An interventional study of Intervention polysomnography and Sham polysomnography in Motor Neuron Disease / Amyotrophic Lateral Sclerosis, sponsored by University of Melbourne. Recruiting at 13 sites in Australia. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-06-23.
Sponsored by University of Melbourne · Not applicable, Interventional, and Treatment
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.
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's planned enrollment of 244 is above the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →University of Melbourne is the lead sponsor of 84 studies on the registry; 22 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
This trial of PSG-assisted commencement of non-invasive ventilation (NIV) in motor neurone disease (MND) follows the methodology of our previous single-site study (Hannan et al 2019 ERJ), with the addition of an open label cohort that extends until (the earlier of) 12 months or death. After empirical NIV set-up and an acclimatisation period (3 weeks), participants will undergo single night in-laboratory polysomnography (PSG). The PSG will be performed and supervised by a sleep scientist. In the intervention group, the "intervention" PSG results will be used to adjust/titrate NIV settings to optimize ventilation and improve synchrony between the patient and the NIV device. Participants will be asked to continue to use NIV as prescribed for the subsequent 7 week intervention period.
Other: Intervention polysomnography
The participants allocated to the control group will also be asked to attend a single night in-laboratory PSG. The NIV settings will not be adjusted throughout the PSG ("sham" PSG). Participants in the control group will retain their original settings after the sham PSG, and will be asked to continue to use NIV in this manner for the subsequent 7 week intervention period.
Other: Sham polysomnography
Please refer to 'Arms: Intervention' section.
Please refer to 'Arms: Control' section.
Adherence 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.
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).
Arterial Blood Gas (ABG) (during polysomnography)
Arterial Blood Gas
Time frame: RCT: During the baseline (following the acclimatisation period) and during the follow-up assessment. Cohort: Not Collected.
Plan to share: Yes — Dataset in de-identified form will be shared on request to corresponding author.
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University of Melbourne