Trial Outcomes & Findings for The Use of Airway Clearance Devices in ALS (NCT NCT02682030)

NCT ID: NCT02682030

Last Updated: 2020-08-25

Results Overview

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.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

5 participants

Primary outcome timeframe

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.

Results posted on

2020-08-25

Participant Flow

Patients were recruited internally at Cedars-Sinai Medical Center ALS multidisciplinary clinic.

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.

Participant milestones

Participant milestones
Measure
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.
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.
Overall Study
STARTED
1
4
Overall Study
COMPLETED
0
2
Overall Study
NOT COMPLETED
1
2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

The Use of Airway Clearance Devices in ALS

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Treatment Group A HFCC Only
n=1 Participants
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.
Treatment Group B HFCC + Cough Assist
n=4 Participants
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.
Total
n=5 Participants
Total of all reporting groups
Age, Continuous
78 years
n=5 Participants
61.5 years
n=7 Participants
64.8 years
n=5 Participants
Sex: Female, Male
Female
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Sex: Female, Male
Male
1 Participants
n=5 Participants
3 Participants
n=7 Participants
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
1 Participants
n=5 Participants
3 Participants
n=7 Participants
4 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
White
1 Participants
n=5 Participants
4 Participants
n=7 Participants
5 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
United States
1 participants
n=5 Participants
4 participants
n=7 Participants
5 participants
n=5 Participants
Height
183 centimeters
n=5 Participants
173.25 centimeters
n=7 Participants
178.125 centimeters
n=5 Participants
Weight
87 kilograms
n=5 Participants
81.15 kilograms
n=7 Participants
84.075 kilograms
n=5 Participants
Forced Vital Capacity
43 % Predicted
n=5 Participants
64.25 % Predicted
n=7 Participants
53.625 % Predicted
n=5 Participants
Forced Expiratory Volume
53 % Predicted
n=5 Participants
67.5 % Predicted
n=7 Participants
60.25 % Predicted
n=5 Participants
Maximum Inspiratory Pressure (MIP)
59 cm/H20
n=5 Participants
72.25 cm/H20
n=7 Participants
65.625 cm/H20
n=5 Participants
Maximum Expiratory Pressure (MEP)
27 cm/H20
n=5 Participants
60 cm/H20
n=7 Participants
43.5 cm/H20
n=5 Participants
Diffusion Capacity
12.7 ml/mmHg/min
n=5 Participants
18.9 ml/mmHg/min
n=7 Participants
15.8 ml/mmHg/min
n=5 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=4 Participants
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.
Number of Subjects With Improved Respiratory Symptoms as Shown by Chest X-Ray Between Baseline and End of Study
0 Participants
0 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=4 Participants
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.
Number of Subjects With Improved Respiratory Symptoms As Shown By Lung Ventilation Scan Between Baseline and End of Study
0 Participants
0 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=2 Participants
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.
Number of Participants With Increased McGill Single Item Quality of Life Scale Question Between Baseline and End of Study
0 Participants
2 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=2 Participants
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.
Number of Participants With Improved Forced Vital Capacity (FVC) Between Baseline and End of Study
0 Participants
0 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=2 Participants
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.
Number of Participants With Improved Maximal Inspiratory Pressure (MIP) Between Baseline and End of Study
0 Participants
0 Participants

PRIMARY outcome

Timeframe: 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.

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.

Outcome measures

Outcome measures
Measure
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.
Treatment Group B- HFCC and Cough Assist
n=2 Participants
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.
Number of Participants With Improved Diffusion Capacity Between Baseline and End of Study
0 Participants
1 Participants

Adverse Events

Treatment Group A - HFCC Only

Serious events: 1 serious events
Other events: 0 other events
Deaths: 1 deaths

Treatment Group B- HFCC and Cough Assist

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Treatment Group A - HFCC Only
n=1 participants at risk
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.
Treatment Group B- HFCC and Cough Assist
n=4 participants at risk
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.
Respiratory, thoracic and mediastinal disorders
Death due to respiratory insufficiency
100.0%
1/1 • Number of events 1 • 25.7 weeks
0.00%
0/4 • 25.7 weeks

Other adverse events

Adverse event data not reported

Additional Information

Dr. Ashraf Elsayegh

Cedars Sinai Medical Center - 8700

Phone: 3105560335

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place