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Experimental gene injection targets dry AMD

NCT ID NCT03846193

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tested a gene therapy called GT005 for dry age-related macular degeneration (AMD), a leading cause of vision loss. The therapy delivers a working gene to the retina via a single injection. The study enrolled 56 people and focused on safety and dosing, but was terminated early. If it works, it could offer a one-time treatment to slow vision loss.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
GT005 (a gene therapy that delivers a working copy of the complement factor I gene via a single injection under the retina)
What this could lead to
If successful, this could lead to a one-time gene therapy that slows or stops vision loss in people with dry age-related macular degeneration.
What could go wrong
This was a very early (Phase 1/2) trial with only 56 participants, and it was terminated early. The therapy involves a surgical injection into the eye, which carries risks like infection or retinal damage.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

56 people

The number who actually took part.

Started

Dec 2018

Finished

Jun 2024

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

55 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. Able and willing to give consent to study participation 2. Have a clinical diagnosis of GA secondary to AMD in the study eye, as determined by the Investigator, and a diagnosis of AMD in the contralateral eye (except if the subject is monocular) 3. Cohorts 1 to 6: GA lesion(s) total size in the study eye must be ≥1.25mm2 and ≤17.5mm2. Cohort 7: GA lesion(s) total size in the study eye must be ≥1.25mm2 4. GA lesion(s) in the study eye must reside completely within the FAF fundus image 5. Cohorts 1 to 3: BCVA of ≤50 letters (6/36 Snellen acuity equivalent or worse) using ETDRS charts in the study eye Cohorts 4 to 7: BCVA of ≥24 letters (6/95 and 20/320 Snellen acuity equivalent or better) using ETDRS charts in the study eye 6. Aged ≥55 years 7. Able to attend all study visits and complete the study procedures 8. Women of child-bearing potential need to have a negative urine pregnancy test within two weeks prior to receiving the drug. A pregnancy test is not required for postmenopausal women (defined as being at least 12 consecutive months without menses) or those surgically sterilised (those having a bilateral tubal ligation/bilateral salpingectomy, bilateral tubal occlusive procedure, hysterectomy, or bilateral oophorectomy) Exclusion Criteria: 1. Have evidence or history of Choroidal Neovascularisation (CNV) in the study eye. Subjects are permitted to have CNV in the fellow eye defined as either: 1. Non-exudative/sub-clinical fellow eye CNV identified at screening, or 2. Known history of fellow eye CNV with either ≥2 years since diagnosis or with no active treatment required in 6 months prior to screening 2. Presence of moderate/severe non-proliferative diabetic retinopathy or worse in the study eye 3. Have history of vitrectomy, sub-macular surgery, or macular photocoagulation in the study eye 4. History of intraocular surgery in the study eye within 12 weeks prior to Screening (Visit 1). Yttrium aluminum garnet capsulotomy is permitted if performed \>10 weeks prior to Visit 1 5. Have clinically significant cataract that may require surgery during the study period in the study eye 6. Presence of moderate to severe glaucomatous optic neuropathy in the study eye; uncontrolled IOP despite the use of more than two topical agents; a history of glaucoma-filtering or valve surgery is also excluded 7. Axial myopia of greater than -8 diopters in the study eye 8. Have received any investigational product for the treatment of GA within the past 6 months or 5 half-lives (whichever is longer), other than nutritional supplements such as the Age-Related Eye Disease Study (AREDS) formula 9. Have received a gene or cell therapy at any time 10. Have a contraindication to the specified protocol corticosteroid regimen 11. Are unwilling to use two forms of contraception (one of which being a barrier method) for 90 days post-dosing, if relevant 12. Active malignancy within the past 12 months, except for: Appropriately treated carcinoma in situ of the cervix, non-melanoma skin carcinoma, or prostate cancer with a stable prostate-specific antigen (PSA) ≥12 months 13. Have any other significant ocular or non-ocular medical or psychiatric condition which, in the opinion of the Investigator, may either put the subject at risk or may influence the results of the study 14. Cohorts 5 to 7 only: presence of metallic objects or implanted stimulator devices in or near the head, including cochlear implants, deep brain stimulators, vagus nerve stimulators, and other implanted electrodes or stimulators

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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.

Contacts and locations

Locations

  • Bristol Eye Hospital

    Bristol, United Kingdom

  • Cincinnati Eye Institute

    Cincinnati, Ohio, 45242, United States

  • Manchester Eye Hospital

    Manchester, United Kingdom

  • Mid-Atlantic Retina

    Philadelphia, Pennsylvania, 19107, United States

  • Midwest Eye Institute

    Indianapolis, Indiana, 46290, United States

  • Moorfields Eye Hospital

    London, United Kingdom

  • Ophthalamic Consultants of Boston (OCB)

    Boston, Massachusetts, 02114, United States

  • Oxford University Hospital

    Oxford, United Kingdom

  • Pepose Vision Institute

    Chesterfield, Missouri, 63017, United States

  • Retina Clinic London

    London, W1G 7LA, United Kingdom

  • Sierra Eye Associates

    Reno, Nevada, 89502, United States

  • Sunderland Eye Infirmary

    Sunderland, United Kingdom

  • Wolfe Eye Clinic

    West Des Moines, Iowa, 50266, United States

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