Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease

NCT05136222 | Recruiting DMC

• 1 other identifier: CT20020
• Study Type: interventional
• Target: 244
• Locations: 1 country
• Sites: 13

Brief Summary

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

Conditions

Motor Neuron Disease / Amyotrophic Lateral Sclerosis

Keywords

Non-invasive ventilation; Polysomnography; Sleep study; Amyotrophic lateral sclerosis; Chronic respiratory failure

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (25)

• Intolerance of NIV

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

• Respiratory function

  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

  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

  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)

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

Other Outcomes (1)

• Arterial Blood Gas (ABG) (during polysomnography)

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

Study Arms (2)

Intervention

EXPERIMENTAL

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

Control

PLACEBO COMPARATOR

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

Interventions

Intervention polysomnography OTHER

Please refer to 'Arms: Intervention' section.

Intervention

Sham polysomnography OTHER

Please refer to 'Arms: Control' section.

Control

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

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

Study Sites (13)

Flinders Medical Centre

Adelaide, Australia

NOT YET RECRUITING

The Prince Charles Hospital

Brisbane, Australia

NOT YET RECRUITING

Motor Neurone Disease Australia

Canberra, Australia

NOT YET RECRUITING

Austin Health

Melbourne, Australia

RECRUITING

Australian MND Registry

Melbourne, Australia

NOT YET RECRUITING

FightMND

Melbourne, Australia

NOT YET RECRUITING

Institute for Breathing and Sleep

Melbourne, Australia

NOT YET RECRUITING

Monash University

Melbourne, Australia

NOT YET RECRUITING

University of Melbourne

Melbourne, Australia

NOT YET RECRUITING

Sir Charles Gairdner Hospital

Perth, Australia

NOT YET RECRUITING

Macquarie University

Sydney, Australia

NOT YET RECRUITING

Royal Prince Alfred Hospital

Sydney, Australia

NOT YET RECRUITING

Westmead Hospital

Sydney, Australia

NOT YET RECRUITING

Related Publications (2)

• Graco M, Berlowitz DJ, Sawyer A, Holland AE, Carey KA, Ahamed Y, Ridgers A, Lannin NA; 3TLA trial Group. Polysomnographic titration of non-invasive ventilation in motor neurone disease (3TLA): protocol for a process evaluation of a clinical trial. Trials. 2025 Mar 6;26(1):79. doi: 10.1186/s13063-025-08784-z.

  PMID: 40050976 DERIVED

• Berlowitz DJ, Rowe D, Howard ME, Piper A, Graco M, Braat S, Singh B, Souza TV, Lannin N, McLean A, Sawyer A, Carey KA, Ahamed Y; 3TLA Trial Group. Polysomnographic titration of non-invasive ventilation in motor neurone disease (3TLA): study protocol for a randomised controlled trial. Trials. 2025 Jan 6;26(1):10. doi: 10.1186/s13063-024-08464-4.

  PMID: 39762986 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

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

Study Officials

• Abbey Sawyer, PhD

  University of Melbourne

  STUDY CHAIR

Central Study Contacts

David Berlowitz, PhD

CONTACT

+613 9496 3871; david.berlowitz@austin.org.au

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: The attending sleep scientist will refer to a database that reveals (statistician-generated) participant's treatment allocation. The sleep scientist will not reveal the treatment allocation to the Clinical team, Research team or the participant. Centralised allocation concealment will be ensured through the Adept/REDCap trial database.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Randomised controlled trial with 12 month cohort follow-up

Study Record Dates

• First Submitted: October 21, 2021
• First Posted: November 29, 2021
• Study Start: December 15, 2021
• Primary Completion: February 28, 2026
• Study Completion (Estimated): February 28, 2028
• Last Updated: June 23, 2022

Record last verified: 2022-06

Data Sharing

• IPD Sharing: Will share
• Time Frame: 5 years from study completion.
• Access Criteria: Subject to optional consent, where participants give permission for data to be used for the purpose of: 1. The ethically approved research project only. 2. This ethically approved research project and any closely related future research projects. 3. This ethically approved research project and any future research projects that may or may not be related to this project.

Locations

AU (13)