MACTEL1

这项研究是一项对黄斑毛细血管扩张症患者玻璃体内注射雷尼比珠单抗的开放性、先导性研究。基因泰克公司将为该试验提供雷尼比珠单抗。五名有新生血管的研究参与者将被选入本研究。根据视力测量、荧光素血管造影术和OCT结果，双眼黄斑毛细血管扩张症患者的研究眼将被指定为受累程度更严重的眼。然而，如果由于不可逆的结构变化(广泛萎缩和色素改变，慢性黄斑疤痕)而判断受影响较严重的眼睛不太可能从治疗中受益，则可以考虑受影响较轻的眼睛。在两只眼睛相同的情况下，研究人员和参与者将确定研究眼睛。另一只眼睛将按照护理标准进行治疗。 由于这种疾病的发病机制尚不清楚，人们只能推测血管内皮生长因子(VEGF)在这种疾病中的可能作用。荧光素血管造影上的渗漏反映了视网膜血管的内皮功能障碍，尽管还不能确定这可能是原发的还是继发性的。黄斑毛细血管扩张症的荧光素渗漏类似于糖尿病视网膜病变，而血管内皮生长因子已被证明在其发病机制中起重要作用。新生血管的发展通常始于视网膜内新生血管，然后发展为视网膜下和脉络膜新生血管，提示血管内皮生长因子可能也参与了这一过程。由于这些原因，我们认为重要的是测试雷尼比珠单抗玻璃体内注射治疗的可能作用。我们将在了解任何安全问题的同时了解这种治疗的可行性。在疾病的早期阶段，我们并不完全了解视力丧失的原因。很明显，新生血管过程会导致视力下降，这一过程与其他导致新生血管形成的原因类似，如老年性黄斑变性。

This study is an open-label, pilot study of intravitreal injections of ranibizumab in participants with macular telangiectasia. Genentech will supply the ranibizumab for this trial. Five study participants, with neovascularization, will be selected for this study. The study eye in participants who present with bilateral macular telangiectasia will be designated as the eye with the greater severity of involvement as determined by visual acuity measurements, fluorescein angiography and OCT results. However, in the case where the more severely affected eye is judged to unlikely benefit from treatment due to irreversible structural changes (extensive atrophy and pigmentary change, chronic macular scarring), then the less severely affected eye may be considered. In cases where both eyes are equivalent, the investigator and participant will determine the study eye. The fellow eye will be treated according to standard-of-care. Because the pathogenesis of this condition is unknown, one can only speculate on the possible role of vascular endothelial growth factor (VEGF) in this condition. The leakage seen on fluorescein angiogram reflects on the endothelial dysfunction of the retinal vessels although it is not certain that this may be a primary or a secondary condition. The fluorescein leakage of macular telangiectasia resembles that of diabetic retinopathy where VEGF has proven to be important in its pathogenesis. The development of neovascularization, which often begins as intra-retinal neovascularization, which then develops into subretinal and choroidal neovascularization, suggests that VEGF may also be implicated in this process. For these reasons, we believe it is important to test the possible role of ranibizumab therapy with intravitreal injections of ranibizumab. We will learn about the feasibility of this treatment while we learn about any safety issues. We do not understand fully the cause of vision loss in the early stages of the disease. It is evident that the neovascular process will decrease vision in a similar process as that of other causes of neovascularization such as age-related macular degeneration.