Pilot Study of Intravitreal Injection of EYE001 (Anti-VEGF Pegylated Aptamer) for Advanced Ocular Disease of Von Hippel-Lindau (VHL) Disease
Von Hippel-Lindau (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 anti-VEGF therapy (EYE001) in 5 patients to investigate the potential
efficacy as a treatment for retinal angiomas associated with VHL. Patients will receive 6
intravitreal injections of study drug over a 30-week period, then return for a follow-up
visit 1 year after initiating injections. The primary outcomes will be improvement in best
corrected visual acuity of 15 letters or more at 1 year, reduction in retinal thickening and
leakage at one year, changes in ERG amplitude and implicit time, and adverse events
including local and systemic toxicities.
Interventional
Endpoint Classification: Safety Study, Primary Purpose: Treatment
United States: Federal Government
030101
NCT00056199
March 2003
November 2005
Name | Location |
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National Eye Institute (NEI) | Bethesda, Maryland 20892 |