NCT01625559已完成1 期
A Phase I, Open-Label, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy(SMD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- safety and tolerance of transplantation
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
详细描述
- to evaluate the safety and tolerability of RPE cellular therapy in patients with SMD
- to evaluate the preliminary efficacy of MA09-hPRE in patients with SMD
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult male or female over 20 years of age.
- •Clinical diagnosis of advanced SMD.
- •The visual acuity of the eye to receive the transplant will be no better than hand movement.
- •The visual acuity of the eye that is not to receive the transplant will be no better than 24 (20/320) Early Treatment of Diabetic Retinopathy Study (ETDRS) letters.
排除标准
- •History of malignancy.
- •History of myocardial infarction in previous 12 months.
- •History of diabetes mellitus.
- •Any immunodeficiency.
- •Any current immunosuppressive therapy other than intermittent or low dose cortico steroids.
- •Serologic evidence of infection with Hepatitis B, Hepatitis C, or HIV.
- •Current participation in any other clinical trial.
- •Participation within previous 6 months in any clinical trial of a drug by ocular or systemic administration.
- •Any other sight-threatening ocular disease.
- •Any chronic ocular medications. Any history of retinal vascular disease (compromised blood-retinal barrier). Glaucoma. Uveitis or other intraocular inflammatory disease. Significant lens opacities or other media opacity. Ocular lens removal within previous 3 months
结局指标
主要结局
safety and tolerance of transplantation
时间窗: 18 months
The transplantation of hESC-derived RPE cells MA09-hRPE will be considered safe and tolerated in the absence of: 1. Any grade 2 (NCI grading system) or greater adverse event related to the cell product 2. Any evidence that the cells are contaminated with an infectious agent 3. Any evidence that the cells show tumorigenic potential
次要结局
- Evidence of successful engraftment(18 months)
研究者
研究点 (2)
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