A Clinical Trial to Evaluate the Safety and Efficacy of F... | NCT03020797

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

NCT03020797

Sponsor

Stony Brook University

Status

Terminated

Last Update Posted

May 6, 2023Actual

Enrollment

12Actual

Phase

Not Applicable

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Perampanel
Placebo Oral Tablet

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2023

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

1) Enrolment below study target and 2) too many subjects had early termination due to disease progression.

Expanded Access Info

Start Date

Jan 12, 2017Actual

Primary Completion Date

Jan 31, 2023Actual

Completion Date

Feb 8, 2023Actual

First Submitted Date

Dec 15, 2016

First Submission Date that Met QC Criteria

Jan 11, 2017

First Posted Date

Jan 13, 2017Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 3, 2023

Last Update Posted Date

May 6, 2023Actual

Name	Class
Eisai Inc.	INDUSTRY

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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 provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Not Applicable

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

12Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Perampanel	Drug		perampanel	Fycompa
Placebo Oral Tablet

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

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

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

80 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

ID	Term
D013118	Spinal Cord Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D016472	Motor Neuron Disease