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UMIN ID:

Recruitment status Completed
Unique ID issued by UMIN UMIN000020715
Receipt No. R000023900
Scientific Title The efficacy of steroid therapy in the patient with Fukuyama congenital muscular dystrophy
Date of disclosure of the study information 2016/02/01
Last modified on 2020/01/29

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Basic information
Public title The efficacy of steroid therapy in the patient with Fukuyama congenital muscular dystrophy
Acronym The efficacy of steroid therapy in the patient with Fukuyama congenital muscular dystrophy
Scientific Title The efficacy of steroid therapy in the patient with Fukuyama congenital muscular dystrophy
Scientific Title:Acronym The efficacy of steroid therapy in the patient with Fukuyama congenital muscular dystrophy
Region
Japan

Condition
Condition Fukuyama congenital muscular dystrophy
Classification by specialty
Neurology Pediatrics
Classification by malignancy Others
Genomic information NO

Objectives
Narrative objectives1 The efficacy of steroid therapy in patient with Fukuyama congenital muscular dystrophy
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2 Pragmatic
Developmental phase Not applicable

Assessment
Primary outcomes Improvement of motor function by Gross motor function scale after steroid therapy.
Key secondary outcomes 1 To evaluate muscle mass by muscle MRI or CT
2 To evaluate muscle mass by BIA

Base
Study type Interventional

Study design
Basic design Single arm
Randomization Non-randomized
Randomization unit
Blinding Open -no one is blinded
Control Historical
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms 1
Purpose of intervention Treatment
Type of intervention
Medicine
Interventions/Control_1 Predonisolone (PSL) is administered at a dose of 0.25mg/kg every other day for the first 2 weeks. Subsequently, the dose of PSL is increased to 0.5mg/kg every other day.
Three months after the start of therapy, the efficacy is evaluated by gross motor function scales. , If the patient shows clear efficacy, the PSL dose is maintained at 0.5mg/kg every other day. If the efficacy of PSL is not clear, PSL is tapered and discontinued. Six months and one year after the start, the safety and efficacy is also evaluated.
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
3 years-old <=
Age-upper limit
10 years-old >=
Gender Male and Female
Key inclusion criteria FCMD patients followed at the Department of Pediatrics, tTokyo Women's Medical University
Genetically confirmed FCMD patients
FCMD patients between 3 to 10 years of age showing clinical evidence of decreasing muscle function
Key exclusion criteria 1. Patients with infections without effective antibacterial agents or patients with systemic fungal disease [Infections may be aggravated because of immunosuppression.]
2. Patients with peptic ulcer [Peptic ulcer may be aggravated because of a reduction in the function of gastric mucosal barrier.]
3. Patients with psychosis [Psychosis may be aggravated because of a possible effect on central nervous system.]
4. Patients with tuberculous disease [Tuberculous disease may be aggravated because of immunosuppression.]
5. Patients with herpes simplex keratitis [Herpes simplex keratitis may be aggravated because of immunosuppression.]
6. Patients with posterior capsular cataract [Posterior capsular cataract may be aggravated because of a possible effect on the lens fibers.]
7. Patients with glaucoma [Glaucoma may be aggravated because of an increase in intraocular pressure.]
8. Patients with hypertension [Hypertension may be aggravated because of retention of sodium and water.]
9. Patients with electrolyte abnormality [Electrolyte abnormality may be aggravated because of retention of sodium and water.]
10. Patients with thrombosis [Thrombosis may be aggravated because of increased blood clotting.]
11. Patients with the unhealed surgical wound of the viscus [Wound healing may be delayed.]
12. Patients with a history of acute myocardial infarction [Cardiac rupture has been reported to occur.]
13. Patients who has been determined to be inappropriate as a subject by principal investigator.
Target sample size 14

Research contact person
Name of lead principal investigator
1st name Satoru
Middle name
Last name Nagata
Organization Tokyo Women's Medical University
Division name Department of Pediatrics
Zip code 162-8666
Address 8-1 Kawada-cho, Shinjuku, Tokyo
TEL 03-3353-8111
Email nagata.satoru@twmu.ac.jp

Public contact
Name of contact person
1st name Terumi
Middle name
Last name Murakami
Organization Tokyo Women's Medical University
Division name Department of Pediatrics
Zip code 162-8666
Address 8-1 Kawada-cho, Shinjuku, Tokyo
TEL 03-3353-8111
Homepage URL
Email murakami.terumi@twmu.ac.jp

Sponsor
Institute The department of Pediatrics, Tokyo Women's Medical University
Institute
Department

Funding Source
Organization None
Organization
Division
Category of Funding Organization Self funding
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization Tokyo Women's Medical University
Address 8-1 Kawada-cho, Shinjuku, Tokyo
Tel 03-5269-7839
Email krinri.bm@twmu.ac.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

Other administrative information
Date of disclosure of the study information
2016 Year 02 Month 01 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 10
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
2016 Year 01 Month 06 Day
Date of IRB
2016 Year 01 Month 09 Day
Anticipated trial start date
2016 Year 02 Month 01 Day
Last follow-up date
2019 Year 03 Month 31 Day
Date of closure to data entry
Date trial data considered complete
2019 Year 03 Month 31 Day
Date analysis concluded

Other
Other related information

Management information
Registered date
2016 Year 01 Month 24 Day
Last modified on
2020 Year 01 Month 29 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023900

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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