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

NCT06119087 | Recruiting DMC FDA Device

• 1 other identifier: 854981
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

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

Outcome Measures

Primary Outcomes (1)

• peak cough flow (PCF)

  PCF in liters/minute as measured by use of a peak flow meter while the patient is in a seated upright position. An alternative device for measuring PCF can be the use of a handheld spirometer and using the measured peak expiratory flow by multiplying by 60 to convert from liters/second to liters/minute. The subject must be seated in an upright position and the interface must include a mouthpiece or an oronasal mask. Subjects are asked to perform a deep inhalation followed by a maximal cough.

  6 months

Secondary Outcomes (9)

• Forced vital capacity (FVC)

  6 months

• Maximal inspiratory pressure (MIP)

  6 months

• Maximal expiratory pressure (MEP)

  6 months

• Maximum insufflation capacity (MIC)

  6 months

• Maximum insufflation capacity assisted peak cough flow

  6 months

Other Outcomes (2)

• Time to start of non-invasive ventilation

  1 year

• Tracheostomy free survival time

  1 year

Study Arms (1)

Mechanical insufflation

EXPERIMENTAL

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

Device: Mechanical insufflation

Interventions

Mechanical insufflation DEVICE

Mechanical insufflation (MI) is a chest physiotherapy exercise that will be performed using a type of mechanical insufflator-exsufflator (MI-E) known as the BiWaze Cough device. The device connects to a tube that can interface with a patient using either a facemask or mouthpiece. Mechanical insufflation is a chest physiotherapy exercise that passively inflates the chest with positive pressure that is delivered in coordination with the patient's own inspiratory timing until maximal inflation capacity (MIC), determined by the patient or maximal chest rise on visual inspection. At MIC, the patient passively exhales, which completes one "cycle". Prior literature has used a "dose" of 5 sets of 5 cycles once or twice daily. The maneuver is usually performed with assistance of a caregiver to hold the mask or mouthpiece in place.

Mechanical insufflation

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• Age \<18 years old.
• Inability to perform a cough peak flow or spirometry manuever
• Current use of non-invasive ventilation (NIV), bi-level positive pressure ventilation, or "Bi-PAP" or physician prescribing NIV on day of potential enrollment.
• 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.
• Active enrollment in hospice.
• Current tracheostomy.
• Presence of cognitive dysfunction that would impair ability to complete study procedures, as determined by neurology attending physician.
• Absence of an able and willing caregiver to assist with MI twice daily as specified in the protocol.
• Pregnancy
• Medical history of any of the following:
• Recent hemoptysis
• Recent barotrauma
• History of emphysema of any kind (including bullous emphysema)
• History of or known susceptibility to pneumothorax
• History of or known susceptibility to pneumomediastinum

Sponsors & Collaborators

• University of Pennsylvania: lead

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

Related Publications (32)

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MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Neuromuscular Diseases

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Jason Ackrivo, MD

  University of Pennsylvania

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jason Ackrivo, MD

CONTACT

2156623202; jason.ackrivo@pennmedicine.upenn.edu

Kelsey Moulton

CONTACT

Kelsey.Moulton@pennmedicine.upenn.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: single-center, single-arm feasibility pilot study of in 20 patients with ALS.

Study Record Dates

• First Submitted: October 31, 2023
• First Posted: November 7, 2023
• Study Start: September 12, 2024
• Primary Completion (Estimated): June 8, 2026
• Study Completion (Estimated): June 8, 2026
• Last Updated: October 21, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)