Safety and Efficacy Study of Novel Gene Therapy AGX-08 for Geographic Atrophy
Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of AGX-08 in Geographic Atrophy
1 other identifier
interventional
24
0 countries
N/A
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for early_phase_1
Started Jul 2026
Typical duration for early_phase_1
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 17, 2026
CompletedFirst Posted
Study publicly available on registry
May 22, 2026
CompletedStudy Start
First participant enrolled
July 1, 2026
ExpectedPrimary Completion
Last participant's last visit for primary outcome
December 30, 2028
Study Completion
Last participant's last visit for all outcomes
December 30, 2029
May 22, 2026
May 1, 2026
2.5 years
May 17, 2026
May 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
baseline to Week 52
Secondary Outcomes (2)
Change in Fundus Autofluorescence (FAF)
baseline to Week 52
Change in best corrected visual acuity (BCVA)
baseline to Week 52
Study Arms (4)
Low Dose
EXPERIMENTALIVT administration of a single low dose AGX-08 injection
Middle Dose
EXPERIMENTALIVT administration of a single middle dose AGX-08 injection
High Dose
EXPERIMENTALIVT administration of a single high dose AGX-08 injection
Control
SHAM COMPARATORSham IVT injection
Interventions
Eligibility Criteria
You may qualify if:
- 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 ≤77 letters (Snellen equivalent ≤20/63), 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.
You may not qualify if:
- 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;
- 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);
- +7 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- early phase 1
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, CARE PROVIDER
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 17, 2026
First Posted
May 22, 2026
Study Start (Estimated)
July 1, 2026
Primary Completion (Estimated)
December 30, 2028
Study Completion (Estimated)
December 30, 2029
Last Updated
May 22, 2026
Record last verified: 2026-05
Data Sharing
- IPD Sharing
- Will not share