UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000020715 |
|---|---|
| Receipt number | 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 | 2021/05/26 00:41:56 |
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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 of0.5mg/kg on alternate day (0.25mg/kg/d) for the first 2 weeks. Subsequently, the dose of PSL is increased to 1.0mg/kg on alternate days (0.5mg/kg/d) .
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 1.0mg/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
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
murakami.terumi@twmu.ac.jp
Sponsor or person
Institute
The department of Pediatrics, Tokyo Women's Medical University
Institute
Department
Personal name
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
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
| 2021 | Year | 05 | Month | 26 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000023900
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