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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000011908
Receipt No. R000013908
Scientific Title A multicentre trial of KN01 in patients with mitochondrial encephalomyopathy (MELAS)
Date of disclosure of the study information 2013/09/30
Last modified on 2019/02/25

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Basic information
Public title A multicentre trial of KN01 in patients with mitochondrial encephalomyopathy (MELAS)
Acronym MELAS-taurine study
Scientific Title A multicentre trial of KN01 in patients with mitochondrial encephalomyopathy (MELAS)
Scientific Title:Acronym MELAS-taurine study
Region
Japan

Condition
Condition motochondrial encephalomyopathy (MELAS)
Classification by specialty
Neurology
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 To investigate the effocacy and safety of taurine supplementation therapy for prevention of stroke-like episodes in patients with MELAS
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2
Developmental phase Phase III

Assessment
Primary outcomes (1) efficacy: 100% respondor rate
(2) safety: adverse events and adverse effects
Key secondary outcomes (1) Japan Mitochondrial Disease Rating Scale
(2) 50% responder rate
(3) the number of abrupt-onset focal neurological deficits
(4) the levels of lactate, pyruvate and taurine in the blood and CSF
(5) brain MRI study
(6) the number of uses intravenous formulation with L-arginine
(7) the number of times in which high intensity lesions detected by brain MRI

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 Prevention
Type of intervention
Medicine
Interventions/Control_1 taurine supplementation
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

Not applicable
Age-upper limit

Not applicable
Gender Male and Female
Key inclusion criteria (1) patients comprehensively diagnosed with MELAS based on clinical manifestations, muscle pathological findings, and genetic testing, as followed by the diagnostic criteria of MELAS
(2) patients with point mutations of either A3243G, T3271C, G3244A, T3258C, or T3291C in the mitochondrial DNA
(3) age at the time of obtaining informed consent , gender , hospitalization or outpatient are unquestioned
(4) patients not using arginine for 78 weeks before obtaining informed consent, or patients continuously using arginine for at least 26 weeks before obtaining informed consent
(5) patients who meet any of the following:
1) patients without arginine use should have stroke-like episodes more than twice in 78 weeks and more than once in 52 weeks before obtaining informed consent (no arginine combination cases )
2) patients with arginine use who meet any of the following, depending on the period of arginine use (arginine combination cases)

If (i) arginine use period within 78 weeks, patients should have stroke-like episodes more than twice in the period of use and at least more than once in 52 weeks before obtaining informed consent
If (ii) arginine use period exceeds 78 weeks, patients should have stroke-like episodes more than twice in 78 weeks and at least more than once in 52 weeks before obtaining informed consent

The definition of stroke-like episodes in the selection criteria , includes any of the following paroxysmal focal neurological symptoms of 1) ~ 6), and
the implementation of the head MRI does not matter .
1) hemiparesis or monoparesis
2) cortical sensory impairment ( sensory extinction )
3) cortical visual impairment (scintilating scotoma, cortical blindness)
4) aphasia
5) apraxia
6) agnosia

(6) patient that has not been treated with oral taurine supplementation
(7) patient capable of being evaluated by clinical findings of stroke-like episodes
Key exclusion criteria (1) patients can not be conducted by head MRI scan because of pacemaker implantation, etc.
(2) patients with severe coma or status epileptics
(3) patients incapable of communication due to dementia, bedridden, etc.,
(4) patients with merger sepsis
(5) patients with severe impairment in cardiac, liver, or renal function
(6) patients in need of systemic administration of steroids for a long period of time (2 weeks or more)
(7) patients with pyruvate in 12 weeks before obtaining informed consent
(8) patients who are pregnant or lactating, or may be pregnant
(9) patients with history of hypersensitivity to the component of study drug
(10) patients with history of drug allergy
(11) patients who participated in clinical trials within 12 weeks before obtaining informed consent
(12) other, patients whom physician or investigator has determined disqualified as participants
Target sample size 15

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Yoshihide Sunada
Organization Kawasaki Medical School
Division name Neurology
Zip code
Address 577 Matsushima,Kurashiki-city,Okayama 701-0192 Japan
TEL 086-462-1111
Email ysunada@med.kawasaki-m.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Mai Moriyama
Organization CTD inc.
Division name Medicinal Products Promotion Division
Zip code
Address 3-3-2, Tsukiji, Chuo-ku, Tokyo
TEL 03-6228-4105
Homepage URL https://www.c-ctd.co.jp/project_sn01/researcher/contents/documents.html
Email taurine@c-ctd.co.jp

Sponsor
Institute Kawasaki Medical School Hospital
Institute
Department

Funding Source
Organization Ministry of Health, Labour and Welfare
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor National Defense Medical College Hospital
Seirei Hamamatsu General Hospital
Fujita Health University School of Medicine
National Hospital Organization Kyoto Medical Center
Hyogo-Chuo National Hospital
Fukuoka University Chikushi Hospital
Kurume University Hospital
Nagasaki University Hospital
Hiroshima University Hospital
Nippon Medical School
National Center of Neurology and Psychiatry
Teikyo University of Science

Name of secondary funder(s)

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 9月13日

Institutions
Institutions 川崎医科大学附属病院/Kawasaki Medical School
防衛医科大学校病院/National Defense Medical College Hospital
聖隷浜松病院/Seirei Hamamatsu General Hospital
藤田保健衛生大学病院/Fujita Health University School of Medicine
独立行政法人国立病院機構京都医療センター/National Hospital Organization Kyoto Medical Center
独立行政法人国立病院機構兵庫中央病院/Hyogo-Chuo National Hospital
福岡大学筑紫病院/Fukuoka University Chikushi Hospital
久留米大学医学部附属病院/Kurume University Hospital
長崎大学病院/Nagasaki University Hospital
広島大学病院/Hiroshima University Hospital

Other administrative information
Date of disclosure of the study information
2013 Year 09 Month 30 Day

Related information
URL releasing protocol https://www.c-ctd.co.jp/project_sn01/researcher/contents/documents.html
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results Look at the attached file, "clinical trial report"
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
2013 Year 07 Month 24 Day
Date of IRB
Anticipated trial start date
2013 Year 10 Month 03 Day
Last follow-up date
2015 Year 01 Month 09 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
2015 Year 03 Month 31 Day

Other
Other related information

Management information
Registered date
2013 Year 09 Month 30 Day
Last modified on
2019 Year 02 Month 25 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000013908

Research Plan
Registered date File name
2017/11/04 Protocol.pdf

Research case data specifications
Registered date File name

Research case data
Registered date File name
2017/11/04 ClinicalTrialReport.pdf


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