Gene therapy injection aims to halt blinding eye disease
NCT ID NCT07602127
First seen Jun 26, 2026 · Last updated Jul 31, 2026 · Updated 2 times
Summary
This early-stage trial tests a gene therapy called AGX-08 for geographic atrophy, an advanced form of age-related macular degeneration that causes vision loss. The study will involve 24 people aged 50 and older, each receiving a single injection into one eye. The main goal is to check safety, but researchers will also measure changes in vision and retinal health.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- AGX-08 (gene therapy injection into the eye)
- What this could lead to
- If it works, this could point toward a treatment that slows or stops vision loss from geographic atrophy.
- What could go wrong
- This is a very early, first-in-human trial with only 24 people. It primarily checks safety, not effectiveness, and may fail or have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Early phase 1
The earliest testing in people: a first look at safety, in a very small group.
- Participants
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About 24 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Jul 2026
An estimate. Start dates often move.
- Expected to finish
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Dec 2029
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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50 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Clinically confirmed diagnosis of geographic atrophy (GA) secondary to age-related macular degeneration (AMD); 2. Age ≥ 50 years, regardless of sex; 3. Best-corrected visual acuity (BCVA) in the study eye measured using the ETDRS chart at a starting distance of 4 meters must be ≤35 letters (Snellen equivalent ≤20/200), with vision no worse than light perception; the fellow eye must not have better visual acuity than the study eye; 4. GA lesion size between 2.5 and 17.5 mm² with clearly defined borders. For multifocal GA, at least one lesion must be ≥1.25 mm² (0.5 disc area) to ensure measurable efficacy assessment; 5. Presence of choroidal neovascularization (CNV) in the fellow eye is allowed; 6. Women of childbearing potential must have a negative pregnancy test (blood or urine); 7. Women of childbearing potential and male participants must agree to use effective contraception during the study and for 3 months after completion, with no plans for reproduction; 8. Willing and able to provide written informed consent and comply fully with the study protocol. Exclusion Criteria: 1. GA caused by conditions other than AMD (e.g., Stargardt disease, cone-rod dystrophy, or other inherited macular dystrophies); 2. Aphakia in the study eye, or cataract surgery/YAG capsulotomy within 3 months prior to screening; 3. Refractive status or axial length outside the following range: spherical equivalent between -6.00D and +5.00D, and axial length between 21 mm and 26 mm; 4. Two baseline BCVA measurements (ETDRS) taken at least 14 days apart during screening differ by \>30%; 5. Current or prior evidence of exudative (wet) AMD in the study eye, including retinal pigment epithelium tear, retinal vascular occlusions, history of corneal transplantation, or any neovascularization confirmed by fluorescein angiography; 6. Active ocular diseases in the study eye, including inflammation, other macular diseases, glaucoma, ocular hypertension, or acute/chronic ocular infections; 7. Major ocular surgery within 3 months prior to screening; 8. History of retinal detachment or other fundus diseases unsuitable for study participation; 9. Prior macular laser photocoagulation with irreversible retinal damage; 10. Pregnant or lactating women, or participants unwilling to use effective contraception for 12 months before and after study intervention; 11. Narrow anterior chamber angle or other contraindications to pupil dilation; 12. Any ocular condition that may interfere with visual acuity assessment, OCT, or other ophthalmic evaluations; 13. History of hypersensitivity to contrast agents, study drugs, or excipients; 14. Allergy to corticosteroids, intolerance to protocol-required corticosteroid therapy, or contraindicated active infections; 15. Severe systemic diseases, psychiatric disorders, uncontrolled chronic conditions, or other medical conditions that may increase study risk (e.g., malignancy, metabolic or autoimmune diseases); 16. articipants with a prior history of malignancy must have completed curative treatment ≥5 years before enrollment and must have no evidence of active disease, recurrence, or metastasis. Participants whose prior malignancy is considered unlikely to affect life expectancy may be eligible based on Investigator assessment in consultation with an oncology specialist. The 5-year disease-free interval requirement may be waived for adequately treated cervical carcinoma in situ or non-melanoma skin cancer with minimal risk of recurrence.; 17. Receiving or likely to receive immunosuppressive therapy outside this study; 18. Participation in another investigational drug study within 3 months prior to screening; 19. Prior gene therapy other than this study; 20. Any condition that may compromise scientific evaluation of the study; 21. Any contraindication to intravitreal (IVT) injection; 22. Any other condition deemed unsuitable by the investigator.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a new eye injection protect vision in geographic atrophy?
- Can genetics predict the worsening of geographic atrophy?
- New eye imaging could reveal hidden clues to vision loss
- A new eye camera may spot stressed cells before vision loss begins
- Gene therapy injection aims to halt blinding eye disease
- Eye imaging showdown: which device measures macular damage best?