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

Phase 1

Terminated

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.

Recruitment & Eligibility

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
Sunitinib Malate	Sunitinib Malate	Participants were expected to receive 9 months of sunitinib malate therapy administered in 6 cycles. Each cycle consisted of a daily oral dose of 50 mg sunitinib malate for 4 weeks followed by a 2-week rest period).

Primary Outcome Measures

Name	Time	Method
Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36	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.

Secondary Outcome Measures

Name	Time	Method
Change in Retinal Thickness From Baseline to Week 36	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.
Reduction in Leakage Intensity as Measured by Fluorescein Angiography From Baseline to Week 36	Baseline and 36 Weeks	Number of participants with improvement in leakage intensity.
Visual Field Changes as Documented by Microperimetry From Baseline to Week 36	Baseline and 36 Weeks
Changes in Non-ocular VHL Lesion Status	Duration of study	Count of participants with new non-ocular lesions present since baseline at any follow-up visit.
Reduction in Leakage Area as Measured by Fluorescein Angiography at the Week 36 Visit	Baseline and 36 Weeks	Number of participants with improvement in leakage area.