Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD)Phase 1/2NCT07158775
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
A Phase 1/2 study testing BS01 in people with Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD), run by Bionic Sight LLC at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 50 to 85 years
- Conditions the study is for
- Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD)
- What is being tested?
- BS01
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 8 Jan 2026to Dec 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- General Inclusion Criteria
- 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.
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- 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.
Who cannot join
- General Exclusion Criteria
- Previous therapeutic radiation in the region of the SE.
- Previous treatment with any ocular or systemic gene transfer product.
- Any treatment with an investigational agent in the past 60 days for any condition.
- Women who are pregnant or nursing.
- Known hypersensitivity to topical ocular anesthetics or diagnostic drops to be used during the study.
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- Any other medical condition, which, in the Investigator's judgment, will interfere with the patient's ability to comply with the protocol, compromises patient safety, or interferes with the interpretation of the study results.
- Ophthalmic Exclusion Criteria
- > 30% difference of BCVA using EDTRS in two baseline visual acuity assessments with at least 14 days apart during screening.
- Any intraocular surgery or thermal laser within 3 months of study entry. Any prior thermal laser in the macular region, regardless of indication.
- Any ocular or periocular infection (including blepharitis), or ocular surface inflammation in the past 12 weeks.
- History of any of the following procedures: Posterior vitrectomy, filtering surgery (e.g., trabeculectomy), glaucoma drainage device, corneal transplant, or retinal detachment.
- Any sign of diabetic retinopathy in either eye.
- Intraocular pressure (IOP) > 25 mmHg in either eye.
- Completely atrophic centrally located lesions based on ellipsoid zone (EZ) loss in spectraldomain optical coherence tomography (SD-OCT).
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
NJ Retina
Edison, New Jersey, 08820
Andy Merino, 732-906-1887, amerino@njretina.com
About this study
Official title: 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.
- Being tested
- BS01, Sham procedure control
- Keywords
- GA
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BS01Biological
a recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP)
Sham procedure controlOther
Sham procedure without needle
| Study group | Role | What they receive |
|---|---|---|
| BS01 (low dose) | Experimental | Not described Receives: Biological: BS01 |
| Sham control | Sham comparator | Not described Receives: Other: Sham procedure control |
| BS01 (high dose) | Experimental | Not described Receives: Biological: BS01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
[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)
Measured: 12 months
[Phase 2] Change from baseline in BCVA (Best Corrected Visual Acuity)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start8 Jan 2026
- Primary completionDec 2027 (estimated)
- Study completionDec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Part 1 is an open label dose-escalation study; Part 2 is a randomized sham-controlled study.
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bionic Sight LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07158775
- Sponsor's protocol ID
- BS01-GA-001
- Status last verified
- Jan 2026
- First submitted
- 4 Sep 2025
- Last updated
- 23 Jan 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 23 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07158775
- Acronym / sponsor ID
- BS01-GA-001
- Keywords
- GA
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Bionic Sight LLCIndustry (pharma, biotech, devices)
- Study start
- 8 Jan 2026 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2031 (estimated)
- First posted
- 8 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
- Model description
- Part 1 is an open label dose-escalation study; Part 2 is a randomized sham-controlled study.
| Arm | Type | Receives |
|---|---|---|
| BS01 (low dose) | Experimental | Biological: BS01 |
| Sham control | Sham comparator | Other: Sham procedure control |
| BS01 (high dose) | Experimental | Biological: BS01 |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (13 listed)
- General Inclusion Criteria
- 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.
Key exclusion criteria (15 listed)
- General Exclusion Criteria
- Previous therapeutic radiation in the region of the SE.
- Previous treatment with any ocular or systemic gene transfer product.
- Any treatment with an investigational agent in the past 60 days for any condition.
- Women who are pregnant or nursing.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BS01a recombinant adeno-associated virus vector expressing ChronosFP (AAV2-CAGChronosFP) | Biological | BS01 (high dose), BS01 (low dose) |
| Sham procedure controlSham procedure without needle | Other | Sham control |
Primary outcome
[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
[Phase 2] Change from baseline in BCVA (Best Corrected Visual Acuity)
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jan 2026.
- Individual participant dataSharing planned: Undecided
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Investigating Geographic Atrophy Insights (i-GAIN) Natural History Study
Dry Age-related Macular Degeneration, Geographic Atrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BS01 in people with Geographic Atrophy (GA) Secondary to Dry Age-related Macular Degeneration (AMD), run by Bionic Sight LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sheila Nirenberg, PhD+ (1+9178425027, snsheilan@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jan 2026.