Oscillation and Lung Expansion (OLE) for Treatment of Neuromuscular Disease Patients

Study Overview
=================
Brief Summary
-----------------
The study will be a non-randomized open label pilot study using an observational design comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.

Detailed Description
-----------------
The study is a decentralized trial. all data will be collected in the patients' homes. The primary objective of the study is to evaluate the impact of OLE to treat respiratory complications of neuromuscular disease patients. The frequency of pulmonary exacerbations and other clinical outcome measures will be assessed to determine the effect of consistent OLE therapy.

Official Title
-----------------
Evaluation of Oscillation and Lung Expansion (OLE) Using The Volara® System for Treatment of Respiratory Complications in Patients With Neuromuscular Disease in the Home Setting

Conditions
-----------------
Neuromuscular Diseases

Intervention / Treatment
-----------------
* Device: Oscillation and Lung Expansion (OLE) therapy


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Documented diagnosis of neuromuscular disease (NMD) Age 5-80 years History of one or more respiratory exacerbations in the past 6 months or two or more respiratory exacerbations in the past 12 months, which required unplanned or unscheduled medical intervention. Ability to perform OLE therapy as directed Signed informed consent (and assent if minor subject) Exclusion Criteria: Diagnosis with rapidly progressing NMD such as certain types of Motor Neuron Disease (MND) Anticipated requirement for hospitalization within the next six months History of pneumothorax within past 6 months History of hemoptysis requiring embolization within past 12 months Pregnancy Use of OLE therapy within the past 12 months Inability or unwillingness to perform OLE therapy or study procedures as required

Ages Eligible for Study
-----------------
Minimum Age: 5 Years
Maximum Age: 80 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Interventional Model Description: Study subjects will serve as their own control. Medical records will be reviewed for each study subject for the twelve-month period prior to initiation of OLE therapy. The study will compare the frequency of exacerbations experienced prior to treatment with OLE to the frequency of exacerbations experienced in the active treatment period, during which study subjects receive treatment with The Volara® System. In addition, specific healthcare utilization indicators including hospitalizations, antibiotic use, and emergency department (ED) visits for pulmonary complications will be documented for each study period.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Intervention<br>Intervention time period, during which all subjects receive OLE therapy as their airway clearance intervention | Device: Oscillation and Lung Expansion (OLE) therapy<br>* Respiratory airway clearance intervention including continuous high-frequency oscillation and continuous positive expiratory pressure<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of exacerbations of pulmonary disease requiring medical intervention | Exacerbation of pulmonary disease is a worsening of pulmonary condition or an event requiring one or more of the following: Hospitalization Emergency Department visit Unscheduled antibiotics Unscheduled outpatient visit | 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Slow Vital Capacity | Slow Vital Capacity is the maximum volume that the subject can exhale in a single breath, following full inspiration. The measure is taken using a slow consistent exhalation. | Monthly for 6 months | 
| Peak Cough Flow | Peak Cough Flow is a pulmonary function measure that evaluates maximum peak flow generated during a cough maneuver. It will be measured with a hand-held spirometer | Monthly for 6 months | 
| Oxygen (O2) saturation | O2 Saturation will be measured while the subject is resting using a hand-held oximeter | Monthly for 6 months | 
| Maximal Inspiratory Pressure | Maximal Inspiratory Pressure is the maximum force (measured in cmH20) that the subject can generate during an inspiratory effort | Monthly for 6 months | 
| Number of hospitalizations | for respiratory complications | 6 months | 
| Number of ICU admissions | for respiratory complications | 6 months | 
| Number of outpatient visits for pulmonary complications | Unscheduled Physician's office, Urgent Care Visits, ED visits | 6 months | 
| Number of antibiotic use days during episodes of pneumonia | Includes any of the following: IV antibiotic days, Oral antibiotic days, Nebulized antibiotic days | 6 months | 
| Adherence to treatment regimen | Adherence to the OLE treatment regimen will be determined by downloading device therapy log data | 6 months | 
| Number of inpatient hospital days | For respiratory complications | 6 months | 
| Impact of OLE therapy on patient quality of life using the Severe Respiratory Insufficiency (SRI) questionnaire | Severe Respiratory Insufficiency (SRI) questionnaire administered at baseline and at 1, 3 and six months. Scoring of the SRI is from 0 - 100. 100 = Best Quality of Life | 6 months | 
| Satisfaction with OLE therapy | Satisfaction assessed by questionnaire administered at baseline and at 1, 3 and six months. Eight satisfaction questions are assessed using a Five-point Likert Scale. Total score is 5 - 40. 40 = Highest Satisfaction | 6 months. |