Amyotrophic Lateral Sclerosis, Motor Neuron Disease
Conditions
Keywords
noninvasive ventilation
Brief summary
Amyotrophic Lateral Sclerosis (ALS), or Lou Gehrig's Disease, is a fatal disorder that causes progressive degeneration and weakening of the muscles of breathing, leading to breathing insufficiency and eventually breathing failure. This breathing insufficiency is commonly treated with a breathing assistance device, known as noninvasive positive pressure ventilation (NIPPV). While generally well tolerated and accepted, it is not clear whether or to what extent NIPPV in fact helps breathing function: some data suggest that NIPPV preserves breathing function over time, whereas other data suggest that it actually causes breathing function to decline more quickly. No studies have shown what the acute effect of NIPPV is on breathing muscle function in ALS patients. This study will test the hypothesis that the acute use of NIPPV, at pressure levels that are in common clinical use, will cause measurable changes in tests of breathing function, compared to baseline and to lower levels of NIPPV. We expect that the results of this study will help to clarify whether and to what extent NIPPV assists respiratory muscle function in patients with ALS.
Interventions
Each subject will undergo 2 hours of high-level noninvasive positive pressure ventilation, with an inspiratory positive airway pressure of 12 cm H2O and an expiratory positive airway pressure of 3 cm H2O.
Sponsors
Columbia University
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
SUPPORTIVE_CARE
Masking
SINGLE (Subject)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* clinical diagnosis of amyotrophic lateral sclerosis * clinical indication to start noninvasive ventilation (forced vital capacity \< 50% predicted or signs/symptoms of respiratory insufficiency) * age 18 to 80 years old
Exclusion criteria
* prior institution of NIPPV * inability to safely use NIPPV * indications for tracheostomy assisted ventilation because of inability to clear secretions from the airway * inability or unwillingness to perform pulmonary function testing * presence of advanced dementia.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Difference in Pulmonary Function and Respiratory Muscle Pressure Testing | 5 hours |
| Difference in Gas Exchange | 5 hours |
| Difference in Subjective Dyspnea Between Baseline and the Two Different Ventilator Modes | 5 hours |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| High Level and Low Level Ventilation All participants | 8 |
| Total | 8 |
Baseline characteristics
| Characteristic | High Level and Low Level Ventilation |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 1 Participants |
| Age, Categorical Between 18 and 65 years | 7 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 7 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Region of Enrollment United States | 8 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 8 |
| other Total, other adverse events | 0 / 8 | 0 / 8 |
| serious Total, serious adverse events | 0 / 8 | 0 / 8 |
Outcome results
Primary
Difference in Gas Exchange
Time frame: 5 hours
Population: Data were collected but not analyzed as specified in the title because the sample size does not generate enough statistical significance. PI no longer has raw data on file. All efforts have been made to retrieve the data and data could not be recovered.
Primary
Difference in Pulmonary Function and Respiratory Muscle Pressure Testing
Time frame: 5 hours
Population: Data were collected but not analyzed as specified in the title because the sample size does not generate enough statistical significance. PI no longer has raw data on file. All efforts have been made to retrieve the data and data could not be recovered.
Primary
Difference in Subjective Dyspnea Between Baseline and the Two Different Ventilator Modes
Time frame: 5 hours
Population: Data were collected but not analyzed as specified in the title because the sample size does not generate enough statistical significance. PI no longer has raw data on file. All efforts have been made to retrieve the data and data could not be recovered.