A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP (AAV2-CAGChronosFP) in Patients With Geographic Atrophy Secondary to Dry AMD
This is a Phase 1/2 study, multi-center, dose-escalation interventional study of BS01 in subjects with GA secondary to dry AMD. Part 1 is an open label dose-escalation study; Part 2 is a dose-expansion study with dose(s) selected from Part 1 based on a benefit/risk assessment, and an untreated (sham injection) group to allow for a controlled comparison of efficacy and safety. This is a seamless Phase 1/2 study in up to 10 patients for Phase 1 and 30 patients Phase 2 in patients with GA secondary to dry AMD.
• Signed informed consent obtained before screening.
• Men or women between 50 and 85 years of age inclusive at the time of signing the informed consent.
⁃ Ophthalmic Inclusion Criteria
• Geographic atrophy with some macula foveal involvement secondary to dry AMD.
• Total GA area ≥ 5 and ≤ 17.5 mm2 (2 and 7 disk areas respectively), based on Heidelberg Region Finder or equivalent automated software.
• If GA is multifocal, at least one focal lesion should measure ≥ 1.25 mm2 (0.5 disk area) to ensure measurable focal effects for efficacy evaluation.
• Composite lesion encompassing confluent GA regions with total area meeting thresholds given in 2 above.
• GA in part within 1200 microns from the foveal center.
• The atrophic lesion must be able to be photographed in its entirety.
• BCVA between 20/50 to 20/400, inclusive, using letter score per EDTRS chart.
• Clear ocular media and adequate pupillary dilatation in both eyes to allow for all imaging procedures, including good quality stereoscopic fundus photography and fundus autofluorescence (FAF).
• Central fixation.