A Pilot Randomized Clinical Trial of Early Ventilation in Amyotrophic Lateral Sclerosis (EVENT ALS)

Launched by UNIVERSITY OF PENNSYLVANIA · Jul 8, 2025

Keywords

ClinConnect Summary

This clinical trial is studying whether starting a breathing support machine called non-invasive ventilation (NIV) earlier than usual can help people with Amyotrophic Lateral Sclerosis (ALS). ALS is a condition that causes muscle weakness and can eventually make it hard to breathe. NIV is a machine that helps people breathe by gently pushing air in and out through a mask. Currently, most insurance plans only cover NIV when breathing problems become quite serious, but this study wants to see if starting NIV sooner can improve health and quality of life for people with ALS.

To take part, individuals must have been diagnosed with ALS within the last six months, be 18 years or older, and able to follow the study procedures. People who have already used or been prescribed NIV or certain breathing devices, have severe breathing weakness, or have other health issues like recent surgery or cognitive problems would not be eligible. Participants will be divided into two groups: one will start NIV early, and the other will receive usual care and begin NIV when it's normally recommended. The study will use a special tool to predict who is at higher risk for breathing problems in the next six months. The goal is to see if early use of NIV is possible and helpful, to better understand how it affects symptoms and breathing, and to learn more about who might have trouble using NIV regularly. This is a small, early study to help plan a larger trial in the future.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Diagnosed with ALS using the Gold Coast Criteria within the last 6 months
• • Age ≥18 years
• • Willingness and ability to participate in study procedures
• • Provision of signed and dated informed consent form
• Exclusion Criteria:
• 1. Current or prior or recommended/prescribed use of NIV including:
• • i) bi-level positive pressure ventilation, such as a respiratory assist device or home ventilator ii) Current or prior use of continuous positive airway pressure, or "CPAP" therapy
• • 2. Forced vital capacity \<50% of predicted normal
• • 3. Maximal inspiratory pressure \> -60 cmH2O (eg, -50 or -40 cmH2O would be excluded)
• • 4. Chronic use of supplemental oxygen at any part of the day
• • 5. Enrollment in hospice
• • 6. Current tracheostomy
• • 7. Prior history of sleep apnea where non-invasive ventilation was used or recommended
• • 8. Thoracic, abdominal, facial or ophthalmic surgery in the prior 6 weeks
• • 9. Coughing up blood
• • 10. Myocardial infarction in the previous 4 weeks
• • 11. Absolute contraindication to NIV, which includes lethargy, obtundation, facial fractures, active pneumothorax, and airway obstruction (such as a tumor)
• • 12. Presence of cognitive dysfunction that would impair ability to complete study procedures, as determined by neurology attending physician