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

NCT03020797 | Terminated DMC

• 1 other identifier: Eisai-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events

  9 months

Secondary Outcomes (1)

• Efficacy as measured by change in ALSFRS-R score (ALS functional rating scale-revised);

  9 months

Study Arms (2)

perampanel

EXPERIMENTAL

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

placebo

PLACEBO COMPARATOR

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

Interventions

Perampanel DRUG

Also known as: Fycompa

perampanel

Placebo Oral Tablet DRUG

placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• diagnosis of ALS
• first clinical weakness within past 3 years
• slow vital capacity \>= 60% of predicted within 1 month of treatment
• may be on stable dose of riluzole for at least 30 days, or otherwise agree to not initiate riluzole for duration of the trial
• may be on stable dose of edaravone for at least 30 days, otherwise agree to not initiate edaravone for duration of the trial
• can travel to Stony Brook to receive medical care
• must have a monitor who can be contacted at regular intervals to report on subject's clinical/psychiatric status

You may not qualify if:

• use of tracheostomy or mechanical ventilation within last 3 months
• hepatic insufficiency or abnormal liver function
• renal insufficiency
• clinically significant psychiatric disorder
• active malignancy
• history of HIV, clinically significant chronic hepatitis, or other active infection
• history of stomach or intestinal surgery or condition that could interfere with absorption, distribution, metabolism or secretion of study drug
• history of alcohol or substance abuse within 3 months prior to entry (subjects will be instructed to refrain from alcohol during the study)
• use of strong cytochrome P4503A inhibitors or inducers, anticonvulsants or other drugs known to interact strongly with perampanel.
• pregnancy or lactation
• clinically significant medical condition (other than ALS) that would pose a risk to the subject if they were to participate
• know hypersensitivity to perampanel
• currently participating, or has participated in a study with an investigation or marketed compound within 3 months of entry

Sponsors & Collaborators

• Stony Brook University: lead
• Eisai Inc.: collaborator

Study Sites (1)

Stony Brook University Medical Center

Stony Brook, New York, 11794-8121, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

perampanel

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Nurcan Gursoy, MD

  Stony Brook Medicine Dept. of Neurology

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Assistant Professor

Study Record Dates

• First Submitted: December 15, 2016
• First Posted: January 13, 2017
• Study Start: January 12, 2017
• Primary Completion: January 31, 2023
• Study Completion: February 8, 2023
• Last Updated: May 6, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)