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

NCT ID: NCT03020797

Last Updated: 2023-05-06

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: NA
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-12
• Study Completion Date: 2023-02-08

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: EXPERIMENTAL

Perampanel

Intervention Type: DRUG

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

Group Type: PLACEBO_COMPARATOR

Placebo Oral Tablet

Intervention Type: DRUG

Interventions

Perampanel

Intervention Type: DRUG

Placebo Oral Tablet

Intervention Type: DRUG

Other Intervention Names

Fycompa

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Eisai Inc.

INDUSTRY

Sponsor Role: collaborator

Stony Brook University

OTHER

Sponsor Role: lead

Responsible Party

Nurcan Gursoy

Clinical Assistant Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Nurcan Gursoy, MD

Role: PRINCIPAL_INVESTIGATOR

Stony Brook Medicine Dept. of Neurology

Locations

Stony Brook University Medical Center

Stony Brook, New York, United States

Countries

United States

Other Identifiers

Eisai-01

Identifier Type: -

Identifier Source: org_study_id