ZVS101e in Patients With Bietti's Crystalline Dystrophy (ZVS101e-BCD)
Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy
The goal of this clinical trial is to evaluate the safety and early effectiveness of ZVS101e in patients with Bietti crystalline dystrophy who meet the eligibility criteria for treatment under the translational application program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone.
Participants will:
undergo screening and baseline assessments to confirm eligibility; receive a single subretinal injection of ZVS101e in the study eye; complete follow-up visits over 4 weeks after treatment for safety monitoring and assessment of early effectiveness.
研究概览
研究类型
注册 (估计的)
阶段
- 不适用
联系人和位置
学习联系方式
- 姓名:Jinlu Zhang, MD
- 电话号码:+86-15810570898
- 邮箱:zhangjinlu@chinagene.cc
研究联系人备份
- 姓名:Tingting Wu, BS
- 电话号码:+86-18500191916
- 邮箱:wutingting@chinagene.cc
学习地点
-
-
Hainan
-
Qionghai、Hainan、中国
- Boao Super Hospital
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Qionghai、Hainan、中国
- Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University
-
-
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
- Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements;
- Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years;
- Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases;
- Target eye BCVA ≤ 60 ETDRS letters.
Exclusion Criteria:
Subjects will be excluded if they meet any 1 of the following exclusion criteria:
- The target eye currently has or has a history of macular lesions; suffers from ocular diseases that may hinder surgery or interfere with the interpretation of study endpoints;
- The target eye has previously undergone retinal reattachment surgery, vitrectomy, or any intraocular surgery within 3 months before enrollment;
- Within 1 month before enrollment, suffered from viral infectious diseases that may affect the evaluation of the efficacy and safety of the investigational drug, or received antiviral vaccines;
- The target eye has previously received gene therapy or stem cell therapy for BCD or other ocular diseases;
- Known allergy to the drugs planned for use in the study;
- The laboratory abnormalities are considered clinically significant;
- Pregnant or lactating females.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
实验性的:ZVS101e Treatment Arm
|
ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.
|
研究衡量的是什么?
主要结果指标
结果测量 |
大体时间 |
|---|---|
|
Change from baseline in Best corrected visual acuity (BCVA)
大体时间:From enrollment to the end of treatment at 52 weeks
|
From enrollment to the end of treatment at 52 weeks
|
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The incidence and severity of adverse events and serious adverse events
大体时间:From enrollment to the end of treatment at 52 weeks
|
From enrollment to the end of treatment at 52 weeks
|
合作者和调查者
出版物和有用的链接
一般刊物
- Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181.
- Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W. Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. Signal Transduct Target Ther. 2024 Apr 24;9(1):95. doi: 10.1038/s41392-024-01806-3.
- Jia R, Chen S, Li W, Zhang J, Qu B, Qiao J, Meng X, Yu S, Liu X, Xu B, Chen T, Shen X, Sun W, Dou H, Mahajan VB, Zhang Q, Yang L. Unravelling CYP4V2: Clinical features, genetic insights, pathogenic mechanisms and therapeutic strategies in Bietti crystalline corneoretinal dystrophy. Prog Retin Eye Res. 2025 Jul;107:101377. doi: 10.1016/j.preteyeres.2025.101377. Epub 2025 Jun 5.
- Yang L, Xing D, Zhang J, Liu B, Jiang X, Chen S, Li W, Qu B, Qiao J, Feng X, Jia H, Hu X, Yu S, Jia R, Wang L, Mahajan VB, Dou H, Lu F, Li X. Gene therapy for Bietti crystalline corneoretinal dystrophy: A phase 1/2 clinical trial. Mol Ther. 2026 Mar 4;34(3):1743-1755. doi: 10.1016/j.ymthe.2026.01.005. Epub 2026 Jan 10.
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
关键字
其他相关的 MeSH 术语
其他研究编号
- ZYA-2025-003
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
在美国制造并从美国出口的产品
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