A Phase I/II Controlled Dose-escalating Trial to Establish the Baseline Safety and Efficacy of a Single Subretinal Injection of rAAV.sFlt-1 Into Eyes of Patients With Exudative Age-related Macular Degeneration (AMD)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection
研究概览
简要总结
The study will involve approximately 40 subjects aged 55 or above who have exudative age-related macular degeneration (wet AMD). Patients will be randomized to receive one of two doses of rAAV.sFlt-1 or assigned to the control group.
详细描述
A new treatment for exudative age-related macular degeneration (wet AMD) is being investigated. The purpose of this Phase I/II clinical research study is to examine the baseline safety and efficacy of an experimental study drug to treat a complication of the disease which leads to vision loss. The name of the study drug is rAAV.sFlt-1.
This experimental study uses a non-pathogenic virus to express a therapeutic protein within the eye. The therapeutic diminishes the growth of abnormal blood vessels under the retina. The duration of effect is thought to be long-term (years) following a single administration.
The clinical research study will look at the baseline safety and efficacy of a single injection of rAAV.sFlt-1 injected directly into the eye.
Approximately forty (40) subjects will participate in Australia. The primary endpoint of the study is at one month, with extended follow up for 3 years.
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
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 55 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age greater than or equal to 55 years;
- •Subfoveal CNV secondary to AMD and with best corrected visual acuity of 3/60 - 6/9 with 6/60 or better in the other eye;
- •Fluorescein angiogram of the study eye must show evidence of a leaking subfoveal choroidal neovascular lesion, or CNV currently under active management with anti-VEGF therapy;
- •Must be a candidate for anti-VEGF intravitreal injections;
- •No previous retinal treatment of photodynamic therapy or laser;
- •Able to provide informed consent;
- •Able to comply with protocol requirements, including follow-up visits.
排除标准
- •Liver enzymes > 2 X upper limit of normal;
- •Any prior treatment for AMD in the study / control eye, excluding anti-VEGF injections;
- •Extensive sub-foveal scarring, extensive geographic atrophy, or thick subretinal blood in the study eye as determined by the investigator;
- •Significant retinal disease other than sub-foveal CNV AMD;
研究组 & 干预措施
Control - ranibizumab only
干预措施: Control (ranibizumab alone) (Other)
Low Dose rAAV.sFlt-1
干预措施: rAAV.sFlt-1 (Biological)
High Dose rAAV.sFlt-1
干预措施: rAAV.sFlt-1 (Biological)
结局指标
主要结局
No sign of unresolved ophthalmic complications, toxicity or systemic complications as measured by laboratory tests from 1 month post injection
时间窗: Primary endpoint at 1 month
1. Ocular examination: * Ocular inflammation * Intraocular pressure * Visual acuity * Retinal bleeding 2. Abnormal laboratory data
次要结局
- Maintenance or improvement of vision without the necessity of ranibizumab re-injections(Up to 3 years)
研究者
Prof. P. Elizabeth Rakoczy
Principal Scientific Investigator
Lions Eye Institute, Perth, Western Australia