Clinical trial NCT03020797: A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Clinical Trial to Evaluate the Safety and Efficacy of Fycompa in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: NA
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: Stony Brook University
Sponsor class: OTHER
Collaborators: name: Eisai Inc.
First posted: 2017-01-13
Enrollment: 12
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: 9 months
Secondary outcomes:
measure: Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);
timeFrame: 9 months
Other outcomes:
Locations:
city: Stony Brook
country: United States
facility: Stony Brook University Medical Center
state: New York
Study officials:
affiliation: Stony Brook Medicine Dept. of Neurology
name: Nurcan Gursoy, MD
role: PRINCIPAL_INVESTIGATOR
References: