NCT07602127尚未招募1 期
Prospective, Dose-Escalating, Investigator Initiated Trial to Evaluate the Safety and Efficacy of AGX-08 in Geographic Atrophy
Zhongmou Therapeutics0 个研究点目标入组 24 人开始时间: 2026年7月1日最近更新:
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 24
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Care Provider)
入排标准
- 年龄范围
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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);
- •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.
研究组 & 干预措施
Low Dose
Experimental
IVT administration of a single low dose AGX-08 injection
干预措施: AGX-08-L (Drug)
High Dose
Experimental
IVT administration of a single high dose AGX-08 injection
干预措施: AGX-08-H (Drug)
Control
Sham Comparator
Sham IVT injection
干预措施: AGX-08-S (Procedure)
研究者
相似试验
招募中
1 期
Safety and Efficacy of GL-2045 in Patients With Immune ThrombocytopeniaImmune ThrombocytopeniaNCT07605845Gliknik Inc.42
招募中
2 期
A Phase 2a Trial to Investigate Safety, Tolerability and Exploratory Efficacy of AGP100 in Patients with Catecholaminergic Polymorphic Ventricular Tachycardia2025-521611-39-00Agiana Pharmaceuticals AS10
招募中
1 期
Safety and Efficacy of GYA01 (CART84) in Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML) and Acute Lymphoblastic T Leukemia Patients (T-ALL).NCT07471789Gyala Therapeutics33
招募中
2 期
A Study to Investigate Efficacy and Safety of FWY003 Compared With Placebo in Participants With Geographic Atrophy Secondary to Age-related Macular DegenerationNCT07441642Novartis Pharmaceuticals272
已完成
1 期
An Ethnobridging TriaL of AGA2118 in Healthy Japanese, Chinese, and Caucasian ParticipantS (ATLAS)NCT07361354Angitia Incorporated Limited28