Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome
Status:
Terminated
Trial end date:
2011-02-01
Target enrollment:
Participant gender:
Summary
This open-label study will pilot the use of systemic sunitinib malate, a dual inhibitor of
vascular endothelial growth factor (VEGF) and platelet derived growth factor (PDGF), in five
participants with Von Hippel-Lindau (VHL) to investigate its potential efficacy as a
treatment for retinal angiomas. Participants will have visual dysfunction with either visual
acuity loss or visual field loss from retinal angiomas secondary to genetically confirmed
VHL. This open-label study will pilot the use of systemic sunitinib malate in five
participants to investigate its potential efficacy as a treatment for retinal angiomas
associated with VHL. Participants will receive nine months of sunitinib malate therapy (six
cycles total - one cycle consists of 50 mg oral dose once daily for four weeks followed by a
two week rest period). The primary outcome will be a change in the best-corrected visual
acuity of more than or equal to 15 letters from baseline to the Week 36 visit. The secondary
ocular outcomes will focus on retinal thickness and leakage of the retinal angioma at the
Week 36 visit. Optical coherence tomography will document changes in retinal thickening and
fluorescein angiography will be used to determine leakage of the retinal angioma.