Clinical trial NCT03797677: MN4000 for Treatment of CF and MND Patients in the Home Setting
Official title: A Pilot Study to Evaluate the Use of the MN4000 for Treatment of Cystic Fibrosis (CF) and Motor Neuron Disease (MND) Patients in the Home Setting
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Cystic Fibrosis
Motor Neuron Disease
Airway Clearance Impairment
Keywords: Airway Clearance
Cystic Fibrosis
Motor Neuron Disease
Home care
Lead sponsor: Baxter Healthcare Corporation
Sponsor class: INDUSTRY
First posted: 2019-01-09
Enrollment: 10
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.

This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period.

Detailed description: The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.

This pilot study evaluated subject satisfaction with the therapy and adherence to the therapy during the 90-day treatment period, and also collected clinical outcome data. Outcomes were assessed before, during and after the MN4000 treatment period. The study did not include a control group. This pilot study was designed to provide initial information that could inform decisions for future larger-scale studies.

Ten (10) patients total were enrolled from CF and NMD clinics. Eligible subjects were adult patients who were able to perform MN4000 therapy using a mouthpiece and who met all inclusion and none of the exclusion criteria.

All patients received therapy with the MN4000 following the labeled instructions for the device.

The MN4000 is an airway clearance and lung expansion therapy device that has been cleared to market by the FDA as The MetaNeb® System for Homecare environment, for clearance of pulmonary secretions and for treatment or prevention of pulmonary atelectasis. It is a Class II device, cleared to market on March 17, 2016 under premarket notification 510(k) K151689 as The MetaNeb® 4 System with application for homecare environment. It is commercially marketed as the MN4000. The device consists of a pneumatic compressor and an air pulse generator that delivers CHFO and CPEP to;

1. facilitate clearance of mucous from the lungs;
2. provide lung expansion therapy and;
3. enhance delivery of aerosol therapy.

This "triple" mode device can provide aerosol therapy while alternating between CPEP for lung expansion and CHFO for airway clearance. Supplemental oxygen therapy may also be delivered when used with compressed oxygen.

The MN4000 has three therapy modes:

* CHFO (Continuous High Frequency Oscillation) - delivers aerosol therapy while providing oscillating pressure pulses to the airway
* CPEP (Continuous Positive Expiratory Pressure) - delivers aerosol therapy while providing continuous positive pressure to help hold open and expand the airways
* Aerosol - for delivery of aerosol only. In this mode, CHFO and CPEP are not available

After assessing baseline status, therapy with the MN4000 was introduced and incorporated into the daily home respiratory care treatment regimen for all patients.

Other airway clearance and/or lung expansion therapies were not to be performed during the three-month study period. The treatment regimen for other respiratory care modalities (e.g. aerosolized medications) was that which was prescribed by the patient's health care team in the routine standard care of each patient.

During the three-month follow-up period, adherence to the daily prescribed therapy regimen was assessed. Subjects/caregivers were asked to provide adherence information for each day during the 90-day study period.

Documentation of efficacy and safety Variables was completed by study staff at the time of occurrence, from review of the patient's medical records and from scores and rankings for questionnaires.

Eligibility criteria: Inclusion Criteria:

* Documented diagnosis of CF or MND
* Age ≥ 18 years
* Signed informed consent

Exclusion Criteria:

* Requirement for continuous mechanical ventilation
* Anticipated requirement for hospitalization within the next three months
* History of pneumothorax within past 6 months
* History of hemoptysis requiring embolization within past 12 months
* Inability to perform MN4000 therapy using a mouthpiece (e.g. inability to create adequate mouth seal)
* Inability to perform MN4000 therapy as directed
* Inability or unwillingness to complete study visits or provide follow-up data as required by the study protocol

Primary outcomes:
measure: Patient / Caregiver Satisfaction Scores
timeFrame: 90 days
measure: Patient / Caregiver Satisfaction Scores
timeFrame: 90 days
Secondary outcomes:
measure: Mean Percentage Adherence to Prescribed Treatment Regimen
timeFrame: 90 days
measure: ALS-Functional Rating Scale (ALS-FRS)
timeFrame: 90 days
measure: Cystic Fibrosis Questionnaire - Revised (CFQ-R)
timeFrame: 90 days
measure: Number of Participants With Exacerbation of Pulmonary Disease
timeFrame: 90 days
measure: FEV1 (Actual)
timeFrame: 90 days
measure: FEV1 (% Predicted)
timeFrame: 90 days
measure: FVC (Actual)
timeFrame: 90 days
measure: FVC (% Predicted)
timeFrame: 90 days
measure: FEV1/FVC Ratio
timeFrame: 90 days
measure: SVC (Actual)
timeFrame: 90 days
measure: SVC (% Predicted)
timeFrame: 90 days
measure: SpO2
timeFrame: 90 days
measure: Maximal Inspiratory Pressure (MIP)
timeFrame: 90 days
measure: PCF
timeFrame: 90 days
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Northwestern
state: Illinois
Study officials:
affiliation: Northwestern University
name: Lisa F Wolfe, MD
role: PRINCIPAL_INVESTIGATOR
References: