UMIN-CTR Clinical Trial

Basic information
Public title	Perampanel for sporadic amyotrophic lateral sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trial
Acronym	Perampanel for sporadic amyotrophic lateral sclerosis (ALS)
Scientific Title	Perampanel for sporadic amyotrophic lateral sclerosis (ALS): A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Phase 2 Trial
Scientific Title:Acronym	Perampanel for sporadic amyotrophic lateral sclerosis (ALS)
Region	Japan

Condition
Condition	Sporadic amyotrophic lateral sclerosis
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To investigate the safety and the efficacy of perampanel in patients with sporadic amyotrophic lateral sclerosis
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase II

Assessment
Primary outcomes	To evaluate the effect of perampanel on progression of the disease in subjects with ALS, as measured by the ALS Functional Rating Scale -Revised (ALSFRS-R). [Time Frame: 48 weeks.] [Designated as safety issue: Yes]
Key secondary outcomes	Change in ALSFRS-R slope [Time Frame: Every 12 weeks.] [Designated as safety issue: Yes] Manual Muscle Test (MMT) [Time Frame: Every 12 weeks.] [Designated as safety issue: Yes] Percent-predicted forced vital capacity (%FVC) [Time Frame: Every 12 weeks.] [Designated as safety issue: Yes]

Base
Study type	Interventional

Study design
Basic design	Parallel
Randomization	Randomized
Randomization unit	Individual
Blinding	Double blind -all involved are blinded
Control	Placebo
Stratification	YES
Dynamic allocation	YES
Institution consideration	Institution is not considered as adjustment factor.
Blocking	NO
Concealment	Central registration

Intervention
No. of arms	3
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Once daily perampanel with dose-escalation tolerable from 2mg to 4mg for 48 weeks (Interventions)
Interventions/Control_2	Once daily perampanel with dose-escalation tolerable from 2mg to 8mg for 48 weeks (Interventions).
Interventions/Control_3	Once daily placebo for 48 weeks (Control)
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	40 years-old <=
Age-upper limit	78 years-old >=
Gender	Male and Female
Key inclusion criteria	[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 [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 3 months 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
Key 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.
Target sample size	60

Research contact person
Name of lead principal investigator	1st name Hitoshi Middle name Last name Aizawa
Organization	Tokyo Medical University Hospital
Division name	Department of Neurology
Zip code	160-0023
Address	Nishishinjuku 6-7-1, Shinjuku-ku, Tokyo, 160-0023, Japan
TEL	03-3342-6111
Email	haizawa@tokyo-med.ac.jp

Public contact
Name of contact person	1st name Haruhisa Middle name Last name Kato
Organization	Tokyo Medical University Hospital
Division name	Department of Neurology
Zip code	160-0023
Address	Nishishinjuku 6-7-1, Shinjuku-ku, Tokyo, 160-0023, Japan
TEL	03-3342-6111
Homepage URL
Email	h-kato@tokyo-med.ac.jp

Sponsor
Institute	Department of Neurology, Tokyo Medical University
Institute
Department

Funding Source
Organization	Japan Medical Association
Organization
Division
Category of Funding Organization	Government offices of other countries
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s)	Perampanel was given by Eizai.Co.

IRB Contact (For public release)
Organization	Tokyo Medical University Hospital
Address	Nishishinjuku 6-7-1, Shinjuku-ku, Tokyo, 160-0023, Japan
Tel	03-3342-6111
Email	yuki-m@tokyo-med.ac.jp

Secondary IDs
Secondary IDs	YES
Study ID_1	NCT03019419
Org. issuing International ID_1	ClinicalTrials.gov
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions

Institutions

東京医科大学病院（東京都）Tokyo Medical University Hospital (Tokyo) 筑波大学附属病院（茨城県）University of Tsukuba Hospital (Ibaraki) 北里大学東病院（神奈川県）Kitasato University East Hospital (Kanagawa) 岡山大学病院（岡山県）Okayama University Hospital (Okayama) 北海道大学病院（北海道）Hokkaido University Hospital (Hokkaido) 東京都立神経病院（東京都）Tokyo Metropolitan Neurological Hospital (Tokyo) 滋賀医科大学医学部附属病院（滋賀県）Shiga University of Medical Science Hospital (Shiga) 東北大学病院（宮城県）Tohoku University Hospital (Miyagi) 山口大学医学部附属病院（山口県）Yamaguchi University Hospital (Yamaguchi) 名古屋大学医学部附属病院（愛知県）Nagoya University Hospital (Aichi) 独立行政法人国立病院機構熊本再春荘病院（熊本県）Kumamoto Saishunso National Hospital (Kumamoto) 東京大学医学部附属病院（東京都）The University of Tokyo Hospital

Other administrative information
Date of disclosure of the study information	2017 Year 02 Month 01 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled	66
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Completed
Date of protocol fixation	2017 Year 01 Month 10 Day
Date of IRB	2017 Year 04 Month 10 Day
Anticipated trial start date	2017 Year 04 Month 24 Day
Last follow-up date	2020 Year 12 Month 23 Day
Date of closure to data entry	2020 Year 12 Month 23 Day
Date trial data considered complete	2020 Year 12 Month 23 Day
Date analysis concluded	2021 Year 12 Month 31 Day

Other
Other related information

Management information
Registered date	2017 Year 01 Month 11 Day
Last modified on	2021 Year 01 Month 14 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000029464