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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BS01 (low dose) | Experimental |
| |
| Sham control | Sham Comparator |
| |
| BS01 (high dose) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BS01 | Biological | a recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP) |
|
| Measure | Description | 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 |
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Inclusion Criteria:
General Inclusion Criteria
Ophthalmic Inclusion Criteria
Exclusion Criteria:
General Exclusion Criteria
Ophthalmic Exclusion Criteria
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Sheila Nirenberg, PhD+ | Contact | 1+9178425027 | snsheilan@gmail.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| NJ Retina | Recruiting | Edison | New Jersey | 08820 | United States |
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Part 1 is an open label dose-escalation study; Part 2 is a randomized sham-controlled study.
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| Sham procedure control | Other | Sham procedure without needle |
|
| ID | Term |
|---|---|
| D057092 | Geographic Atrophy |
| ID | Term |
|---|---|
| D008268 | Macular Degeneration |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D005128 | Eye Diseases |
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