A Phase 1/ Phase 2 Study Evaluating the Safety and Efficacy of the Investigational New Drug TTHX1114(NM141) on the Regeneration of Corneal Endothelial Cells in Patients With Corneal Endothelial Dystrophies Following Intracameral Delivery
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 8
- 主要终点
- DLTs/ Adverse Reactions
研究概览
简要总结
Prospective, multicenter, randomized, masked, vehicle-controlled, dose-escalation study
详细描述
This is a prospective, multi-center, randomized, masked, vehicle-controlled, dose-escalation study that will include an observational (no intervention) sub-study. Eligible subjects with moderate to severe corneal endothelial dystrophy (defined as Endothelial Cell Density < 2000 mm^2) in at least one eye will be enrolled and randomized.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fuchs Endothelial Corneal Dystrophy, pseudophakic bullous keratopathy, or endothelial dysfunction/ insufficiency due to surgical intervention diagnosed more than 6 months prior to Study Day 0
- •Central endothelial cell count of < 2000 mm^2 in at least one eye as determined by the central reading facility
排除标准
- •Conditions that would impair examination of the anterior chamber structure
- •Documented repeated elevated intra ocular pressure (in either eye)
- •Corneal transplant (in either eye)
- •Posterior Polymorphous Corneal Dystrophy (PPCD)
- •History of uveitis or herpetic keratitis
- •Cataract surgery within the past 3 months
- •Refractive surgery (in the Study Eye)
- •Anterior Chamber IOL placement (in the Study Eye)
- •Active extra-ocular inflammation from any non-infectious or infectious cause within the past 6 months
- •Expected or planned ocular surgery within the next 3 months
- •Use of cytotoxic chemotherapy within the last 1 month
- •Treatment with a rho kinase inhibitor within the last 3 months
- •Use of cyclosporine ophthalmic emulsion or lifitegrast ophthalmic solution in the last 30 days
- •Systemic or ophthalmic corticosteroid use in the 30 days prior to Study Day 0 unless approved by the Medical Monitor
- •History of significant allergy, hypersensitivity, or intolerance to any drug compound, food, or other substance
- •Unwilling to use birth control
研究组 & 干预措施
Low dose
TTHX1114(NM141) low-dose weekly x 4
干预措施: TTHX1114(NM141) (Drug)
Vehicle (placebo)
Placebo weekly x 4
干预措施: Vehicle (placebo) (Other)
Mid-dose
TTHX1114(NM141) mid-dose weekly x 4
干预措施: TTHX1114(NM141) (Drug)
High-dose
TTHX1114(NM141) high-dose weekly x 4
干预措施: TTHX1114(NM141) (Drug)
结局指标
主要结局
DLTs/ Adverse Reactions
时间窗: Day 90
Suspected Adverse Reactions Reported Following Study Drug Adminsitration
次要结局
未报告次要终点