Amyotrophic Lateral Sclerosis
Conditions
Keywords
Airway Clearance Devices, High Frequency Chest Compression Devices, Cough Assist
Brief summary
The investigator is examining the use of one airway clearance medical device compared to the use of two airway clearance medical devices together in patients with amyotrophic lateral sclerosis (ALS). More specifically, the investigator wants to know how effective the use of either a mechanical High Frequency Chest Compression (HFCC) device is on its own or the use of both a mechanical High Frequency Chest Compression (HFCC) device and Cough Assist together to maintain a healthy airway and clear secretions. The first device is a passive form of mechanical High Frequency Chest Compression (HFCC), which was designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area. The second device, called a Cough Assist, aids patients to clear mucus and secretions that they would otherwise be unable to clear with coughing. This study will enroll up to 20 people in total at CSMC.
Detailed description
This 180 day (25.7 weeks) pilot study is designed to evaluate the effectiveness of airway clearance devices in adults with ALS. Subjects will be randomized in a 1:1 ratio to one of two treatment groups: treatment with a mechanical HCFF device alone or treatment with both a mechanical HFCC device and a cough assist device. This outpatient study includes a screening/baseline visit followed by a 180 day (25.7 weeks) treatment period with three scheduled clinic visits (day 30, day 90, day 180). Pulmonary assessments and ALS outcome measures will be collected at each visit in addition to quality of life assessments and device usage diaries.
Interventions
DEVICEHigh Frequency Chest Compression Device (HFCC)
A vest designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area.
DEVICECough Assist
A device that aids patients to clear mucus and secretions that they would otherwise be unable to clear with coughing.
Sponsors
Cedars-Sinai Medical Center
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Suspected, possible, probable, Probable (Lab-Supported), or Definite ALS according to El Escorial Criteria 2. Males and females age 18 and above 3. Novel to airway clearance device use 4. Forced vital capacity ≤ 75% of predicted
Exclusion criteria
1. Any contraindication for pulmonary ventilation scan including allergy to radioisotopes 2. Any contraindication for use of a pulmonary clearance device * Susceptibility to pneumothorax * Recent (within 30 days) barotrauma * Unstable head or neck injury * Active hemorrhage with hemodynamic instability
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects With Improved Respiratory Symptoms as Shown by Chest X-Ray Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 X-rays over 6 months. | A projection radiograph of the chest used to diagnose conditions affecting the chest. Chest X-Ray performed at each study visit. Investigator looked at clinical impression of Chest X-ray to be normal versus abnormal. If there was presence of atelectasis, this indicated a worsening of subject's respiratory symptoms. If subject's Chest X-Ray impression remained the same and continually normal, this indicated no change in respiratory symptoms. |
| Number of Subjects With Improved Respiratory Symptoms As Shown By Lung Ventilation Scan Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 lung ventilation scans over 6 months. | A nuclear scanning test commonly used to detect abnormalities in air flow. A radioactive tracer gas or mist is inhaled into the lungs. Pictures from the scan indicate areas of the lungs that are not receiving enough air or that retain too much air. Areas of the lung that retain too much air are brighter spots on the film and areas not receiving enough air are dark. Lung scan performed at each study visit. Investigator looked at clinical impression of lung scan to be normal versus abnormal. If there was indication of decreased ventilation, this indicated a worsening of subject's respiratory symptoms. If subject's lung scan impression remained the same and continually normal, this indicated no change in respiratory symptoms. |
| Number of Participants With Increased McGill Single Item Quality of Life Scale Question Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 questionnaires over 6 months. | Assess the improvement of/ rate of deterioration of the subject's quality of life from baseline to end of study on a rated scale of 1 to 10. |
| Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months. | Spirometry will be used to measure FVC. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled. |
| Number of Participants With Improved Maximal Inspiratory Pressure (MIP) Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months. | Spirometry will be used to measure MIP. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled. |
| Number of Participants With Improved Diffusion Capacity Between Baseline and End of Study | 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months. | Spirometry will be used to measure Diffusion Capacity. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled. |
Countries
United States
Participant flow
Recruitment details
Patients were recruited internally at Cedars-Sinai Medical Center ALS multidisciplinary clinic.
Pre-assignment details
5 participants met inclusion criteria and were enrolled into the study. One participant was randomized to the Treatment Group A HFCC Only arm while 4 participants were randomized to the Treatment Group B HFCC + Cough Assist Arm.
Participants by arm
| Arm | Count |
|---|---|
| Treatment Group A HFCC Only Subjects randomized to group A will be fitted for and issued a mechanical High Frequency Chest Compression Device (HFCC) device for home use. Subjects will be instructed to use the HFCC device 2-3 times per day for 15-30 minutes each. Subjects will be instructed to keep a device usage, concomitant medication, and adverse event diary.
High Frequency Chest Compression Device (HFCC): A vest designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area. | 1 |
| Treatment Group B HFCC + Cough Assist Subjects randomized to group B will be fitted for and issued a mechanical High Frequency Chest Compression Device (HFCC) device and will also be issued a cough assist device. Subjects will be instructed in the use of both devices. The HFCC device should be utilized 2-3 times per day for 15-30 minutes each followed by a session with the cough assist. Subjects will be instructed to keep a device usage, concomitant medication, and adverse event diary.
High Frequency Chest Compression Device (HFCC): A vest designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area.
Cough Assist: A device that aids patients to clear mucus and secretions that they would otherwise be unable to clear with coughing. | 4 |
| Total | 5 |
Baseline characteristics
| Characteristic | Treatment Group A HFCC Only | Treatment Group B HFCC + Cough Assist | Total |
|---|---|---|---|
| Age, Continuous | 78 years | 61.5 years | 64.8 years |
| Diffusion Capacity | 12.7 ml/mmHg/min | 18.9 ml/mmHg/min | 15.8 ml/mmHg/min |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 1 Participants | 3 Participants | 4 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Forced Expiratory Volume | 53 % Predicted | 67.5 % Predicted | 60.25 % Predicted |
| Forced Vital Capacity | 43 % Predicted | 64.25 % Predicted | 53.625 % Predicted |
| Height | 183 centimeters | 173.25 centimeters | 178.125 centimeters |
| Maximum Expiratory Pressure (MEP) | 27 cm/H20 | 60 cm/H20 | 43.5 cm/H20 |
| Maximum Inspiratory Pressure (MIP) | 59 cm/H20 | 72.25 cm/H20 | 65.625 cm/H20 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 1 Participants | 4 Participants | 5 Participants |
| Region of Enrollment United States | 1 participants | 4 participants | 5 participants |
| Sex: Female, Male Female | 0 Participants | 1 Participants | 1 Participants |
| Sex: Female, Male Male | 1 Participants | 3 Participants | 4 Participants |
| Weight | 87 kilograms | 81.15 kilograms | 84.075 kilograms |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 1 | 0 / 4 |
| other Total, other adverse events | 0 / 1 | 0 / 4 |
| serious Total, serious adverse events | 1 / 1 | 0 / 4 |
Outcome results
Primary
Number of Participants With Improved Diffusion Capacity Between Baseline and End of Study
Spirometry will be used to measure Diffusion Capacity. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months.
Population: Treatment Group A subject 003 did not complete study. Treatment Group B subjects 001 and 005 were only subjects to complete study and there was a worsening of diffusion capacity values for subject 005 and an improvement of subject 005 from 23.1 ml/mmHg/min to 28.7 ml/mmHg/min Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Participants With Improved Diffusion Capacity Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Participants With Improved Diffusion Capacity Between Baseline and End of Study | 1 Participants |
Primary
Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study
Spirometry will be used to measure FVC. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months.
Population: Treatment Group A subject 003 did not complete study. Treatment Group B subjects 001 and 005 were only subjects to complete study and there was a worsening of FVC values for both subjects.~Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study | 0 Participants |
Primary
Number of Participants With Improved Maximal Inspiratory Pressure (MIP) Between Baseline and End of Study
Spirometry will be used to measure MIP. It is the most common of the pulmonary function tests and measures lung function, specifically the measurement of the amount (volume) and/or speed (flow) of air that can be inhaled and exhaled.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 testing sessions over 6 months.
Population: Treatment Group A subject 003 did not complete study. Treatment Group B subjects 001 and 005 were only subjects to complete study and there was a worsening of MIP values for both subjects.~Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Participants With Improved Maximal Inspiratory Pressure (MIP) Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Participants With Improved Maximal Inspiratory Pressure (MIP) Between Baseline and End of Study | 0 Participants |
Primary
Number of Participants With Increased McGill Single Item Quality of Life Scale Question Between Baseline and End of Study
Assess the improvement of/ rate of deterioration of the subject's quality of life from baseline to end of study on a rated scale of 1 to 10.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 questionnaires over 6 months.
Population: Treatment Group A subject 003 did not complete study. Treatment Group B subjects 001 and 005 were only subjects to complete study and reported upward trend of McGill Quality of Life Scale.~Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Participants With Increased McGill Single Item Quality of Life Scale Question Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Participants With Increased McGill Single Item Quality of Life Scale Question Between Baseline and End of Study | 2 Participants |
Primary
Number of Subjects With Improved Respiratory Symptoms as Shown by Chest X-Ray Between Baseline and End of Study
A projection radiograph of the chest used to diagnose conditions affecting the chest. Chest X-Ray performed at each study visit. Investigator looked at clinical impression of Chest X-ray to be normal versus abnormal. If there was presence of atelectasis, this indicated a worsening of subject's respiratory symptoms. If subject's Chest X-Ray impression remained the same and continually normal, this indicated no change in respiratory symptoms.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 X-rays over 6 months.
Population: Chest X-ray was not performed for Subject 003 of treatment group A at any visit and therefore was not analyzed.~Chest X-ray was performed for Subjects 001, 002, 004, and 005. There was not found to be any improvement of respiratory symptoms across all participants.~Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Subjects With Improved Respiratory Symptoms as Shown by Chest X-Ray Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Subjects With Improved Respiratory Symptoms as Shown by Chest X-Ray Between Baseline and End of Study | 0 Participants |
Primary
Number of Subjects With Improved Respiratory Symptoms As Shown By Lung Ventilation Scan Between Baseline and End of Study
A nuclear scanning test commonly used to detect abnormalities in air flow. A radioactive tracer gas or mist is inhaled into the lungs. Pictures from the scan indicate areas of the lungs that are not receiving enough air or that retain too much air. Areas of the lung that retain too much air are brighter spots on the film and areas not receiving enough air are dark. Lung scan performed at each study visit. Investigator looked at clinical impression of lung scan to be normal versus abnormal. If there was indication of decreased ventilation, this indicated a worsening of subject's respiratory symptoms. If subject's lung scan impression remained the same and continually normal, this indicated no change in respiratory symptoms.
Time frame: 1 time at screening/baseline and then at the month 1, month 3, and month 6 study visit for a total of 4 lung ventilation scans over 6 months.
Population: Lung scan was not performed for Subject 003 of treatment group A at any visit and therefore was not analyzed.~Lung scan was performed for Subjects 001, 002, 004, and 005. There was not found to be any improvement of respiratory symptoms across all participants.~Not enough data to draw statistical inferences between groups.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Treatment Group A | Number of Subjects With Improved Respiratory Symptoms As Shown By Lung Ventilation Scan Between Baseline and End of Study | 0 Participants |
| Treatment Group B- HFCC and Cough Assist | Number of Subjects With Improved Respiratory Symptoms As Shown By Lung Ventilation Scan Between Baseline and End of Study | 0 Participants |