A Multicenter, Randomized，Controlled，Phase 3 Clinical Trial to Evaluate the Efficacy and Safety of ZVS101e Administered as a Single Subretinal Injection in Subjects With Bietti's Crystalline Dystrophy (BCD)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 62
- 试验地点
- 8
- 主要终点
- Proportion of study eyes with a ≥15-letter improvement from baseline in BCVA
研究概览
简要总结
This is a multi-center, randomized and controlled phase 3 clinical trial.
详细描述
Subjects who have signed the written informed consent form and who were eligible for enrollment after screening will be randomly assigned to the treatment group or untreated control group. Each Participant in the treatment group will receive a unilateral subretinal injection of ZVS101e in the study eye.
Participants in the control group will receive no treatment during the first 52 weeks of enrollment.
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 (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All of the following inclusion criteria should be met for enrollment into the trial:
- •Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial procedures as required by the protocol;
- •Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age≥18 years ;
- •Genetic testing confirmed biallelic CYP4V2 mutations without other ophthalmic genetic diseases;
- •Best-corrected visual acuity of 5-60 ETDRS letters.
排除标准
- •The study eye has or has had macular lesions such as macular hole or macular neovascularization; glaucoma, diabetic retinopathy, or any other ocular disease that may preclude surgery or interfere with interpretation of the study endpoints
- •The study eye had received the following intraocular surgical treatments: retinal reattachment, vitrectomy;
- •The study eye had received any intraocular surgery, such as phacoemulsification 3 months prior to enrollment;
- •Previously treatment of either eye with gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to viral vector gene therapy, RNA therapy;
- •Pregnant or lactating women;
研究组 & 干预措施
Treatment group
Each Participant in this group will receive a unilateral subretinal injection of ZVS101e in the study eye
干预措施: ZVS101e (Drug)
结局指标
主要结局
Proportion of study eyes with a ≥15-letter improvement from baseline in BCVA
时间窗: From baseline to Week 52
Best corrected visual acuity (BCVA) will be assessed using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart. A 15-letter improvement over the baseline is clinical meaningful.
次要结局
- Change from Baseline in BCVA(From baseline to Week 52)
- Proportion of study eyes with a ≥10-letter improvement from baseline in BCVA(From baseline to Week 52)
- Change from baseline in multi-luminance mobility test (MLMT) score(From baseline to Week 52)
- Change from baseline in 25-Item visual function questionnaire (VFQ-25) score(From baseline to Week 52)
- Change from Baseline in microperimetry(From baseline to Week 52)
- Change from baseline in OCT(From baseline to Week 52)
- Incidence and severity of ocular and systemic adverse events(From baseline to Week 52)