Fukuyama-type congenital muscular dystrophy
Conditions
Interventions
1.0mg/kg of prednisolone every other day for 6 months
Sponsors
Ishigaki Keiko
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients with homozygous 3kb insertion in the fukutin gene and a definitive diagnosis of FCMD 2) Patients aged 5 to 13 at the time of consent 3) Patients whose legal guardian capable of providing informed consent has provided written informed consent upon thorough understanding of the study procedure. Efforts should be made so that the subjects themselves give voluntary assent after having been provided with an explanation according to their ability to understand. 4) Patients who are expected to survive over one year 5) Patients whose motor function has clearly deteriorated by confirming upper extremity function, shuffling distance, or sitting time at 2 points in the pre-observation period and at least 3 months before
Exclusion criteria
Exclusion criteria: 1) History of hypersensitivity to the Prednisolone 2) Patients with infections or systemic mycoses for which there are no effective antibacterial agents 3) Patients with peptic ulcer 4) Patients with tuberculous infections 5) Patients with electrolyte abnormality 6) Patients with thrombosis 7) Patients with a history of acute myocardial infarction 8) Patients with insomnia and panic reaction 9) Patients who received other investigational drugs , study drugs or aspirin within 3 months before the start of administration of the Prednisolone 10) Previous exposure to Prednisolone (Short-term history of steroid treatment as acute treatment for bronchial asthma and short-term history of topical drug treatment not intended for FCMD treatment are excluded.) 11) Patients who are judged by the investigator (or subinvestigator) to be inappropriate for this clinical trial for any reason
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Gross Motor Function Measure | — |
Secondary
| Measure | Time frame |
|---|---|
| Change in muscle mass Physical examination Vital signs Electrocardiogram Echocardiography Ophthalmological examination Clinical examination Immunological test Adverse events | — |
Contacts
Public ContactHiroko Harada
National Center Hospital, National Center of Neurology and Psychiatry
Outcome results
None listed