NCT06088992招募中早期 1 期
An Investigator-Initiated Open-Label, Multiple-Dose Clinical Study to Evaluate the Safety,Tolerability, and Efficacy of Gene Therapy for 2Leber's Congenital Amaurosis With RPE65 Mutation (LCA2)
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine1 个研究点 分布在 1 个国家目标入组 9 人开始时间: 2023年1月10日最近更新:
适应症
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 9
- 试验地点
- 1
- 主要终点
- Incidence and severity of ocular and systemic adverse events
研究概览
简要总结
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutationsin RPE65 gene.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 8 Years 至 50 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or females between 8 and 50 years of age at the time of signing theinformed consent form.
- •Willing to adhere to protocol as evidenced by written informed consent orparental permission and subject assent.
- •Clinical confirmed diagnosis of Leber congenital amaurosis (LCA) andmolecular diagnosis of LCA due to RPE65 mutations.
- •Ability to perform tests of visual and retinal function.
- •Visual acuity of ≤ 20/160 or visual field less than 20 degrees in the eye to beinjected.
- •Acceptable hematology, clinical chemistry, and urine laboratory parameters.
排除标准
- •OCT examination determined that the outer nuclear layer was not visible inthe planned injection area (Bleb) in the study eye.
- •Presence of epiretinal membrane by OCT.
- •Complicating systemic diseases or clinically significant abnormal baselinelaboratory values.
- •Complicating systemic diseases would include those in which the diseaseitself, or the treatment for the disease, can alter ocular function.
- •Prior ocular surgery within six months.
- •Prior gene therapy or oligonucleotide therapy treatments.
- •Any other condition that would not allow the potential subject to completefollow-up examinations during the study and would, in the opinion of theinvestigator, make the potential subject unsuitable for the study.
结局指标
主要结局
Incidence and severity of ocular and systemic adverse events
时间窗: 26 weeks
Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)
次要结局
未报告次要终点
研究者
Peiquan Zhao
Professor
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
研究点 (1)
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