NCT00999609View on ClinicalTrials.gov

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

31

Participants

Timeline

Start Date

October 31, 2012

Primary Completion Date

July 31, 2015

Study Completion Date

January 31, 2030

Conditions
Inherited Retinal Dystrophy Due to RPE65 MutationsLeber Congenital Amaurosis
Interventions
BIOLOGICAL

AAV2-hRPE65v2,voretigene neparvovec-rzyl

Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector genomes per eye) to both eyes via surgical procedures on separate days.

Trial Locations (2)

19104

Children's Hospital of Philadelphia, Philadelphia

52242

University of Iowa, Iowa City

All Listed Sponsors
collaborator

Children's Hospital of Philadelphia

OTHER

collaborator

University of Iowa

OTHER

lead

Spark Therapeutics, Inc.

INDUSTRY