Phase I/II Study of the Safety and Preliminary Efficacy of Human Central Nervous System Stem Cells (HuCNS-SC) Subretinal Transplantation in Subjects With Geographic Atrophy of Age-Related Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 5
- 主要终点
- Number of subjects with adverse events
研究概览
简要总结
The purpose of this Phase I/II study is to investigate the safety and preliminary efficacy of unilateral subretinal transplantation of HuCNS-SC cells in subjects with geographic atrophy secondary to age-related macular degeneration.
详细描述
This study is an open-label dose-escalation investigation of the safety and preliminary efficacy of unilateral subretinal transplantation of HuCNS-SC cells in subjects with Geographic Atrophy secondary to Age-Related Macular Degeneration (AMD). Subjects will be enrolled based on specific inclusion/exclusion criteria and evaluated at regular post transplant intervals. The investigation will be divided into two sequential cohorts. Subjects will be enrolled into each cohort based on best-corrected visual acuity (BCVA) visual acuity as determined by the Electronic Early Treatment Diabetic Retinopathy Study (E-ETDRS) acuity test. BCVA of less than or equal to 20/400 in the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be enrolled in Cohort II. Cohort I will consist of four subjects who will undergo transplant with 200,000 cells followed by four subjects who will undergo transplant with 1 million cells. Cohort II will consist of 7 subjects who will undergo transplant with 1 million cells. An independent Data Monitoring Committee (DMC) will review accruing safety data for all subjects.
HuCNS-SC cells will be transplanted by a board-certified vitreoretinal surgeon. The transplantation will be conducted in the eye with the inferior best-corrected visual acuity (BCVA) (i.e., the Study Eye). Only the Study Eye will undergo transplantation. The HuCNS-SC cells will be administered into the subretinal space through a standard surgical approach.
Immunosuppressive agents will be administered orally to all subjects for a period of three months after surgery.
Subjects will be monitored frequently for a total of one year after HuCNS-SC cell transplantation. An additional four years of monitoring will be conducted in a separate follow-up study that will commence with the termination visit of the Phase I/II investigation. The follow-up study will be conducted as a separate investigation.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of age-related macular degeneration with geographic atrophy (GA)
- •Only patients with a specific degree and extent of GA will be eligible
- •Subjects with best-corrected visual acuity (BCVA) of less than or equal to 20/400 in the Study Eye will be enrolled in Cohort I. Subjects with BCVA of 20/320 to 20/100 in the Study Eye will be enrolled in Cohort II
- •No prior or current choroidal neovascularization in either eye
- •Must have adequate care-giver support and access to medical care in the local community
- •Able to provide written informed consent prior to any study related procedures
- •Agree to comply in good faith with all conditions of the study and to attend all required study visits
排除标准
- •Prior vitreal or retinal surgery
- •Atrophic macular disease of any other cause
- •Diabetic retinopathy or diabetic macular edema in either eye
- •Previous organ, tissue or bone marrow transplantation
- •Previous participation in a gene transfer or a cell transplant trial
- •Autoimmune disease
- •Allergy to tacrolimus, mycophenolate mofetil (MMF), scopolamine, Cyclogyl, Moxifloxacin, or Gatifloxacin
- •Current or prior malignancy (or is on chemotherapy)
研究组 & 干预措施
HuCNS-SC
HuCNS-SC cells
干预措施: HuCNS-SC cells (Drug)
结局指标
主要结局
Number of subjects with adverse events
时间窗: One year following transplant surgery
Descriptive analysis of frequency and types of adverse events experienced by each subject during the study period.
次要结局
- Assessment of visual function changes from baseline(At frequent intervals for one year following transplantation)