Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease (VT-NMD)

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Neuromuscular Diseases
• Intervention / Treatment: Device: Volume-targeted non-invasive ventilation

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 Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Patrick Murphy; Phone Number: 02071888070; Email: Patrick.murphy@gstt.nhs.uk

Study Locations

• United Kingdom
  • London, United Kingdom, SE1 9RT
    • Guy's & St Thomas' NHS Foundation Trust
  • London, United Kingdom, SE1 7EH
    • Guy's and St. Thomas NHS Foundation Trust

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: VT-NMD arm; all patients will be trialed on volume targeted non invasive ventilation	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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean overnight transcutaneous carbon dioxide	Average carbon dioxide level overnight	2 night

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum overnight transcutaneous carbon dioxide	Maximum carbon dioxide level reached overnight	2 night
Overnight desaturation index	The number of desaturation episodes per night during sleep	2 night
Adherence to ventilation	average hours per night the patient uses the ventilator,	3 months
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
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

Collaborators and Investigators

• Sponsor: Guy's and St Thomas' NHS Foundation Trust

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): April 1, 2026

Study Registration Dates

• First Submitted: March 25, 2024
• First Submitted That Met QC Criteria: March 29, 2024
• First Posted (Actual): April 1, 2024

Study Record Updates

• Last Update Posted (Actual): April 25, 2024
• Last Update Submitted That Met QC Criteria: April 23, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: 324456

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No