Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study

University of Pennsylvania

Status: Enrolling

Conditions: Chronic Respiratory Failure; Neuromuscular Diseases; Amyotrophic Lateral Sclerosis; Airway Clearance Impairment

Treatments: Device: Mechanical insufflation

Study type: Interventional

Funder types: Other

Identifiers

NCT06119087

854981

Details and patient eligibility

About

The goal of this clinical trial is to learn how doing mechanical insufflation (MI) using a mechanical insufflator-exsufflator (MI-E) device affects breathing in early amyotrophic lateral sclerosis (ALS). This will be a single-center, single-arm study of MI in 20 patients with ALS at Penn.

Based on prior research, we believe that 6-months of MI may slow decline in cough strength, measured as peak cough flow (PCF).

Participants will perform MI using a device designed for mechanical insufflation-exsufflation (MI-E) known as the BiWaze Cough system. The BiWaze Cough is used for mucus clearance . It is connected to tubing and mouthpiece (or mask). The device will use programmed pressure and timing settings. An insufflation includes inflating the lungs for a maximal size inhalation before exhaling. The daily routine for the device includes 5 sets of 5 insufflations twice daily.

Researchers will compare how use of MI in early ALS affects peak cough flow compared to 20 subjects who did not use MI in early ALS.

Enrollment: 20 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Provision of signed and dated informed consent form.
2. Stated willingness to comply with all study procedures and availability for the duration of the study.
3. Age ≥18 years.
4. Diagnosed with amyotrophic lateral sclerosis using the Gold Coast Criteria.
5. Have an able and willing caregiver to assist with mechanical insufflation on a daily basis.
6. Willingness and ability to participate in study procedures.

Exclusion criteria

1. Age <18 years old.

2. Inability to perform a cough peak flow or spirometry manuever

3. Current use of non-invasive ventilation (NIV), bi-level positive pressure ventilation, or "Bi-PAP" or physician prescribing NIV on day of potential enrollment.

4. Current use of MI-E (also known as a "cough assist device") for airway clearance. Please note that patients can start use of a MI-E device subsequent to enrollment while currently being followed for the study.

5. Active enrollment in hospice.

6. Current tracheostomy.

7. Presence of cognitive dysfunction that would impair ability to complete study procedures, as determined by neurology attending physician.

8. Absence of an able and willing caregiver to assist with MI twice daily as specified in the protocol.

9. Pregnancy

10. Medical history of any of the following:

    1. Recent hemoptysis
    2. Recent barotrauma
    3. History of emphysema of any kind (including bullous emphysema)
    4. History of or known susceptibility to pneumothorax
    5. History of or known susceptibility to pneumomediastinum
    6. Chronic obstructive pulmonary disease
    7. Uncontrolled asthma (defined as recent exacerbation requiring corticosteroids in the previous 30 days)
    8. Symptomatic cardiomyopathy (heart failure) with left ventricular ejection fraction less than 50%
    9. History of right heart failure or pulmonary hypertension

11. Current smoker or tobacco use within the last 30 days.

Trial design

Primary purpose: Supportive Care

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Mechanical insufflation

Experimental group

Description:

Participants will be asked to perform mechanical insufflation using the BiWaze cough device as 5 sets of 5 insufflations twice daily for 6 months.

Treatment:

Device: Mechanical insufflation