Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)

Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trials

To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis

Study Overview

• Status: Completed
• Conditions: ALS
• Intervention / Treatment: Drug: placebo; Drug: Perampanel

Detailed Description

To evaluate the effect of peramanel for 48 weeks on progression of disease in subjects with ALS, as measured by ALS Functional Rating Scale-Revised (ALSFRS-R)

• Study Type: Interventional
• Enrollment (Actual): 66
• Phase: Phase 2

Contacts and Locations

Study Locations

• Japan
  • Kanagawa, Japan
    • Kitasato University East Hospital
  • Kumamoto, Japan
    • Kumamoto Saishunso National Hospital
  • Nagoya, Japan
    • Nagoya University Hospital
  • Okayama, Japan
    • Okayama University Hospital
  • Sapporo, Japan
    • Hokkaido University Hospital
  • Sendai, Japan
    • Tohoku University Hospital
  • Shiga, Japan
    • Shiga University of Medical Science Hospital
  • Tokyo, Japan, 160-0023
    • Tokyo Medical University
  • Tokyo, Japan
    • The University of Tokyo Hospital
  • Tokyo, Japan
    • Tokyo Metropolitan Neurological Hospital
  • Tsukuba, Japan
    • University of Tsukuba Hospital
  • Yamaguchi, Japan
    • Yamaguchi University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 78 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

[Eligibility Criteria for Interim Registration]

• Patients who are able to submit written informed consent. If patients are duly capable of study consent but are unable to sign by themselves due to aggravation of disease condition, written informed consent can be obtained from a legally authorized representative who can sign on behalf of the patients after confirming the patients' agreement to study participation.
• Patients who are male or female aged 40 years to 78 years at the time of obtaining informed consent
• Patients who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised El Escorial Airlie House diagnostic criteria.
• The sum of the 3 respiratory items of the ALSFRS-R must total 12 points or more
• Patients within 2-year elapsed time period from disease onset at the time of obtaining informed consent
• Patients who can visit study site for out-patient treatment

[Eligibility Criteria for Registration]

Subjects who meet the following criteria in addition to the inclusion criteria for the interim registration

• The progression on score of ALSFRS-R during 12 weeks of observation period must be between -2 and -5
• Patients who has not initiated newly introduced riluzole therapy after starting the observation period. Or those who has not received dose escalation or resumed administration of riluzole therapy after previous down titration or discontinuation
• Patients who has not initiated newly introduced edaravone therapy after starting the observation period
• Patients who are judged to be eligible for continuation of the study by the investigators

[Exclusion Criteria]

• Patients who underwent tracheostomy.
• Patients who experienced non-invasive positive pressure ventilation.
• Patients whose percent-predicted forced vital capacity (%FVC) is ≤80%.
• Patients with progressive bulbar palsy type.
• Patients with cognitive impairment, severe disease in the renal, cardiovascular, or hematological system.
• Patients with hepatic disease.
• Patients with malignant tumor.
• Pregnant women or women with a possibility of becoming pregnant.
• Patients who participated in another clinical study within 12 weeks before starting the observation period.
• Patients who has initiated perampanel therapy in the past or at present.
• Patients who are judged to be ineligible for study entry by the investigators.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Perampanel 4mg; Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks	Drug: Perampanel; 4mg/d or 8mg/d; Other Names: non-competitive AMAP antagonist
Experimental: Perampanel 8mg; Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks	Drug: Perampanel; 4mg/d or 8mg/d; Other Names: non-competitive AMAP antagonist
Placebo Comparator: Placebo; Once daily placebo for control for 48 weeks	Drug: placebo; placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in ALS Functional rating scale	48 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Change in ALS Functional rating scale	12, 24, 36, and 48 weeks
Manual Muscle Test	12, 24, 36, and 48 weeks
Percent-predicted forced vital capacity	12, 24, 36, and 48 weeks

Collaborators and Investigators

• Sponsor: Tokyo Medical University
• Investigators: Study Director: Tomohiro Haga, The University of Tokyo Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 24, 2017
• Primary Completion (Actual): December 23, 2020
• Study Completion (Actual): December 23, 2020

Study Registration Dates

• First Submitted: January 10, 2017
• First Submitted That Met QC Criteria: January 10, 2017
• First Posted (Estimate): January 12, 2017

Study Record Updates

• Last Update Posted (Actual): January 15, 2021
• Last Update Submitted That Met QC Criteria: January 14, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Keywords: perampanel; sporadic ALS
• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: A2016-J000-001

Plan for Individual participant data (IPD)

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

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No