A Phase 1/2 Study to Evaluate the Safety and Efficacy of Intravitreal Administration of BS01 in Patients With Geographic Atrophy Secondary to Dry AMD

Conditions

• Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD)

Eligibility Criteria

Sex

ALL

Age

50 Years - 85 Years

Healthy Volunteers

Not accepted

Interventions

• BS01 — BIOLOGICAL
  a recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP)
• Sham procedure control — OTHER
  Sham procedure without needle

Study Details

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.

Key Dates

Start date

Jan 8, 2026

Status verified

Jan 2026

Primary completion

Dec 31, 2027

Completion

Dec 31, 2031

Study Design

Enrollment

40 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: BS01 (low dose)
• Sham Comparator: Sham control
• Experimental: BS01 (high dose)

Primary Outcome Measure

[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) [ Time Frame: 12 months ]

Central Contacts

• Sheila Nirenberg, PhD+
  1+9178425027
  [email protected]

Locations (1)

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