Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
Amyotrophic Lateral Sclerosis, Noninvasive Ventilation, Respiratory Dysfunction, Positive Pressure Ventilation
Brief summary
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.
Interventions
OTHERSleep study-guided adjustment of NIV
Sleep studies will be performed at baseline, within 2 weeks to initially titrate NIV, and at 1, 3 and 6 months to assess NIV performance and adjust it as necessary based on oxygenation and ventilation parameters.
OTHERStandard initiation of NIV
NIV will be initiated and managed as per current standard of practice. Sleep studies will be performed at baseline, 2 weeks, 1, 3 and 6 months to gather data but will not influence NIV management. NIV will be adjusted by a respiratory therapist or the subject's primary physician per waking symptoms.
Sponsors
ALS Association
Columbia University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* Diagnosis of probable or definite Amyotrophic Lateral Sclerosis (ALS) per El Escorial criteria * Between ages of 18 and 80 yrs old
Exclusion criteria
* 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline in spirometric, respiratory muscle strength, and gas exchange measures | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Whether sleep study-titrated NIV is associated with trends to improved survival, compared with standardly prescribed (non-titrated) NIV |
| Nocturnal oxygenation and ventilation | Up to 6 months after starting NIV | Nadir oxygen saturation, number of oxygen desaturations of 3%/hr (ODI3), % time of sleep spent with oxygen saturation \<90%, apnea-hypopnea index, asynchrony index |
| Modified Borg dyspnea score (see description) | Up to 6 months after baseline | The Modified Borg Dyspnea Scale is a 10-point Likert scale asking subjects to rate perceived shortness of breath, ranging from 0 (no breathlessness at all) to 10 (maximal breathlessness) |
Countries
United States
Outcome results
None listed