UMIN-CTR Clinical Trial

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
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	2021 Year 05 Month 26 Day

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