UMIN-CTR Clinical Trial

Unique ID issued by UMIN UMIN000017322
Receipt number 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 10:30:27

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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 or person

Institute

National Hospital Organization

Institute

Department

Personal name



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

Value
https://center6.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