UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Open label, efficacy and safety study of long-term 6-methylsulfinylhexyl Isothiocyanate(6-MSITC) administration to Duchenne muscular dystrophy
Title of the study (Brief title)	Long-term 6-MSITC administration to Duchenne muscular dystrophy
Region	Japan

Condition
Condition	Duchenne muscular dystrophy
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Efficacy of 6-MSITC
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1	Exploratory
Trial characteristics_2
Developmental phase	Phase I

Assessment
Primary outcomes	Urinary titin concentration
Key secondary outcomes	Serum CK level, Urinary 8OhDG Serum AST,ALT,BUN and Cr WBC,Hb,Plt Urinary NAG, Urinary beta2MG and Urinary Protein to Cr ratio.

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Food
Interventions/Control_1	6-methylsulfinylhexyl Isothiocyanate; 6-MSITC Oral administraion, 3 times a day 9mg/day for 8 weeks
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	13 years-old <=
Age-upper limit	Not applicable
Gender	Male
Key inclusion criteria	A patient with Duchenne muscular dystrophy confirmed by genetic test and/or immunohistological findings in muscle biopsy DMD patient who is taking a steroid therapy with consistent dose.
Key exclusion criteria	DMD patient who has a severe complication DMD patient who joined other clinical trials within 6 months DMD patient who cannot swallow a capsule
Target sample size	3

Research contact person
Name of lead principal investigator	Hiroyuki Awano
Organization	Kobe University Graduate School of Medicine
Division name	Department of Pediatrics
Address	7-5-1, Kusucnoki-cho, Chuo-ku, Kobe, Japan
TEL	+81-78-382-6090
Email	awahiro@med.kobe-u.ac.jp

Public contact
Name of contact person	Hiroyuki Awano
Organization	Kobe University Graduate School of Medicine
Division name	Department of Pediatrics
Address	7-5-1, Kusucnoki-cho, Chuo-ku, Kobe, Japan
TEL	+81-78-382-6090
Homepage URL
Email	awahiro@med.kobe-u.ac.jp

Sponsor
Institute	Kobe University
Institute
Department

Funding Source
Organization	Productive Aging Laboratory,CO., Ltd.
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization

Other related organizations
Co-sponsor	The Faculty of Rehabilitation, Kobe Gakuin University Laboratory of Molecular Life Science, Foundation for Biomedical Research and Innovation
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

Other administrative information
Date of disclosure of the study information	2018 Year 08 Month 01 Day

Progress
Recruitment status	Terminated
Date of protocol fixation	2018 Year 04 Month 20 Day
Anticipated trial start date	2018 Year 08 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	2018 Year 06 Month 15 Day
Last modified on	2018 Year 12 Month 14 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000037017