Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)

Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome) - Placebo controlled double blind randomized Phase 2 clinical trial of sodium pyruvate on lactic acidosis associated with mitochondrial disorders (Leigh syndrome)

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-UMIN000025872

Enrollment

Registered

2017-02-01

Start date

2017-02-01

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Leigh disorders

Interventions

sodium pyruvate 0.5g/kg/day TID 24 weeks Placebo (lactate+NaCl) 0.17/kg/day TID 24 weeks

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Leigh disease who fulfilled the following criteria 1)neurological findings (all matched) progressive central neurological sings symptoms suggesting bilateral brain stem abnormality bilateral necrosis of brain stem by MRI 2)defined mitochondrial dysfunction (one of the following ) by repeated abnormality of serum lactate by respiratory chain enzyme activity by muscle pathology by genetic abnormality

Exclusion criteria

Exclusion criteria: 1) patients who is not evaluated by this protocol of the clinical trial 2)severe unconsciousness 3)sepsis 4)renal dysfunction by eGFR lower than 60 mL/min/1.73m2 5)heart failure (EF<40%) 6)status epileptics 7)pregnant 8)allergy against the sodium pyruvate 9)enrolled to the other clinical trial 24 weeks before this trial 10)patient who cannot evaluate the muscle activity by device 11)patients who are not recommended by the doctor

Design outcomes

Primary

Measure	Time frame
Total score of section 1-3 in NMDPS at 24 weeks after oral administration	—

Secondary

Measure	Time frame
Total score of NMDAS section 1-3, Score of section 1, 2, 3, 4. brain lactate by MRS, GDF15 at 24 weeks after oral administration. Duration of in-patient, Emergency visit, mortality	—

Countries

Japan

Contacts

Public ContactYasutoshi Koga

Kurume University Department of Pediatrics and Child Health

yasukoga@med.kurume-u.ac.jp0942-31-7565

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026