Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 1
- 主要终点
- Number of subjects with evidence of graft failure or rejection.
研究概览
简要总结
To evaluate the safety of 3 regimens of short-term, low-dose systemic IMT as rejection prophylaxis prior to and/or following transplant of MA09-hRPE cells.
详细描述
This study will be a Phase 2, double-masked, randomized, parallel group, sham surgery/placebo control, multi-center trial. Subjects will be randomized in a 3:1 ratio to either the treatment or control group respectively. Subjects randomized to the treatment group will receive transplantation with 200,000 MA09-hRPE (human embryonic stem cell derived retinal pigmented epithelial)cells in one eye. Subjects randomized to the control group will have a sham surgery without transplantation of MA09-hRPE cells. The study eye must meet all eligibility criteria. If both eyes meet all eligibility criteria, then the study eye will be the eye with the worst Best Corrected Visual Acuity (BCVA) score at screening. If both eyes have identical BCVA scores, then the study eye will be chosen by the Investigator and the subject. There will be 3 cohorts, each with a different regimen of low-dose IMT [tacrolimus and mycophenolate mofetil (MMF)]. Subjects will be randomized to treatment or control within cohorts, defined by severity of BCVA in the study eye at Screening. Enrollment in Cohort 1 and 2 will be concurrent. Enrollment into Cohort 3 will begin once Cohort 2 is fully enrolled.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 50 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Geographic atrophy (GA) secondary to AMD with no evidence of prior to active choroidal neovascularization (CNV) in the study eye.
- •BCVA in the study eye must be between 4 and 58 Early Treatment of Diabetic Retinopathy Study (EDTRS) letters (20/800 to 20/80; 0.025-0.25)
- •Subjects must be willing to take IMT and willing to discontinue any medication that has a known strong interaction with tacrolimus or mycophenolate mofetil (MMF)
排除标准
- •Macular atrophy due to causes other than AMD
- •Other sight-threatening ocular disease
- •Current or prior history of optic neuropathy, retinal dystrophy, retinitis pigmentosa, chorioretinitis, vasoocclusive disease, retinal vascular disease or retinal degenerative disease OTHER than AMD
- •History of uveitis
- •History of allergic reaction to sulfa drugs
- •Solid organ or bone marrow transplant recipient
- •History of malignancy within the previous 5 years (except for BCC (basal cell carcinoma), SCC (squamous cell carcinoma) or in-situ cervical)
- •History of myocardial infarction with past 12 months
- •History of clinically significant cardiac dysrhythmia
- •History of diabetes mellitus, bowel disease, tuberculosis
- •Prior treatment for non-exudative AMD
- •Intraocular, refractive or cataract surgery in the last 12 weeks
- •Prior retinal surgery, vitrectomy, macular laser photocoagulation, external beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery (except cataract surgery)
- •Receipt of gene transfer of cell transplant therapy in a prior clinical trial
- •Participation in any other interventional clinical trial within the last 12 weeks
研究组 & 干预措施
Control group
Sham surgery and placebo immunosuppressive therapy (IMT)
干预措施: Placebo tacrolimus and mycophenolate mofetil (Drug)
Control group
Sham surgery and placebo immunosuppressive therapy (IMT)
干预措施: Sham Surgery (Procedure)
Active Group
Sub-retinal transplantation of MA09-hRPE cells and IMT
干预措施: Sub-retinal transplantation of MA09-hRPE cells (Biological)
Active Group
Sub-retinal transplantation of MA09-hRPE cells and IMT
干预措施: tacrolimus and mycophenolate mofetil (Drug)
结局指标
主要结局
Number of subjects with evidence of graft failure or rejection.
时间窗: 18 months
次要结局
- Change in area of geographic atrophy by Autofluorescence(18 months)
- Change in area of geographic atrophy by Optical coherence tomography(18 months)
- Change in average Best Corrected Visual Acuity (BCVA)(18 months)