Clinical trial NCT05366010: 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
Overall status: TERMINATED
Phase: NA
Conditions: Neuromuscular Diseases
Lead sponsor: Baxter Healthcare Corporation
Sponsor class: INDUSTRY
First posted: 2022-05-09
Enrollment: 41
Sex: ALL
Minimum age (years): 5
Maximum age (years): 80
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention 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.
Primary purpose: PREVENTION

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.

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

Primary outcomes:
measure: Frequency of exacerbations of pulmonary disease requiring medical intervention
timeFrame: 6 months
Secondary outcomes:
measure: Slow Vital Capacity
timeFrame: Monthly for 6 months
measure: Peak Cough Flow
timeFrame: Monthly for 6 months
measure: Oxygen (O2) saturation
timeFrame: Monthly for 6 months
measure: Maximal Inspiratory Pressure
timeFrame: Monthly for 6 months
measure: Number of hospitalizations
timeFrame: 6 months
measure: Number of ICU admissions
timeFrame: 6 months
measure: Number of outpatient visits for pulmonary complications
timeFrame: 6 months
measure: Number of antibiotic use days during episodes of pneumonia
timeFrame: 6 months
measure: Adherence to treatment regimen
timeFrame: 6 months
measure: Number of inpatient hospital days
timeFrame: 6 months
measure: Impact of OLE therapy on patient quality of life using the Severe Respiratory Insufficiency (SRI) questionnaire
timeFrame: 6 months
measure: Satisfaction with OLE therapy
timeFrame: 6 months.
Other outcomes:
Locations:
city: Durham
country: United States
facility: Science 37
state: North Carolina
Study officials:
References: