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

Not Applicable

Terminated

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Placebo Oral Tablet; Drug: Perampanel

• Registration Number: NCT03020797
• Lead Sponsor: Stony Brook University

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-12-15
• Study First Submitted QC: 2017-01-11
• Study Start: 2017-01-12
• Study First Posted: 2017-01-13
• Primary Completion: 2023-01-31
• Study Completion: 2023-02-08
• Status Verified: 2023-05
• Last Update Submitted: 2023-05-03
• Last Update Posted: 2023-05-06

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 12

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
placebo	Placebo Oral Tablet	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
perampanel	Perampanel	perampanel 2mg QD for 2 weeks perampanel 4mg QD for 2 weeks perampanel 6mg QD for 2 weeks perampanel 8mg QD for 30 weeks

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events	9 months

Secondary Outcome Measures

Name	Time	Method
Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);	9 months

Trial Locations

Locations (1)

Stony Brook University Medical Center; 🇺🇸 Stony Brook, New York, United States