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Placebo controlled double blind randomized Phase II Clinical Trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders

Phase 2
Conditions
lactic acidosis associated with mitochondrial disorders
Registration Number
JPRN-jRCT1091220093
Lead Sponsor
Yasutoshi KogaDepartment of Pediatrics and Child Health, Kurume University School of Medicine
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
PENDING
Sex
All
Target Recruitment
12
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

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

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
JMDRS section 1 & 2
Secondary Outcome Measures
NameTimeMethod
MDAS section 1-3, brain lactate by MRS, GDF15
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