| Active, not recruiting | 3 | 31 | US | AAV2-hRPE65v2,voretigene neparvovec-rzyl, AAV2-hRPE65v2, voretigene neparvovec-rzyl, gene therapy vector | Spark Therapeutics, Inc., Children's Hospital of Philadelphia, University of Iowa | Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis | 07/15 | 07/29 | | |