An Open Label Phase 1 Study of ADVM-022 (AAV.7m8-aflibercept) in Neovascular (Wet) Age-Related Macular Degeneration
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 30
- 试验地点
- 12
- 主要终点
- Type, severity, and incidence of ocular and systemic adverse events (AEs)
研究概览
简要总结
ADVM-022 (AAV.7m8-aflibercept) is a gene therapy product developed for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is a serious condition and the leading cause of blindness in the elderly. The available therapies for treating wet AMD require life-long intravitreal (IVT) injections every 4-12 weeks to maintain efficacy. A one-time IVT administration of ADVM-022 has the potential to treat wet AMD by providing durable expression of therapeutic levels of intraocular anti-VEGF protein (aflibercept) and maintaining the vision of patients. ADVM-022 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with wet AMD receiving anti-VEGF therapy in clinical practice.
详细描述
This open-label, multicenter, dose-ranging study will evaluate 2 dose levels in up to 30 subjects (15 per dose) with active choroidal neovascularization (CNV) secondary to AMD. Subjects who are under active anti-VEGF treatment and have demonstrated a meaningful response to anti-VEGF therapy will be considered for participation in this study. The primary endpoint for this study is safety and tolerability of ADVM-022. All subjects will continue to be assessed for 104 weeks following treatment with ADVM-022.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
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
- •Diagnosis of neovascular (wet) AMD
- •BCVA ETDRS Snellen equivalent between ≤20/32 and ≥20/320 for each cohort
- •Subjects must be under active anti-VEGF treatment for wAMD and received a minimum of 2 injections within 4 months prior to screening
- •Demonstrated a meaningful response to anti-VEGF therapy
- •Willing and able to provide consent
排除标准
- •History of retinal disease in the study eye other than wet AMD
- •Fibrosis or atrophy, retinal epithelial tear in the center of the fovea in the study eye, or any condition preventing visual acuity improvement
- •History of retinal detachment (with or without repair) in the study eye
- •History of vitrectomy, trabeculectomy, or other filtration surgery in the study eye
- •Uncontrolled glaucoma in the study eye
- •Any prior treatment with photodynamic therapy or retinal laser for the treatment of wet AMD and any previous therapeutic radiation in the region of the study eye
- •Any previous intraocular or periocular surgery on the study eye within 6 months
- •Acute coronary syndrome, myocardial infarction or coronary artery revascularization, CVA, TIA in the last 6 months
- •Uncontrolled hypertension defined as average SBP ≥160 mmHg or an average DBP ≥100 mmHg
结局指标
主要结局
Type, severity, and incidence of ocular and systemic adverse events (AEs)
时间窗: 104 weeks
Type, severity, and incidence of ocular and systemic adverse events (AEs)
次要结局
- Change in central subfield thickness (CST) and macular volume measured by SD-OCT(104 weeks)
- Percentage of subjects without intraretinal fluid over time(104 weeks)
- Change in best corrected visual acuity (BCVA)(104 weeks)
- Mean number of anti-VEGF injections over time(104 weeks)
- Percentage of subjects requiring anti-VEGF injections over time(104 weeks)
- Percentage of subjects without subretinal fluid over time(104 weeks)