Clinical Trial: Promising ROd-cone DYstrophy Gene therapY

Study Status: RECRUITING
Recruit Status: RECRUITING
Study Type: INTERVENTIONAL




Official Title: A Phase I/II Study to Assess the Safety and Tolerability of a Single Subretinal Administration of SPVN06 Gene Therapy in Subjects With Rod-Cone Dystrophy (RCD) Due to a Mutation in the RHO, PDE6A

Brief Summary: This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm controlled double-masked randomized extension phase (Step 2), in subjects with advanced RCD due to a mutation in the RHO, PDE6A, or PDE6B gene.