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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05136222




Registration number
NCT05136222
Ethics application status
Date submitted
21/10/2021
Date registered
29/11/2021

Titles & IDs
Public title
Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease
Scientific title
A Multi-centre Randomised Controlled Trial of Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (PSG4NIVinMND; 3, Three Letter Acronyms [3TLA])
Secondary ID [1] 0 0
CT20020
Universal Trial Number (UTN)
Trial acronym
3TLA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Motor Neuron Disease / Amyotrophic Lateral Sclerosis 0 0
Condition category
Condition code
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Other interventions - Intervention polysomnography
Other interventions - Sham polysomnography

Experimental: Intervention - 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.

Placebo comparator: Control - 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 interventions: Intervention polysomnography
Please refer to 'Arms: Intervention' section.

Other interventions: Sham polysomnography
Please refer to 'Arms: Control' section.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adherence with NIV
Timepoint [1] 0 0
Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
Secondary outcome [1] 0 0
Intolerance of NIV
Timepoint [1] 0 0
Change during the acclimatization period (~ 3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
Secondary outcome [2] 0 0
Respiratory function
Timepoint [2] 0 0
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.
Secondary outcome [3] 0 0
Maximal inspiratory/expiratory pressure
Timepoint [3] 0 0
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.
Secondary outcome [4] 0 0
Sniff nasal pressure
Timepoint [4] 0 0
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.
Secondary outcome [5] 0 0
Arousal index (during polysomnography)
Timepoint [5] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [6] 0 0
Asynchrony index (during polysomnography)
Timepoint [6] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [7] 0 0
Oxygen indices (during polysomnography)
Timepoint [7] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [8] 0 0
Total sleep time (during polysomnography)
Timepoint [8] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [9] 0 0
% rapid eye movement (REM) sleep (during polysomnography)
Timepoint [9] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [10] 0 0
% slow wave sleep (SWS) (during polysomnography)
Timepoint [10] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [11] 0 0
Asynchrony sub-indices (during polysomnography)
Timepoint [11] 0 0
During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
Secondary outcome [12] 0 0
Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)
Timepoint [12] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [13] 0 0
Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)
Timepoint [13] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [14] 0 0
Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)
Timepoint [14] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [15] 0 0
Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)
Timepoint [15] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [16] 0 0
Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)
Timepoint [16] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [17] 0 0
Sleep quality - Pittsburgh Sleep Quality Index (PSQI)
Timepoint [17] 0 0
RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [18] 0 0
Daytime somnolence - Epworth Sleepiness Scale (ESS)
Timepoint [18] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [19] 0 0
Daytime somnolence - Karolinska Sleepiness Scales (KSS)
Timepoint [19] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [20] 0 0
Carer burden - Caregiver Burden Scale (CBS)
Timepoint [20] 0 0
During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
Secondary outcome [21] 0 0
Cost effectiveness of the intervention
Timepoint [21] 0 0
Throughout the trial period (approx. 5 years) (retrospective analysis).
Secondary outcome [22] 0 0
Usual clinical care practices
Timepoint [22] 0 0
At trial commencement and trial end.
Secondary outcome [23] 0 0
Usual care and the barriers and enablers to undertaking the intervention
Timepoint [23] 0 0
At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).
Secondary outcome [24] 0 0
Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage
Timepoint [24] 0 0
At trial end (end of RCT; approx. 4 to 5 years)
Secondary outcome [25] 0 0
Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage
Timepoint [25] 0 0
At trial end (end of RCT; approx. 4 to 5 years).

Eligibility
Key inclusion criteria
* Age >18 years
* Clinical indication to commence long term NIV
* Confirmed clinical diagnosis of underlying condition
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
NA
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
28/02/2028
Actual
Sample size
Target
244
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Flinders Medical Centre - Adelaide
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Brisbane
Recruitment hospital [3] 0 0
Motor Neurone Disease Australia - Canberra
Recruitment hospital [4] 0 0
Austin Health - Melbourne
Recruitment hospital [5] 0 0
Australian MND Registry - Melbourne
Recruitment hospital [6] 0 0
FightMND - Melbourne
Recruitment hospital [7] 0 0
Institute for Breathing and Sleep - Melbourne
Recruitment hospital [8] 0 0
Monash University - Melbourne
Recruitment hospital [9] 0 0
University of Melbourne - Melbourne
Recruitment hospital [10] 0 0
Sir Charles Gairdner Hospital - Perth
Recruitment hospital [11] 0 0
Macquarie University - Sydney
Recruitment hospital [12] 0 0
Royal Prince Alfred Hospital - Sydney
Recruitment hospital [13] 0 0
Westmead Hospital - Sydney
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Canberra
Recruitment postcode(s) [4] 0 0
- Melbourne
Recruitment postcode(s) [5] 0 0
- Perth
Recruitment postcode(s) [6] 0 0
- Sydney

Funding & Sponsors
Primary sponsor type
Other
Name
University of Melbourne
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Austin Hospital, Melbourne Australia
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Institute for Breathing and Sleep, Australia
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Royal Prince Alfred Hospital, Sydney, Australia
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
Macquarie University, Australia
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
Macquarie Health
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
Western Sydney Local Health District
Address [6] 0 0
Country [6] 0 0
Other collaborator category [7] 0 0
Government body
Name [7] 0 0
Flinders Medical Centre
Address [7] 0 0
Country [7] 0 0
Other collaborator category [8] 0 0
Other
Name [8] 0 0
Sir Charles Gairdner Hospital
Address [8] 0 0
Country [8] 0 0
Other collaborator category [9] 0 0
Government body
Name [9] 0 0
The Prince Charles Hospital
Address [9] 0 0
Country [9] 0 0
Other collaborator category [10] 0 0
Other
Name [10] 0 0
Monash University
Address [10] 0 0
Country [10] 0 0
Other collaborator category [11] 0 0
Other
Name [11] 0 0
Motor Neurone Disease Australia
Address [11] 0 0
Country [11] 0 0
Other collaborator category [12] 0 0
Other
Name [12] 0 0
FightMND
Address [12] 0 0
Country [12] 0 0
Other collaborator category [13] 0 0
Other
Name [13] 0 0
Australian Motor Neurone Disease Registry
Address [13] 0 0
Country [13] 0 0
Other collaborator category [14] 0 0
Other
Name [14] 0 0
Calvary Bethlehem
Address [14] 0 0
Country [14] 0 0
Other collaborator category [15] 0 0
Other
Name [15] 0 0
Perron Institute for Neurological and Translational Science
Address [15] 0 0
Country [15] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Abbey Sawyer, PhD
Address 0 0
University of Melbourne
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
David Berlowitz, PhD
Address 0 0
Country 0 0
Phone 0 0
+613 9496 3871
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Dataset in de-identified form will be shared on request to corresponding author.
When will data be available (start and end dates)?
5 years from study completion.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05136222