Share
Save
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.
Study details:
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.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 18 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2021-12-15
Primary completion: 2026-02-28
Study completion finish: 2028-02-28
Study type
TREATMENT
Phase
NA
Trial ID
NCT05136222
Intervention or treatment
OTHER: Intervention polysomnography
OTHER: Sham polysomnography
Conditions
- • Motor Neuron Disease / Amyotrophic Lateral Sclerosis
Find a site
Closest Location:
Austin Health
Research sites nearby
Select from list below to view details:
Austin Health
Melbourne, Not Specified, Australia
Royal Prince Alfred Hospital
Sydney, Not Specified, Australia
Flinders Medical Centre
Adelaide, Not Specified, Australia
The Prince Charles Hospital
Brisbane, Not Specified, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: Intervention
| OTHER: Intervention polysomnography
|
PLACEBO_COMPARATOR: Control
| OTHER: Sham polysomnography
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| Adherence with NIV | Defined as using NIV \> 4 hours/day during the NIV treatment period. | Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant). |
Secondary outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| Intolerance of NIV | Defined as cessation of NIV during the NIV treatment period and/or \< 4 hours. | 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 \[FVC\] | 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'. | 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'. | 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). | 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. | 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. | 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. | 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 | 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'. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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). | 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. | 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. | Throughout the trial period (approx. 5 years) (retrospective analysis). |
| Usual clinical care practices | Multidisciplinary clinician surveys at each recruitment site. | 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. | 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. | 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. | At trial end (end of RCT; approx. 4 to 5 years). |
Frequently Asked Questions
Please note: some questions and answers are submitted by anonymous patients or using AI, and have not been verified by Clinrol
No questions submitted. Be the first to ask a question!