Phase 2 Clinical Trial of Ciliary Neurotrophic Factor (CNTF) for Macular Telangiectasia Type 2 (MacTel) (14-EI-0078, 17-EI-0136)

我们在2014财年在NEI启动了这个项目，作为第二阶段临床试验(14-EI-0078)的一个地点，测试CNTF治疗黄斑毛细血管扩张(Mactel)。我们达到了NEI网站的应计目标，研究分析已经完成，初步结果公布。这项试验达到了其主要终点，在24个月时，接受假治疗的眼睛比接受CNTF治疗的眼睛显示出比接受CNTF治疗的眼睛多31%的神经变性进展，其中光感受器丢失的平均面积在24个月时相差0.05±0.03mm2(p=0.04)。第二阶段协议在完成后已终止。 第二阶段试验的参与者参加了一项扩展研究(17-EI-0136)，以研究CNTF植入物在3、4、5和6年的效果，并继续对该方案的现场参与者进行随访。 2018年，我们作为第三阶段临床试验(18-EI-0055)的一个站点被激活，测试CNTF(NT-501，雷克萨斯)的Mactel(作为一个单独的项目列出)。

We initiated this project at the NEI in fiscal year 2014 as a site in the phase 2 clinical trial (14-EI-0078) testing CNTF for macular telangiectasia (MacTel). We met the accrual goal at the NEI site and study analysis has been completed, with publication of the primary results. The trial met its primary endpoint, with eyes receiving sham treatment showing 31% greater progression of neurodegeneration than the CNTF-treated eyes using an en face OCT measure of change in area of outer retinal disruption, with difference in mean area of photoreceptor loss of 0.05 0.03 mm2 (p = 0.04) at 24 months. The phase 2 protocol has been terminated following completion. Participants in the phase 2 trial were enrolled in an extension study (17-EI-0136) to study effects of the CNTF implant at years 3, 4, 5, and 6, and follow up continues for site participants in this protocol. In 2018, we were activated as a site in the phase 3 clinical trial (18-EI-0055) testing CNTF (NT-501, Renexus) for Mactel (listed as a separate project).