Fukuyama congenital muscular dystrophy
Conditions
Interventions
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) .
Sponsors
The department of Pediatrics, Tokyo Women's Medical University
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvement of motor function by Gross motor function scale after steroid therapy. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1 To evaluate muscle mass by muscle MRI or CT 2 To evaluate muscle mass by BIA | — |
Countries
Japan
Contacts
Public ContactTerumi Murakami
Tokyo Women's Medical University Department of Pediatrics
Outcome results
None listed