Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease

Neuromuscular Diseases Clinical Trial

— VT-NMD  

Official title:

A Proof-on-concept Study to Investigate the Potential Use of Volume-targeted Ventilation in Patients With Slowly Progressive Neuromuscular Disease

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06339580
• Other study ID #: 324456
• Status: Recruiting
• Phase: Phase 2
• Start date: April 9, 2024
• Est. completion date: April 2026

Study information

• Verified date: May 2024
• Source: Guy's and St Thomas' NHS Foundation Trust
• Contact: Patrick Murphy
• Phone: 02071888070
• Email: Patrick.murphy[@]gstt.nhs.uk
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Assessment of safety and efficacy of volume-targeted ventilation in patients with neuromuscular disease.

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 information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: April 2026
• Est. primary completion date: March 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

Eligibility

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

Related Conditions & MeSH terms

• Neuromuscular Diseases

Intervention

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.

Locations

Country	Name	City	State
United Kingdom	Guy's & St Thomas' NHS Foundation Trust	London
United Kingdom	Guy's and St. Thomas NHS Foundation Trust	London

Sponsors (1)

Lead Sponsor	Collaborator
Guy's and St Thomas' NHS Foundation Trust

Country where clinical trial is conducted

United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean overnight transcutaneous carbon dioxide	Average carbon dioxide level overnight	2 night
Secondary	Maximum overnight transcutaneous carbon dioxide	Maximum carbon dioxide level reached overnight	2 night
Secondary	Overnight desaturation index	The number of desaturation episodes per night during sleep	2 night
Secondary	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)	2 night
Secondary	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.	3 months
Secondary	Adherence to ventilation	average hours per night the patient uses the ventilator,	3 months