Clinical trial NCT05136222: Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease
Official title: A Multi-centre Randomised Controlled Trial of Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (PSG4NIVinMND; 3, Three Letter Acronyms [3TLA])
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Motor Neuron Disease / Amyotrophic Lateral Sclerosis
Keywords: Non-invasive ventilation
Polysomnography
Sleep study
Amyotrophic lateral sclerosis
Chronic respiratory failure
Lead sponsor: University of Melbourne
Sponsor class: OTHER
Collaborators: name: Austin Hospital, Melbourne Australia
name: Institute for Breathing and Sleep, Australia
name: Royal Prince Alfred Hospital, Sydney, Australia
name: Macquarie University, Australia
name: Macquarie Health
name: Western Sydney Local Health District
name: Flinders Medical Centre
name: Sir Charles Gairdner Hospital
name: The Prince Charles Hospital
name: Monash University
name: Motor Neurone Disease Australia
name: FightMND
name: Australian Motor Neurone Disease Registry
name: Calvary Bethlehem
name: Perron Institute for Neurological and Translational Science
First posted: 2021-11-29
Enrollment: 244
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomised controlled trial with 12 month cohort follow-up
Primary purpose: TREATMENT

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

Detailed description: 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: 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

Primary outcomes:
measure: Adherence with NIV
timeFrame: Change during the acclimatization period (~3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
Secondary outcomes:
measure: Intolerance of NIV
timeFrame: Change during the acclimatization period (~ 3 weeks) and during the NIV treatment period (~7-8 weeks) (approx. 10 weeks total per participant).
measure: Respiratory function
timeFrame: 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.
measure: Maximal inspiratory/expiratory pressure
timeFrame: 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.
measure: Sniff nasal pressure
timeFrame: 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.
measure: Arousal index (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: Asynchrony index (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: Oxygen indices (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: Total sleep time (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: % rapid eye movement (REM) sleep (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: % slow wave sleep (SWS) (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: Asynchrony sub-indices (during polysomnography)
timeFrame: During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.
measure: Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Sleep quality - Pittsburgh Sleep Quality Index (PSQI)
timeFrame: RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Daytime somnolence - Epworth Sleepiness Scale (ESS)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Daytime somnolence - Karolinska Sleepiness Scales (KSS)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Carer burden - Caregiver Burden Scale (CBS)
timeFrame: During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.
measure: Cost effectiveness of the intervention
timeFrame: Throughout the trial period (approx. 5 years) (retrospective analysis).
measure: Usual clinical care practices
timeFrame: At trial commencement and trial end.
measure: Usual care and the barriers and enablers to undertaking the intervention
timeFrame: At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).
measure: Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage
timeFrame: At trial end (end of RCT; approx. 4 to 5 years)
measure: Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage
timeFrame: At trial end (end of RCT; approx. 4 to 5 years).
Other outcomes:
measure: Arterial Blood Gas (ABG) (during polysomnography)
timeFrame: RCT: During the baseline (following the acclimatisation period) and during the follow-up assessment. Cohort: Not Collected.
Locations:
city: Adelaide
country: Australia
facility: Flinders Medical Centre
city: Brisbane
country: Australia
facility: The Prince Charles Hospital
city: Canberra
country: Australia
facility: Motor Neurone Disease Australia
city: Melbourne
country: Australia
facility: Austin Health
city: Melbourne
country: Australia
facility: Australian MND Registry
city: Melbourne
country: Australia
facility: FightMND
city: Melbourne
country: Australia
facility: Institute for Breathing and Sleep
city: Melbourne
country: Australia
facility: Monash University
city: Melbourne
country: Australia
facility: University of Melbourne
city: Perth
country: Australia
facility: Sir Charles Gairdner Hospital
city: Sydney
country: Australia
facility: Macquarie University
city: Sydney
country: Australia
facility: Royal Prince Alfred Hospital
city: Sydney
country: Australia
facility: Westmead Hospital
Study officials:
affiliation: University of Melbourne
name: Abbey Sawyer, PhD
role: STUDY_CHAIR
References: