Trial Outcomes & Findings for MN4000 for Treatment of CF and MND Patients in the Home Setting (NCT NCT03797677)

MN4000 for Treatment of CF and MND Patients in the Home Setting

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.

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.

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.

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: * Speech * Salivation * Swallowing * Handwriting * Cutting food and handling utensils (with or without gastrostomy) * Dressing and hygiene * Turning in bed and adjusting bed clothes * Walking * Breathing * Climbing stairs 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.

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.

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.

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

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

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 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.

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.

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.

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.

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.

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.

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