UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000030562 |
|---|---|
| Receipt number | R000034885 |
| Scientific Title | The natural history study of ambulatory patients with Duchenne muscular dystrophy |
| Date of disclosure of the study information | 2017/12/25 |
| Last modified on | 2023/07/01 00:53:21 |
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Basic information
Public title
The natural history study of ambulatory patients with Duchenne muscular dystrophy
Acronym
NH-ambulatory DMD
Scientific Title
The natural history study of ambulatory patients with Duchenne muscular dystrophy
Scientific Title:Acronym
NH-ambulatory DMD
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 investigate the natural history data of motor function in ambulatory patients with Duchenne muscular dystrophy (DMD) receiving standard care in Japan until they become non-ambulatory.
Basic objectives2
Others
Basic objectives -Others
This study is aimed to select avairable outcome measure of motor function test for drug development of DMD in the future.
Trial characteristics_1
Others
Trial characteristics_2
Others
Developmental phase
Not applicable
Assessment
Primary outcomes
Motor function test
Key secondary outcomes
Activities of daily living (ADL)
Patient reported outcome (PRO)
Acceleration meter/activity meter
Subject demographics
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
| 5 | years-old | <= |
Age-upper limit
| Not applicable |
Gender
Male
Key inclusion criteria
1) Patients with a dystrophin abnormality proven by dystrophin gene testing or muscle pathology and a definitive diagnosis of DMD
2) Sex: male
3) Aged 5 or older (at the time of informed consent)
4) Able to rise from the floor and walk independently at baseline
5) Patients receiving oral glucocorticosteroids for at least 6 months before baseline without change in dosage or dosing regimen within 3 months and having stable symptoms
6) If participating in another clinical study, at least 6 months has passed since starting this study at baseline
Key exclusion criteria
1) Patients with clinically apparent symptoms of cardiac failure and/or respiratory failure as determined by the investigators
2) Patients continuously using a ventilator (use of non-invasive positive-pressure ventilation [NPPV] during sleep is allowed)
3) Patients with behavior abnormalities including intellectual disability, autistic tendency, and attention deficit hyperactivity disorder that, in the opinion of the investigators, could prevent to conduct the assessment of motor function
4) Other patients as determined by the investigators to be ineligible to participate in this study
5) Patients participating in another clinical trial at baseline
Target sample size
80
Research contact person
Name of lead principal investigator
| 1st name | Eri |
| Middle name | |
| Last name | Takeshita |
Organization
National Center Hospital, National Center of Neurology and Psychiatry (NCNP)
Division name
Department of Child Neurology
Zip code
1878551
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 | Mikiko |
| Middle name | |
| Last name | Shigemori |
Organization
Muscular Dystrophy Clinical Trial Network (MDCTN)
Division name
Secretariat Office
Zip code
1878551
Address
4-1-1 Ogawahigashi-cho, Kodaira, Tokyo 187-8551, Japan
TEL
042-341-2711
Homepage URL
shigemori@ncnp.go.jp
Sponsor or person
Institute
National Center of Neurology and Psychiatry (NCNP)
Institute
Department
Personal name
Funding Source
Organization
National Center of Neurology and Psychiatry (NCNP)
Organization
Division
Category of Funding Organization
Self funding
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
pharmaceutical company
IRB Contact (For public release)
Organization
National Center of Neurology and Psychiatry (NCNP)
Address
4-1-1 Ogawahigashi-cho, Kodaira, Tokyo 187-8551, Japan
Tel
042-341-2711
ml_rinrijimu@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
国立病院機構鈴鹿病院（三重県）
国立病院機構八雲病院（北海道）
熊本大学医学部附属病院（熊本県）
国立病院機構刀根山病院（大阪府）
国立病院機構宇多野病院（京都府）
国立病院機構東埼玉病院（埼玉県）
国立病院機構大牟田病院（福岡県）
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）
岐阜大学医学部附属病院（岐阜県）
信州大学医学部附属病院（長野県）
東京女子医科大学病院（東京都）
兵庫医科大学病院（兵庫県）
長崎大学病院（長崎県）
名古屋市立大学病院（愛知県）
国立精神・神経医療研究センター病院（東京都）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 12 | Month | 25 | 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
33
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
| 2017 | Year | 09 | Month | 25 | Day |
Date of IRB
| 2017 | Year | 10 | Month | 06 | Day |
Anticipated trial start date
| 2018 | Year | 01 | Month | 25 | Day |
Last follow-up date
| 2025 | Year | 09 | Month | 30 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Prospective observational study now underway.
Management information
Registered date
| 2017 | Year | 12 | Month | 25 | Day |
Last modified on
| 2023 | Year | 07 | Month | 01 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034885
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| Registered date | File name |
| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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| Registered date | File name |
