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Name:
UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000017322
Receipt No. R000019733
Scientific Title Clinical features of long-term survivors over 40 years old with Duchenne muscular dystrophy
Date of disclosure of the study information 2015/04/29
Last modified on 2015/04/29

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Basic information
Public title Clinical features of long-term survivors over 40 years old with Duchenne muscular dystrophy
Acronym Clinical features of long-term survivors over 40 years old with Duchenne muscular dystrophy
Scientific Title Clinical features of long-term survivors over 40 years old with Duchenne muscular dystrophy
Scientific Title:Acronym Clinical features of long-term survivors over 40 years old with Duchenne muscular dystrophy
Region
Japan

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

Objectives
Narrative objectives1 To elucidate the clinical condition and treatment of patients with DMD over 40 years old.
Basic objectives2 Others
Basic objectives -Others To explore the key to long survive of DMD patients
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Method and result of gene analysis.
Muscle biopsy findings.
Age of loss of independent ambulation.
Status of cardiomyopathy ( EF of echocardiograph and plasma BNP).
Kinds of agents used for treatment for cardiomyopathy.
History of steroid therapy (used, not used, or now using).
History of spinal fusion operation (done, not done).
Central nervous symptom (mental retardation, autistic disorder, epilepsy, and so on).
Other matter worthy of special mention.
Key secondary outcomes

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
40 years-old <=
Age-upper limit

Not applicable
Gender Male
Key inclusion criteria DMD patients aged over 40 year of age at the time of the search on July 1, 2013. They are recruited based on the Muscular dystrophy wards database of 2012.
Key exclusion criteria DMD patients under 40 years of age at the time of the database search in July 2013 are excluded.
Target sample size 118

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Toshio Saito
Organization National Hospital Organization Toneyama National Hospital
Division name Division of Child Neurology, Department of Neurology
Zip code
Address 5-1-1 Toneyama, Toyonaka, Osaka, 560-8552 Japan
TEL 06-6853-2001
Email saitot@toneyama.go.jp

Public contact
Name of contact person
1st name
Middle name
Last name Toshio Saito
Organization National Hospital Organization Toneyama National Hospital
Division name Division of Child Neurology, Department of Neurology
Zip code
Address 5-1-1 Toneyama, Toyonaka, Osaka, 560-8552 Japan
TEL 06-6853-2001
Homepage URL
Email saitot@toneyama.go.jp

Sponsor
Institute National Hospital Organization
Institute
Department

Funding Source
Organization Ministry of Health, Labour and Welfare of Japan
Organization
Division
Category of Funding Organization
Nationality of Funding Organization

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

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 旭川医療センター Asahikawa Medical Center
八雲病院 Yakumo Hospital
青森病院 Aomori National Hospital
あきた病院 Akita National Hospital
西多賀病院 Nishitaga National Hospital
精神・神経医療研究センター National Center of Neurology and Psychiatry
東埼玉病院East Saitama National Hospital
下志津病院Shimoshizu National Hospital
箱根病院National Hakone Hospital
新潟病院Niigata National Hospital
医王病院Iou National Hospital
長良医療センターNagara Medical Center
鈴鹿病院Suzuka National Hospital
兵庫中央病院Hyogo-cyuo National Hospital
奈良医療センターNara Cedical Center
宇多野病院Utano Hospital
刀根山病院 Toneyama National Hospital
松江病院 Matsue Medical Center
広島西医療センター Hiroshima-Nishi Medical Center
徳島病院Tokushima National Hospital
南九州病院Minami Kyushu National Hospital
大牟田病院 Oomuta Hospital
長崎神経医療センター Nagasaki Kawatana Medical Center
熊本再春荘病院 Kumamoto Saishunso National Hospital
西別府病院 Nishibeppu National
宮崎東病院 Miyazaki Higashi Hospital
沖縄病院 Okinawa National Hospital

Other administrative information
Date of disclosure of the study information
2015 Year 04 Month 29 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
Mean age at study time is 43.6(SD 3.0) years.
Mean age of loss of independent ambulation is 10.6 (SD1.5) years.
Mean age of introduction of artificial ventilation is 24.1 (SD 5.5) years.
Cases with NPPV and those with TIV are comparable.
Mean EF of echocardiograph is 45.0 (SD 15.4) %.
Twenty one cases are administered >=3 agents for cardiomyopathy, while 9 cases no agent.
Seventy three percent of patients preserved oral nutritional function.
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
2013 Year 07 Month 01 Day
Date of IRB
Anticipated trial start date
2013 Year 08 Month 01 Day
Last follow-up date
2013 Year 12 Month 31 Day
Date of closure to data entry
2013 Year 12 Month 31 Day
Date trial data considered complete
2013 Year 12 Month 31 Day
Date analysis concluded
2015 Year 06 Month 30 Day

Other
Other related information Age in July 2013 (years)
Mean (SD) = 43.6(3.0), Range = 40.2-51.0
Number: (age>=40-41), 12; (>=41-43), 18; (>=43-46), 12; (>=46), 13

Age at loss of independent ambulation (years)
Mean (SD) = 10.6(1.5) , Range = 7.0-15.0
Number: (age=7 years), 1; (8), 1; (9), 9; (10), 17; (11), 13; (12), 11; (14), 2; (15), 1

Age at introduction of artificial ventilation (years)
Mean (SD) = 24.1(5.5) , Range = 15.0-40.0

Respiratory method
Number: (NPPV), 27; (TIV), 28

Cardioprotective Agent
Number: (Beta-blocker), 18; (ACEI), 29; (ARB), 4; (Diuretics), 26; (Antiarrhythmic agent), 11; (Digitalis), 18; (Pimobendan), 2

Number of agents for cardiomyopathy
Number: (Number of agents = 0), 9; (1), 13; (2), 12; (3), 8; ( 4), 13

Nutritional method
Number: (Oral nutritional supply), 40; (Oral nutritional supply + gastrostomy feeding), 1; (Gastrostomy feeding), 6; (Tube feeding), 8

Management information
Registered date
2015 Year 04 Month 29 Day
Last modified on
2015 Year 04 Month 29 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000019733

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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