A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 332
- 试验地点
- 1
- 主要终点
- Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
研究概览
简要总结
Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF).
详细描述
The study will assess the rate of change of the area of GA secondary to AMD by measuring FAF in up to 330 participants with GA due to AMD being treated with IONIS-FB-LRx. The study is a Phase 2, double-masked, randomized, stratified, placebo-controlled study conducted at multiple centers. It is an adaptive design in which three dose levels will be evaluated in a subset of participants (Stage 1) and, following an interim analysis, the number of participants in two of the dose cohorts will be expanded (Stage 2).
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
- •Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
- •Well-demarcated geographic atrophy (GA) due to AMD
- •Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
- •Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging
排除标准
- •Clinically-significant abnormalities in medical history
- •A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
- •Chronic treatment with steroids, including topically or intravitreally administered
- •History or presence of diabetic retinopathy or diabetic macular edema (DME)
- •History or presence of a disease other than AMD that could affect vision or safety assessments
- •Prior treatment with another investigational drug, biological agent, or device
- •Other protocol-specified inclusion/exclusion criteria may apply
研究组 & 干预措施
IONIS-FB-LRx
Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
干预措施: IONIS-FB-LRx (Drug)
Placebo
Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.
干预措施: Placebo (Drug)
结局指标
主要结局
Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
时间窗: Week 49
次要结局
- Percentage Change from Baseline in Levels of Factor B (FB) in Plasma(Baseline and up to Week 49)
- Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)(Baseline and up to Week 49)
- Absolute change in the GA area from Baseline as measured by Fundus Autofluorescence (FAF)(Baseline and up to Week 57)
- Percentage Change from Baseline in Levels of Serum AH50 Activity(Baseline and up to Week 49)