Oscillation and Lung Expansion (OLE) for Treatment of Neuromuscular Disease Patients
Primary Purpose
Neuromuscular Diseases
Status
Recruiting
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Oscillation and Lung Expansion (OLE) therapy
Sponsored by
About this trial
This is an interventional prevention trial for Neuromuscular Diseases
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
Sites / Locations
- Science 37Recruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Intervention
Arm Description
Intervention time period, during which all subjects receive OLE therapy as their airway clearance intervention
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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.
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
Oxygen (O2) saturation
O2 Saturation will be measured while the subject is resting using a hand-held oximeter
Maximal Inspiratory Pressure
Maximal Inspiratory Pressure is the maximum force (measured in cmH20) that the subject can generate during an inspiratory effort
Number of hospitalizations
for respiratory complications
Number of ICU admissions
for respiratory complications
Number of outpatient visits for pulmonary complications
Unscheduled Physician's office, Urgent Care Visits, ED visits
Number of antibiotic use days during episodes of pneumonia
Includes any of the following: IV antibiotic days, Oral antibiotic days, Nebulized antibiotic days
Adherence to treatment regimen
Adherence to the OLE treatment regimen will be determined by downloading device therapy log data
Number of inpatient hospital days
For respiratory complications
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
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05366010
Brief Title
Oscillation and Lung Expansion (OLE) for Treatment of Neuromuscular Disease Patients
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
Study Type
Interventional
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 4, 2021 (Actual)
Primary Completion Date
April 2024 (Anticipated)
Study Completion Date
April 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hill-Rom
4. Oversight
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
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuromuscular Diseases
7. Study Design
Primary Purpose
Prevention
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
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)
Allocation
N/A
Enrollment
70 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Intervention
Arm Type
Experimental
Arm Description
Intervention time period, during which all subjects receive OLE therapy as their airway clearance intervention
Intervention Type
Device
Intervention Name(s)
Oscillation and Lung Expansion (OLE) therapy
Intervention Description
Respiratory airway clearance intervention including continuous high-frequency oscillation and continuous positive expiratory pressure
Primary Outcome Measure Information:
Title
Frequency of exacerbations of pulmonary disease requiring medical intervention
Description
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
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Slow Vital Capacity
Description
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.
Time Frame
Monthly for 6 months
Title
Peak Cough Flow
Description
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
Time Frame
Monthly for 6 months
Title
Oxygen (O2) saturation
Description
O2 Saturation will be measured while the subject is resting using a hand-held oximeter
Time Frame
Monthly for 6 months
Title
Maximal Inspiratory Pressure
Description
Maximal Inspiratory Pressure is the maximum force (measured in cmH20) that the subject can generate during an inspiratory effort
Time Frame
Monthly for 6 months
Title
Number of hospitalizations
Description
for respiratory complications
Time Frame
6 months
Title
Number of ICU admissions
Description
for respiratory complications
Time Frame
6 months
Title
Number of outpatient visits for pulmonary complications
Description
Unscheduled Physician's office, Urgent Care Visits, ED visits
Time Frame
6 months
Title
Number of antibiotic use days during episodes of pneumonia
Description
Includes any of the following: IV antibiotic days, Oral antibiotic days, Nebulized antibiotic days
Time Frame
6 months
Title
Adherence to treatment regimen
Description
Adherence to the OLE treatment regimen will be determined by downloading device therapy log data
Time Frame
6 months
Title
Number of inpatient hospital days
Description
For respiratory complications
Time Frame
6 months
Title
Impact of OLE therapy on patient quality of life using the Severe Respiratory Insufficiency (SRI) questionnaire
Description
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
Time Frame
6 months
Title
Satisfaction with OLE therapy
Description
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
Time Frame
6 months.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Brian Becker, MEd, RRT
Phone
16126001301
Email
brian.becker@hillrom.com
First Name & Middle Initial & Last Name or Official Title & Degree
Victoria Taylor
Phone
13103652885
Email
victoria.taylor@science37.com
Facility Information:
Facility Name
Science 37
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27709
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Victoria Taylor, MBA
Phone
310-365-2885
Email
p-cooper@northwestern.edu
First Name & Middle Initial & Last Name & Degree
Brent Hsu
Phone
1-310-935-5030
Email
brent@science37.com
First Name & Middle Initial & Last Name & Degree
Debra Weinstein, MD
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Oscillation and Lung Expansion (OLE) for Treatment of Neuromuscular Disease Patients
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