- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05136222
Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (3TLA)
A Multi-centre Randomised Controlled Trial of Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (PSG4NIVinMND; 3, Three Letter Acronyms [3TLA])
Study Overview
Status
Intervention / Treatment
Detailed Description
Study Type
Enrollment (Anticipated)
Phase
- Not Applicable
Contacts and Locations
Study Contact
- Name: David Berlowitz, PhD
- Phone Number: +613 9496 3871
- Email: david.berlowitz@austin.org.au
Study Locations
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Adelaide, Australia
- Not yet recruiting
- Flinders Medical Centre
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Contact:
- Dr Vinod Aiyappan
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Brisbane, Australia
- Not yet recruiting
- The Prince Charles Hospital
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Contact:
- Dr Deanne Curtin
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Canberra, Australia
- Not yet recruiting
- Motor Neurone Disease Australia
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Contact:
- Dr Gethin Thomas
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Melbourne, Australia
- Recruiting
- Austin Health
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Contact:
- Associate Professor Mark Howard
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Melbourne, Australia
- Not yet recruiting
- Monash University
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Contact:
- Professor Natasha Lannin
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Melbourne, Australia
- Not yet recruiting
- Australian MND Registry
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Contact:
- A/Professor Paul Talman
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Melbourne, Australia
- Not yet recruiting
- FightMND
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Contact:
- Dr Bec Sheean
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Melbourne, Australia
- Not yet recruiting
- Institute for Breathing and Sleep
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Contact:
- Associate Professor Mark Howard
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Melbourne, Australia
- Not yet recruiting
- University of Melbourne
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Contact:
- Professor David Berlowitz
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Perth, Australia
- Not yet recruiting
- Sir Charles Gairdner Hospital
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Contact:
- Dr Bhajan Singh
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Sydney, Australia
- Not yet recruiting
- Macquarie University
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Contact:
- Professor Dominic Rowe
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Sydney, Australia
- Not yet recruiting
- Royal Prince Alfred Hospital
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Contact:
- Dr Amanda Piper
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Sydney, Australia
- Not yet recruiting
- Westmead Hospital
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Contact:
- Dr John Wheatley
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Age >18 years
- Clinical indication to commence long term NIV
- Confirmed clinical diagnosis of underlying condition
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 Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: RANDOMIZED
- Interventional Model: PARALLEL
- Masking: QUADRUPLE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
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. |
Please refer to 'Arms: Intervention' section.
|
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.
|
Please refer to 'Arms: Control' section.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Adherence with NIV
Time Frame: Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
|
Defined as using NIV > 4 hours/day during the NIV treatment period.
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Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Intolerance of NIV
Time Frame: Change during the acclimatization period (~ 3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
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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).
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Respiratory function
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.
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Forced expiratory volume in 1 second [FEV1], forced vital capacity [FVC]
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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
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.
|
'MIPs/MEPs'.
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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
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.
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'SNIP'.
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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)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Defined as the number of electroencephalogram (EEG) arousals observed per hour of total sleep time (TST).
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Asynchrony index (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Defined as the number of asynchrony events per hour of sleep.
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Oxygen indices (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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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.
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Total sleep time (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Total amount of time asleep in minutes.
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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% rapid eye movement (REM) sleep (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Percentage of sleep characterised by eye movement, relaxation of the body, faster.
respiration, and increased brain activity
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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% slow wave sleep (SWS) (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Percentage of 'deep sleep'.
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Asynchrony sub-indices (during polysomnography)
Time Frame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Ineffective efforts, double-trigger etc.
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During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
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Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)
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.
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A measure of breathlessness in people with ALS/MND.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)
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.
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A measure of health-related quality of life.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)
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.
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A measure of health-related quality of life.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)
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.
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A measure of health-related quality of life.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)
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.
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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.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Sleep quality - Pittsburgh Sleep Quality Index (PSQI)
Time Frame: RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.
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A measure of sleep quality.
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RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.
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Daytime somnolence - Epworth Sleepiness Scale (ESS)
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.
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A measure of daytime sleepiness.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Daytime somnolence - Karolinska Sleepiness Scales (KSS)
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.
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The KSS is rating of the current daytime sleepiness state using a 9-point scale (1 = very alert to 9 = very sleepy, fighting sleep).
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Carer burden - Caregiver Burden Scale (CBS)
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.
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A measure of caregiver burden.
Rated ona scale from 0 (never) to 4 (nearly always), with higher scores indicating greater carer burden.
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During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
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Cost effectiveness of the intervention
Time Frame: Throughout the trial period (approx. 5 years) (retrospective analysis).
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Economic evaluation using MBS/PBS data.
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Throughout the trial period (approx. 5 years) (retrospective analysis).
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Usual clinical care practices
Time Frame: At trial commencement and trial end.
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Multidisciplinary clinician surveys at each recruitment site.
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At trial commencement and trial end.
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Usual care and the barriers and enablers to undertaking the intervention
Time Frame: At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).
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Multidisciplinary clinician focus groups at each recruitment site.
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At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).
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Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage
Time Frame: At trial end (end of RCT; approx. 4 to 5 years)
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Participant semi-structured interviews.
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At trial end (end of RCT; approx. 4 to 5 years)
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Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage
Time Frame: At trial end (end of RCT; approx. 4 to 5 years).
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Caregiver semi-structured interviews.
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At trial end (end of RCT; approx. 4 to 5 years).
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Arterial Blood Gas (ABG) (during polysomnography)
Time Frame: RCT: During the baseline (following the acclimatisation period) and during the follow-up assessment. Cohort: Not Collected.
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Arterial Blood Gas
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RCT: During the baseline (following the acclimatisation period) and during the follow-up assessment. Cohort: Not Collected.
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Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Chair: Abbey Sawyer, PhD, University of Melbourne
Study record dates
Study Major Dates
Study Start (ACTUAL)
Primary Completion (ANTICIPATED)
Study Completion (ANTICIPATED)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ACTUAL)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CT20020
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Time Frame
IPD Sharing Access Criteria
Subject to optional consent, where participants give permission for data to be used for the purpose of:
- The ethically approved research project only.
- This ethically approved research project and any closely related future research projects.
- This ethically approved research project and any future research projects that may or may not be related to this project.
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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