A Phase II Study to Evaluate the Safety and Preliminary Efficacy of Subretinal Administration of JWK001 in Patients With Neovascular Age-Related Macular Degeneration (nAMD)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 16
- 主要终点
- Change from baseline in best-corrected visual acuity (BCVA)
研究概览
简要总结
This is a Phase II, randomized, open-label, active-controlled study designed to evaluate the preliminary efficacy and safety of a single subretinal administration of JWK001 at different dose levels in patients with neovascular age-related macular degeneration.
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
- None
入排标准
- 年龄范围
- 50 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent and ability to comply with the study protocol.
- •Male or female, aged 50-85 years.
- •Active subfoveal MNV secondary to nAMD requiring anti-VEGF treatment.
- •Demonstrated clinical response to aflibercept treatments in the study eye confirmed by the Reading Center;
- •Study-eye BCVA of 19-78 ETDRS letters.
- •Pseudophakic study eye.
排除标准
- •Any ocular condition other than nAMD that may affect vision, study assessments, treatment safety, or interpretation of results.
- •Intraocular surgery or intraocular corticosteroid treatment within 4 weeks at screening.
- •Any ocular condition making the study eye unsuitable for subretinal injection.
- •Active ocular infection or inflammation, or a history of idiopathic or autoimmune uveitis.
- •Monocular status or non-study-eye BCVA <19 ETDRS letters.
- •Clinically significant systemic disease or infection, malignancy, prohibited concomitant treatment, prior gene therapy, or recent participation in another clinical trial.
- •Any other condition considered by the investigator to make the subject unsuitable for participation.
研究组 & 干预措施
Aflibercept Active Comparator Group
Participants assigned to this arm will receive aflibercept 2 mg by intravitreal injection at Weeks 2, 10, 18, 26, 34, 42, and 50 after randomization.
干预措施: Aflibercept (2.0 mg) (Drug)
JWK001 6×10^10 vg/eye group
Participants assigned to this arm will receive a single subretinal injection of JWK001 at a dose of 6 × 10^10 vector genomes (vg) in one study eye.
干预措施: JWK001 (Drug)
JWK001 3×10^10 vg/eye group
Participants assigned to this arm will receive a single subretinal injection of JWK001 at a dose of 3 × 10^10 vector genomes (vg) in one study eye.
干预措施: JWK001 (Drug)
结局指标
主要结局
Change from baseline in best-corrected visual acuity (BCVA)
时间窗: Week 34
BCVA measured via the Early Treatment Diabetic Retinopathy Study (ETDRS) letter score
次要结局
- Annualized mean number of rescue treatments(Week 34, Week 50 and Year 5)
- Change from baseline in BCVA(Week 50 and Year 5)
- Change from baseline in central retinal thickness (CRT)(Week 34, Week 50 and Year 5)
- Change from baseline in the area of Macular Neovascularization (MNV)(Week 34 and Week 50)
- Change from baseline in leakage area(Week 34 and Week 50)
- Change from baseline in the 25-item National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) score(Week 34 and Week 50)
- Frequencies and severities of all safety events(Week 34 and Year 5)
研究者
Fang Lu
Professor and Chief Physician
West China Hospital