A Multi-centre Randomised Controlled Trial of Polysomnographic Titration of Non-invasive Ventilation in Motor Neurone Disease (PSG4NIVinMND; 3, Three Letter Acronyms [3TLA])
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 244
- 试验地点
- 13
- 主要终点
- Adherence with NIV
研究概览
简要总结
A two-arm, individual participant randomised controlled, assessor-blinded trial in 7 MND care centres across Australia will be undertaken.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
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.
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age >18 years
- •Clinical indication to commence long term NIV
- •Confirmed clinical diagnosis of underlying condition
排除标准
- •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
研究组 & 干预措施
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.
干预措施: Intervention polysomnography (Other)
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.
干预措施: Sham polysomnography (Other)
结局指标
主要结局
Adherence with NIV
时间窗: 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.
次要结局
- 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.)
- 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.)
- Asynchrony index (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- Health-related quality of life - Severe Respiratory Insufficient Questionnaire (SRI)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Usual care and the barriers and enablers to undertaking the intervention(At trial commencement (start of RCT) and trial end (end of RCT; approx. 4 to 5 years).)
- Carer burden - Caregiver Burden Scale (CBS)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Cost effectiveness of the intervention(Throughout the trial period (approx. 5 years) (retrospective analysis).)
- Experience of receiving the intervention and the barriers and enablers to the PSG and NIV usage(At trial end (end of RCT; approx. 4 to 5 years))
- 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.)
- Total sleep time (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- Asynchrony sub-indices (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- Dyspnoea Amyotrophic Lateral Sclerosis (DALS-15)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Functional rating - Amyotrophic Lateral Sclerosis Functional Rating Scale (Revised) (ALSFRS)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Daytime somnolence - Karolinska Sleepiness Scales (KSS)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Usual clinical care practices(At trial commencement and trial end.)
- Oxygen indices (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- % rapid eye movement (REM) sleep (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- Health-related quality of life - Assessment of Quality of Life (8-Dimension-AQoL)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Health-related quality of life - Calgary Sleep Apnoea Quality of Life Index (SAQLI)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- Daytime somnolence - Epworth Sleepiness Scale (ESS)(During the baseline (~week 0) and during the follow-up assessment (~ week 3 + 7). Cohort: At 3, 6 and 12 months following RCT commencement.)
- % slow wave sleep (SWS) (during polysomnography)(During the baseline (following the ~3 week acclimatisation period) and during the follow-up assessment (~ week 3 + 7). Cohort: Not Collected.)
- Sleep quality - Pittsburgh Sleep Quality Index (PSQI)(RCT: During the baseline and during the follow-up assessment. Cohort: At 3, 6 and 12 months following RCT commencement.)
- Experience of the person they are caring for receiving the intervention and the barriers and enablers to the PSG and NIV usage(At trial end (end of RCT; approx. 4 to 5 years).)