4 results
The primary objective of the study is to demonstrate clinically relevant statistical superiority in progression-free survival (PFS) with GClb compared to RClb and Clb alone and RClb compared to Clb [GClb vs Clb; GClb vs RClb; RClb vs Clb] in…
Primary ObjectivesThe primary objectives of this study are:Phase I, Part A - Dose Escalation:• To determine the maximum tolerated dose (MTD) or recommended phase 2 dose (RP2D) of CB-103 as a single agent when administered orally and with repeat…
Main objective:To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRPTo assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of functional vision…
Primary:To assess the effect of bilateral treatment with AAV5-hRKp.RPGR on functional vision as measured by vision-guided mobility assessment.Secondary:To assess changes after treatment administration in retinal function, functional vision, visual…