UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Study of outcome measures in muscular dystrophy clinical trials
Title of the study (Brief title)	MDCTN-OM
Region	Japan

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

Objectives
Narrative objectives1	To create reliable and valid outcome measures of motor function in Duchenne muscular dystrophy clinical trials alternative to 6-minute walk test.
Basic objectives2	Others
Basic objectives -Others	Reliability Validity
Trial characteristics_1	Others
Trial characteristics_2	Others
Developmental phase	Not applicable

Assessment
Primary outcomes	Test-retest reliability Concurrent validity Minimal clinically important differences; MCIDs
Key secondary outcomes	Relationship between subjective estimate and motor function test.

Base
Study type	Observational

Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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	12 years-old >
Gender	Male
Key inclusion criteria	a) Patients with Duchenne muscular dystrophy confirmed by dystrophin gene analysis and/or muscle pathology. b) Sex; males. c) Age; 6 to <12 years old. d) Patients who are able to stand up from the floor and complete 6-minute walk test with minimal distance of at least 75m (allowing wheelchair user in his daily life). e) Patients receiving steroid for a minimum of 6 months immediately prior to baseline evaluation, with no significant change in dosing regimen or total daily dosage for a minimum of 3 months immediately prior to baseline evaluation when used. Patients must meet all of the inclusion criteria a)-e) and able to give informed assent (where appropriate) and/or consent in writing signed by the patients and/or legally authorized representative for this study.
Key exclusion criteria	a) Patients with obvious heart failure and respiratory failure in the judgement of principal investigator or sub-investigator. b) Patients with persistent artificial ventilation (excepting the use of Noninvasive Positive Pressure Ventilation (NPPV) at night). c) Patients with behavioral problems interfered with motor function test such as intellectual disability, autism, attention-deficit hyperactivity disorder in the judgement of principal investigator or sub-investigator. d) Patients who were considered inappropriate for this study by principal investigator or sub-investigator for other reason. e) Patients who are part of other clinical trials now.
Target sample size	50

Research contact person
Name of lead principal investigator	Eri Takeshita
Organization	National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
Division name	Department of Child Neurology
Address	4-1-1 Ogawahigashi-cho, Kodaira, Tokyo 187-8551, Japan
TEL	042-341-2711
Email	erit@ncnp.go.jp

Public contact
Name of contact person	Mikiko Shigemori
Organization	Muscular Dystrophy Clinical Trial Network
Division name	Secretariat Office
Address	4-1-1 Ogawahigashi-cho, Kodaira, Tokyo 187-8551, Japan
TEL	042-341-2711
Homepage URL	http://www.mdctn.jp/
Email	shigemori@ncnp.go.jp

Sponsor
Institute	Muscular Dystrophy Clinical Trial Network
Institute
Department

Funding Source
Organization	Taiho Pharmaceutical Co. Ltd. Daiichi Sankyo Company, Limited.
Organization
Division
Category of Funding Organization	Profit organization
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	National Center of Neurology and Psychiatry (NCNP)
Name of secondary funｄer(s)	National Center of Neurology and Psychiatry (NCNP) Japan Agency for Medical Research and Development (AMED)

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	国立病院機構八雲病院（北海道） 国立病院機構東埼玉病院（埼玉県） 国立病院機構下志津病院（千葉県） 東京女子医科大学病院小児科（東京都） 国立精神・神経医療研究センター病院（東京都） 信濃医療福祉センター（長野県） 国立病院機構医王病院（石川県） 国立病院機構長良医療センター（岐阜県） 滋賀県立小児保健医療センター（滋賀県） 国立病院機構宇多野病院（京都府） 国立病院機構刀根山病院（大阪府） 兵庫医科大学病院（兵庫県） 鳥取大学医学部附属病院（鳥取県） 文佑会原病院（福岡県） 国立病院機構大牟田病院（福岡県）

Other administrative information
Date of disclosure of the study information	2015 Year 07 Month 07 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 04 Month 02 Day
Anticipated trial start date	2015 Year 07 Month 08 Day
Last follow-up date	2017 Year 03 Month 31 Day
Date of closure to data entry	2017 Year 03 Month 31 Day
Date trial data considered complete	2017 Year 03 Month 31 Day
Date analysis concluded	2017 Year 03 Month 31 Day

Related information
URL releasing protocol
Publication of results	Partially published
URL releasing results
Results
Other related information	Prospective observational study in operation.

Management information
Registered date	2015 Year 07 Month 07 Day
Last modified on	2017 Year 07 Month 08 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000021109