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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment group A | Active Comparator | 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. |
|
| Treatment group B- HFCC and Cough Assist | Active Comparator | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| High Frequency Chest Compression Device (HFCC) | Device | A vest designed to help clear the airway of mucus and other secretions through mechanical knocking of the chest area. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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. |
| 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. | 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. |
| 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. | 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. |
| Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study |
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Inclusion Criteria:
Exclusion Criteria:
Any contraindication for pulmonary ventilation scan including allergy to radioisotopes
Any contraindication for use of a pulmonary clearance device
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| Name | Affiliation | Role |
|---|---|---|
| Ashraf Elsayegh, MD, FCCP | Cedars-Sinai Medical Center | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | United States |
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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.
Patients were recruited internally at Cedars-Sinai Medical Center ALS multidisciplinary clinic.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. |
| FG001 | Treatment Group B- HFCC and 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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Subjects randomized to group A will be issued a mechanical High Frequency Chest Compression Device (HFCC) device for home use. Subjects randomized to group B will be issued a mechanical High Frequency Chest Compression Device (HFCC) device and will also be issued a cough assist device. Please see Baseline Characteristics for more information.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
25.7 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Death due to respiratory insufficiency | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment | Patient with ALS died due to respiratory insufficiency during study |
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Data too limited to address the aims and perform statistical analyses between the groups. 5 subjects were enrolled, only two completed the study, and the one that was randomized for HFCC only did not have any follow up visit.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Ashraf Elsayegh | Cedars Sinai Medical Center - 8700 | 3105560335 | ashraf.elsayegh@cshs.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Apr 18, 2016 | Jul 14, 2020 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Cough Assist | Device | A device that aids patients to clear mucus and secretions that they would otherwise be unable to clear with coughing. |
|
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. |
| 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. |
| 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. | 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. |
| 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. | 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. |
| BG001 | 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. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Height | Mean | Full Range | centimeters |
|
| Weight | Mean | Full Range | kilograms |
|
| Forced Vital Capacity | Mean | Full Range | % Predicted |
|
| Forced Expiratory Volume | Mean | Full Range | % Predicted |
|
| Maximum Inspiratory Pressure (MIP) | Mean | Full Range | cm/H20 |
|
| Maximum Expiratory Pressure (MEP) | Mean | Full Range | cm/H20 |
|
| Diffusion Capacity | Mean | Full Range | ml/mmHg/min |
|
| OG000 |
| Treatment Group A |
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. |
| OG001 | Treatment Group B- HFCC and 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. |
|
|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
|
|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
|
|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
|
|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
|
|
| 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. | 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. | Posted | Count of Participants | Participants | 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. |
|
|
|
| 1 |
| 1 |
| 1 |
| 1 |
| 0 |
| 1 |
| EG001 | Treatment Group B- HFCC and 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. | 0 | 4 | 0 | 4 | 0 | 4 |
|
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| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |