Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Pilot Study of Intravitreal Injection of EYE001 (Anti-VEGF Pegylated Aptamer) for Advanced Ocular Disease of Von Hippel-Lindau (VHL) Disease
Brief Summary:
This study will test the ability of the experimental drug EYE001 to reduce retinal thickening and improve vision in patients with Von Hippel-Lindau syndrome (VHL). Angiomas (blood vessel tumors) commonly develop in the back of the eye on the retina and the optic nerve in patients with VHL. Although the tumors are not cancerous, they may cause significant vision loss. Current treatments, including laser therapy, cryotherapy, and vitrectomy, may not be successful or possible for all patients. EYE001 decreases production of VEGF, a growth factor that is important for the formation of new blood vessels and that is elevated in VHL. Preliminary findings from studies of other retinal diseases suggest that EYE001 can reduce retinal thickening and improve vision.
Patients 18 years of age and older with retinal angiomas due to VHL in one or both eyes and central vision loss of 20/40 or worse may be eligible for this study. Participants will undergo the following tests and procedures: