Clinical Trial: Sunitinib Malate to Treat Advanced Eye Disease in Patients With Von Hippel-Lindau Syndrome

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: Pilot Study of Sunitinib Malate for Advanced Ocular Disease of Von Hippel-Lindau Syndrome

Brief 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.

Detailed Summary:
Sponsor: National Eye Institute (NEI)

Current Primary Outcome: Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36 [ Time Frame: Baseline and 36 Weeks ]

Visual acuity was measured using the Early Treatment Diabetic Retinopathy Study (ETDRS) protocol. Acuity is measured as letters read on an ETDRS eye chart and the letters read equate to Snellen measurements. For example, if a participant reads between 84 and 88 letters, the equivalent Snellen measurement is 20/20.


Original Primary Outcome: Change in best corrected visual acuity of 15 letters or more three months after completion of drug therapy (at 1-year follow-up)

Current Secondary Outcome:

  • Change in Retinal Thickness From Baseline to Week 36 [ Time Frame: Baseline and 36 Weeks ]
    Retinal thickness was assessed by spectral-domain optical coherence tomography (Cirrus HD-OCT; Carl Zeiss Meditec, Dublin, CA), a non-invasive imaging technique that uses long-wavelength light to capture micrometer-resolution cross-sectional images from biological tissue.
  • Change in Retinal Angioma Leakage From Baseline to Week 36 [ Time Frame: Baseline and 36 Weeks ]
    Leakage of the retinal angioma was calculated after manually outlining the inner and outer borders of the subretinal fluid packet in the optical coherence tomography (OCT) images using the "Edit Segmentation" function of the Cirrus HD-OCT software. In cases where a pigment epithelial detachment was present, the volume of the pigment epithelial detachment was included in the calculation of leakage volume.


Original Secondary Outcome: Reduction in lesion size as recorded by OCT

Information By: National Institutes of Health Clinical Center (CC)

Dates:
Date Received: May 6, 2008
Date Started: May 2008
Date Completion:
Last Updated: November 14, 2011
Last Verified: November 2011