NCT01721733View on ClinicalTrials.gov

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

PHASE2CompletedINTERVENTIONAL

Enrollment

35

Participants

Timeline

Start Date

October 31, 2012

Primary Completion Date

February 28, 2015

Study Completion Date

May 31, 2015

Conditions

Leigh Syndrome

Interventions

DRUG

Placebo

DRUG

EPI-743 15 mg/kg

DRUG

EPI-743 5 mg/kg

Trial Locations (4)

44308

Akron Children's Hospital, Akron

77030

Baylor College of Medicine, Houston

94304

Stanford University, Palo Alto

98105

Seattle Children's Hospital, Seattle

Sponsors

Lead Sponsor

PTC Therapeutics

Collaborators (1)

Axio Research. LLC

All Listed Sponsors

collaborator

Axio Research. LLC

INDUSTRY

lead

PTC Therapeutics

INDUSTRY

NCT01721733 - Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome | Biotech Hunter | Biotech Hunter