UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000018235
Receipt number R000021109
Scientific Title Study of outcome measures in muscular dystrophy clinical trials
Date of disclosure of the study information 2015/07/07
Last modified on 2017/07/08 17:05:50

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Basic information

Public title

Study of outcome measures in muscular dystrophy clinical trials

Acronym

MDCTN-OM

Scientific Title

Study of outcome measures in muscular dystrophy clinical trials

Scientific Title:Acronym

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

1st name
Middle name
Last name Eri Takeshita

Organization

National Center Hospital, National Center of Neurology and Psychiatry (NCNP)

Division name

Department of Child Neurology

Zip code


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

1st name
Middle name
Last name Mikiko Shigemori

Organization

Muscular Dystrophy Clinical Trial Network

Division name

Secretariat Office

Zip code


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 or person

Institute

Muscular Dystrophy Clinical Trial Network

Institute

Department

Personal name



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)


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



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


Related information

URL releasing protocol


Publication of results

Partially published


Result

URL related to results and publications


Number of participants that the trial has enrolled


Results


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

2015 Year 04 Month 02 Day

Date of IRB


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


Other

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

Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000021109


Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name