Non-Invasive Ventilation for ALS

(EVENT ALS Trial)

Not yet recruiting at 2 trial locations

Overseen By Rebecca Lang Gallagher, MSEd

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Pennsylvania

Disqualifiers: NIV use, Low vital capacity, Oxygen use, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the use of non-invasive ventilation (NIV) for individuals with amyotrophic lateral sclerosis (ALS), a condition that weakens muscles and can cause serious breathing problems. Researchers aim to determine if starting NIV earlier can benefit those at high risk of breathing issues, even before insurance typically covers it. Participants will join one of two groups: one starting early NIV and the other receiving usual care, which introduces NIV later. The study seeks to find the best way to use NIV, track symptoms and outcomes, and identify characteristics of individuals who may struggle with using NIV. Those recently diagnosed with ALS and not currently using or recommended to use NIV could be a good fit for this trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important findings.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

What prior data suggests that non-invasive ventilation is safe for ALS patients?

Studies have shown that non-invasive ventilation (NIV) is generally safe for people with ALS, a condition affecting the nerves and muscles. Research indicates that NIV can extend life and enhance quality of life. Even patients in the early stages of ALS benefit from starting NIV sooner rather than later. Although some patients may initially find it challenging to adjust to the machine, serious side effects are rare. Most people adapt well to the treatment over time. These findings suggest that NIV is a well-tolerated option for managing breathing problems in ALS, with a good safety record. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about using non-invasive ventilation (NIV) for ALS because it offers a way to support breathing without surgery or more invasive procedures. Unlike traditional treatments that might require a tracheostomy or rely solely on medications to manage symptoms, NIV provides respiratory support through a mask, which can enhance quality of life and potentially slow respiratory decline. This method is particularly appealing because it aims to improve comfort and ease of use for people with ALS, possibly allowing them to maintain independence for longer.

What evidence suggests that non-invasive ventilation is effective for ALS?

Research has shown that non-invasive ventilation (NIV) can extend the lives of people with amyotrophic lateral sclerosis (ALS). One study found that ALS patients who used NIV for at least 4 hours daily lived longer and experienced a better quality of life. Another study demonstrated that NIV reduces carbon dioxide (CO2) levels in the blood, which is crucial because high CO2 can be harmful to people with ALS. This trial will compare early initiation of NIV with usual care. Evidence suggests that starting NIV earlier might be more beneficial, especially for those at high risk of breathing problems. While NIV doesn't work the same for everyone, many patients experience significant benefits. ^{4 6 7 8 9}

Who Is on the Research Team?

Jason T Ackrivo, MD, MSCE

Principal Investigator

Perelman School of Medicine at the University of Pennsylvania

Are You a Good Fit for This Trial?

This trial is for individuals with ALS who are at high risk of developing severe breathing problems within the next 6 months but do not yet qualify for non-invasive ventilation (NIV) coverage by insurance. The study aims to identify those who could benefit from starting NIV early.

Inclusion Criteria

Provision of signed and dated informed consent form

I was diagnosed with ALS in the last 6 months.

I am willing and able to follow the study's procedures.

Exclusion Criteria

I cannot use a non-invasive ventilator due to specific health issues.

I use or have been advised to use a CPAP or BiPAP machine.

I have not had surgery on my chest, abdomen, face, or eyes in the last 6 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Randomization and Early NIV Initiation

Participants are randomized to either early non-invasive ventilation (NIV) or usual care. Early NIV is initiated for those in the experimental group.

6 months

Regular visits for NIV adjustment and monitoring

Follow-up

Participants are monitored for safety, effectiveness, and adherence to NIV. Data on quality of life, CO2 levels, and other outcomes are collected.

1 year

Periodic in-person and virtual follow-ups

Data Analysis and Outcome Assessment

Analysis of collected data to assess feasibility, effect sizes, and factors influencing NIV adherence.

What Are the Treatments Tested in This Trial?

Interventions

• Non-invasive ventilation

Trial Overview The trial is testing the benefits of starting non-invasive ventilation (NIV) earlier than current standards in the United States. Participants will be randomly assigned to either begin early NIV or receive usual care, with outcomes compared to plan a larger future trial.

How Is the Trial Designed?

Group I: Early Non-invasive Ventilation Experimental Treatment 1 Intervention

Participants randomized to this arm will be assigned to start therapy with non-invasive ventilation.

Group II: Usual care Active Control 1 Intervention

Participants randomized to this arm will receive usual care for amyotrophic lateral sclerosis.

Who Is Running the Clinical Trial?

University of Pennsylvania

Lead Sponsor

Trials

2,118

Recruited

45,270,000+

United States Department of Defense

Collaborator

Trials

940

Recruited

339,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7919153/

Noninvasive Ventilation Use Is Associated with Better ... Rationale: Noninvasive ventilation (NIV) is standard of care in amyotrophic lateral sclerosis (ALS), yet few data exist regarding its benefits.

2. jcsm.aasm.org jcsm.aasm.org/doi/10.5664/jcsm.2586

Effects of Noninvasive Ventilation on Sleep Outcomes in ... NIV improved oxygenation but showed no significant effects on sleep efficiency, sleep arousals, restful sleep, or sleep architecture.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT00537641

Efficacy of Noninvasive Ventilation in Amyotrophic Lateral ... This study will test the hypothesis that noninvasive ventilation (NIV) as prescribed in current medical practice for use in amyotrophic Lateral Sclerosis (ALS) ...

4. publications.ersnet.org publications.ersnet.org/content/erj/50/suppl61/pa2151

NIV (Non Invasive Ventilation) criteria and Amyotrophic ... The aim of our study was to assess different criteria used to introduce NIV in ALS patients, including polysomnography (PSG) values and how it can affecttheir ...

5. mdpi.com mdpi.com/2075-1729/14/12/1664

Survival Prognostic Factors of Non-Invasive Ventilation in ... According to the evidence base available to date, NIV favors survival compared to non-NIV in patients with bulbar onset ALS.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6589990/

Non-invasive ventilation in amyotrophic lateral sclerosis - PMC Non-invasive ventilation (NIV) has become an important cornerstone of symptomatic treatment in amyotrophic lateral sclerosis (ALS), improving survival and ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT07071935

A Pilot Randomized Clinical Trial of Early Ventilation in ... Impact of Early Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis: A multicenter Randomized Controlled Trial. J Neuromuscul Dis ...

8. jwatch.org jwatch.org/na57227/2024/03/18/als-progression-slows-with-use-noninvasive-ventilation

ALS Progression Slows with Use of Noninvasive Ventilation In an observational study, noninvasive respiratory support was associated with disease slowing for ALS patients, regardless of stage or site of onset.

9. publications.ersnet.org publications.ersnet.org/content/erj/54/3/1900261

The optimisation of noninvasive ventilation in amyotrophic ... Noninvasive ventilation (NIV) prolongs survival and quality of life in amyotrophic lateral sclerosis (ALS); however, its benefits depend upon the optimisation ...