Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
试验速览
- 阶段
- 早期 1 期
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Number of subjects with adverse events
研究概览
简要总结
Early Phase I Study of the Safety and Preliminary Efficacy of Human Fetal Retinal Pigment Epithelial(fRPE) Cells Subretinal Transplantation in Age-Related Macular Degeneration(AMD) Patients
详细描述
This study is a perspective, single-arm and open-labeled investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of fRPE cells in subjects with dry Age-Related Macular Degeneration and non-exudative AMD. The investigators will recruit and enroll 6 patients based on specific inclusive/exclusive criteria. Experimental and self-controlled eye will be determined based on best-corrected visual acuity (BCVA). The eye with BCVA of no more than 20/400 will be determined as experimental eye, which will be divided into 3 groups and undergo subretinal injection of 3 different dosages of fRPE cells (100.000, 200,000 or 500,000) respectively, while the other one (BCVA of more than or equal to 20/400) as control eye, will not receive the surgery.
fRPE cells will be obtained from the fetuses aborted in the Department of Obstetrics and Gynecology in Jiangsu Province Hospital from 2015 to 2018. The obtained fRPE cells will meet specific inclusive/exclusive criteria and conform to Good manufacturing practices (GMP). fRPE will be transplanted by a board-certified vitreoretinal surgeon, which will be administered into the subretinal space of experimental eye through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects after transplantation. Dosage and time duration of immunosuppressive agents will be regulated strictly relying on the condition of immune rejection. Subjects will be monitored with ophthalmologic and systemic examinations frequently at regular post-transplant intervals after fRPE cell transplantation.
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 90 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Diagnosis of dry AMD or non-exudative AMD (geographical atrophy>250μm, involving central fovea of macular; or AMD with fibrous disciform scar and maintained stationary for at least 3 months)
- •Experimental eye with best-corrected visual acuity (BCVA) of no more than 20/400 and control eye with BCVA of more than or equal to 20/400
- •Patients are in good state
- •Patient understand and sign the consent form.
排除标准
- •Blood routine examination is abnormal (hemoglobin<10gm/dL; blood platelet count<100k/mm³; neutrophil count<1000/mm³)
- •Blood biochemistry is abnormal (ALT／AST>1.5; creatinine>1.3mg/dL)
- •Experimental eye has optic nerve atrophy caused by glaucoma
- •Patients need cataract surgery within a year
- •Patients have received cataract surgery and other ocular surgery in recent 3 months
- •Experimental eye has retinal detachment, or has received retinal detachment surgery.
- •Patients with uveitis and other endophthalmitis
- •Patients with other ocular disease affecting vision.
- •Patients have participated in clinical study of ocular or systemic drug use in recent 6 months.
- •Patients with medical history of malignant cancer (except resected basal cell carcinoma and squamous-cell carcinoma).
- •Patients with medical history of myocardial infarction
- •Patient with diabetes
- •Patient with Parkinson disease or Alzheimer's disease
- •Patients are under the treatment of immunosuppressive agent (except intermittent, low-dose corticosteroid treatment).
- •Patients with other medical conditions that restricts the compliance and safety of patients, or affects experimental results.
研究组 & 干预措施
fRPE cells
Subretinal transplantation of fRPE cells in experimental eye
干预措施: fRPE cells (Drug)
结局指标
主要结局
Number of subjects with adverse events
时间窗: Two years following transplantation
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
次要结局
- Assessment of standardized ETDRS acuity testing(Two years following transplantation)
- Assessment of visual function changes(Two years following transplantation)
- Assessment of systemic condition(Two years following transplantation)