UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000046838 |
|---|---|
| Receipt number | R000053409 |
| Scientific Title | Natural history study of Duchenne muscular dystrophy focusing on upper limb motor function |
| Date of disclosure of the study information | 2022/02/07 |
| Last modified on | 2025/01/08 12:25:03 |
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Basic information
Public title
Natural history study of Duchenne muscular dystrophy focusing on upper limb motor function
Acronym
Natural history study of upper limb function of Duchenne muscular dystrophy
Scientific Title
Natural history study of Duchenne muscular dystrophy focusing on upper limb motor function
Scientific Title:Acronym
Natural history study of Duchenne muscular dystrophy
Region
| Japan |
Condition
Condition
Duchenne muscular dystrophy
Classification by specialty
| Neurology | Pediatrics | Rehabilitation medicine |
Classification by malignancy
Others
Genomic information
YES
Objectives
Narrative objectives1
The purpose of this survey is to obtain natural history data of upper limb motor function in DMD patients.
Basic objectives2
Others
Basic objectives -Others
The purpose is to select appropriate evaluation indicators that can be used in drug development in the future.
Trial characteristics_1
Exploratory
Trial characteristics_2
Explanatory
Developmental phase
Not applicable
Assessment
Primary outcomes
We will perform a multifaceted evaluation of upper limb function obtained from natural history.
Key secondary outcomes
We will Evaluate ADL, QOL, and swallowing function.
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
| 8 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male
Key inclusion criteria
1. A dystrophin abnormality has been proved by dystrophin genetic test or myopathology, and a definitive diagnosis of DMD has been made.
2. Upper limb motor dysfunction classification at baseline 2007 grade 12 or lower
3. If you are taking steroids, start using them at least 6 months before baseline, and within 3 months, the dosage and administration of steroids will not change and the symptoms will be stable.
4. If you have participated in clinical trials / clinical trials, more than 6 months have passed since the end of study / investigational drug administration at baseline.
Key exclusion criteria
1. The principal investigator or the research coordinator determined that he / she had behavioral abnormalities such as intellectual disability, autistic tendency, and attention deficit hyperactivity disorder that interfered with the implementation of motor function evaluation.
2. Severe heart failure or respiratory failure makes it difficult to visit an outpatient clinic
3. In addition, the principal investigator or the research coordinator judged that it was inappropriate as the subject of this research.
4. Participating in clinical trials / clinical trials at baseline
Target sample size
100
Research contact person
Name of lead principal investigator
| 1st name | Takahashi |
| Middle name | |
| Last name | Hara |
Organization
National Center of Neurology and Psychiatry
Division name
Department of Rehabilitation Medicine
Zip code
1870031
Address
Ogawahigashimachi4-1-1, Tokyo
TEL
042-341-2711
mizuno.katsuhiro@ncnp.go.jp
Public contact
Name of contact person
| 1st name | Takatoshi |
| Middle name | |
| Last name | Hara |
Organization
National Center of Neurology and Psychiatry
Division name
Department of Rehabilitation Medicine
Zip code
1870031
Address
Ogawahigashimachi4-1-1, Tokyo
TEL
042-341-2711
Homepage URL
t_hara1019@ncnp.go.jp
Sponsor or person
Institute
National Center of Neurology and Psychiatry
Institute
Department
Personal name
Funding Source
Organization
Nippon Shinyaku
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Osaka Toneyama Medical Center, Higashisaitama National Hospital, Matsumoto Medical Center, National Hospital Organization Suzuka National Hospital, National Hospital Organization Shimoshizu National Hospital, National Hospital Organization Akita Hospital, National Hospital Organization Hakone National Hospital, National Hospital Organization Nagara Medical Center, Nara Medical University Hospital, Nagano Children's Hospital, Nagasaki University Hospital, NHO Iou National Hospital
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Institutional Review Board
Address
Ogawahigashimachi4-1-1, Tokyo
Tel
042-346-2190
rinri@ncnp.go.jp
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
国立精神・神経医療研究センター（東京都）、国立病院機構大阪刀根山医療センター、国立病院機構東埼玉病院、国立病院機構まつもと医療センター、国立病院機構鈴鹿病院、国立病院機構下志津病院、国立病院機構あきた病院、国立病院機構箱根病院、国立病院機構長良医療センター、奈良県立医科大学附属病院、長野県立こども病院、長崎大学病院、国立病院機構医王病院
Other administrative information
Date of disclosure of the study information
| 2022 | Year | 02 | Month | 07 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
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
No longer recruiting
Date of protocol fixation
| 2021 | Year | 12 | Month | 15 | Day |
Date of IRB
| 2022 | Year | 06 | Month | 07 | Day |
Anticipated trial start date
| 2022 | Year | 08 | Month | 01 | Day |
Last follow-up date
| 2027 | Year | 03 | Month | 01 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Recruitment closed
Management information
Registered date
| 2022 | Year | 02 | Month | 05 | Day |
Last modified on
| 2025 | Year | 01 | Month | 08 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000053409
