad for your overall health. Keeping it controlled helps you feel better and stay healthier.

Will my regular doctor know I'm in the study?

Yes, we would share information with your regular medical team to ensure your care is coordinated.

What if I feel worse on the new machine setting?

If you have any concerns or feel unwell, you should contact the study team immediately. You can also stop using the new setting and return to your usual one, and you can withdraw from the study at any time.

How to find out more

Patrick Murphy

patrick.murphy@gstt.nhs.uk 02071888070 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.