Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

NCT01721733 · clinicaltrials.gov ↗

PHASE2

Phase

COMPLETED

Status

35

Enrollment

INDUSTRY

Sponsor class

Conditions

Leigh Syndrome

Interventions

DRUG: Placebo
DRUG: EPI-743 15 mg/kg
DRUG: EPI-743 5 mg/kg

Sponsor

PTC Therapeutics

Collaborators

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