NCT02352896View on ClinicalTrials.gov

Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome

PHASE2CompletedINTERVENTIONAL
Enrollment

30

Participants

Timeline

Start Date

January 31, 2014

Primary Completion Date

October 30, 2023

Study Completion Date

October 30, 2023

Conditions
Leigh Syndrome
Interventions
DRUG

EPI-743

EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm.

Trial Locations (4)

44308

Akron Children's Hospital, Akron

77030

Baylor College of Medicine, Houston

94305

Gregory Enns, Stanford University

98105

Seattle Children's Hospital, Seattle

Sponsors

Lead Sponsor

PTC Therapeutics
All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT02352896 - Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome | Biotech Hunter | Biotech Hunter