Using a device to help breathing in early amyotrophic lateral sclerosis
Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study
This study is testing if a breathing device can help people with early amyotrophic lateral sclerosis (ALS) improve their cough strength over six months.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | University of Pennsylvania Academic / other |
| Locations | 1 site (Philadelphia, Pennsylvania) |
| Trial ID | NCT06119087 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the effects of mechanical insufflation (MI) on breathing in patients with early amyotrophic lateral sclerosis (ALS). It will be a single-center, single-arm study involving 20 participants at the University of Pennsylvania. Participants will use the BiWaze Cough system, a mechanical insufflator-exsufflator device, to perform daily breathing exercises designed to improve cough strength over a six-month period. The primary outcome will be measured by peak cough flow (PCF) to assess the effectiveness of the intervention.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with early-stage amyotrophic lateral sclerosis who have a caregiver to assist with the device.
Not a fit: Patients currently using non-invasive ventilation or those with advanced ALS may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could improve respiratory function and quality of life for patients with early ALS.
How similar studies have performed: Previous studies have shown promise in using mechanical insufflation for respiratory support in neuromuscular diseases, suggesting potential success for this approach.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Philadelphia, Pennsylvania
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (Recruiting)
Study contacts
- Principal investigator: Jason Ackrivo, MD — University of Pennsylvania
- Study coordinator: Jason Ackrivo, MD
- Email: jason.ackrivo@pennmedicine.upenn.edu
- Phone: 2156623202
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.