MELAS/MELA
Conditions
Interventions
24 weeks of sodium pyruvate 0.5g/kg/day TID
24 weeks of Placebo (Lactose + NaCl) 0.17g/kg/day TID
Sponsors
Japan Agency of Medical Research and Development
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Mitochondrial disease who fulfilled the following criteria 1) patient whoes age is over than 6 years-old 2) patient who are fulfilled the diagnostic criteria of MELAS/MELA 3) patientwho has an A3243G mutation. 4) no past history of sodium pyruvate administration for the therapeutic purpose 5) patient whose score of section 1+2 by JMDRS is more than 10, and one of the specific score is more than 2. 6) patient who have a written informed conscent or ascent by his/her own opinion.
Exclusion criteria
Exclusion criteria: 1) patient who has bed ridden status 2) patient who has dementic status and cannot be evaluated the JMDRS/NMDAS by medical doctor 3) patient who has uncontrolled status epileptics 4) patient who has septic status 5) patient who has renal failre, and less than 60mL/min/1.73m by eGFR 6) patient who has hypertrophic cardiomyopathy or cardiac failre 7) patient who has allergic state against sodium pyruvate 8) patient who enrolled to the other clinical trial within 6 months 9) patient who has pregnant 10) patient who is not recommended to the entry by medical doctor
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| JMDRS section 1 & 2 change at 24 weeks after the administration of sodium pyruvate | — |
Secondary
| Measure | Time frame |
|---|---|
| NMDAS section 1-3, brain lactate by MRS, GDF15 changes at 24 weeks after the administration of sodium pyruvate | — |
Countries
Japan
Contacts
Public ContactYasutoshi Koga
Kurume University Department of Pediatrics and Child Health
Outcome results
None listed