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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000010783
Receipt No. R000012615
Scientific Title Exploratory Clinical Trial of EPI-743 in Japanese MELAS Syndrome Patients
Date of disclosure of the study information 2013/05/23
Last modified on 2013/05/22

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Basic information
Public title Exploratory Clinical Trial of EPI-743 in
Japanese MELAS Syndrome Patients
Acronym EPI-743 Therapy for Japanese MELAS
Scientific Title Exploratory Clinical Trial of EPI-743 in
Japanese MELAS Syndrome Patients
Scientific Title:Acronym EPI-743 Therapy for Japanese MELAS
Region
Japan

Condition
Condition MELAS: mitochondrial
myopathy, encephalopathy, lactic acidosis and stroke-like episodes
Classification by specialty
Medicine in general Cardiology Pneumology
Endocrinology and Metabolism Hematology and clinical oncology Neurology
Pediatrics Radiology
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 1) Pharmacokinetics of EPI-743 in affected Japanese juvenile and adult patients
2) Efficacy and safety evaluation
Basic objectives2 Pharmacokinetics
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Tabulate EPI-743 blood concentrations and calculate fundamental pharmacokinetic parameters
Key secondary outcomes 1) Lactate levels at MRS testing
2) blood and CSF concentrations of lactate and pyruvate
3) QOL values (SF-36, PedsQL)
4) Mitochondrial disease evaluation scale scores (NPMDS or NMDAS)
5) Incidence of abnormal brain waves
6) Incidence of epileptiform seisures
7) Fatigue level
8) Safety evaluation

Base
Study type Interventional

Study design
Basic design Single arm
Randomization Non-randomized
Randomization unit
Blinding Open -no one is blinded
Control Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 Single-dose as Step 1 (Table 1), repeated-dose as Step 2 (Table 2). In Step 1, the drug is administered orally during breakfast, and after confirming no safety problems during 7 days of observation, the trial proceeds to Step 2. In Step 2, the drug is administered orally three times daily, once each during breakfast, lunch, and dinner, for a period of 12 weeks.

Table 1 Step 1: Single-Dose
Children (aged 6-15): Dosage 100mg; No. of Doses, Once; No. of Cases, 5; Observation after dosing, 7 days
Adults (aged 16-59): Dosage 100mg; No. of Doses, Once; No. of Cases, 5; Observation after dosing, 7 days

Table 2 Step2: Repeated-Dose
Children (aged 6-15): Dosage 15mg/kg up to 200 mg; No. of Dosing days, 3 times per day for 12 weeks; No. of Cases, 5; Final Observation, 1 day after last dose
Adults (aged 16-59): Dosage 300mg each time; No. of Dosing days, 3 times per day for 12 weeks; No. of Cases, 5; Final Observation, 1 day after last dose
Interventions/Control_2
Interventions/Control_3
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
6 years-old <=
Age-upper limit
60 years-old >
Gender Male and Female
Key inclusion criteria 1) Patients diagnosed with MELAS syndrome
2) Patients aged 6 to 59 at the time of consent
3) After adequate explanation regarding trial participation, patients who have
adequately understood the explanation and have freely provided written
agreement (or assent) and for whom a legal representative's written
consent has been obtained
4) Patients that have a care provider who can ensure that the trial protocol is
followed
5) Patients for whom the use of general pharmaceuticals can be prohibited
6) Patients for whom the ingestion of foods or beverages fortified with
coenzyme Q10 or vitamin E can be prohibited for over 8 weeks prior to the
administration start and for the duration of the trial.
7) Patients for whom the use of ubidecarenone, idebenone, and dichloroacetate can be prohibited for over 8 weeks prior to the
administration start and for the duration of the trial.
Key exclusion criteria 1) Patients for whom the MELAS syndrome genetic mutation has not been
confirmed
2) Patients who cannot undergo magnetic resonance spectroscopy (MRS)
3) Patients allergic to EPI-743 or to sesame oil
4) Patients with a bleeding tendency, or with abnormal PT/PTT values (INR
1.5 or higher)
5) Patients with AST or ALT values of more than double the institutional limit
6) Patients with renal failure and who require dialysis
7) Patients with fat malabsorption syndrome that impedes drug absorption
8) Patients with pyruvate dehydrogenase (PDH) deficiency or other inborn
error of metabolism
9) Patients who are pregnant or planning to become pregnant
10) Patients with an alcohol dependency or who are illegal drug users
11) Patients with severe heart failure accompanied by lactate acidosis
12) Patients who have received another trial drug or test drug within three
months of the start of dosing of the test drug in this trial
13) Patients with a history of severe drug hypersensitivity
14) Other patients deemed by the researchers to be unsuitable for this trial
Target sample size 10

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Yuichi Goto
Organization National Center Hospital, National Center of Neurology and Psychiatry
Division name Pediatric Neurology
Zip code
Address 4-1-1 Ogawahigashi, Kodaira, Tokyo 187-8551, Japan
TEL
Email

Public contact
Name of contact person
1st name
Middle name
Last name Akemi Tamaura
Organization National Center Hospital, National Center of Neurology and Psychiatry
Division name Clinical Research Unit
Zip code
Address 4-1-1 Ogawahigashi, Kodaira, Tokyo 187-8551, Japan
TEL 042-346-2711
Homepage URL
Email tamaura@ncnp.go.jp

Sponsor
Institute National Center of Neurology and Psychiatry, Japan
Institute
Department

Funding Source
Organization Edison Pharmaceuticals, Inc
Organization
Division
Category of Funding Organization Outside Japan
Nationality of Funding Organization USA

Other related organizations
Co-sponsor Edison Pharmaceuticals, Inc
Name of secondary funder(s) Ministry of Health, Labour and Welfare, Japan

IRB Contact (For public release)
Organization
Address
Tel
Email

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions 独立行政法人 国立精神・神経医療研究センター(東京都)

Other administrative information
Date of disclosure of the study information
2013 Year 05 Month 23 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
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 Open public recruiting
Date of protocol fixation
2013 Year 05 Month 23 Day
Date of IRB
Anticipated trial start date
2013 Year 05 Month 23 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2013 Year 05 Month 22 Day
Last modified on
2013 Year 05 Month 22 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000012615

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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