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The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period.
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period. The study did not include a control group. This pilot study was designed to provide initial information that could inform decisions for future larger-scale studies.
Ten (10) patients total were enrolled from CF and NMD clinics. Eligible subjects were adult patients who were able to perform MN4000 therapy using a mouthpiece and who met all inclusion and none of the exclusion criteria.
All patients received therapy with the MN4000 following the labeled instructions for the device.
The MN4000 is an airway clearance and lung expansion therapy device that has been cleared to market by the FDA as The MetaNeb® System for Homecare environment, for clearance of pulmonary secretions and for treatment or prevention of pulmonary atelectasis. It is a Class II device, cleared to market on March 17, 2016 under premarket notification 510(k) K151689 as The MetaNeb® 4 System with application for homecare environment. It is commercially marketed as the MN4000. The device consists of a pneumatic compressor and an air pulse generator that delivers CHFO and CPEP to;
This "triple" mode device can provide aerosol therapy while alternating between CPEP for lung expansion and CHFO for airway clearance. Supplemental oxygen therapy may also be delivered when used with compressed oxygen.
The MN4000 has three therapy modes:
After assessing baseline status, therapy with the MN4000 was introduced and incorporated into the daily home respiratory care treatment regimen for all patients.
Other airway clearance and/or lung expansion therapies were not to be performed during the three-month study period. The treatment regimen for other respiratory care modalities (e.g. aerosolized medications) was that which was prescribed by the patient's health care team in the routine standard care of each patient.
During the three-month follow-up period, adherence to the daily prescribed therapy regimen was assessed. Subjects/caregivers were asked to provide adherence information for each day during the 90-day study period.
Documentation of efficacy and safety Variables was completed by study staff at the time of occurrence, from review of the patient's medical records and from scores and rankings for questionnaires.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Home based airway clearance with Metaneb | Experimental | Patients with CF and MND who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. The MN4000 is an airway clearance and lung expansion therapy device that has been cleared to market by the FDA as The MetaNeb® System for Homecare environment, for clearance of pulmonary secretions and for treatment or prevention of pulmonary atelectasis. It is a Class II device, cleared to market on March 17, 2016 under premarket notification 510(k) K151689 as The MetaNeb® 4 System with application for homecare environment. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MN 4000 | Device | Patients who required regular home airway clearance therapy were enrolled in the study and were prescribed therapy with the MN4000. Adherence to the prescribed therapy regimen and patient/caregiver satisfaction with the therapy was assessed. Pulmonary function, was assessed for each subject at baseline, after 1 month and after 3 months of home therapy. Results from the therapy period was compared to the baseline period, during which the subject received their regular airway clearance regimen. Airway Clearance Satisfaction surveys were conducted at baseline, after 1 month, and after 3 months of therapy. Results from the MN4000 therapy period were compared to the baseline period, during which the subject received their regular regimen |
| Measure | Description | Time Frame |
|---|---|---|
| Patient / Caregiver Satisfaction Scores | The primary efficacy variable was patient and/or caregiver satisfaction with therapy, as evaluated using a Therapy Use Rating Questionnaire. The questionnaire was used to assess change in satisfaction or effectiveness on a variety of parameters. The satisfaction score was completed by the patient, if possible. Some of the patients in this study had Motor Neuron Disease. This disease can compromise the ability of the patient to interact and to respond to questions. In situations where patients could not respond, caregivers were asked to respond on their behalf. | 90 days |
| Patient / Caregiver Satisfaction Scores | The Therapy Use Rating Questionnaire is an assessment of patient/caregiver satisfaction with the therapy and their subjective assessment of the benefit of the therapy. The questionnaire was used to assess change in satisfaction or effectiveness on a variety of parameters. The satisfaction score was completed by the patient, if possible. Some of the patients in this study had Motor Neuron Disease This disease can compromise the ability of the patient to interact and to respond to questions. In situations where patients could not respond, caregivers were asked to respond on their behalf. | 90 days |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Percentage Adherence to Prescribed Treatment Regimen | Adherence to the treatment regimen was calculated for each patient (number of minutes of therapy performed / number of minutes of therapy prescribed). The adherence percentage calculated for each study subject was then used to calculate the of Mean adherence to prescribed treatment regimen for the group or groups. | 90 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Lisa F Wolfe, MD | Northwestern University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Northwestern | Chicago | Illinois | 60208 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Home Based Airway Clearance With Metaneb | Patients with CF and MND who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Home Based Airway Clearance With Metaneb in Patients With Cystic Fibrosis. | Patients with CF who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. |
| BG001 | Home Based Airway Clearance With Metaneb in Patients With Motor Neuron Disease. |
| 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 | Patient / Caregiver Satisfaction Scores | The primary efficacy variable was patient and/or caregiver satisfaction with therapy, as evaluated using a Therapy Use Rating Questionnaire. The questionnaire was used to assess change in satisfaction or effectiveness on a variety of parameters. The satisfaction score was completed by the patient, if possible. Some of the patients in this study had Motor Neuron Disease. This disease can compromise the ability of the patient to interact and to respond to questions. In situations where patients could not respond, caregivers were asked to respond on their behalf. | Posted | Count of Participants | Participants | 90 days |
|
90 days
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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 | Patients With CF Treated With Home Based Airway Clearance With Metaneb | Patients with CF who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Baxter Clinical Trials Disclosure Call Center | Baxter Healthcare | (224) 948-7359 | Global_CORP_ClinicalTrialsDisclosure@baxter.com |
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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 | Dec 5, 2016 | Mar 1, 2024 | Prot_SAP_001.pdf |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D016472 | Motor Neuron Disease |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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|
| ALS-Functional Rating Scale (ALS-FRS) | MND patients only. The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) is an instrument for evaluating the functional status of patients with Amyotrophic Lateral Sclerosis. It can be used to monitor functional change in a patient over time. It contains 10 distinct measures:
These 10 parameters are scored on a scale of 0-4, based on the patients' ability to perform tasks. The minimum score is 0 and maximum 40.The higher the score the more function is retained. | 90 days |
| Cystic Fibrosis Questionnaire - Revised (CFQ-R) | CF patients only. The Cystic Fibrosis Questionnaire (CFQ) is a disease-specific instrument that measures health-related quality of life (HRQoL) for adolescents and adults with cystic fibrosis (CF) > or = 14 years, consisting of 44 items on 12 generic and disease-specific scales. It offers 5 distinct 4-point Likert scales (e.g., always/often/ sometime/never). Scores for each HRQoL domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health. | 90 days |
| Number of Participants With Exacerbation of Pulmonary Disease | Hospitalization and/or antibiotics for respiratory infection or complication. Exacerbations of pulmonary disease were defined as respiratory infections that resulted in the requirement for hospitalization and/or antibiotics to treat the respiratory infection or complication. Hospitalizations that are part of routine care (e.g. hospital admissions for annual "tune-up") or antibiotics that are part of the regular treatment regimen were not documented as exacerbations. Occurrence of exacerbation related hospitalization OR the necessity of a prescription for antibiotics qualify as worsening disease. | 90 days |
| FEV1 (Actual) | FEV1 is the actual volume measured in liters. Forced expiratory volume in one second (FEV1): The forced expiratory volume-one second is the total volume of air a patient exhales in the first second during maximal effort. Normal range is >80%. The patient takes a full breath in and exhale as hard and fast as they can. FEV1 refers to the volume of air that an individual can exhale during a forced breath in the first 1 second. Decreasing percentage is associated with worsening / severe disease | 90 days |
| FEV1 (% Predicted) | FEV1 Percent Predicted (FEV1 % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." FEV1: The forced expiratory volume-one second is the total volume of air a patient exhales in the first second during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease | 90 days |
| FVC (Actual) | FVC is the actual volume measured in liters. The functional vital capacity (FVC) is the total volume of air a patient exhales for the total duration of the test during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease. FVC was only done in CF subjects because performance of the test requires intact respiratory muscle function. CF patients have intact respiratory muscle function and can therefore perform this maneuver. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | 90 days |
| FVC (% Predicted) | FVC Percent Predicted (FVC % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." The functional vital capacity is the total volume of air a patient exhales for the total duration of the test during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease. FVC was only done in CF subjects because performance of the test requires intact respiratory muscle function. CF patients have intact respiratory muscle function and can therefore perform this maneuver. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | 90 days |
| FEV1/FVC Ratio | The proportion of the FVC expired in one second Results are given in both actual values (litres, litres per second) and %predicted-the test result as a percent of the "predicted values" for the patients of similar characteristics. Results over 80% are considered normal. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | 90 days |
| SVC (Actual) | Slow Vital Capacity (SVC) is the actual volume measured in liters. Slow Vital Capacity displays the volume of gas measured on a complete expiration after a maximal inspiration without forced or rapid effort. Useful measurement when FVC is reduced, and airway obstruction is present. Educed SVC is associated with worsening respiratory disease. Slow Vital Capacity provides the same measure as FVC - i.e. "Vital Capacity." It does not, however, require a "Forced expiratory maneuver (it requires a slow inspiration and slow expiration) and therefore is easier to achieve a valid result in patients with Motor Neuron Disease who suffer from progressive weakness of all muscles including the respiratory muscles. | 90 days |
| SVC (% Predicted) | SVC Percent Predicted (SVC % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." Slow Vital Capacity displays the volume of gas measured on a complete expiration after a maximal inspiration without forced or rapid effort. Useful measurement when FVC is reduced and airway obstruction is present. Educed SVC is associated with worsening respiratory disease. Slow Vital Capacity provides the same measure as FVC - i.e. "Vital Capacity." It does not, however, require a "Forced expiratory maneuver (it requires a slow inspiration and slow expiration) and therefore is easier to achieve a valid result in patients with Motor Neuron Disease who suffer from progressive weakness of all muscles including the respiratory muscles. | 90 days |
| SpO2 | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and Peak Cough Flow) are commonly followed in the clinical care of patients with MND. SpO2 stands for peripheral capillary oxygen saturation, an estimate of the amount of oxygen in the blood. Low SpO2 is associated with worsening respiratory function. | 90 days |
| Maximal Inspiratory Pressure (MIP) | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and Peak Cough Flow) are commonly followed in the clinical care of patients with MND. Maximal inspiratory pressure (MIP) is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. Reduction in MIP is asociated with worsening neuromuscular status and poor ability to cough. | 90 days |
| PCF | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and PCF) are commonly followed in the clinical care of patients with MND. The Peak Cough Flow (PCF) is the maximum air flow generated during a cough. Decreasing air flow generated is associated with weakening neuromuscular status and associated poor cough impulse | 90 days |
Patients with MND who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. |
| 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 |
|
| OG001 | Home Based Airway Clearance With Metaneb in Patients With Motor Neuron Disease. | Patients with MND who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. |
|
|
| Primary | Patient / Caregiver Satisfaction Scores | The Therapy Use Rating Questionnaire is an assessment of patient/caregiver satisfaction with the therapy and their subjective assessment of the benefit of the therapy. The questionnaire was used to assess change in satisfaction or effectiveness on a variety of parameters. The satisfaction score was completed by the patient, if possible. Some of the patients in this study had Motor Neuron Disease This disease can compromise the ability of the patient to interact and to respond to questions. In situations where patients could not respond, caregivers were asked to respond on their behalf. | Posted | Count of Participants | Participants | 90 days |
|
|
|
| Secondary | Mean Percentage Adherence to Prescribed Treatment Regimen | Adherence to the treatment regimen was calculated for each patient (number of minutes of therapy performed / number of minutes of therapy prescribed). The adherence percentage calculated for each study subject was then used to calculate the of Mean adherence to prescribed treatment regimen for the group or groups. | CF and MND population | Posted | Mean | Full Range | Percentage of adherence to therapy | 90 days |
|
|
|
| Secondary | ALS-Functional Rating Scale (ALS-FRS) | MND patients only. The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) is an instrument for evaluating the functional status of patients with Amyotrophic Lateral Sclerosis. It can be used to monitor functional change in a patient over time. It contains 10 distinct measures:
These 10 parameters are scored on a scale of 0-4, based on the patients' ability to perform tasks. The minimum score is 0 and maximum 40.The higher the score the more function is retained. | MND subpopulation | Posted | Mean | Standard Deviation | score on a scale | 90 days |
|
|
|
| Secondary | Cystic Fibrosis Questionnaire - Revised (CFQ-R) | CF patients only. The Cystic Fibrosis Questionnaire (CFQ) is a disease-specific instrument that measures health-related quality of life (HRQoL) for adolescents and adults with cystic fibrosis (CF) > or = 14 years, consisting of 44 items on 12 generic and disease-specific scales. It offers 5 distinct 4-point Likert scales (e.g., always/often/ sometime/never). Scores for each HRQoL domain; after recoding, each item is summed to generate a domain score and standardized. Scores range from 0 to 100, with higher scores indicating better health. | Cystic Fibrosis Questionnaire - Revised (CFQ-R) administered on the CF patient subpopulation | Posted | Mean | Standard Deviation | Score on a scale | 90 days |
|
|
|
| Secondary | Number of Participants With Exacerbation of Pulmonary Disease | Hospitalization and/or antibiotics for respiratory infection or complication. Exacerbations of pulmonary disease were defined as respiratory infections that resulted in the requirement for hospitalization and/or antibiotics to treat the respiratory infection or complication. Hospitalizations that are part of routine care (e.g. hospital admissions for annual "tune-up") or antibiotics that are part of the regular treatment regimen were not documented as exacerbations. Occurrence of exacerbation related hospitalization OR the necessity of a prescription for antibiotics qualify as worsening disease. | Posted | Count of Participants | Participants | 90 days |
|
|
|
| Secondary | FEV1 (Actual) | FEV1 is the actual volume measured in liters. Forced expiratory volume in one second (FEV1): The forced expiratory volume-one second is the total volume of air a patient exhales in the first second during maximal effort. Normal range is >80%. The patient takes a full breath in and exhale as hard and fast as they can. FEV1 refers to the volume of air that an individual can exhale during a forced breath in the first 1 second. Decreasing percentage is associated with worsening / severe disease | Posted | Mean | Standard Deviation | Liters | 90 days |
|
|
|
| Secondary | FEV1 (% Predicted) | FEV1 Percent Predicted (FEV1 % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." FEV1: The forced expiratory volume-one second is the total volume of air a patient exhales in the first second during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease | Posted | Mean | Standard Deviation | percentage of predicted normal | 90 days |
|
|
|
| Secondary | FVC (Actual) | FVC is the actual volume measured in liters. The functional vital capacity (FVC) is the total volume of air a patient exhales for the total duration of the test during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease. FVC was only done in CF subjects because performance of the test requires intact respiratory muscle function. CF patients have intact respiratory muscle function and can therefore perform this maneuver. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from MND patients for this outcome measure. | Posted | Mean | Standard Deviation | Liters | 90 days |
|
|
|
| Secondary | FVC (% Predicted) | FVC Percent Predicted (FVC % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." The functional vital capacity is the total volume of air a patient exhales for the total duration of the test during maximal effort. Normal range is >80%. Decreasing percentage is associated with worsening / severe disease. FVC was only done in CF subjects because performance of the test requires intact respiratory muscle function. CF patients have intact respiratory muscle function and can therefore perform this maneuver. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from MND patients for this outcome measure. | Posted | Mean | Standard Deviation | percentage of predicted normal | 90 days |
|
|
|
| Secondary | FEV1/FVC Ratio | The proportion of the FVC expired in one second Results are given in both actual values (litres, litres per second) and %predicted-the test result as a percent of the "predicted values" for the patients of similar characteristics. Results over 80% are considered normal. Patients with Motor Neuron Disease suffer from progressive weakness of all muscles including the respiratory muscles. The test results for FVC are affected muscle weakness and are therefore not a valid representation of their pulmonary function. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from MND patients for this outcome measure. | Posted | Mean | Standard Deviation | FEV1/FVC ratio | 90 days |
|
|
|
| Secondary | SVC (Actual) | Slow Vital Capacity (SVC) is the actual volume measured in liters. Slow Vital Capacity displays the volume of gas measured on a complete expiration after a maximal inspiration without forced or rapid effort. Useful measurement when FVC is reduced, and airway obstruction is present. Educed SVC is associated with worsening respiratory disease. Slow Vital Capacity provides the same measure as FVC - i.e. "Vital Capacity." It does not, however, require a "Forced expiratory maneuver (it requires a slow inspiration and slow expiration) and therefore is easier to achieve a valid result in patients with Motor Neuron Disease who suffer from progressive weakness of all muscles including the respiratory muscles. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from CF patients for this outcome measure. | Posted | Mean | Standard Deviation | Liters | 90 days |
|
|
|
| Secondary | SVC (% Predicted) | SVC Percent Predicted (SVC % Predicted) is the actual volume exhaled in the first 1 second, divided by the "Normal" value for that actual value for a person of that age, gender, height and weight. A nomogram based on population norms provides a series of "Normal Values." Slow Vital Capacity displays the volume of gas measured on a complete expiration after a maximal inspiration without forced or rapid effort. Useful measurement when FVC is reduced and airway obstruction is present. Educed SVC is associated with worsening respiratory disease. Slow Vital Capacity provides the same measure as FVC - i.e. "Vital Capacity." It does not, however, require a "Forced expiratory maneuver (it requires a slow inspiration and slow expiration) and therefore is easier to achieve a valid result in patients with Motor Neuron Disease who suffer from progressive weakness of all muscles including the respiratory muscles. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from CF patients for this outcome measure. | Posted | Mean | Standard Deviation | percentage of predicted normal | 90 days |
|
|
|
| Secondary | SpO2 | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and Peak Cough Flow) are commonly followed in the clinical care of patients with MND. SpO2 stands for peripheral capillary oxygen saturation, an estimate of the amount of oxygen in the blood. Low SpO2 is associated with worsening respiratory function. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from CF patients for this outcome measure. | Posted | Mean | Standard Deviation | Percent of oxygen saturation | 90 days |
|
|
|
| Secondary | Maximal Inspiratory Pressure (MIP) | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and Peak Cough Flow) are commonly followed in the clinical care of patients with MND. Maximal inspiratory pressure (MIP) is a measure of the strength of inspiratory muscles, primarily the diaphragm, and allows for the assessment of ventilatory failure, restrictive lung disease and respiratory muscle strength. Reduction in MIP is asociated with worsening neuromuscular status and poor ability to cough. | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from CF patients for this outcome measure. | Posted | Mean | Standard Deviation | cmH2O | 90 days |
|
|
|
| Secondary | PCF | Standard pulmonary function measures (FEV1, FVC, and FEV1/FVC) are commonly followed in the clinical care of patients with CF. Standard pulmonary function measures (SVC, SpO2, MIP, and PCF) are commonly followed in the clinical care of patients with MND. The Peak Cough Flow (PCF) is the maximum air flow generated during a cough. Decreasing air flow generated is associated with weakening neuromuscular status and associated poor cough impulse | FVC and FEV1/FVC ratio are pulmonary function tests appropriate to assess lung function in patients with CF. These tests could not be physically performed by the patients in the MND population and thus these patients were not included in these analysis. For the MND population, SVC, SpO2, MIP, and PCF were performed instead of FVC and FEV1/FVC ratio. Hence, no data were collected from CF patients for this outcome measure. | Posted | Mean | Standard Deviation | liters per minute | 90 days |
|
|
|
| 0 |
| 5 |
| 0 |
| 5 |
| 0 |
| 5 |
| EG001 | Patients With MND Treated With Home Based Airway Clearance With Metaneb | Patients with MND who required regular home airway clearance therapy were enrolled to use the Metaneb device in the home setting. | 0 | 5 | 0 | 5 | 0 | 5 |
Not provided
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| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D019636 | Neurodegenerative Diseases |
| D009422 | Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Worsened |
|
| Missing |
|
| Same |
|
| More |
|
| Much More |
|
| Easy to perform (Day 30) |
|
| Like to continue MN4000 (Day 30) |
|
| Effective (Day 90) |
|
| Easy to perform (Day 90) |
|
| Like to continue MN4000 (Day 90) |
|
|
| Respiratory Sub-score at baseline |
|
| Respiratory Sub-score after 30 days of therapy |
|
| Respiratory Sub-score after 90 days of therapy |
|
| Total Score at Baseline |
|
| Total Score after 30 days of therapy |
|
| Total Score after 90 days of therapy |
|
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| Role domain at baseline |
|
| Role domain after 30 days of therapy |
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| Role domain after 90 days of therapy |
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| Vitality domain at baseline |
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| Vitality domain after 30 days of therapy |
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| Vitality domain after 90 days of therapy |
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| Emotion domain at baseline |
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| Emotion domain after 30 days of therapy |
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| Emotion domain after 90 days of therapy |
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| Social domain at baseline |
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| Social domain after 30 days of therapy |
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| Social domain after 90 days of therapy |
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| Body domain at baseline |
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| Body domain after 30 days of therapy |
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| Body domain after 90 days of therapy |
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| Eat domain at baseline |
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| Eat domain after 30 days of therapy |
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| Eat domain after 90 days of therapy |
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| Treat domain at baseline |
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| Treat domain after 30 days of therapy |
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| Treat domain after 90 days of therapy |
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| Health domain at baseline |
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| Health domain after 30 days of therapy |
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| Health domain after 90 days of therapy |
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| Weight domain at baseline |
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| Weight domain after 30 days of therapy |
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| Weight domain after 90 days of therapy |
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| Respiratory domain at baseline |
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| Respiratory domain after 30 days of therapy |
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| Respiratory domain after 90 days of therapy |
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| Digestion domain at baseline |
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| Digestion domain after 30 days of therapy |
|
| Digestion domain after 90 days of therapy |
|
|
| FEV1 measure after 90 days of therapy (Actual) |
|
| FEV1 measure after 90 days of therapy (% Predicted) |
|
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| Title | Measurements |
|---|---|
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| Title | Measurements |
|---|---|
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| Title | Measurements |
|---|---|
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| PCF after 90 days of therapy |
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