Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD)
Conditions
Keywords
GA
Brief summary
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.
Interventions
BIOLOGICALBS01
a recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP)
Sham procedure without needle
Sponsors
Bionic Sight LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Intervention model description
Part 1 is an open label dose-escalation study; Part 2 is a randomized sham-controlled study.
Eligibility
Sex/Gender
ALL
Age
50 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
General Inclusion Criteria 1. Signed informed consent obtained before screening. 2. Men or women between 50 and 85 years of age inclusive at the time of signing the informed consent. Ophthalmic Inclusion Criteria 1. Geographic atrophy with some macula foveal involvement secondary to dry AMD. 2. Total GA area ≥ 5 and ≤ 17.5 mm2 (2 and 7 disk areas respectively), based on Heidelberg Region Finder or equivalent automated software. 3. 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. 4. Composite lesion encompassing confluent GA regions with total area meeting thresholds given in 2 above. 5. GA in part within 1200 microns from the foveal center. 6. The atrophic lesion must be able to be photographed in its entirety. 7. BCVA between 20/50 to 20/400, inclusive, using letter score per EDTRS chart. 8. 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). 9. Central fixation.
Exclusion criteria
General
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| [Phase 1] Number of the dose limiting toxicities (DLTs), incident and severity of ocular and non-ocular Study Drug-related adverse events (SDAE), treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | 12 months |
| [Phase 2] Change from baseline in BCVA (Best Corrected Visual Acuity) | 12 months |
Countries
United States
Contacts
CONTACTSheila Nirenberg, PhD+
Outcome results
None listed