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

Amyotrophic Lateral Sclerosis Clinical Trial

— MI-PALS  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06119087
• Other study ID #: 854981
• Status: Not yet recruiting
• Phase: N/A
• Start date: June 1, 2024
• Est. completion date: December 31, 2025

Study information

• Verified date: April 2024
• Source: University of Pennsylvania
• Contact: Jason Ackrivo, MD
• Phone: 2156623202
• Email: jason.ackrivo[@]pennmedicine.upenn.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: December 31, 2025
• Est. primary completion date: September 8, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Airway Clearance Impairment
• Amyotrophic Lateral Sclerosis
• Chronic Respiratory Failure
• Motor Neuron Disease
• Neuromuscular Diseases
• Respiratory Insufficiency
• Sclerosis

Intervention

Device:
• Mechanical insufflation
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.

Locations

Country	Name	City	State
United States	University of Pennsylvania	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
University of Pennsylvania

Country where clinical trial is conducted

United States, 

References & Publications (32)

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* Note: There are 32 references in all — Click here to view all references

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Time to start of non-invasive ventilation	Defined by the time period between enrollment for the MI trial and date of prescription for non-invasive ventilation; Non-invasive ventilation start date will be defined by date of prescription for non-invasive ventilation as recorded in the medical record or patient-reported date if a prescription is not recorded in the medical record.	1 year
Other	Tracheostomy free survival time	Defined by the time period between enrollment for the MI trial and death as long as the subject never had a tracheostomy placed.; Death date will be determined by family report, electronic medical record review, or publicly available obituaries.; Tracheostomy placement will be determined by patient or family report; or as documented in the electronic medical record.	1 year
Primary	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	Forced vital capacity (FVC)	Defined as maximal volume, in liters, that a person can exhale forcefully after a complete inhalation.; Measured using a spirometer connected to a face mask or mouthpiece in the sitting and upright position.	6 months
Secondary	Maximal inspiratory pressure (MIP)	Defined as the maximum pressure, in cmH2O, generated when the subject exhales as much air as possible and then immediately inhales as forcefully as possible.	6 months
Secondary	Maximal expiratory pressure (MEP)	Defined as the maximum pressure, in cmH2O, generated when the subject inhales as much air as possible and then immediately exhales as forcefully as possible.; Measured using a hand-held manual or digital manometer connected to a mouthpiece or mask in the sitting and upright position.	6 months
Secondary	Maximum insufflation capacity (MIC)	Defined as exhaled volume, in liters, immediately following a MI maneuver to maximum insufflation capacity.; Measured using a spirometer connected to a face mask or mouthpiece, in the sitting and upright position.	6 months
Secondary	Maximum insufflation capacity assisted peak cough flow	Defined as the peak cough flow generated from MIC.; Lungs are inflated to MIC using MI and before the subject exhales they insert a peak flow meter in their mouth and follow with a peak cough flow as described above.	6 months
Secondary	MIC-FVC difference	defined by subtracting the FVC from the MIC	6 months
Secondary	Transcutaneous carbon dioxide	Defined as the average value in mmHg from a daytime in-clinic measurement over a 15-minute transcutaneous recording.; Measured using a Sentec transcutaneous digital monitoring system while the subject is sitting upright in a chair or wheelchair. The transcutaneous sensor can be placed on the subject forehead, cheek, or earlobe.	6 months
Secondary	ALS Functional Rating Scale - Revised (ALSFRS-R) dyspnea and orthopnea scores	The ALSFRS-R is a 12-item standardized questionnaire to assess the motor function status of an individual with ALS. The ALSFRS-R is assessed by a research staff member who has been certified for performance of the ALSFRS-R.; The dyspnea score is one of the questions focusing on level of shortness of breath, scored on a scale of 0 to 4, with 4 being no symptoms at all and 0 being severely symptomatic with consideration of mechanical respiratory support.; The orthopnea score is one of the questions focused on breathing symptoms and difficulty sleeping while lying supine, scored on a scale of 0 to 4, with 4 being no symptoms at all and 0 being severely symptomatic with inability to sleep.	6 months
Secondary	Global rate of change score for peak cough flow	The global rate of change score for the peak cough flow will ask patients how effective they think their cough is today by rating it on a Likert scale from -7 (extremely impaired) to +7 (extremely strong).	6 months