Amyotrophic Lateral Sclerosis
Conditions
Brief summary
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.
Interventions
DRUGPerampanel
DRUGPlacebo Oral Tablet
Sponsors
Eisai Inc.
Stony Brook University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. diagnosis of ALS 2. first clinical weakness within past 3 years 3. slow vital capacity \>= 60% of predicted within 1 month of treatment 4. may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial 5. may be on stable dose of edaravone for at least 30 days, otherwise agree to not initiate edaravone for duration of the trial 6. can travel to Stony Brook to receive medical care 7. must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status
Exclusion criteria
1. use of tracheostomy or mechanical ventilation within last 3 months 2. hepatic insufficiency or abnormal liver function 3. renal insufficiency 4. clinically significant psychiatric disorder 5. active malignancy 6. history of HIV, clinically significant chronic hepatitis, or other active infection 7. history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug 8. history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study) 9. use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel. 10. pregnancy or lactation 11. clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate 12. know hypersensitivity to perampanel 13. currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events | 9 months |
Secondary
| Measure | Time frame |
|---|---|
| Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised); | 9 months |
Countries
United States
Outcome results
None listed