Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis

Sponsor

University Hospitals Coventry and Warwickshire NHS Trust (Other)

Overall Status

Recruiting

CT.gov ID

NCT05328492

Collaborator

(none)

Enrollment

Location

Arms

Anticipated Duration (Months)

1.7

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to assess the efficacy of using intelligent volume assured pressure support (iVAPS-AE) versus spontaneous timed (ST) modes of non-invasive ventilation (NIV) in patients diagnosed with amyotrophic lateral sclerosis (ALS).

The investigators believe that the use of iVAPS-AE mode NIV over a 90 day period will produce NIV compliance data and health-related quality of life (HRQOL) scores that are equivalent or no worse compared to ST mode NIV.

Condition or Disease	Intervention/Treatment	Phase
Amyotrophic Lateral Sclerosis Respiratory Failure Respiratory Insufficiency Sleep-Disordered Breathing Neuro-Degenerative Disease Neuron Disease, Motor Nervous System Diseases Neuromuscular Diseases	Device: iVAPS-AE Device: ST-mode	N/A

Detailed Description

Amyotrophic lateral sclerosis (ALS) is one of five motor neurone diseases (MNDs). It is a rare, incurable disease characterised by progressive destruction of nerve cells called motor neurones that instruct a patient's muscles to contract, to enable all movements, including walking, talking, speaking and swallowing.

Over time as more motor neurone cells are damaged, the muscles used to breathe will weaken and patients will develop breathlessness and sleep disturbances. This can be very distressing and reduce a patient's health- related quality of life (HRQOL). Eventually the condition may progress to the extent that the patient will develop respiratory failure, which is the leading cause of death in ALS.

Respiratory failure is the most frequent cause of death in ALS and as such a significant proportion of ALS cases are complicated by respiratory and bulbar symptoms which can reduce HRQOL from breathlessness, impaired cough and sleep, and can also shorten life expectancy. The use of respiratory support, provided via non-invasive ventilation (NIV), has been shown to be beneficial.

NIV is a safe treatment in ALS and as such current National Institute for Health and Care Excellence (NICE) guidelines recommend a trial of NIV in those ALS patients who develop respiratory impairment

NIV compliance is of significant importance in ALS as it is directly linked to improved survival and health related quality of life. Compliance is affected by various factors including non-invasive ventilation mode, disease type (bulbar vs limb) and baseline physiology.

Various NIV modes exist including pressure support (ST mode) and volume assured pressure support (iVAPS-AE). Evidence suggests that not one mode is more superior, but both have advantages and disadvantages in clinical practice.

This study will follow a standard care pathway and aim to recruit 40 ALS patients randomised to receive ST mode or iVAPS-AE mode. Each patient will enrol onto the study for 90 days and attend 5 hospital visits. The study will assess if iVAPS-AE improves a patient's symptoms sooner and allows a patient to use the NIV for longer periods thereby improving HRQOL.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

40 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Single (Participant)

Primary Purpose:

Treatment

Official Title:

Volume Targeted Versus Pressure Targeted Non-invasive Ventilation in Amyotrophic Lateral Sclerosis: a Randomised Control Trial.

Actual Study Start Date :

Mar 15, 2022

Anticipated Primary Completion Date :

Dec 15, 2023

Anticipated Study Completion Date :

Mar 15, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: iVAPS-AE Patients with ALS and respiratory insufficiency randomised to this arm will be treated with home NIV using the Intelligent Volume-Assured Pressure Support with automatic EPAP (iVAPS-AE) mode.	Device: iVAPS-AE Patients randomised to this intervention will commence home NIV in iVAPS-AE mode according to the clinical operating procedures used at the Respiratory and Sleep Science Department. Patients will follow a NHS standard care pathway as described in the study protocol. Other Names: ResMed Lumis 150 VPAP ST-A
Active Comparator: ST-mode Patients with ALS and respiratory insufficiency randomised to this arm will be treated with home NIV using the spontaneous timed (ST) home NIV mode.	Device: ST-mode Patients randomised to this intervention will commence home NIV in ST-mode according to the clinical operating procedures used at the Respiratory and Sleep Science Department. Patients will follow a NHS standard care pathway as described in the study protocol. Other Names: ResMed Lumis 100 VPAP ST-A

Arm

Intervention/Treatment

Experimental: iVAPS-AE

Patients with ALS and respiratory insufficiency randomised to this arm will be treated with home NIV using the Intelligent Volume-Assured Pressure Support with automatic EPAP (iVAPS-AE) mode.

Device: iVAPS-AE

Patients randomised to this intervention will commence home NIV in iVAPS-AE mode according to the clinical operating procedures used at the Respiratory and Sleep Science Department. Patients will follow a NHS standard care pathway as described in the study protocol.

Other Names:

ResMed Lumis 150 VPAP ST-A

Active Comparator: ST-mode

Patients with ALS and respiratory insufficiency randomised to this arm will be treated with home NIV using the spontaneous timed (ST) home NIV mode.

Device: ST-mode

Patients randomised to this intervention will commence home NIV in ST-mode according to the clinical operating procedures used at the Respiratory and Sleep Science Department. Patients will follow a NHS standard care pathway as described in the study protocol.

Other Names:

ResMed Lumis 100 VPAP ST-A

Outcome Measures

Primary Outcome Measures

Home NIV Compliance [90 days]
Measured in hours per night

Secondary Outcome Measures

Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) [Baseline, 14, 30, 60, 90 days]
A 12-item questionnaire to monitor the disease progression covering bulbar, motor and respiratory function. Total scores range from 0 to 48. Higher scores indicate better function.
Severe Respiratory Insufficiency Questionnaire (SRI) [Baseline, 14, 30, 60, 90 days]
A 49-item questionnaire to measure health related quality of life in patients receiving long term non-invasive ventilation (NIV). Total scores range from 49 to 245. Higher scores indicate worse outcomes.
Modified Hospital Anxiety and Depression Score (mHADS) [Baseline, 14, 30, 60, 90 days]
A 14-item questionnaire to measure anxiety and depression levels. The questionnaire consists of anxiety sub-scale and depression sub-scale. Scores for each sub-scale range from 0 to 21. Higher scores indicate greater anxiety/depression levels. This version is modified for use in patients diagnosed with amyotrophic lateral sclerosis (ALS).

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 100 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients with respiratory failure secondary to ALS (diagnosed either at an MND MDT or specialist neurology clinic) according to criteria set out in the NICE guideline (NG42) (2016); Motor neurone disease: assessment and management.

Patients able to provide informed consent to take part in the research study.
Patients not contraindicated to commence NIV in accordance with local protocol.
Patients not currently enrolled in another research study that could alter disease progression.

Exclusion Criteria:

Acutely unwell or medically complicated patients as assessed by lead investigator. These patients will be urgently reviewed by a dedicated Consultant Physician. The Principal Investigator will be immediately informed.
An inability to provide informed consent.
An inability to use NIV.
Patients whom are contraindicated to commence NIV in accordance with local protocol.