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
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/
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
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
