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

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.

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: Placebo Oral Tablet; Drug: Perampanel

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Diana Kaell, BA; Phone Number: 631-444-7832; Email: diana.kaell@stonybrookmedicine.edu
• Study Contact Backup: Name: Nurcan Gursoy, MD; Phone Number: 631-444-2599; Email: nurcan.gursoy@stonybrookmedicine.edu

Study Locations

• United States
  • New York
    • Stony Brook, New York, United States, 11794-8121
      • Stony Brook University Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: Perampanel; Other Names: Fycompa
Placebo Comparator: placebo; 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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Stony Brook University
• Collaborators: Eisai Inc.
• Investigators: Principal Investigator: Nurcan Gursoy, MD, Stony Brook Medicine Dept. of Neurology

Study record dates

Study Major Dates

• Study Start (Actual): January 12, 2017
• Primary Completion (Actual): January 31, 2023
• Study Completion (Actual): February 8, 2023

Study Registration Dates

• First Submitted: December 15, 2016
• First Submitted That Met QC Criteria: January 11, 2017
• First Posted (Estimate): January 13, 2017

Study Record Updates

• Last Update Posted (Estimate): May 5, 2023
• Last Update Submitted That Met QC Criteria: May 3, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: Eisai-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO