Safety and Efficacy of Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium in the Treatment of Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Safety measure
研究概览
简要总结
This project intends to perform autologous transplantation of induced pluripotent stem cell-derived retinal pigment epithelium (iPSC-RPE). The clinical-grade RPE will be transplanted into subretinal space to treat refractory age-related macular degeneration. The efficacy and safety of RPE transplants to treat macular degeneration will be monitored and analyzed with results from EDTRS, BCVA, OCT, ERG, microperimetry, and fluorescein angiography, before and after the treatment.
详细描述
The investigators will generate iPSC lines from recruited participants and differentiate the iPSC into RPE. Autologous iPSC-derived RPE will be transplanted into participants eyes by subretinal injections. The safety and efficacy will be closely monitored and analyzed.
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
- 50 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 50-75 years;
- •Clinical diagnosis is consistent with the definition of late dry AMD in the age-related eye disease study (AREDS), with one or more >250 um geographic atrophy in the fovea;
- •Clinical diagnosis is wet AMD, but no obvious efficacy after conventional treatment;
- •The BCVA of the target eye will be 0.05 to 0.3;
- •Voluntary as test subjects, informed consent, regular follow-up on time.
排除标准
- •One-eyed subjects;
- •Macular atrophy caused by other diseases in addition to AMD;
- •Suffer from retinitis pigmentosa, choroidal retinitis, central serous choroiditis, diabetic retinopathy, or other retinal vascular and degenerative diseases besides AMD;
- •Lens opacities (affecting the central vision), glaucoma, uveitis, retinal detachment, optic neuropathy, and other ocular histories;
- •Other intraocular surgery histories besides cataract surgery;
- •Combined with severe systemic diseases, such as heart failure, liver disease, renal insufficiency, cor pulmonale, COPD in the previous 12 months;
- •Combined with severe infectious diseases, such as HIV, HBV, HCV, syphilis, tuberculosis, etc;
- •Abnormal blood coagulation function or other laboratory tests;
- •If female and of childbearing potential, pregnant, breastfeeding, or planning to become pregnant through the study;
- •If male, refuse to use barrier and spermicide contraception during the study;
- •Malignant tumor and history of malignancy;
- •Any immune deficiency;
- •Allergy to tacrolimus or other macrolides;
- •Any immune deficiency;
- •Use glucocorticoids, immunosuppressive drugs, or antipsychotic drugs in the previous 3 months;
- •Use anticoagulant, or the platelet function is still not restored to normal after stopping antiplatelet drugs for 10 days;
- •A history of addiction to alcoholism or prohibited drugs;
- •Be participating in other intervention clinical trials or receiving other study medications;
- •Poor compliance, difficulty to complete the study, or refusal to informed consent;
- •Some other situations which might increase the risks of the subjects or interfere with clinical trials, such as mental disorders, cognitive dysfunction, etc.
结局指标
主要结局
Safety measure
时间窗: 12 months
Safety will be assessed by Adverse Events (AEs) of special interest in regards to the investigational product. This will include obtaining information about Serious Adverse Events (SAEs) that are neurologic, infectious, hematologic or fatal, any AE that causes the subject to withdraw from the study, any new diagnosis of an ocular or immune-mediated disorder, cancer (irrespective of prior history), ectopic or proliferative cell growth (RPE or non-RPE) with adverse clinical consequence, unexpected, clinically significant AE possibly related to the cell transplant procedure or the investigational product (autologous iPSC-RPE), pregnancy in a female subject or the partner of a male subject and pregnancy outcome.
次要结局
- Best Corrected Visual Acuity (BCVA)(12 months)
- Optical coherence tomography (OCT) imaging(12 months)
- Electroretinography (ERG)(12 months)
- Color and autofluorescence imaging(12 months)
- Fundus autofluorescence(12 months)
- Fluorescein angiography(12 months)
- Microperimetry(12 months)