Oscillation and Lung Expansion (OLE) for Treatment of Neuromuscular Disease 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 using an observational design comparing a retrospective control period to an active treatment period with oscillation and lung expansion (OLE) therapy.

Study Overview

• Status: Recruiting
• 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 (Estimated): 70
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Brian Becker, MEd, RRT; Phone Number: 16126001301; Email: brian.becker@hillrom.com
• Study Contact Backup: Name: Victoria Taylor; Phone Number: 13103652885; Email: victoria.taylor@science37.com

Study Locations

• United States
  • North Carolina
    • Durham, North Carolina, United States, 27709
      • Recruiting
      • Science 37
      • Contact: Victoria Taylor, MBA; Phone Number: 310-365-2885; Email: p-cooper@northwestern.edu
      • Contact: Brent Hsu; Phone Number: 1-310-935-5030; Email: brent@science37.com
      • Principal Investigator: Debra Weinstein, MD

Participation Criteria

Eligibility Criteria

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

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• 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

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.

Collaborators and Investigators

• Sponsor: Hill-Rom

Study record dates

Study Major Dates

• Study Start (Actual): November 4, 2021
• Primary Completion (Estimated): April 1, 2024
• Study Completion (Estimated): April 1, 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): October 12, 2023
• Last Update Submitted That Met QC Criteria: October 10, 2023
• Last Verified: October 1, 2023

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