Progression of Respiratory Dysfunction in Amyotrophic Lateral Sclerosis (ALS) Patients: A Comparison of Standard of Practice vs Polysomnography-Directed Nocturnal Non-Invasive Positive Pressure Ventilation
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 25
- 试验地点
- 1
- 主要终点
- Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
研究概览
简要总结
Use of noninvasive ventilation (NIV, also known colloquially as "Bipap") has been associated in some studies with improvement in pulmonary function, quality of life and survival. NIV is typically applied during sleep, and without the benefit of sleep study to determine the optimal settings. The investigators have shown that when NIV is used in this fashion, failure of nocturnal oxygenation and ventilation is prominent. This study is randomizing patients to standard application of NIV vs application guided by use of sleep study data to determine the effect of titrated therapy on pulmonary function, quality of life and survival.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Supportive Care
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria
- •Between ages of 18 and 80 yrs old
排除标准
- •Inability to clear secretions from the airway
- •Life expectancy < 6 months from a comorbid condition
- •Dementia sufficient to impair ability to use NIV, perform respiratory muscle pressure testing (PFTs), or complete Health-related Quality of Life (HRQOL) instruments
- •Inability to follow up at the ALS Center on a regular basis
- •Previously diagnosed obstructive Sleep Apnea
研究组 & 干预措施
Standard NIV
Noninvasive ventilation (NIV) will be initiated and managed as per current standard of practice guided by the American Academy of Neurology (AAN) Practice Parameters (updated in 2009), in all subjects with amyotrophic lateral sclerosis (ALS) and a forced vital capacity of <50% predicted. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months, but will not influence management of the NIV.
干预措施: Standard initiation of NIV (Other)
Sleep study titrated NIV
ALS subjects in this arm, who are offered NIV for Forced Vital Capacity (FVC) <50% as per AAN Practice Parameters, will have their initial level of NIV determined polysomnographically. They will be followed with sleep studies at 1 month, 3 months and 6 months to reassess NIV efficacy and NIV will be adjusted as necessary to optimize parameters of oxygenation and ventilation.
干预措施: Sleep study-guided adjustment of NIV (Other)
结局指标
主要结局
Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures
时间窗: Up to 6 months after starting NIV
FVC (forced vital capacity), FEV1 (forced expiratory volume in 1 second), MIP (maximum inspiratory pressure) and MEP (maximum expiratory pressure
次要结局
- Duration that the Mental Component Summary (MCS) is maintained above 75% of baseline score for the Medical Outcomes Study Health Survey (SF-12)(Up to 6 months after starting NIV)
- Survival(Up to 6 months after baseline)
- Nocturnal oxygenation and ventilation(Up to 6 months after starting NIV)
- Modified Borg dyspnea score (see description)(Up to 6 months after baseline)