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

Conditions

• Amyotrophic Lateral Sclerosis (ALS)
• Chronic Respiratory Failure
• Neuromuscular Disease
• Neuromuscular Disease Patients
• Non-invasive Ventilation
• Non-invasive Ventilation Support
• Non-invasive Ventilatory Support
• Positive Pressure Ventilation
• Respiratory Insufficiency
• Respiratory Insufficiency Requiring Mechanical Ventilation

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Non-invasive ventilation — DEVICE
  Non-invasive ventilation will be started earlier than usual respiratory care guidelines for ALS in the United States.

Study Details

Amyotrophic lateral sclerosis (ALS) is a disease that causes weakness of the muscles of the body. The disease can eventually lead to severe breathing problems, which is the most common cause of death from ALS. The treatment for breathing is non-invasive ventilation (NIV). It is a machine that helps a person breathe by pushing air in and out of their lungs through a mask worn over the face. Research has shown that NIV can improve the quality of life and survival of someone with ALS. Unfortunately, NIV is not equally beneficial for everyone. The investigators do not yet know the best time or method for starting NIV in ALS. Europe and Canada allow starting NIV much earlier in ALS than the United States. Current recommendations for starting NIV are based on the opinion of experts rather than large research studies. Medical insurance companies will not cover NIV until significant breathing weakness occurs. After NIV is started, there is no evidence-based guidance on the best way to adjust NIV to benefit patients as much as possible. Some patients have difficulty tolerating NIV, but it is not clear how to identify these individuals ahead of time. The investigators have created a new prediction tool that can identify patients at high risk of breathing problems within the next 6 months. This may help the study team identify who is more likely to benefit from starting NIV early. The investigators have published a paper that shows that NIV helps people with ALS live longer. This paper also showed that patients get more benefit with use NIV for at least 4 hours per day. The investigators published another paper that measured a gas called carbon dioxide (CO2), which goes high if someone's breathing is weakened. This paper showed that patients with ALS may live longer when CO2 levels are lowered using NIV. The investigators also have data suggesting that certain characteristics may predict who is less likely to use NIV at least 4 hours per day. In this study, the investigators will collect pilot data on starting early NIV in individuals with ALS who do not yet meet insurance criteria for covering NIV. The research team will first use their previously published prediction tool to identify patient risk. Then, subjects would be randomized to start early NIV or to usual care. The usual care group would eventually start NIV as would occur if the participants were not in the study. The purpose of this study is to collect data to help the investigators plan a larger randomized clinical trial. This study has 4 objectives. First, the project aims to identify individuals who would benefit from earlier NIV. The research team will use the original prediction tool to identify risk of severe breathing problems within the next 6 months. Second, the project aims to show that it is feasible to start NIV early. Third, the project aims to gather data on the effect of randomization on symptoms, CO2 levels, and outcomes. Fourth, the project aims to identify traits that may make someone less likely to use NIV.

Key Dates

Start date

May 31, 2026

Status verified

Apr 2026

Primary completion

May 31, 2029

Completion

May 31, 2029

Study Design

Enrollment

48 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Early Non-invasive Ventilation
  Participants randomized to this arm will be assigned to start therapy with non-invasive ventilation.
• No Intervention: Usual care
  Participants randomized to this arm will receive usual care for amyotrophic lateral sclerosis.

Primary Outcome Measure

Time to first use of non-invasive ventilation [ Time Frame: From enrollment to end of follow-up at approximately 1 year ]

Central Contacts

• Jason T Ackrivo, MD, MSCE
  215-829-3053
  [email protected]
• Rebecca Lang Gallagher, MSEd
  215-662-4088
  [email protected]

Locations (3)

Find similar trials in Hershey, PA

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