Therapeutic application of sodium pyruvate to patients of severe mitochondrial disorders
Phase 2
- Conditions
- mitochondrial disorders
- Registration Number
- JPRN-UMIN000008341
- Lead Sponsor
- Department of Pediatrics, Hiroshima University Hospital
- Brief Summary
Five patients were enrolled in the study. All had withdrawn by the end of 2014.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 20
Inclusion Criteria
Not provided
Exclusion Criteria
1) Women who are pregnant, breastfeeding, or of possible pregnancy. 2) Patients who are judged to be inappropriate for any other reason by their doctor in charge.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Changes in scores of Mitochondrial Disease Adult Scale (NMDAS; for adults), or Newcastle Paediatric Mitochondrial Disease Scale (NPMDS; for children) before and after 6-month administration.
- Secondary Outcome Measures
Name Time Method Reduction rate of lactate/pyruvate ratio in blood as effectiveness, and rate of adverse events as safety.