A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Study Overview
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Brief Summary
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This is a pilot trial to test perampanel (Fycompa; Eisai, Inc.) in ALS patients. The investigators will focus on safety and preliminary signs of efficacy. Perampanel is approved by the FDA for treatment of seizures in patients with epilepsy. In this study, perampanel will be used off-label for adults with ALS at an oral medication dose on the low end of the recommended dose range for epilepsy. This study will consist of two treatments arms: perampanel and matching placebo randomized at a 1:1 ratio. Subjects will receive medication for 9 months.

Official Title
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A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: Perampanel
* Drug: Placebo Oral Tablet


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: diagnosis of ALS first clinical weakness within past 3 years slow vital capacity >= 60% of predicted within 1 month of treatment may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial may be on stable dose of edaravone for at least 30 days, otherwise agree to not initiate edaravone for duration of the trial can travel to Stony Brook to receive medical care must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status Exclusion Criteria: use of tracheostomy or mechanical ventilation within last 3 months hepatic insufficiency or abnormal liver function renal insufficiency clinically significant psychiatric disorder active malignancy history of HIV, clinically significant chronic hepatitis, or other active infection history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study) use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel. pregnancy or lactation clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate know hypersensitivity to perampanel currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: perampanel<br>perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks | Drug: Perampanel<br> <br> * Other names: Fycompa;|
| Placebo Comparator: placebo<br>placebo 1 tablet QD for 2 weeks placebo 2 tablets QD for 2 weeks placebo 3 tablets QD for 2 weeks placebo 4 tablets QD for 2 weeks | Drug: Placebo Oral Tablet<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of treatment-emergent adverse events |  | 9 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised); |  | 9 months |