Eculizumab for the Treatment of Non-Exudative Age-Related Macular Degeneration: An Exploratory Study to Evaluate the Effects of C5 Inhibition on Drusen and Geographic Atrophy
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 60
- 试验地点
- 1
- 主要终点
- Decrease in Drusen Volume
研究概览
简要总结
To evaluate the safety and efficacy of eculizumab for the treatment of dry AMD as evaluated by the change in drusen volume and area of geographic atrophy.
详细描述
This is a randomized, double-arm, double-masked study designed to evaluate the safety and efficacy of eculizumab for the treatment of patients with dry AMD. There are three stages in the study: the screening period, the treatment period, and the follow-up period.During the screening period patients will be evaluated for eligibility. Eligible patients will receive either eculizumab or placebo for 24 weeks.
A total of 60 patients will be enrolled and divided equally between the drusen cohort and the GA cohort. A 2:1 randomization will result in 20 patients in each cohort receiving eculizumab while 10 patients receive placebo.
The treatment period will begin two weeks after administration of the meningococcal vaccine. During the treatment period, patients will receive eculizumab or placebo over a period of approximately 26 weeks. Patient will treatment according to the following regimen:
Induction Period: patient will receive eculizumab 600 mg or 900 mg via IV infusion over approximately 30 minutes once a week (7 ± 2 days) for 4 weeks followed by 900 mg eculizumab for the fifth dose 7 days later (7 ± 2 days).
Maintenance Period: patient will receive eculizumab 900 mg or 1200 mg via IV infusion over approximately 30 minutes every 2 weeks (14 ± 2 days).
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
- 50 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ability to provide written informed consent and comply with study assessments for the full duration of the study
- •Age > 50 years
- •In the study eye(s), the presence of non-exudative AMD documented by fundus photography, autofluorescence, fluorescein angiography, and spectral domain OCT.
- •Visual acuity of 20/63 or better (BCVA score of at least 59 letters) as measured on an ETDRS chart.
- •Able and willing to comply with study procedures.
排除标准
- •Visual acuity worse than 20/63
- •Any history of choroidal neovascularization in the study eye
- •Unresolved meningococcal disease.
- •Confounding ocular conditions such as amblyopia; aphakia; myopia requiring >6 diopters of correction; pigment epithelial detachment; uncontrolled glaucoma (intraocular pressure ≥ 25 mmHg despite treatment with anti-glaucoma medication); steroid-induced ocular hypertension; retinal inflammatory disease; central serous choroidopathy; prior or current retinal detachment; macular edema; cystic lesion (individual cysts or cystoid macular edema); ocular herpes simplex virus; severe non-proliferative or worse diabetic retinopathy; anterior ischemic optic neuropathy; RPE tear involving the macula; pseudovitelliform macular degeneration; vitreo-retinal traction maculopathy; vitreous hemorrhage, history of or current rhegmatogenous retinal detachment or macular hole; uveitis; diffuse choroidal atrophy; optic atrophy (as evidenced by pallor); intraocular inflammation; ocular or periocular infection; moderate or worse dry eye syndrome; clinically significant cataract or opacification of the posterior capsule which, in the Investigator's opinion, would progress during the course of the study and could affect central vision; other ocular conditions that the Investigator believes may be a confounding factor in this study
- •Refusal to be vaccinated against Neisseria meningitides or an active Neisseria meningitides infection
研究组 & 干预措施
Saline
Randomized patients in the drusen or the GA cohort will receive placebo saline infusions as a comparator
干预措施: Saline (Drug)
Eculizumab
Randomized patients in the drusen or the GA cohort will receive active treatment with eculizumab
干预措施: Eculizumab (Drug)
结局指标
主要结局
Decrease in Drusen Volume
时间窗: 6 Months
Growth of Geographic Atrophy
时间窗: 6 months
次要结局
- Change in Visual Acuity for Drusen Group(Baseline/ 6 Months)
- Change in Visual Acuity for Geographic Atrophy Group(Baseline/ 6 Months)
研究者
Philip J. Rosenfeld, MD, PhD
Professor of Ophthalmology
University of Miami