UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	A multicentre, long-term administration study of KN01 in patients with mitochondrial encephalomyopathy(MELAS)
Title of the study (Brief title)	MELAS-taurine long-term 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 safety of long-term taurine supplementation therapy for prevention of stroke-like episodes in patients with MELAS
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase	Phase III

Assessment
Primary outcomes	safety: adverse events and adverse effects
Key secondary outcomes

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)MELAS patients who participated in the multiceentre trial of KN01 (KN01-MELAS-01:UMIN000011908). (2)Adult patients who agree to participate in the clinical trial and sign the written informed consent document. Children patients who cannot autonomously provide informed consent are required to give informed assent by themselves. Instead, their parents sign the written informed consent document.
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)Ppatients 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	10

Research contact person
Name of lead principal investigator	Yoshihide Sunada
Organization	Kawasaki Medical School
Division name	Neurology
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	Mai Moriyama
Organization	CTD inc.
Division name	Medicinal Products Promotion Division
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	Kawasaki Medical School
Organization
Division
Category of Funding Organization	Other
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 Nagasaki University Hospital Hiroshima University Hospital Nippon Medical School National Center of Neurology and Psychiatry Teikyo University of Science
Name of secondary funｄer(s)

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8日

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2014 Year 10 Month 27 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2014 Year 07 Month 16 Day
Anticipated trial start date	2014 Year 10 Month 02 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Unpublished
URL releasing results
Results
Other related information

Management information
Registered date	2014 Year 10 Month 27 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=R000017746