Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis

NCT ID: NCT05328492

Last Updated: 2023-06-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-15

Study Completion Date

2024-03-15

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Amyotrophic Lateral Sclerosis Respiratory Failure Respiratory Insufficiency Sleep-Disordered Breathing Neuro-Degenerative Disease Neuron Disease, Motor Nervous System Diseases Neuromuscular Diseases

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Participants

Study Groups

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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.

Group Type EXPERIMENTAL

iVAPS-AE

Intervention Type DEVICE

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.

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.

Group Type ACTIVE_COMPARATOR

ST-mode

Intervention Type DEVICE

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.

Interventions

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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.

Intervention Type 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.

Intervention Type DEVICE

Other Intervention Names

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ResMed Lumis 150 VPAP ST-A ResMed Lumis 100 VPAP ST-A

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

100 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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University Hospitals Coventry and Warwickshire NHS Trust

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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David G Parr, MD

Role: PRINCIPAL_INVESTIGATOR

University Hospitals Coventry and Warwickshire NHS Trust

Locations

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University Hospital Coventry and Warwickshire NHS Trust

Coventry, West Midlands, United Kingdom

Site Status RECRUITING

Countries

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United Kingdom

Central Contacts

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Edward Parkes, MSc

Role: CONTACT

02476966734

Joanna Shakespeare, MSc

Role: CONTACT

02476966734

Facility Contacts

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Claire Finnie

Role: primary

02476 966195

Sonia Kandola

Role: backup

024 7696 6195

References

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Reference Type BACKGROUND

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Reference Type BACKGROUND

Nicholson TT, Smith SB, Siddique T, Sufit R, Ajroud-Driss S, Coleman JM 3rd, Wolfe LF. Respiratory Pattern and Tidal Volumes Differ for Pressure Support and Volume-assured Pressure Support in Amyotrophic Lateral Sclerosis. Ann Am Thorac Soc. 2017 Jul;14(7):1139-1146. doi: 10.1513/AnnalsATS.201605-346OC.

Reference Type BACKGROUND
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Reference Type BACKGROUND
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Reference Type BACKGROUND
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Czudaj KP, Suchi S, Schonhofer B. [Physiological parameters of breathing and the impact of non-invasive ventilation (NIV) on patients with amyotrophic lateral sclerosis (ALS)]. Pneumologie. 2009 Dec;63(12):687-92. doi: 10.1055/s-0029-1215130. Epub 2009 Nov 3. German.

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Other Identifiers

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EP545921

Identifier Type: -

Identifier Source: org_study_id

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