Clinical trial NCT06119087: Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study
Official title: Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
Chronic Respiratory Failure
Airway Clearance Impairment
Neuromuscular Diseases
Keywords: amyotrophic lateral sclerosis
chronic respiratory failure
neuromuscular weakness
mechanical insufflation-exsufflation
peak cough flow
Lead sponsor: University of Pennsylvania
Sponsor class: OTHER
First posted: 2023-11-07
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: single-center, single-arm feasibility pilot study of in 20 patients with ALS.
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: peak cough flow (PCF)
timeFrame: 6 months
Secondary outcomes:
measure: Forced vital capacity (FVC)
timeFrame: 6 months
measure: Maximal inspiratory pressure (MIP)
timeFrame: 6 months
measure: Maximal expiratory pressure (MEP)
timeFrame: 6 months
measure: Maximum insufflation capacity (MIC)
timeFrame: 6 months
measure: Maximum insufflation capacity assisted peak cough flow
timeFrame: 6 months
measure: MIC-FVC difference
timeFrame: 6 months
measure: Transcutaneous carbon dioxide
timeFrame: 6 months
measure: ALS Functional Rating Scale - Revised (ALSFRS-R) dyspnea and orthopnea scores
timeFrame: 6 months
measure: Global rate of change score for peak cough flow
timeFrame: 6 months
Other outcomes:
measure: Time to start of non-invasive ventilation
timeFrame: 1 year
measure: Tracheostomy free survival time
timeFrame: 1 year
Locations:
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
Study officials:
affiliation: University of Pennsylvania
name: Jason Ackrivo, MD
role: PRINCIPAL_INVESTIGATOR
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