Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT04437368) enrolled 98 people in total across three groups: 52 people received a lower dose of an investigational gene therapy called GT005, 9 received a higher dose, and 37 were in an untreated control group. The trial was looking at a condition called geographic atrophy (GA) — a progressive eye condition where patches of cells in the back of the eye gradually die off. The main thing being measured was how much those damaged patches grew over time, specifically the change in their size (measured in square millimetres) from the start of the trial to 48 weeks later. The reported data shows that, for the primary measure at 48 weeks, the damaged area in the low-dose group grew by approximately 2.12 mm², the high-dose group by approximately 2.38 mm², and the untreated control group by approximately 1.14 mm². For the secondary measure looking at longer-term changes, the reported data shows that by 96 weeks the low-dose group had an increase of approximately 4.84 mm², the high-dose group approximately 4.07 mm², and the untreated control group approximately 2.80 mm². These numbers represent how much the affected area changed from where each group started — they do not on their own indicate whether one group was better or worse off overall, as individual starting points varied. The reported data also shows that across both parts of the trial combined, 18 people in the low-dose group, 9 in the high-dose group, and 2 in the untreated control group experienced what are described as ocular (eye-related) adverse events — that is, undesirable medical occurrences that happened after joining the trial. Non-ocular (non-eye-related) adverse events were recorded in 13 people in the low-dose group, 8 in the high-dose group, and 14 in the untreated control group. These are counts of events that were recorded and tracked, and no further detail about their nature or severity is provided in the submitted data. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 37314061) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 2 Macular Degeneration Trial, Terminated
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are willing and able to sign a consent form agreeing to take part
- You are 55 years of age or older
- You have been diagnosed with geographic atrophy (a form of advanced dry age-related macular degeneration, or AMD) in one eye, and have AMD in the other eye (unless you only have vision in one eye)
- The affected area in your eye falls within a specific size range — not too small and not too large (confirm with trial site)
- The affected area in your eye is fully visible within a special eye imaging test
- Your vision in the study eye meets a minimum level on a standard eye chart test (confirm with trial site)
- You are able to attend all required appointments and complete all study procedures
- If you are a woman who could become pregnant, you must have a negative pregnancy test shortly before the study begins (this is not needed if you have gone through menopause or have had certain surgical procedures)
- Part 1 of the trial only: You carry a specific rare gene variation linked to lower levels of a particular protein in your blood (confirm with trial site)
Who may not be able to join:
- You have been diagnosed with a different inherited eye condition called Stargardt Disease or another retinal dystrophy
- You have a history of, or currently have, a type of abnormal blood vessel growth (wet AMD) in the study eye
- You have moderate or severe diabetic eye disease in the study eye
- You have previously had certain eye surgeries in the study eye, including surgery under the retina or laser treatment to the macula
- You have had eye surgery in the study eye within the past 12 weeks (a specific type of laser capsule procedure may be allowed if done more than 10 weeks ago — confirm with trial site)
- You have a significant cataract in the study eye that may need surgery during the trial
- You have moderate to severe glaucoma damage to the optic nerve, uncontrolled eye pressure even with two or more eye drop treatments, or a history of certain glaucoma surgeries in the study eye
- You have a high level of short-sightedness (greater than -8 dioptres) in the study eye
- You have any other significant eye or general health condition, or a mental health condition, that the doctor feels could put you at risk or affect the results
- You have a medical reason why you cannot take the steroid medication required by this trial
- You have received any treatment (investigational or approved) for geographic atrophy in the past 6 months or longer (standard eye vitamins such as AREDS formula are allowed)
- You have ever received a gene therapy or cell therapy at any time
- You are not willing to use two forms of contraception (one being a barrier method) for 90 days after receiving the treatment, if this applies to you
- You have had an active cancer diagnosis in the past 12 months (some exceptions may apply — confirm with trial site)
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
2 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
The Change From Baseline to Week 48 in Geographic Atrophy (GA) - Part 1
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.