An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Tolerability of GS010 (rAAV2/2-ND4) in Patients With Leber Hereditary Optic Neuropathy Due to Mutations in the Mitochondrial NADH Dehydrogenase 4 Gene

GenSight Biologics1 个研究点分布在 1 个国家目标入组 19 人2014年2月13日

适应症Leber Hereditary Optic Neuropathy

干预措施GS010

概览

阶段: 1 期
干预措施: GS010
疾病 / 适应症: Leber Hereditary Optic Neuropathy
发起方: GenSight Biologics
入组人数: 19
试验地点: 1
主要终点: Incidence of local and general adverse events and Serious Adverse Events
状态: 已完成
最后更新: 2个月前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

The purpose of this study is to evaluate the safety and tolerability profile of ascending doses of GS010 in Leber Hereditary Optic Neuropathy (LHON) patients.

注册库

clinicaltrials.gov

开始日期

2014年2月13日

结束日期

2020年6月25日

最后更新

2个月前

研究类型

Interventional

研究设计

Single Group

性别

All

研究者

发起方

GenSight Biologics

责任方

Sponsor

入排标准

入选标准

•Documented diagnosis of LHON based on a genetic test confirming the presence of the G11778A mutation in the mitochondrial ND4
•Age 18 years old or older at the time of study entry (informed consent signature)
•Visual acuity ≤ 1/10 of the less functional eye

排除标准

•Any known allergy or hypersensibility to one of the product used during the trial
•Contraindication to IVT surgery (anaemia Hb \<8g/dl, severe cardiovascular disease, severe coagulopathy…)
•Disorder of the ocular humors and of the internal retina involving visual disability
•Presence of other pathology whose symptoms or associated treatments might affect the retina or the optic nerve Vascular retinal occlusion
•Narrow angle contra-indicating pupillary dilation
•Other cause of optic neuropathy (inflammatory conditions or exposure to toxins...)
•Patients presenting known mutation of other genes implicated in pathological retinal conditions