An Open-label, Multicenter, Two Part, Ascending Dose Followed by a Controlled Trial to Assess the Safety and Efficacy of a Subretinal Administration of AAVB-039 in Participants with Stargardt Disease (STGD1) (CELESTE)

Stargardt Disease (STGD1)

Part A: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial, Part B: Rate of change of area of ellipsoid zone (EZ) loss at 12 months

Part A: Rate of change at 12, 24, and 60 months as measured by: Area of ellipsoid zone (EZ) loss, Area of atrophy as assessed via short wavelength fundus autofluorescence (SW-FAF), Macular sensitivity assessed via microperimetry, Best corrected visual acuity (BCVA) letter score, Low-luminance visual acuity (LLVA) letter score, Part B: Incidence and severity of adverse events (AEs) including serious adverse events (SAEs), treatment-emergent adverse events (TEAEs), dose limiting toxicities (DLTs), and changes in clinical laboratory parameters, vital signs, physical examination findings and immunogenicity parameters from Day 0 to End of Trial, Part B: Rate of change at 12, 24, and 60 months as measured by: Area of ellipsoid zone (EZ) loss (24 and 60 months), Area of atrophy as assessed via short wavelength fundus autofluorescence (SW-FAF), Macular sensitivity assessed via microperimetry, Best corrected visual acuity (BCVA) letter score, Low-luminance visual acuity (LLVA) letter score

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