Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis

NCT05328492 | Unknown

• 1 other identifier: EP545921
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Non-invasive ventilation; Home ventilation; iVAPS-AE

Outcome Measures

Primary Outcomes (1)

• Home NIV Compliance

  Measured in hours per night

  90 days

Secondary Outcomes (3)

• Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)

  Baseline, 14, 30, 60, 90 days

• Severe Respiratory Insufficiency Questionnaire (SRI)

  Baseline, 14, 30, 60, 90 days

• Modified Hospital Anxiety and Depression Score (mHADS)

  Baseline, 14, 30, 60, 90 days

Study Arms (2)

iVAPS-AE

EXPERIMENTAL

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

ST-mode

ACTIVE COMPARATOR

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

Interventions

iVAPS-AE 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.

Also known as: ResMed Lumis 150 VPAP ST-A

iVAPS-AE

ST-mode 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.

Also known as: ResMed Lumis 100 VPAP ST-A

ST-mode

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University Hospitals Coventry and Warwickshire NHS Trust: lead

Study Sites (1)

University Hospital Coventry and Warwickshire NHS Trust

Coventry, West Midlands, CV2 2DX, United Kingdom

RECRUITING

Related Publications (20)

• Wolf J, Safer A, Wohrle JC, Palm F, Nix WA, Maschke M, Grau AJ. [Causes of death in amyotrophic lateral sclerosis : Results from the Rhineland-Palatinate ALS registry]. Nervenarzt. 2017 Aug;88(8):911-918. doi: 10.1007/s00115-017-0293-3. German.

  PMID: 28184974 BACKGROUND

• Goetz CG. Amyotrophic lateral sclerosis: early contributions of Jean-Martin Charcot. Muscle Nerve. 2000 Mar;23(3):336-43. doi: 10.1002/(sici)1097-4598(200003)23:33.0.co;2-l.

  PMID: 10679709 BACKGROUND

• Raheja D, Stephens HE, Lehman E, Walsh S, Yang C, Simmons Z. Patient-reported problematic symptoms in an ALS treatment trial. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17(3-4):198-205. doi: 10.3109/21678421.2015.1131831. Epub 2016 Jan 29.

  PMID: 26824413 BACKGROUND

• Arnulf I, Similowski T, Salachas F, Garma L, Mehiri S, Attali V, Behin-Bellhesen V, Meininger V, Derenne JP. Sleep disorders and diaphragmatic function in patients with amyotrophic lateral sclerosis. Am J Respir Crit Care Med. 2000 Mar;161(3 Pt 1):849-56. doi: 10.1164/ajrccm.161.3.9805008.

  PMID: 10712332 BACKGROUND

• NICE. Recommendations | Motor neurone disease: assessment and management | Guidance | NICE [Internet]. NICE; 2016 [cited 2021 May 10]. Available from: https://www.nice.org.uk/guidance/NG42/chapter/Recommendations#prognostic-factors

  BACKGROUND

• Ristell HV, Parkes E, Shakespeare J, Bishopp A, Ali A, Parr D. Non-invasive Ventilation Compliance and Survival Trends in Motor Neurone Disease. European Respiratory Journal [Internet]. 2019 Sep 28 [cited 2021 May 5];54(suppl 63). Available from: https://erj.ersjournals.com/content/54/suppl_63/PA3705

  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.

  PMID: 28410001 BACKGROUND

• Gruis KL, Brown DL, Lisabeth LD, Zebarah VA, Chervin RD, Feldman EL. Longitudinal assessment of noninvasive positive pressure ventilation adjustments in ALS patients. J Neurol Sci. 2006 Aug 15;247(1):59-63. doi: 10.1016/j.jns.2006.03.007. Epub 2006 Apr 24.

  PMID: 16631799 BACKGROUND

• Lo Coco D, Marchese S, Pesco MC, La Bella V, Piccoli F, Lo Coco A. Noninvasive positive-pressure ventilation in ALS: predictors of tolerance and survival. Neurology. 2006 Sep 12;67(5):761-5. doi: 10.1212/01.wnl.0000227785.73714.64. Epub 2006 Aug 9.

  PMID: 16899545 BACKGROUND

• Kim SM, Park KS, Nam H, Ahn SW, Kim S, Sung JJ, Lee KW. Capnography for assessing nocturnal hypoventilation and predicting compliance with subsequent noninvasive ventilation in patients with ALS. PLoS One. 2011 Mar 30;6(3):e17893. doi: 10.1371/journal.pone.0017893.

  PMID: 21479202 BACKGROUND

• Mansell SK, Cutts S, Hackney I, Wood MJ, Hawksworth K, Creer DD, Kilbride C, Mandal S. Using domiciliary non-invasive ventilator data downloads to inform clinical decision-making to optimise ventilation delivery and patient compliance. BMJ Open Respir Res. 2018 Mar 3;5(1):e000238. doi: 10.1136/bmjresp-2017-000238. eCollection 2018.

  PMID: 29531743 BACKGROUND

• Rudnicki SA, Andrews JA, Bian A, Cockroft BM, Cudkowicz ME, Hardiman O, Malik FI, Meng L, Wolff AA, Shefner JM; VITALITY-ALS STUDY GROUP. Noninvasive ventilation use by patients enrolled in VITALITY-ALS. Amyotroph Lateral Scler Frontotemporal Degener. 2021 Nov;22(7-8):486-494. doi: 10.1080/21678421.2021.1904993. Epub 2021 Apr 1.

  PMID: 33792451 BACKGROUND

• Vitacca M, Banfi P, Montini A, Paneroni M. Does timing of initiation influence acceptance and adherence to NIV in patients with ALS? Pulmonology. 2020 Jan-Feb;26(1):45-48. doi: 10.1016/j.pulmoe.2019.05.007. Epub 2019 Jun 5.

  PMID: 31175073 BACKGROUND

• Kleopa KA, Sherman M, Neal B, Romano GJ, Heiman-Patterson T. Bipap improves survival and rate of pulmonary function decline in patients with ALS. J Neurol Sci. 1999 Mar 15;164(1):82-8. doi: 10.1016/s0022-510x(99)00045-3.

  PMID: 10385053 BACKGROUND

• 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.

  PMID: 19890778 BACKGROUND

• Bach JR. Amyotrophic lateral sclerosis. Communication status and survival with ventilatory support. Am J Phys Med Rehabil. 1993 Dec;72(6):343-9.

  PMID: 8260126 BACKGROUND

• Pinto AC, Evangelista T, Carvalho M, Alves MA, Sales Luis ML. Respiratory assistance with a non-invasive ventilator (Bipap) in MND/ALS patients: survival rates in a controlled trial. J Neurol Sci. 1995 May;129 Suppl:19-26. doi: 10.1016/0022-510x(95)00052-4.

  PMID: 7595610 BACKGROUND

• Rabec C, Rodenstein D, Leger P, Rouault S, Perrin C, Gonzalez-Bermejo J; SomnoNIV group. Ventilator modes and settings during non-invasive ventilation: effects on respiratory events and implications for their identification. Thorax. 2011 Feb;66(2):170-8. doi: 10.1136/thx.2010.142661. Epub 2010 Oct 14.

  PMID: 20947891 BACKGROUND

• Sancho J, Servera E, Morelot-Panzini C, Salachas F, Similowski T, Gonzalez-Bermejo J. Non-invasive ventilation effectiveness and the effect of ventilatory mode on survival in ALS patients. Amyotroph Lateral Scler Frontotemporal Degener. 2014 Mar;15(1-2):55-61. doi: 10.3109/21678421.2013.855790. Epub 2013 Nov 25.

  PMID: 24266679 BACKGROUND

• Morelot-Panzini C, Bruneteau G, Gonzalez-Bermejo J. NIV in amyotrophic lateral sclerosis: The 'when' and 'how' of the matter. Respirology. 2019 Jun;24(6):521-530. doi: 10.1111/resp.13525. Epub 2019 Mar 25.

  PMID: 30912216 BACKGROUND

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Respiratory Insufficiency; Sleep Apnea Syndromes; Motor Neuron Disease; Nervous System Diseases; Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Respiration Disorders; Respiratory Tract Diseases; Apnea; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders

Study Officials

• David G Parr, MD

  University Hospitals Coventry and Warwickshire NHS Trust

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Edward Parkes, MSc

CONTACT

02476966734; edward.parkes@uhcw.nhs.uk

Joanna Shakespeare, MSc

CONTACT

02476966734; joanna.shakespeare@uhcw.nhs.uk

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: PARTICIPANT
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2022
• First Posted: April 14, 2022
• Study Start: March 15, 2022
• Primary Completion: December 15, 2023
• Study Completion: March 15, 2024
• Last Updated: June 1, 2023

Record last verified: 2023-05

Locations

GB (1)