Safety and Efficacy Study of Novel Gene Therapy AGX-08 for Geographic Atrophy

May 20, 2026 updated by: Zhongmou Therapeutics

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.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

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 12 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 Type

Interventional

Enrollment (Estimated)

24

Phase

  • Early Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 ≤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;
  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. History of malignancy within 5 years (except cured basal cell carcinoma of the skin or cervical carcinoma in situ);
  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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low Dose
IVT administration of a single low dose AGX-08 injection
Drug: AGX-08-L
rAAV2.AG-eNCRV intravitreal injection of low dose
Other Names:
  • Low dose
Experimental: Middle Dose
IVT administration of a single middle dose AGX-08 injection
Drug: AGX-08-M
rAAV2.AG-eNCRV intravitreal injection of middle dose
Other Names:
  • Middle dose
Experimental: High Dose
IVT administration of a single high dose AGX-08 injection
Drug: AGX-08-H
rAAV2.AG-eNCRV intravitreal injection of high dose
Other Names:
  • High dose
Sham Comparator: Control
Sham IVT injection
Procedure: AGX-08-S
sham intravitreal injection of AGX-08 (not actual injection)
Other Names:
  • Sham control

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
baseline to Week 52

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change in Fundus Autofluorescence (FAF)
Time Frame: baseline to Week 52
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.
baseline to Week 52
Change in best corrected visual acuity (BCVA)
Time Frame: baseline to Week 52
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.
baseline to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Zhongmou Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

July 1, 2026

Primary Completion (Estimated)

December 30, 2028

Study Completion (Estimated)

December 30, 2029

Study Registration Dates

First Submitted

May 17, 2026

First Submitted That Met QC Criteria

May 20, 2026

First Posted (Actual)

May 22, 2026

Study Record Updates

Last Update Posted (Actual)

May 22, 2026

Last Update Submitted That Met QC Criteria

May 20, 2026

Last Verified

May 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AGX-08

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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