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

睫状神经营养因子 (CNTF) 治疗 2 型黄斑毛细血管扩张症 (MacTel) 的 2 期临床试验 (14-EI-0078、17-EI-0136)

• 批准号: 10266905
• 负责人: Henry Wiley
• 金额: $ 7.31万
• 依托单位: NATIONAL EYE INSTITUTE
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10266905
• 关键词: Affect; Area; Blindness; Ciliary Neurotrophic Factor; Degenerative Disorder; Devices; Disease; Enrollment; Eye; Goals; Implant; Measures; Nerve Degeneration; Participant; Phase; Phase II Clinical Trials; Phase III Clinical Trials; Photoreceptors; Placebos; Protocols documentation; Publications; Retina; Safety; Site; Telangiectasis; Testing; follow-up; implantable device; implantation; macula; phase II trial; primary endpoint; sham surgery

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. We opened a sub-study (19-EI-0045) to allow implantation of the CNTF-secreting device (NT-501; Renexus) in participants who received only the sham-surgery in the phase 2 trial, and have implanted the device in two participants. 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).

我们于2014财政年度在NEI启动了该项目，作为测试CNTF治疗黄斑毛细血管扩张（MacTel）的2期临床试验（14-EI-0078）的一个站点。我们在NEI现场达到了预期目标，研究分析已经完成，并发表了主要结果。试验达到了主要终点，在24个月时，接受假治疗的眼睛比接受cntf治疗的眼睛神经退行性变的进展要大31%，使用表面OCT测量外视网膜破坏面积的变化，平均光感受器损失面积的差异为0.05 0.03 mm2 （p = 0.04）。第二阶段协议在完成后终止。