Ranibizumab (rhuFAB V2) for Advanced Ocular Disease of V

雷珠单抗 (rhuFAB V2) 治疗 V 型晚期眼病

• 批准号: 7141780
• 负责人: EMILY Y CHEW
• 金额: --
• 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/7141780
• 关键词: Von Hippel Lindau syndrome; angiogenesis inhibitors; edema; eye disorder chemotherapy; eye neoplasms; gene mutation; human subject; human therapy evaluation; injection /infusion; patient oriented research; retina disorder; vascular endothelial growth factors; vision tests; visual perception

Von Hippel-Lindau Syndrome (VHL) is an autosomal dominant heritable disorder in which multiple benign and malignant neoplasms and cysts of specific histopathologies develop in the kidney, adrenal gland, pancreas, brain, spinal cord, eye, inner ear, epididymis, and broad ligament. Retinal angioma may be one of the earliest manifestations of VHL disease and may lead to a significant decrease in visual acuity of the affected individual. These tumors rarely regress spontaneously. The main cause of vision loss is retinal edema, specifically macular edema secondary to enlargement of peripheral retinal angiomas or angiomas found on or around the optic disk. Treatment of retinal angiomas depends on the location and size of the lesions but typically consists of photocoagulation or cryotherapy. However, there is no proven effective therapy for the treatment of VHL ocular lesions on or surrounding the optic nerve or lesions in the peripheral retina too large to respond to the traditional therapies. The genetic mutation found in VHL disease up-regulates the production of vascular endothelial growth factor (VEGF). Immunochemical studies of the VHL ocular lesions, as well as others found elsewhere in the body show marked increase in VEGF. This open-label study will pilot the use of an anti-VEGF therapy, ranibizumab (rhuFab V2) in 5 participants to investigate the potential efficacy as a treatment for retinal angiomas associated with VHL. Participants will receive 7 intravitreal injections of study drug over a 6-month period, with the option of up to seven additional injections at the same dose and schedule during follow-up for a period of 1 year after the initiation of treatment. The primary outcome will be a change in the best-corrected visual acuity of 15 letters or more at 1 year. The secondary outcomes will be a reduction in retinal thickening and leakage at one year, and adverse events including local and systemic toxicities. Recruitment continues.