Amyotrophic Lateral Sclerosis, Respiratory Muscle Weakness
Conditions
Keywords
Amyotrophic Lateral Sclerosis, Airway Clearance, Mechanical Insufflation Exsufflation, High Frequency Chest Wall Compression Oscillation
Brief summary
Patients will receive one of three respiratory therapy interventions for airway clearance assistance: 1) High frequency chest wall oscillation (HFCWO) and mechanical insufflation/exsufflation (MIE), 2) HFCWO or 3) MIE. The study period will be six months and include three clinic visits, baseline and follow-up visits at 3 and 6 months, and 6 monthly home visits by the respiratory therapist.
Detailed description
The proposed project includes both the HFCWO and MIE and will capitalize on the specific goals of each therapy and address the problem as a whole rather than piece-meal. The HFCWO aims to mobilize the secretions to the pharynx to allow the patient to expel the secretions. However, many patients diagnosed with ALS are unable to expel the secretions due to atrophied expiratory muscles. The HFCWO device uses a small air compressor with a vest that wraps around the chest to induce airflows to pull secretions from the walls of the airways, thin the secretions and move them up the airways towards the larger airways and pharynx. The MIE, a noninvasive therapy, removes secretions in patients who have an ineffective cough because the peak cough flows are less than 270 L/min. This device applies a positive pressure to the airway and rapidly switches to a negative pressure applied to the airway. The rapid switch between the two types of pressure simulates a natural cough, thus assisting with expulsion of the secretions. Logically, the two mechanisms of action of these devices should work synergistically to produce effective airway clearance to keep the lungs clear, healthy and reduce the risk of infections from stagnant secretions. Respiratory infections are especially serious for patients with ALS because the patient is not able to recuperate from infections as quickly as a person without ALS. The objective is to determine if changing the use of existing respiratory therapy devices can improve the physical and psychosocial health and quality of life for patients diagnosed with ALS and caregivers.
Interventions
The HFCWO aims to mobilize the secretions to the pharynx to allow the patient to expel the secretions. However, many ALS patients are unable to expel their secretions due to atrophied expiratory muscles. The HFCWO device uses a small air compressor with a vest that wraps around the chest to induce airflows that pull secretions from the walls of the airways, thin the secretions and move them up the airways towards the larger airways and pharynx.
A noninvasive therapy, removes secretions in patients who have an ineffective cough because their peak cough flows are less than 270 L/min. This device applies a positive pressure to the airway and rapidly switches to a negative pressure applied to the airway. The rapid switch between the two types of pressure simulates a natural cough, thus assisting with expulsion of the secretions.
Sponsors
ALS Association
The University of Texas Health Science Center at San Antonio
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Subject)
Masking description
Participants will be blinded to the intervention
Intervention model description
A single-blind parallel group design in which 60 patients with probable or definite ALS will be randomized equally into three groups (20 per group) over a period of 12 months
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Age 21 and above, 2. Probable or definite ALS diagnosis, 3. Peak Cough Flow of \<160L/min or complain of issues clearing airway secretions 4. Caregiver must also consent to participate
Exclusion criteria
1. Current use of HFCWO or MIE 2. Tracheostomy 3. Congestive heart failure 4. All contraindications for the HFCWO 5. Head and/or neck injury that has not been stabilized; 6. Active hemorrhage with hemodynamic instability; 7. Uncontrolled hypertension; 8. Active or recent gross hemoptysis; and 9. All contraindications for the MIE 10. History of bullous emphysema; 11. Known susceptibility to pneumothorax; 12. Pneumomediastinum; and 13. Recent barotrauma 14. Frontal Temporal Dementia (FTD) - suspected or diagnosed . FTD is a form of dementia found in about 20% of Familial ALS. This dementia interferes with the ability to follow commands and follow through with therapies due to forgetfulness. 15. Patients not able to provide informed consent.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Respiratory Complications Severity | Baseline to 6 months | A 9-item instrument will be used to record change from baseline to 6 months in respiratory complications. These are scored on a scale from best (no complications) to worst (death due to respiratory complications) throughout the study period. Scores range from 0-9 with a score of a 9 indicating no complications. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Patient Global Impression of Change (PGIC) | Baseline to 6 months | This outcome is assessed and recorded as a number from 1-5 with 1 indicating the worst outcome (markedly worse) and 5 indicating best outcome (markedly better) since starting the study. |
Countries
United States
Participant flow
Recruitment details
Patients and their caregivers (PAC) were randomized into one of three groups. The PAC completed perception of problem and quality of life surveys.
Participants by arm
| Arm | Count |
|---|---|
| High Frequency Chest Wall Oscillation High Frequency Chest Wall Oscillation: The HFCWO aims to mobilize the secretions to the pharynx to allow the patient to expel the secretions. However, many ALS patients are unable to expel their secretions due to atrophied expiratory muscles. The HFCWO device uses a small air compressor with a vest that wraps around the chest to induce airflows that pull secretions from the walls of the airways, thin the secretions and move them up the airways towards the larger airways and pharynx. | 7 |
| High Frequency Chest Wall Oscillation and Mechanical Insufflation/ Exsufflation High Frequency Chest Wall Oscillation: The HFCWO aims to mobilize the secretions to the pharynx to allow the patient to expel the secretions. However, many ALS patients are unable to expel their secretions due to atrophied expiratory muscles. The HFCWO device uses a small air compressor with a vest that wraps around the chest to induce airflows that pull secretions from the walls of the airways, thin the secretions and move them up the airways towards the larger airways and pharynx.
Mechanical insufflation/exsufflation: A noninvasive therapy, removes secretions in patients who have an ineffective cough because their peak cough flows are less than 270 L/min. This device applies a positive pressure to the airway and rapidly switches to a negative pressure applied to the airway. The rapid switch between the two types of pressure simulates a natural cough, thus assisting with expulsion of the secretions. | 11 |
| Mechanical Insufflation/ Exsufflation Mechanical insufflation/exsufflation: A noninvasive therapy, removes secretions in patients who have an ineffective cough because their peak cough flows are less than 270 L/min. This device applies a positive pressure to the airway and rapidly switches to a negative pressure applied to the airway. The rapid switch between the two types of pressure simulates a natural cough, thus assisting with expulsion of the secretions. | 10 |
| Total | 28 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Death | 1 | 1 | 1 |
| Overall Study | Lost to Follow-up | 3 | 7 | 5 |
Baseline characteristics
| Characteristic | High Frequency Chest Wall Oscillation | High Frequency Chest Wall Oscillation and Mechanical Insufflation/ Exsufflation | Mechanical Insufflation/ Exsufflation | Total |
|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 3 Participants | 4 Participants | 7 Participants | 14 Participants |
| Age, Categorical Between 18 and 65 years | 4 Participants | 7 Participants | 3 Participants | 14 Participants |
| Race/Ethnicity, Customized Black | 0 Participants | 2 Participants | 1 Participants | 3 Participants |
| Race/Ethnicity, Customized Hispanic-Non White | 2 Participants | 3 Participants | 2 Participants | 7 Participants |
| Race/Ethnicity, Customized White | 5 Participants | 6 Participants | 7 Participants | 18 Participants |
| Sex: Female, Male Female | 4 Participants | 4 Participants | 5 Participants | 13 Participants |
| Sex: Female, Male Male | 3 Participants | 7 Participants | 5 Participants | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 1 / 7 | 1 / 10 | 1 / 11 |
| other Total, other adverse events | 0 / 7 | 0 / 10 | 0 / 11 |
| serious Total, serious adverse events | 0 / 7 | 0 / 10 | 0 / 11 |
Outcome results
Primary
Respiratory Complications Severity
A 9-item instrument will be used to record change from baseline to 6 months in respiratory complications. These are scored on a scale from best (no complications) to worst (death due to respiratory complications) throughout the study period. Scores range from 0-9 with a score of a 9 indicating no complications.
Time frame: Baseline to 6 months
Population: Half (50%) of patients were randomized to the group with two devices, while 30% to only the HFCWO device and 20% to only the Mechanical Insufflation/exsufflation device.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| High Frequency Chest Wall Oscillation | Respiratory Complications Severity | 8.33 score on a scale | Standard Deviation 1.15 |
| Mechanical Insufflation/ Exsufflation | Respiratory Complications Severity | 9.0 score on a scale | Standard Deviation 0 |
| High Frequency Chest Wall Oscillation and Mechanical Insufflation/ Exsufflation | Respiratory Complications Severity | 7.8 score on a scale | Standard Deviation 1.64 |
Secondary
Patient Global Impression of Change (PGIC)
This outcome is assessed and recorded as a number from 1-5 with 1 indicating the worst outcome (markedly worse) and 5 indicating best outcome (markedly better) since starting the study.
Time frame: Baseline to 6 months
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| High Frequency Chest Wall Oscillation | Patient Global Impression of Change (PGIC) | 3.33 score on a scale | Standard Deviation 0.58 |
| Mechanical Insufflation/ Exsufflation | Patient Global Impression of Change (PGIC) | 3.00 score on a scale | Standard Deviation 0 |
| High Frequency Chest Wall Oscillation and Mechanical Insufflation/ Exsufflation | Patient Global Impression of Change (PGIC) | 4.40 score on a scale | Standard Deviation 0.96 |