Geographic AtrophyEarly Phase 1NCT07602127
Safety and Efficacy Study of Novel Gene Therapy AGX-08 for Geographic Atrophy
A Early Phase 1 study testing AGX-08-L, AGX-08-H and AGX-08-S in people with Geographic Atrophy, run by Augelux Therapeutics.
Trial summary
This is AGX-08's safety, tolerability, and efficacy in Geographic Atrophy first-in-human study. This trial is meant to evaluate the safety and efficacy of AGX-08 in Geographic Atrophy patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Geographic Atrophy
- What is being tested?
- AGX-08-L, AGX-08-H and AGX-08-S
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2026to 30 Dec 2029
- Phase
- Early Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinically confirmed diagnosis of geographic atrophy (GA) secondary to age-related macular degeneration (AMD);
- Age ≥ 50 years, regardless of sex;
- 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;
- 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;
- Presence of choroidal neovascularization (CNV) in the fellow eye is allowed;
- Women of childbearing potential must have a negative pregnancy test (blood or urine);
- 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;
- Willing and able to provide written informed consent and comply fully with the study protocol.
Who cannot join
- GA caused by conditions other than AMD (e.g., Stargardt disease, cone-rod dystrophy, or other inherited macular dystrophies);
- Aphakia in the study eye, or cataract surgery/YAG capsulotomy within 3 months prior to screening;
- 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;
- Two baseline BCVA measurements (ETDRS) taken at least 14 days apart during screening differ by >30%;
- 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;
- Active ocular diseases in the study eye, including inflammation, other macular diseases, glaucoma, ocular hypertension, or acute/chronic ocular infections;
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- Major ocular surgery within 3 months prior to screening;
- History of retinal detachment or other fundus diseases unsuitable for study participation;
- Prior macular laser photocoagulation with irreversible retinal damage;
- Pregnant or lactating women, or participants unwilling to use effective contraception for 12 months before and after study intervention;
- Narrow anterior chamber angle or other contraindications to pupil dilation;
- Any ocular condition that may interfere with visual acuity assessment, OCT, or other ophthalmic evaluations;
- History of hypersensitivity to contrast agents, study drugs, or excipients;
- Allergy to corticosteroids, intolerance to protocol-required corticosteroid therapy, or contraindicated active infections;
- Severe systemic diseases, psychiatric disorders, uncontrolled chronic conditions, or other medical conditions that may increase study risk (e.g., malignancy, metabolic or autoimmune diseases);
- 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.;
- Receiving or likely to receive immunosuppressive therapy outside this study;
- Participation in another investigational drug study within 3 months prior to screening;
- Prior gene therapy other than this study;
- Any condition that may compromise scientific evaluation of the study;
- Any contraindication to intravitreal (IVT) injection;
- Any other condition deemed unsuitable by the investigator.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of AGX-08 in Geographic Atrophy
This is AGX-08's safety, tolerability, and efficacy in Geographic Atrophy first-in-human study. This trial is meant to evaluate the safety and efficacy of AGX-08 in Geographic Atrophy patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Read the detailed description
Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) is a leading cause of irreversible vision loss in the elderly population. Patients with GA experience progressive degeneration of retinal cells, resulting in gradual and permanent visual decline. Currently, there are limited effective treatment options available. We have developed an innovative adeno-associated virus (AAV)-based gene therapy for patients with GA, regardless of underlying genetic background. Approximately 8 to 24 subjects with GA will be recruited, among whom a subset will receive a single unilateral intravitreal injection of AGX-08 at ascending doses, while a proportion of subjects will receive sham injections as the control group.
- Conditions
- Geographic Atrophy
- Being tested
- AGX-08-L, AGX-08-H, AGX-08-S
- Keywords
- GA, AMD, dry AMD
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AGX-08-LDrug
Also called Low dose
rAAV2.AG-eNCRV intravitreal injection of low dose
AGX-08-HDrug
Also called High dose
rAAV2.AG-eNCRV intravitreal injection of high dose
AGX-08-SProcedure or surgery
Also called Sham control
sham intravitreal injection of AGX-08 (not actual injection)
| Study group | Role | What they receive |
|---|---|---|
| Low Dose | Experimental | IVT administration of a single low dose AGX-08 injection Receives: Drug: AGX-08-L |
| High Dose | Experimental | IVT administration of a single high dose AGX-08 injection Receives: Drug: AGX-08-H |
| Control | Sham comparator | Sham IVT injection Receives: Procedure: AGX-08-S |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events,adverse events, serious adverse events and dose-limiting toxicities
Incidence of severity of ocular and systemic treatment-emergent adverse events, (TEAEs), adverse events (AEs) , serious adverse events (SAEs) and dose-limiting toxicities(DLTs) following a single intravitreal injection of AGX-08.
Measured: baseline to Week 52
Secondary outcomes (2)
Change in Fundus Autofluorescence (FAF)
FAF is a non-invasive imaging technique that provides critical information about the health of the retina, which is vital for the functioning of the retina.
Measured: baseline to Week 52
Change in best corrected visual acuity (BCVA)
BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart or other avaliable measurement. This approach was chosen to facilitate visual acuity testing in subject who cannot recognize letters.
Measured: baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2026
- Study start1 Jul 2026 (estimated)
- Primary completion30 Dec 2028 (estimated)
- Study completion30 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic AtrophyStudies 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Augelux Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07602127
- Sponsor's protocol ID
- AGX-08
- Status last verified
- Jul 2026
- First submitted
- 17 May 2026
- Last updated
- 31 Jul 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of AGX-08 in Geographic Atrophy
- ClinicalTrials.gov ID
- NCT07602127
- Acronym / sponsor ID
- AGX-08
- Conditions
- Geographic Atrophy
- Keywords
- GA, AMD, dry AMD
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- Augelux TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 1 Jul 2026 (estimated)
- Primary completion
- 30 Dec 2028 (estimated)
- Study completion
- 30 Dec 2029 (estimated)
- First posted
- 22 May 2026 (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 AGX-08's safety, tolerability, and efficacy in Geographic Atrophy first-in-human study. This trial is meant to evaluate the safety and efficacy of AGX-08 in Geographic Atrophy patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.
Detailed description
Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) is a leading cause of irreversible vision loss in the elderly population. Patients with GA experience progressive degeneration of retinal cells, resulting in gradual and permanent visual decline. Currently, there are limited effective treatment options available. We have developed an innovative adeno-associated virus (AAV)-based gene therapy for patients with GA, regardless of underlying genetic background. Approximately 8 to 24 subjects with GA will be recruited, among whom a subset will receive a single unilateral intravitreal injection of AGX-08 at ascending doses, while a proportion of subjects will receive sham injections as the control group.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Low Dose | Experimental | Drug: AGX-08-LIVT administration of a single low dose AGX-08 injection |
| High Dose | Experimental | Drug: AGX-08-HIVT administration of a single high dose AGX-08 injection |
| Control | Sham comparator | Procedure: AGX-08-SSham IVT injection |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Clinically confirmed diagnosis of geographic atrophy (GA) secondary to age-related macular degeneration (AMD);
- Age ≥ 50 years, regardless of sex;
- 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;
- 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;
- Presence of choroidal neovascularization (CNV) in the fellow eye is allowed;
Key exclusion criteria (22 listed)
- GA caused by conditions other than AMD (e.g., Stargardt disease, cone-rod dystrophy, or other inherited macular dystrophies);
- Aphakia in the study eye, or cataract surgery/YAG capsulotomy within 3 months prior to screening;
- 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;
- Two baseline BCVA measurements (ETDRS) taken at least 14 days apart during screening differ by >30%;
- 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;
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 |
|---|---|---|
| AGX-08-LAlso: Low doserAAV2.AG-eNCRV intravitreal injection of low dose | Drug | Low Dose |
| AGX-08-HAlso: High doserAAV2.AG-eNCRV intravitreal injection of high dose | Drug | High Dose |
| AGX-08-SAlso: Sham controlsham intravitreal injection of AGX-08 (not actual injection) | Procedure or surgery | Control |
Primary outcome
Incidence of treatment-emergent adverse events,adverse events, serious adverse events and dose-limiting toxicities
Time frame: baseline to Week 52
Incidence of severity of ocular and systemic treatment-emergent adverse events, (TEAEs), adverse events (AEs) , serious adverse events (SAEs) and dose-limiting toxicities(DLTs) following a single intravitreal injection of AGX-08.
2 secondary outcomes
Change in Fundus Autofluorescence (FAF)
Time frame: baseline to Week 52
Change in best corrected visual acuity (BCVA)
Time frame: baseline to Week 52
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 31 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Geographic Atrophy
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age-related Macular Degeneration (AMD), Geographic Atrophy (GA)
RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
Geographic Atrophy, Macular Degeneration
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.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing AGX-08-L, AGX-08-H and AGX-08-S in people with Geographic Atrophy, run by Augelux Therapeutics.
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 8 inclusion and 22 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: opening soon, as of the sponsor's last update on 31 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
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 phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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 Windy Zhou (+86 18986214263, zmt@zmtherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events,adverse events, serious adverse events and dose-limiting toxicities (time frame: baseline to Week 52). 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 31 Jul 2026.