Fukuyama-type congenital muscular dystrophy
Conditions
Interventions
For patients with FCMD caused by a homozygous
1.0mg/kg of prednisolone every other day for 6 months
For patients with FCMD caused by a hetrozygous
1.0mg/kg of prednisolone every other day for 12 mo
Sponsors
Ishigaki Keiko
Eligibility
Inclusion criteria
Inclusion criteria: For patients with FCMD caused by a homozygous 1) Patients who completed the TWMU-FCMD-01 study 2) 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. For patients with FCMD caused by a hetrozygous 1) Patient with FCMD and a compound heterozygous type with a confirmed diagnosis by genetic testing and a unilateral genetic mutation 2) Patients aged 3 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 |
|---|---|
| Physical examination Vital signs Electrocardiogram Echocardiography Ophthalmological examination Clinical examination Immunological test Adverse events | — |
Secondary
| Measure | Time frame |
|---|---|
| Gross Motor Function Measure Change in muscle mass | — |
Contacts
Public ContactHiroko Harada
National Center Hospital, National Center of Neurology and Psychiatry
Outcome results
None listed