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NCT Number: NCT05136222

Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease

A two-arm, individual participant randomised controlled, assessor-blinded trial in 7 MND care centres across Australia will be undertaken.

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Key information

Conditions

Motor Neuron Disease / Amyotrophic Lateral Sclerosis Amyotrophic Lateral Sclerosis Central Nervous System Diseases Metabolic Diseases Motor Neuron Disease Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies Spinal Cord Diseases TDP-43 Proteinopathies

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Flinders Medical Centre, Adelaide, Australia

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About this study

Non-invasive ventilation (NIV) is a treatment that uses positive pressure delivered via a face mask or mouthpiece to assist a person to breathe. It can be used as a long-term treatment for people whose breathing is failing - usually due to chronic conditions that produce weakness of the respiratory muscles such as motor neurone disease / amyotrophic lateral sclerosis [MND/ALS]chronic obstructive pulmonary disease). Most people with MND/ALS use NIV at night initially. Even though NIV may improve survival and function, many are unable to use it for more than 4 hours per day (which is considered a threshold amount of use in order to gain a benefit) and many others are unable to tolerate it at all. Our team has recently provided evidence that specific and individualised titration of NIV leads to better outcomes in people with MND. This previous trial determined that the use of a sleep study (also called 'polysomnography') can improve the way people are initially set up with NIV. This study will replicate and extend the single site study in a large, multi-centre randomised controlled trial (RCT) across multiple sites This multi-centre RCT will also include a 12-month follow-up period to evaluate longer-term outcomes.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Age >18 years
  • Clinical indication to commence long term NIV
  • Confirmed clinical diagnosis of underlying condition

Exclusion criteria

  • Medically unstable
  • Hypoventilation attributable to medications with sedative/respiratory depressant side- effects
  • Use of NIV for more than 1 month in the previous 3 months
  • Inability to provide informed consent
  • Previous intolerance of NIV

Treatment and study plan

Intervention polysomnography

Other

Please refer to 'Arms: Intervention' section.

Sham polysomnography

Other

Please refer to 'Arms: Control' section.

Primary outcomes

  1. Adherence with NIV

    Time frame: Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).

    Defined as using NIV > 4 hours/day during the NIV treatment period.

Secondary outcomes

  1. Intolerance of NIV

    Time frame: Change during the acclimatization period (~ 3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).

    Defined as cessation of NIV during the NIV treatment period and/or < 4 hours.

  2. Respiratory function

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement as able.

    Forced expiratory volume in 1 second [FEV1], forced vital capacity [FVC]

  3. Maximal inspiratory/expiratory pressure

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement as able.

    'MIPs/MEPs'.

  4. Sniff nasal pressure

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement as able.

    'SNIP'.

  5. Arousal index (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Defined as the number of electroencephalogram (EEG) arousals observed per hour of total sleep time (TST).

  6. Asynchrony index (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Defined as the number of asynchrony events per hour of sleep.

  7. Oxygen indices (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Multiple measures to summarise oxygenation as one single outcome including oxygen desaturation index (defined as the total number of oxygen desaturation episodes [= 4%] per hour of total), sleep time, nadir SpO2, and time with SpO2 < 90%, area under the curve and others.

  8. Total sleep time (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Total amount of time asleep in minutes.

  9. % rapid eye movement (REM) sleep (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Percentage of sleep characterised by eye movement, relaxation of the body, faster. respiration, and increased brain activity

  10. % slow wave sleep (SWS) (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Percentage of 'deep sleep'.

  11. Asynchrony sub-indices (during polysomnography)

    Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.

    Ineffective efforts, double-trigger etc.

  12. Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of breathlessness in people with ALS/MND.

  13. Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of health-related quality of life.

  14. Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of health-related quality of life.

  15. Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of health-related quality of life.

  16. Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A clinical measure of functional rating in people with ALS/MND. Minimum score: 0, maximum score: 40. The higher the score the more function is retained.

  17. Sleep quality - Pittsburgh Sleep Quality Index (PSQI)

    Time frame: RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of sleep quality.

  18. Daytime somnolence - Epworth Sleepiness Scale (ESS)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of daytime sleepiness.

  19. Daytime somnolence - Karolinska Sleepiness Scales (KSS)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    The KSS is rating of the current daytime sleepiness state using a 9-point scale (1 = very alert to 9 = very sleepy, fighting sleep).

  20. Carer burden - Caregiver Burden Scale (CBS)

    Time frame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.

    A measure of caregiver burden. Rated ona scale from 0 (never) to 4 (nearly always), with higher scores indicating greater carer burden.

  21. Cost effectiveness of the intervention

    Time frame: Throughout the trial period (approx. 5 years) (retrospective analysis).

    Economic evaluation using MBS/PBS data.

  22. Usual clinical care practices

    Time frame: At trial commencement and trial end.

    Multidisciplinary clinician surveys at each recruitment site.

  23. Usual care and the barriers and enablers to undertaking the intervention

    Time frame: At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).

    Multidisciplinary clinician focus groups at each recruitment site.

  24. Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage

    Time frame: At trial end (end of RCT; approx. 4 to 5 years)

    Participant semi-structured interviews.

  25. Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage

    Time frame: At trial end (end of RCT; approx. 4 to 5 years).

    Caregiver semi-structured interviews.

Other outcomes

  1. Arterial Blood Gas (ABG) (during polysomnography)

    Time frame: RCT: During the baseline (following the acclimatisation period) and during the follow-up assessment. Cohort: Not Collected.

    Arterial Blood Gas

Study contacts

Contact information is provided by the study sponsor or research team.

David Berlowitz, PhD

CONTACT

[email protected]

+613 9496 3871

Sponsors and collaborators

Lead sponsor

University of Melbourne

Other

Collaborators

  • Austin Hospital, Melbourne Australia
  • Australian Motor Neurone Disease Registry
  • Calvary Bethlehem
  • FightMND
  • Flinders Medical Centre
  • Institute for Breathing and Sleep, Australia
  • Macquarie Health
  • Macquarie University, Australia
  • Monash University
  • Motor Neurone Disease Australia
  • Perron Institute for Neurological and Translational Science
  • Royal Prince Alfred Hospital, Sydney, Australia
  • Sir Charles Gairdner Hospital
  • The Prince Charles Hospital
  • Western Sydney Local Health District

Registry information

Official study title

A Multi-centre Randomised Controlled Trial of Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (PSG4NIVinMND; 3, Three Letter Acronyms [3TLA])

Acronym: 3TLA

Important dates

Study start
2021
Primary completion
2026
Study completion
2028
First posted
Nov 29, 2021
Registry last updated
Jun 23, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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