Polysomnographic titration of non-invasive ventilation in motor neurone disease (3TLA): study protocol for a randomised controlled trial.

Background: Non-invasive ventilation (NIV) uses positive pressure to assist people with respiratory muscle weakness or severe respiratory compromise to breathe. Most people use this treatment during sleep when breathing is most susceptible to instability. The benefits of using NIV in motor neurone disease (MND) are well-established. However, uptake and usage are low (~ 19%) and there is no consensus on how to best implement NIV in MND in Australia. Consequently, clinical practice models are highly variable. Our team has recently provided evidence that specific and individualised NIV titration using a sleep study (polysomnography; PSG) leads to better outcomes in people with MND. However, for this clinical practice model to result in sustained benefits, evidence of effectiveness across multiple sites, as well as culture and practice change, must occur.
Methods: A two-arm, assessor-blinded, individual participant randomised controlled trial in MND care centres across Australia will be undertaken. Two-hundred and forty-four participants will be randomised (1:1) to either the intervention group (PSG-assisted commencement of NIV settings; PSG) or a control group (sham PSG). Participants will be asked to use their NIV device for 7 weeks and will then return for follow-up assessments. Respiratory, sleep and patient-reported outcome measures will be collected at baseline and follow-up. The primary aim is to determine if the proportion of participants using NIV for > 4 h/day during the intervention period is higher in the PSG than the control group. A process evaluation, health economic evaluation and 12-month cohort follow-up will be undertaken and reported separately.
Discussion: The results of this trial will demonstrate the effects of PSG-assisted titration of NIV on usage of NIV in people with MND. We hypothesise that the PSG intervention will improve synchrony between the user and the machine, which will lead to greater NIV usage compared to the control group.
Trial Registration: ClinicalTrials.gov NCT05136222. Registered on November 25, 2021.
Competing Interests: Declarations. Ethics approval and consent to participate: Ethical approval for all sites has been granted by Austin Health Human Research Ethics Committee (HREC/68088/Austin-2021). Written, informed consent to participate will be obtained from all participants. Any proposed amendments to the project including any changes to the protocol, Participant Information and Consent Form/s, agreed upon by the Trial Steering Committee, will be submitted to the reviewing HREC for approval prior to implementation. These changes will be communicated to the trial participants by members of the research team and re-consent for participation in the research will be collected if required. Consent for publication: Not applicable—no identifying images or other personal or clinical details of participants are presented here or will be presented in reports of the trial results. Competing interests: The authors declare that they have no competing interests.
(© 2025. The Author(s).)