Neuromuscular DiseasesPhase 2VT-NMDNCT06339580
Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease
A Phase 2 study testing Volume-targeted non-invasive ventilation in people with Neuromuscular Diseases, run by Guy's and St Thomas' NHS Foundation Trust at 2 sites in United Kingdom.
Trial summary
Assessment of safety and efficacy of volume-targeted ventilation in patients with neuromuscular disease. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Neuromuscular Diseases
- What is being tested?
- Volume-targeted non-invasive ventilation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 9 Apr 2024to 30 Sep 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom2 sites, 2 recruiting
- Recruiting
Guy's & St Thomas' NHS Foundation Trust
London, London, SE1 9RT
Gillian Radcliffe, 02071888070, Gillian.Radcliffe@gstt.nhs.uk
- Recruiting
Guy's and St. Thomas NHS Foundation Trust
London, SE1 7EH
Neeraj Shah, neeraj.shah@gstt.nhs.uk
Gillian Radcliffe, Gillian.Radcliffe@gstt.nhs.uk
About this study
Official title: A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease
Assessment of safety and efficacy of volume-targeted ventilation in patients with neuromuscular disease.
Read the 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.
- Conditions
- Neuromuscular Diseases
- Being tested
- Volume-targeted non-invasive ventilation
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Volume-targeted non-invasive ventilationDevice
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.
| Study group | Role | What they receive |
|---|---|---|
| VT-NMD arm | Experimental | all patients will be trialed on volume targeted non invasive ventilation Receives: Device: Volume-targeted non-invasive ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean overnight transcutaneous carbon dioxide
Average carbon dioxide level overnight
Measured: 2 night
Secondary outcomes (5)
Maximum overnight transcutaneous carbon dioxide
Maximum carbon dioxide level reached overnight
Measured: 2 night
Overnight desaturation index
The number of desaturation episodes per night during sleep
Measured: 2 night
Visual analogue scale of sleep comfort
Assessment of sleep comfort on each ventilator on 100mm scale. Min score 0mm (worse sleep comfort), Maximum score 100mm (best sleep comfort)
Measured: 2 night
Health-related quality of life
- Quality of Life Measure for people with slowly progressive and genetic neuromuscular disease questionnaire. Minimum score (worst QoL) 0, Maximum score (best QoL) 100.
Measured: 3 months
Adherence to ventilation
average hours per night the patient uses the ventilator,
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2024
- Study start9 Apr 2024
- Primary completion30 Sep 2026 (estimated)
- Study completion30 Sep 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseasesStudies of the same condition, open and under way.
- More trials run by Guy's and St Thomas' NHS Foundation TrustThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Guy's and St Thomas' NHS Foundation Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06339580
- Sponsor's protocol ID
- 324456
- Acronym
- VT-NMD
- Status last verified
- Dec 2025
- First submitted
- 25 Mar 2024
- Last updated
- 8 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease
- ClinicalTrials.gov ID
- NCT06339580
- Acronym / sponsor ID
- VT-NMD / 324456
- Conditions
- Neuromuscular Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Guy's and St Thomas' NHS Foundation TrustUniversity, hospital or other
- Study start
- 9 Apr 2024 (actual)
- Primary completion
- 30 Sep 2026 (estimated)
- Study completion
- 30 Sep 2026 (estimated)
- First posted
- 1 Apr 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| VT-NMD arm | Experimental | Device: Volume-targeted non-invasive ventilationall patients will be trialed on volume targeted non invasive ventilation |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- 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)
Key exclusion criteria (6 listed)
- Rapidly progressive neuromuscular disease
- Decompensated respiratory failure (pH < 7.35)
- Pregnancy
- Aged <18, >80
- Poor adherence to NIV (<4hrs per night)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Volume-targeted non-invasive ventilationA target tidal volume is set on the ventilation device, and the device will then automatically adjust delivered pressures to reach this target tidal volume. | Device | VT-NMD arm |
Primary outcome
Mean overnight transcutaneous carbon dioxide
Time frame: 2 night
Average carbon dioxide level overnight
5 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Volume-targeted non-invasive ventilation in people with Neuromuscular Diseases, run by Guy's and St Thomas' NHS Foundation Trust at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 8 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Patrick Murphy (02071888070, Patrick.murphy@gstt.nhs.uk), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean overnight transcutaneous carbon dioxide (time frame: 2 night). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jun 2026.