A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome

Status

Unknown

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080222300

Enrollment

Registered

2013-11-21

Start date

2013-11-21

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 Syndrome

Interventions

investigational material(s) Generic name etc : EPI-743 INN of investigational material : Therapeutic category code : 399 Agents affecting metabolism, n.e.c. Dosage and Administration for Investigatio

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Clinical and MRI diagnosis of Leigh syndrome etc.

Exclusion criteria

Exclusion criteria: - Allergy to EPI-743 or sesame oil - Allergy to vitamin E - Diagnosis of any other concurrent inborn error of metabolism etc.

Design outcomes

Primary

Measure	Time frame
Newcastle Pediatric Mitochondrial Disease Scale(NPMDS) (Sections 1-3) Amount of change in NPMDS(Sections 1-3) total score of each evaluation period from baseline	—

Secondary

Measure	Time frame
Barry-Albright Dystonia Scale Gross Motor Function Measure Need for tracheostomy Number of deaths Total number of hospitalizations Glutathione cycle biomarkers Adverse events etc. Amount of change in Barry-Albright Dystonia Scale total score of each evaluation period from baseline etc.	—

Outcome results

None listed

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