Clinical trial NCT03019419: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: ALS
Keywords: sporadic ALS
perampanel
Lead sponsor: Tokyo Medical University
Sponsor class: OTHER
First posted: 2017-01-12
Enrollment: 66
Sex: ALL
Minimum age (years): 40
Maximum age (years): 78
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)

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.

Primary outcomes:
measure: Change in ALS Functional rating scale
timeFrame: 48 weeks
Secondary outcomes:
measure: Change in ALS Functional rating scale
timeFrame: 12, 24, 36, and 48 weeks
measure: Manual Muscle Test
timeFrame: 12, 24, 36, and 48 weeks
measure: Percent-predicted forced vital capacity
timeFrame: 12, 24, 36, and 48 weeks
Other outcomes:
Locations:
city: Kanagawa
country: Japan
facility: Kitasato University East Hospital
city: Kumamoto
country: Japan
facility: Kumamoto Saishunso National Hospital
city: Nagoya
country: Japan
facility: Nagoya University Hospital
city: Okayama
country: Japan
facility: Okayama University Hospital
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
city: Sendai
country: Japan
facility: Tohoku University Hospital
city: Shiga
country: Japan
facility: Shiga University of Medical Science Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical University
city: Tokyo
country: Japan
facility: The University of Tokyo Hospital
city: Tokyo
country: Japan
facility: Tokyo Metropolitan Neurological Hospital
city: Tsukuba
country: Japan
facility: University of Tsukuba Hospital
city: Yamaguchi
country: Japan
facility: Yamaguchi University Hospital
Study officials:
affiliation: The University of Tokyo Hospital
name: Tomohiro Haga
role: STUDY_DIRECTOR
References: