Evaluating a new breathing device for patients with neuromuscular diseases
A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease
This study is testing a new breathing device that adjusts to patients' needs to see if it helps people with neuromuscular diseases breathe better than their current equipment.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | Guy's and St Thomas' NHS Foundation Trust Academic / other |
| Locations | 2 sites (London and 1 other locations) |
| Trial ID | NCT06339580 on ClinicalTrials.gov |
What this trial studies
This study assesses the safety and efficacy of volume-targeted non-invasive ventilation (VT-NIV) in patients with neuromuscular diseases who require breathing support. Participants will be admitted for two nights, during which their carbon dioxide control will be evaluated using their current fixed non-invasive ventilation device. On the second night, they will switch to the VT-NIV mode, which adjusts pressures based on patient needs to deliver a target volume of air. The goal is to gather data that will inform a future randomized controlled trial comparing VT-NIV with traditional fixed ventilation methods.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 80 with slowly progressive neuromuscular diseases who are stable on fixed bi-level ventilation.
Not a fit: Patients with rapidly progressive neuromuscular diseases or those experiencing decompensated respiratory failure may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could lead to improved respiratory outcomes and quality of life for patients with neuromuscular diseases.
How similar studies have performed: There have been a few studies suggesting that auto-titrating ventilation may improve carbon dioxide control, but further investigation is needed to confirm these findings.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
London and 1 other locations
- Guy's and St. Thomas NHS Foundation Trust — London, United Kingdom (Recruiting)
- Guy's & St Thomas' NHS Foundation Trust — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Patrick Murphy
- Email: Patrick.murphy@gstt.nhs.uk
- Phone: 02071888070
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.