Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis

May 31, 2023 updated by: University Hospitals Coventry and Warwickshire NHS Trust

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Estimated)

40

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United Kingdom
    • West Midlands
      • Coventry, West Midlands, United Kingdom, CV2 2DX
        • Recruiting
        • University Hospital Coventry and Warwickshire NHS Trust
        • Contact:
        • Contact:
        • Principal Investigator:
          • David G Parr, MD
        • Sub-Investigator:
          • Edward Parkes, MSc

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 100 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Single

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Home NIV Compliance
Time Frame: 90 days
Measured in hours per night
90 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Time Frame: 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.
Baseline, 14, 30, 60, 90 days
Severe Respiratory Insufficiency Questionnaire (SRI)
Time Frame: 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.
Baseline, 14, 30, 60, 90 days
Modified Hospital Anxiety and Depression Score (mHADS)
Time Frame: 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).
Baseline, 14, 30, 60, 90 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospitals Coventry and Warwickshire NHS Trust

Investigators

  • Principal Investigator: David G Parr, MD, University Hospitals Coventry and Warwickshire Nhs Trust

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 15, 2022

Primary Completion (Estimated)

December 15, 2023

Study Completion (Estimated)

March 15, 2024

Study Registration Dates

First Submitted

April 7, 2022

First Submitted That Met QC Criteria

April 7, 2022

First Posted (Actual)

April 14, 2022

Study Record Updates

Last Update Posted (Actual)

June 1, 2023

Last Update Submitted That Met QC Criteria

May 31, 2023

Last Verified

May 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EP545921

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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