NCT07002398招募中1 期
A Single Arm, Ph1/2, Open-label, Multicenter Trial With Dose-exploration Via Subretinal Injection to Evaluate the Safety and Preliminary Efficacy of VG801 for Treatment of ABCA4 Mutation-associated Recessive Hereditary Retinal Dystrophy (Stargardt Disease)
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
This is a single-arm, open-label, non-randomized, single dose-escalation, first-in-human (FIH) clinical trial to evaluate the safety and preliminary efficacy of VG801 for treatment of patients with retinal dystrophy (Stargardt disease) due to biallelic ABCA4 mutations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •To be eligible for study entry, subjects must satisfy all the following criteria:
- •Written informed consent.
- •Subjects aged ≥ 6 years.
- •Clinical diagnosis of a macular lesion phenotypically consistent with a recessive hereditary macular dystrophy (Stargardt disease).
- •Confirmed molecular diagnosis of ABCA4 mutations (homozygotes or compound heterozygotes).
- •Poor vision in the study eye.
排除标准
- •Subjects will be excluded from the study if one or more of the following statements are applicable to either eye:
- •Pre-existing eye conditions such as uveitis, glaucoma, or diabetic retinopathy or implantation of a medical device in the vitreous cavity or subretinal space.
- •Systemic diseases that would preclude the planned surgery or interfere with the interpretation of study results.
- •History of intraocular surgery within the previous 6 months.
- •Previous participation in a gene therapy trial.
- •Participation in a clinical trial (investigational drug or medical device) within the previous 6 months.
- •Any other eye disease that may affect the outcome of the study (e.g., ocular opacities, advanced cataracts, amblyopia, etc.).
研究组 & 干预措施
VG801
Experimental
Participants will receive a single dose of subretinal injection of VG801at Day 0.
干预措施: VG801 (Drug)
结局指标
主要结局
Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Baseline to Month 12
Abnormal examination results will be recorded.
次要结局
- Best Corrected Visual Acuity (BCVA)(Screening to Month 12)
- Optical coherence tomography (OCT)(Baseline to Month 12)
- Microperimetry(Baseline to Month 12)
- Best Corrected Visual Acuity (BCVA)(Screening to Month 12)
- Optical coherence tomography (OCT)(Baseline to Month 12)
- Microperimetry(Baseline to Month 12)
- Novel Virtual Reality Visual Test (Exploratory)(Baseline to Month 12)
- Fundus autofluorescence(Baseline to Month 12)
研究者
研究点 (2)
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