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

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

The study will be a non-randomized open label pilot study comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.

Study Overview

• Status: Terminated
• Conditions: Neuromuscular Diseases
• Intervention / Treatment: Device: 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.

• Study Type: Interventional
• Enrollment (Actual): 41
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27709
      • Science 37

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years to 80 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Patients who met all the following inclusion criteria are included in the study:

• Documented diagnosis of neuromuscular disease (NMD) or neurological disorder.
• Age 5 to 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 Oscillation and Lung Expansion (OLE) therapy as directed.
• Signed informed consent (and assent if minor patient).

Patients who meet any of the following criteria will be excluded from the study:

• Diagnosis with rapidly progressing NMD such as certain types of Motor Neuron Disease (MND).
• Requirement for continuous mechanical ventilation.
• Anticipated requirement for respiratory related hospitalization within the next six months.
• History of pneumothorax within the past 6 months.
• History of hemoptysis requiring embolization within past 12 months.
• Pregnancy.
• Home use of OLE therapy within the past 12 months.
• Inability or unwillingness to perform OLE therapy or study procedures as required.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

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 pre-treatment and 6 months post-treatment with the Volara System

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.	Baseline and 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.	Baseline and monthly for 6 months
Resting Oxygen Saturation	Oxygen Saturation will be measured while the subject is resting using a hand-held oximeter.	Baseline and monthly for 6 months
Maximal Inspiratory Pressure	Maximal Inspiratory Pressure is the maximum force (measured in centimeters of water [cmH2O]) that the subject can generate during an inspiratory effort.	Baseline and monthly for 6 months
Number of Hospital Admissions	Number of hospital admissions for respiratory complications.	6 months pre-treatment with the Volara System and 6 months post-treatment with the Volara System
Total Hospital Length of Stay	Number of inpatient hospital days for respiratory complications	6 months pre-treatment with the Volara System and 6 months post-treatment with the Volara System
Number of Intensive Care Unit (ICU) Admissions	Number of ICU admissions for respiratory complications.	6 months pre-treatment with the Volara System and 6 months post-treatment with the Volara System
Total Intensive Care Unit (ICU) Length of Stay	Number of ICU hospital days for respiratory complications	6 months pre-treatment with the Volara System and 6 months post-treatment with the Volara System
Number of Outpatient Visits for Pulmonary Complications	Number of unscheduled Physician's office visits, urgent care visits, and emergency department (ED) visits.	6 months pre-treatment and 6 months post-treatment with the Volara System
Total Number of Antibiotic Use Days During Episodes for Respiratory Infection	The total number of antibiotic use days includes any of the following: intravenous (IV) antibiotic days, oral antibiotic days, and nebulized antibiotic days.	6 months pre-treatment and 6 months post-treatment with the Volara System
Adherence to Treatment Regimen: Average Daily Volara System Usage	Adherence to the OLE treatment regimen was presented as Average Daily Volara System Usage. This was calculated by dividing the total minutes of device use by the total number of days the subject had the device.	Total number of days the subject had the device (approximately up to 6 months of OLE therapy)
Impact of OLE Therapy on Patient Quality of Life Using the Respiratory Complaints Domain of the Severe Respiratory Insufficiency (SRI) Questionnaire	Severe Respiratory Insufficiency (SRI) questionnaire administered at baseline, and at 1, 3 and 6 months. The total score can range from a minimum of 0 to a maximum of 100 with higher values indicating a better health-related quality of life according to content of the scale.	Baseline, and after 1 month, 3 months, and 6 months of therapy using the Volara System
Satisfaction With Current Airway Clearance Therapy at Baseline	Satisfaction is assessed through a questionnaire administered at baseline. Seven satisfaction questions are assessed using a Five-point Likert Scale. Total score is from 5 to 35, with 35 indicating the highest satisfaction.	Baseline
Satisfaction With OLE Therapy After 1 Month of Using the Volara System	Satisfaction is assessed through a questionnaire administered after 1 month of therapy using the Volara System. Eight satisfaction questions are assessed using a Five-point Likert Scale. Total score is from 5 to 40, with 40 indicating the highest satisfaction.	After 1 month of using the Volara System
Satisfaction With OLE Therapy After 3 Months of Using the Volara System	Satisfaction is assessed through a questionnaire administered after 3 months of therapy using the Volara System. Eight satisfaction questions are assessed using a Five-point Likert Scale. Total score is from 5 to 40, with 40 indicating the highest satisfaction.	After 3 months of using the Volara System
Satisfaction With OLE Therapy After 6 Months of Using the Volara System	Satisfaction is assessed through a questionnaire administered after 6 months of therapy using the Volara System. Eight satisfaction questions are assessed using a Five-point Likert Scale. Total score is from 5 to 40, with 40 indicating the highest satisfaction.	After 6 months of using the Volara System

Collaborators and Investigators

• Sponsor: Baxter Healthcare Corporation
• Collaborators: Science 37

Study record dates

Study Major Dates

• Study Start (Actual): November 4, 2021
• Primary Completion (Actual): February 27, 2024
• Study Completion (Actual): February 27, 2024

Study Registration Dates

• First Submitted: April 27, 2022
• First Submitted That Met QC Criteria: May 4, 2022
• First Posted (Actual): May 9, 2022

Study Record Updates

• Last Update Posted (Actual): July 31, 2025
• Last Update Submitted That Met QC Criteria: July 30, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: CR-RR-2020-005

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes
• product manufactured in and exported from the U.S.: Yes