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

Stony Brook University

Status: Terminated

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo Oral Tablet; Drug: Perampanel

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT03020797

Eisai-01

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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12 participants in 2 patient groups, including a placebo group

perampanel

Experimental group

Description:

perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks

Treatment:

Drug: Perampanel

placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo Oral Tablet