A two-arm, individual participant randomised controlled, assessor-blinded trial in 7 MND care centres across Australia will be undertaken.
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
244
Please refer to 'Arms: Intervention' section.
Please refer to 'Arms: Control' section.
Flinders Medical Centre
Adelaide, Australia
NOT_YET_RECRUITINGThe Prince Charles Hospital
Brisbane, Australia
NOT_YET_RECRUITINGAdherence with NIV
Defined as using NIV \> 4 hours/day during the NIV treatment period.
Time frame: Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
Intolerance of NIV
Defined as cessation of NIV during the NIV treatment period and/or \< 4 hours.
Time frame: Change during the acclimatization period (~ 3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
Respiratory function
Forced expiratory volume in 1 second \[FEV1\], forced vital capacity \[FVC\]
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.
Maximal inspiratory/expiratory pressure
'MIPs/MEPs'.
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.
Sniff nasal pressure
'SNIP'.
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.
Arousal index (during polysomnography)
Defined as the number of electroencephalogram (EEG) arousals observed per hour of total sleep time (TST).
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Motor Neurone Disease Australia
Canberra, Australia
NOT_YET_RECRUITINGAustin Health
Melbourne, Australia
RECRUITINGAustralian MND Registry
Melbourne, Australia
NOT_YET_RECRUITINGFightMND
Melbourne, Australia
NOT_YET_RECRUITINGInstitute for Breathing and Sleep
Melbourne, Australia
NOT_YET_RECRUITINGMonash University
Melbourne, Australia
NOT_YET_RECRUITINGUniversity of Melbourne
Melbourne, Australia
NOT_YET_RECRUITINGSir Charles Gairdner Hospital
Perth, Australia
NOT_YET_RECRUITING...and 3 more locations
Asynchrony index (during polysomnography)
Defined as the number of asynchrony events per hour of sleep.
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Oxygen indices (during polysomnography)
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.
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Total sleep time (during polysomnography)
Total amount of time asleep in minutes.
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
% rapid eye movement (REM) sleep (during polysomnography)
Percentage of sleep characterised by eye movement, relaxation of the body, faster. respiration, and increased brain activity
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
% slow wave sleep (SWS) (during polysomnography)
Percentage of 'deep sleep'.
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Asynchrony sub-indices (during polysomnography)
Ineffective efforts, double-trigger etc.
Time frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)
A measure of breathlessness in people with ALS/MND.
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.
Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)
A measure of health-related quality of life.
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.
Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)
A measure of health-related quality of life.
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.
Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)
A measure of health-related quality of life.
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.
Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)
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.
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.
Sleep quality - Pittsburgh Sleep Quality Index (PSQI)
A measure of sleep quality.
Time frame: RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.
Daytime somnolence - Epworth Sleepiness Scale (ESS)
A measure of daytime sleepiness.
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.
Daytime somnolence - Karolinska Sleepiness Scales (KSS)
The KSS is rating of the current daytime sleepiness state using a 9-point scale (1 = very alert to 9 = very sleepy, fighting sleep).
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.
Carer burden - Caregiver Burden Scale (CBS)
A measure of caregiver burden. Rated ona scale from 0 (never) to 4 (nearly always), with higher scores indicating greater carer burden.
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.
Cost effectiveness of the intervention
Economic evaluation using MBS/PBS data.
Time frame: Throughout the trial period (approx. 5 years) (retrospective analysis).
Usual clinical care practices
Multidisciplinary clinician surveys at each recruitment site.
Time frame: At trial commencement and trial end.
Usual care and the barriers and enablers to undertaking the intervention
Multidisciplinary clinician focus groups at each recruitment site.
Time frame: At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).
Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage
Participant semi-structured interviews.
Time frame: At trial end (end of RCT; approx. 4 to 5 years)
Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage
Caregiver semi-structured interviews.
Time frame: At trial end (end of RCT; approx. 4 to 5 years).