UMIN-CTR Clinical Trial

Basic information
Public title	A study of the factor related to life-prolongation of patients with Duchenne muscular dystrophy
Acronym	A study of the factor related to life-prolongation of DMD patients
Scientific Title	A study of the factor related to life-prolongation of patients with Duchenne muscular dystrophy
Scientific Title:Acronym	A study of the factor related to life-prolongation of DMD patients
Region	Japan

Condition
Condition	Duchenne muscular dystrophy
Classification by specialty	Neurology Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To clarify the factor that prolongs life expectancy of patients with Duchenne muscular dystrophy
Basic objectives2	Others
Basic objectives -Others	Time to death
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Time to death
Key secondary outcomes	Time at loss of ambulation

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	Not applicable
Age-upper limit	Not applicable
Gender	Male
Key inclusion criteria	Genetically confirmed as Duchenne muscular dystrophy No limitation of disease stage. No limitation of general condition. No limitation of organ function (motor function, respiratory failure, cardiac failure, and dysphagia). Patients with written informed consent. In case of unable to make decision because of the age under 16 years or mental retardation, patient with written informed consent by parents or legal guardian.
Key exclusion criteria	Subject meets any of the following criteria is not enrolled in the study. Not confirmed the diagnosis of DMD. Subject considered to be an unsuitable candidate for the study by the Principal investigator or sub-investigator.
Target sample size	300

Research contact person
Name of lead principal investigator	1st name Toshio Middle name Last name Saito
Organization	National Hospital Organization Osaka Toneyama Medical Center
Division name	Child Neurology, Neurology
Zip code	5608552
Address	5-1-1 Toneyama, Toyonaka, Osaka, Japan
TEL	06-6853-2001
Email	saitot@toneyama.go.jp

Public contact
Name of contact person	1st name Toshio Middle name Last name Saito
Organization	National Hospital Organization Osaka Toneyama Medical Center
Division name	Clinical research center
Zip code	5608552
Address	5-1-1 Toneyama, Toyonaka, Osaka, Japan
TEL	06-6853-2001
Homepage URL
Email	saitot@toneyama.go.jp

Sponsor
Institute	National Hospital Organization
Institute
Department

Funding Source
Organization	National Hospital Organization
Organization
Division
Category of Funding Organization	Other
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor	National Center of Neurology and Psychiatry
Name of secondary funder(s)

IRB Contact (For public release)
Organization	National Hospital Organization
Address	2-5-23 Higasigaoka, Meguroku, Tokyo
Tel	03-5712-5075
Email	kenkyu2004@hosp.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

国立病院機構大阪刀根山医療センター（大阪），National Hospital Organization Osaka Toneyama Medical Center (Osaka) 国立病院機構東埼玉病院（埼玉），National Hospital Organization National Higashisaitama Hospital (Saitama) 国立病院機構あきた病院（秋田），National Hospital Organization National Akita Hospital (Akita) 国立病院機構仙台西多賀病院（宮城），National Hospital Organization National Sendai Nishitaga Hospital (Miyagi) 国立病院機構鈴鹿病院（三重），National Hospital Organization National Suzuka Hospital (Mie) 国立病院機構下志津病院（千葉），National Hospital Organization National Shimoshizu Hospital (Chiba) 国立病院機構長良医療センター（岐阜），National Hospital Organization Nagara Medical Center (Gifu) 国立病院機構長崎川棚医療センター（長崎），National Hospital Organization Nagasaki Kawatana Medical Center（Nagasaki） 国立病院機構大牟田病院（福岡）, National Hospital Organization Omuta National Hospital (Fukuoka)

Other administrative information
Date of disclosure of the study information	2015 Year 03 Month 03 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	Completed
Date of protocol fixation	2017 Year 02 Month 09 Day
Date of IRB	2015 Year 01 Month 15 Day
Anticipated trial start date	2015 Year 03 Month 10 Day
Last follow-up date	2020 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete	2020 Year 03 Month 31 Day
Date analysis concluded

Other
Other related information	In this study, we evaluate the following factor; Effects of artificial ventilation and cardioprotection therapy, Influence of modifier, Influence of mutation of Dystrophin gene, Gain of motor function and life prognosis.

Management information
Registered date	2015 Year 03 Month 03 Day
Last modified on	2020 Year 07 Month 12 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019367