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

Korábbi kezelés

• Aphakia in the study eye, or cataract surgery/YAG capsulotomy within 3 months prior to screening;
• 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;
• Major ocular surgery within 3 months prior to screening;
• 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;
• 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;

Más betegség

• GA caused by conditions other than AMD (e.g., Stargardt disease, cone-rod dystrophy, or other inherited macular dystrophies);
• Active ocular diseases in the study eye, including inflammation, other macular diseases, glaucoma, ocular hypertension, or acute/chronic ocular infections;
• History of retinal detachment or other fundus diseases unsuitable for study participation;
• 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);
• Any condition that may compromise scientific evaluation of the study;
• Any other condition deemed unsuitable by the investigator.

Életkor

• 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.;

Egyéb

• 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%;
• Narrow anterior chamber angle or other contraindications to pupil dilation;
• Any contraindication to intravitreal (IVT) injection;

• 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.