A Phase I/II Clinical Study Evaluating the Safety and Preliminary Efficacy of EXG202 in Patients With Neovascular Age-related Macular Degeneration (nAMD)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 60
- 主要终点
- Safety and tolerability after EXG202 injection
研究概览
简要总结
VEGF inhibitors (anti-VEGF),such as aflibercept has been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients.
EXG 202 is a recombinant adeno-associated virus (rAAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit.
详细描述
This Phase I/II study was designed to evaluate the safety and Preliminary Efficacy of EXG202 gene therapy in subjects with nAMD. Subjects who met the inclusion/exclusion criteria and had response to an initial anti-VEGF injection received a single dose of EXG202 administered . Safety was the primary focus for the phase I/II trial ,at the same time ,preliminary Efficacy also is another goal for the trial.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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
- •Male or female, aged ≥50 years old;
- •The study eye must be diagnosed of wAMD and current active lesions;
- •Subjects have clear refractive media and sufficient pupil dilation at the time of screening to obtain high-quality retinal images for confirmation of diagnosis;
- •Subjects (including male subjects) have no pregnancy plans during the screening period and the entire trial period and voluntarily take effective contraceptive measures and have no sperm or egg donation plans;
- •Subjects are Voluntarily participate in this clinical trial, understand the research procedures and sign the informed consent form before screening; subjects have good compliance and are willing to abide by the research procedures.
排除标准
- •The study eye has any eye disease other than wAMD that may affect central vision and/or macular detection ;
- •The study eye has a history of retinal detachment or retinal detachment during the screening period;
- •The study eye has MNV caused by reasons other than wAMD (such as diabetic retinopathy, pathological myopia, retinal vein occlusion, angioid streak disease, ocular histoplasmosis, trauma, etc.), and a history of macular pathology unrelated to wAMD;
- •Any intraocular surgery is planned for the study eye during the study period;
- •The study eye currently has retinal angiomatous proliferation (RAP), central serous chorioretinopathy or symptomatic vitreomacular traction syndrome;
- •Presence of glaucoma or optic neuropathy that involves or compromises the central visual field of the study eye or presence of uncontrolled high intraocular pressure in the study eye；
- •The fellow eye meets the definition of legal blindness；
- •Hormone-induced increased intraocular pressure in any eye；
- •Active infection in any eye this trial (such as those who cannot understand and comply with the trial requirements or are deemed unsuitable for safety reasons).
研究组 & 干预措施
Dose escalation-Cohort 1
Dose 1 :Administered via intravitreal injection
干预措施: EXG202 injection (Biological)
Dose escalation-Cohort 2
Dose 2 :Administered via intravitreal injection
干预措施: EXG202 injection (Biological)
Dose escalation-Cohort 3
Dose 3 :Administered via intravitreal injection
干预措施: EXG202 injection (Biological)
Dose escalation-Cohort 4
Dose 4 :Administered via intravitreal injection
干预措施: EXG202 injection (Biological)
结局指标
主要结局
Safety and tolerability after EXG202 injection
时间窗: Up to 52 weeks after treatment
Type, severity, and incidence of adverse events (AEs) and serious adverse events (SAEs) from Week 0 to 52, and dose limited toxicity
Preliminary Efficancy after EXG202 injection
时间窗: Up to 52 weeks after treatment
The Best Corrected Visual Acuity (BCVA) change from baseline
次要结局
- Safety and tolerability after EXG202 injection(Up to 24 weeks after treatment)