A Multicenter, Randomized, Sham-control, Proof-of-concept Study of Intravitreal LFG316 in Patients With Geographic Atrophy Associated With Age-related Macular Degeneration
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 158
- 试验地点
- 1
- 主要终点
- Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505
研究概览
简要总结
This study was conducted in two parts; Part A and Part B: Part B was initially planned to include two cohorts. Cohort 2 was cancelled following an interim analysis for efficacy in Part A of the study, and not due to any safety issues or concerns. Cohort 2 is not referred to again and part B cohort 1 is referred to as part B alone in the remainder of the document and is the subject of this report.
Part B was conducted to assess the safety and tolerability of a single intravitreal (IVT) LFG316 10 mg/100 µL injection. There was no efficacy evaluation in Part B. The study employed a multicenter, randomized, sham - controlled, single masked design. Eight patients with advanced AMD were planned to be randomized in a 3:1 ratio to receive a single IVT dose of LFG316 (10 mg/100 µL) or sham injection. Patients assigned to a sham injection were treated the same as those assigned to LFG316, except that the hub of an empty syringe (without needle) was placed against the eye instead of the IVT injection.
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
- Single (Participant)
入排标准
- 年龄范围
- 55 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of AMD if enrolled in Part B of study
- •Geographic atrophy in at least one eye if enrolled in Part A of study
- •ETDRS best corrected visual acuity of 60 letters or worse (~≤ 20/63)
排除标准
- •Retinal disease other than AMD
- •History of choroidal neovascularization
- •Severe cataract
- •History of infectious uveitis or endophthalmitis
- •Eye surgery in the non-study eye within 30 days prior to study
- •Eye surgery or IVT injection in the study eye within 90 days prior to study
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
LFG316 higher dose
LFG316 10 mg/100 μL
干预措施: LFG316 (Drug)
Sham
Sham injection
干预措施: Sham (Drug)
LFG316 lower dose
LFG316 5 mg/ 50 μL
干预措施: LFG316 Lower dose (Drug)
结局指标
主要结局
Part A: Geographic Atrophy (GA) Lesion Growth Measured by Fundus Autofluorescence (FAF) From Baseline to Day 505
时间窗: Day 1 to Day 505 (starting from the day of first intravitreal injection until Day 505)
Geographic atrophy (GA) lesion growth measured by fundus autofluorescence (FAF) from baseline to Day 505.
Part A: Sensitivity Analysis of the Primary End Point: Mixed Effects Model for Repeated Measurements on GA Lesion Growth Measured by Fundus Autoflourescence
时间窗: The primary objective was from Day 1 to Day 337, however data was captured to Day 505 as exploratory objective
Number is the Estimated Difference (95% CI) in lesion size.
Part B: Safety and Tolerability of a Single Intravitreal (IVT) Dose of 10 mg/100 μL of LFG316 in Patients With Advanced AMD).
时间窗: Day 1 to Day 85
This primary outcome (for Part B) is reported under the Adverse Events section.
次要结局
- Part A: Change in Best Corrected Visual Acuity (BCVA) as Measured by the EDTRS (Early Treatment of Diabetic Retinopathy Study) Scale From Baseline to Days 169, 337 & 505 in Patients Receiving Every 28 Days, Successive IVT Doses of LFG316 Compared to Sham(Baseline Day 1, Day 169, Day 337 to Day 505)
- Part A: Change From Baseline in GA Lesions Growth Measured by Fundus Autofluorescence(Day 1 to Day 169 and Day 505 (starting from the day of first intravitreal injection until Day 505))
- Part A: Summary of Best Corrected Visual Acuity Over Time, Statistical Analysis of Change in Best Corrected Visual Acuity Over Time Parameter: Visual Acuity (EDTRS Letter) BCVA Scale is 0-100, Worst is 0 and Best 100 Eye: FELLOW(Baseline Day 1, Day 169, Day 337 to Day 505)
- Part A: Concentrations of Total C5 in Blood During the Course of the Study(Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559))
- Part B: AUC (Area Under the Curve) - Summary Statistics for PK Parameters(Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85))
- Tmax (hr)(Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85))
- Part A: Concentrations of Total LFG316 in Blood During the Course of the Study(Day 1 to Day 559 (starting from the day of first intravitreal injection to day 559))
- Part B: Cmax - Summary Statistic for PK Parameters(Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85))
- Part B: Cmax_D - Summary Statistic for PK Parameters(Day 1 to Day 85 (starting from the day of first intravitreal injection to day 85))