Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis
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| ClinicalTrials.gov Identifier: NCT05328492 |
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Recruitment Status :
Recruiting
First Posted : April 14, 2022
Last Update Posted : April 29, 2022
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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.
| 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 | Not Applicable |
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 (Clinical Trial) |
| Estimated 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 : | March 15, 2022 |
| Estimated Primary Completion Date : | December 15, 2023 |
| Estimated Study Completion Date : | March 15, 2024 |
| Arm | Intervention/treatment |
|---|---|
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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.
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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 Name: ResMed Lumis 150 VPAP ST-A |
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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.
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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 Name: ResMed Lumis 100 VPAP ST-A |
- Home NIV Compliance [ Time Frame: 90 days ]Measured in hours per night
- 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.
- 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.
- 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).
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 100 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05328492
| Contact: Edward Parkes, MSc | 02476966734 | edward.parkes@uhcw.nhs.uk | |
| Contact: Joanna Shakespeare, MSc | 02476966734 | joanna.shakespeare@uhcw.nhs.uk |
| United Kingdom | |
| University Hospital Coventry and Warwickshire NHS Trust | Recruiting |
| Coventry, West Midlands, United Kingdom, CV2 2DX | |
| Contact: Claire Finnie 02476 966195 ResearchSponsorship@uhcw.nhs.uk | |
| Contact: Sonia Kandola 024 7696 6195 ResearchSponsorship@uhcw.nhs.uk | |
| Principal Investigator: David G Parr, MD | |
| Sub-Investigator: Edward Parkes, MSc | |
| Principal Investigator: | David G Parr, MD | University Hospitals Coventry and Warwickshire NHS Trust |
| Responsible Party: | University Hospitals Coventry and Warwickshire NHS Trust |
| ClinicalTrials.gov Identifier: | NCT05328492 |
| Other Study ID Numbers: |
EP545921 |
| First Posted: | April 14, 2022 Key Record Dates |
| Last Update Posted: | April 29, 2022 |
| Last Verified: | April 2022 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Non-invasive ventilation Home ventilation iVAPS-AE |
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Respiratory Insufficiency Sleep Apnea Syndromes Motor Neuron Disease Amyotrophic Lateral Sclerosis Nervous System Diseases Neuromuscular Diseases Neurodegenerative Diseases Sclerosis Pathologic Processes Respiration Disorders |
Respiratory Tract Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Apnea Sleep Disorders, Intrinsic Dyssomnias Sleep Wake Disorders |