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Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)

Primary Purpose

ALS

Status
Completed
Phase
Phase 2
Locations
Japan
Study Type
Interventional
Intervention
Perampanel
placebo
Sponsored by
Tokyo Medical University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for ALS focused on measuring sporadic ALS, perampanel

Eligibility Criteria

40 Years - 78 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

[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.

Sites / Locations

  • Kitasato University East Hospital
  • Kumamoto Saishunso National Hospital
  • Nagoya University Hospital
  • Okayama University Hospital
  • Hokkaido University Hospital
  • Tohoku University Hospital
  • Shiga University of Medical Science Hospital
  • Tokyo Medical University
  • The University of Tokyo Hospital
  • Tokyo Metropolitan Neurological Hospital
  • University of Tsukuba Hospital
  • Yamaguchi University Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Perampanel 4mg

Perampanel 8mg

Placebo

Arm Description

Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks

Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks

Once daily placebo for control for 48 weeks

Outcomes

Primary Outcome Measures

Change in ALS Functional rating scale

Secondary Outcome Measures

Change in ALS Functional rating scale
Manual Muscle Test
Percent-predicted forced vital capacity

Full Information

First Posted
January 10, 2017
Last Updated
January 14, 2021
Sponsor
Tokyo Medical University
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1. Study Identification

Unique Protocol Identification Number
NCT03019419
Brief Title
Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)
Official Title
Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trials
Study Type
Interventional

2. Study Status

Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
April 24, 2017 (Actual)
Primary Completion Date
December 23, 2020 (Actual)
Study Completion Date
December 23, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Tokyo Medical University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ALS
Keywords
sporadic ALS, perampanel

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
66 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Perampanel 4mg
Arm Type
Experimental
Arm Description
Once daily 4mg of perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks
Arm Title
Perampanel 8mg
Arm Type
Experimental
Arm Description
Once daily 8mg of perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Once daily placebo for control for 48 weeks
Intervention Type
Drug
Intervention Name(s)
Perampanel
Other Intervention Name(s)
non-competitive AMAP antagonist
Intervention Description
4mg/d or 8mg/d
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
placebo
Primary Outcome Measure Information:
Title
Change in ALS Functional rating scale
Time Frame
48 weeks
Secondary Outcome Measure Information:
Title
Change in ALS Functional rating scale
Time Frame
12, 24, 36, and 48 weeks
Title
Manual Muscle Test
Time Frame
12, 24, 36, and 48 weeks
Title
Percent-predicted forced vital capacity
Time Frame
12, 24, 36, and 48 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
78 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
[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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Tomohiro Haga
Organizational Affiliation
The University of Tokyo Hospital
Official's Role
Study Director
Facility Information:
Facility Name
Kitasato University East Hospital
City
Kanagawa
Country
Japan
Facility Name
Kumamoto Saishunso National Hospital
City
Kumamoto
Country
Japan
Facility Name
Nagoya University Hospital
City
Nagoya
Country
Japan
Facility Name
Okayama University Hospital
City
Okayama
Country
Japan
Facility Name
Hokkaido University Hospital
City
Sapporo
Country
Japan
Facility Name
Tohoku University Hospital
City
Sendai
Country
Japan
Facility Name
Shiga University of Medical Science Hospital
City
Shiga
Country
Japan
Facility Name
Tokyo Medical University
City
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
The University of Tokyo Hospital
City
Tokyo
Country
Japan
Facility Name
Tokyo Metropolitan Neurological Hospital
City
Tokyo
Country
Japan
Facility Name
University of Tsukuba Hospital
City
Tsukuba
Country
Japan
Facility Name
Yamaguchi University Hospital
City
Yamaguchi
Country
Japan

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Perampanel for Sporadic Amyotrophic Lateral Sclerosis (ALS)

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