Gene therapy injection aims to halt blinding eye disease

NCT ID NCT07602127

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jul 2026

An estimate. Start dates often move.

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

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

50 years and older

Sex

Anyone

Healthy volunteers

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

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.

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