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Name:
UMIN ID:

Recruitment status Enrolling by invitation
Unique ID issued by UMIN UMIN000030562
Receipt No. 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 2018/06/26

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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
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 (MDCTN)
Division name Secretariat Office
Zip code
Address 4-1-1 Ogawahigashi-cho, Kodaira, Tokyo 187-8551, Japan
TEL 042-341-2711
Homepage URL
Email shigemori@ncnp.go.jp

Sponsor
Institute National Center of Neurology and Psychiatry (NCNP)
Institute
Department

Funding Source
Organization Japan Agency for Medical Research and Development (AMED)
Organization
Division
Category of Funding Organization Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funder(s) pharmaceutical company

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
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 Enrolling by invitation
Date of protocol fixation
2017 Year 09 Month 25 Day
Date of IRB
Anticipated trial start date
2018 Year 01 Month 25 Day
Last follow-up date
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
2018 Year 06 Month 26 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034885

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


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