UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Development of oral taurine therapy for the treatment of MERRF
Title of the study (Brief title)	Taurine therapy for MERRF
Region	Japan

Condition
Condition	MERRF
Classification by specialty	Neurology
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To evaluate the efficacy of taurine to prevent disease progression of MERRF
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	-Frequency of seizure -Clinical score of cerebellar ataxia
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	Treatment
Type of intervention	Medicine
Interventions/Control_1	Ten patients with MERRF having A8344G mutations in the mitochondrial DNA are enrolled in the study. Oral taurine (12g/d) is administered to the patients for 24 months to evaluate its effect on preventing epileptic symptoms and the progression of cerebellar ataxia.
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	16 years-old <=
Age-upper limit	Not applicable
Gender	Male and Female
Key inclusion criteria	Male and female patients over 16 years old
Key exclusion criteria	none
Target sample size	10

Research contact person
Name of lead principal investigator	Yutaka Ohsawa
Organization	Kawasaki Medical School
Division name	Neurology
Address	577 Matsusima, Kurashiki-city, Okayama 701-0192, Japan
TEL	086-462-1111
Email	yosawa@med.kawasaki-m.ac.jp

Public contact
Name of contact person	Yutaka Ohsawa
Organization	Kawasaki Medical School
Division name	Neurology
Address	577 Matsusima, Kurashiki-city, Okayama 701-0192, Japan
TEL	086-462-1111
Homepage URL
Email	yosawa@med.kawasaki-m.ac.jp

Sponsor
Institute	Department of Neurology, Kawasaki Medical School
Institute
Department

Funding Source
Organization	Department of Neurology, Kawasaki Medical School
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	Department of Biochemistry and Cell Biology, Institute of Development and Aging Sciences, Nippon Medical School
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

Institutions
Institutions	川崎医科大学附属病院(岡山県)

Other administrative information
Date of disclosure of the study information	2012 Year 11 Month 19 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2012 Year 11 Month 19 Day
Anticipated trial start date	2012 Year 12 Month 01 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	2012 Year 11 Month 19 Day
Last modified on	2018 Year 10 Month 09 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000010995