TerminatedPhase 1/2

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

Stopped: Inability to recruit and adequate number of participants

United States2 participantsStarted 2008-05

Plain-language 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.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Participant must understand and sign the informed consent.
✓. Participant must be at least 18 years of age.
✓. Participant must have genetically confirmed VHL disease.
✓. Participant must have an optic nerve angioma secondary to VHL in one or both eyes.
✓. Participant must have an optic nerve tumor that has caused any visual field depression on microperimetry-1 that correlates with the retinal angioma OR the participant clinically may have hard exudates correlating with the retinal angioma OR has best-corrected visual acuity of 20/40 or worse in the study eye.
✓. Participant must have clear ocular media and adequate pupillary dilation to permit good quality stereoscopic fundus photography.
✓. All women of childbearing potential must have a negative urine pregnancy test at baseline, and have regular negative pregnancy testing while taking sunitinib malate. (Sunitinib malate has the potential for teratogenic or abortifacient effects, and no data regarding its safety in pregnant women are available).
✓. All women of childbearing potential who are sexually active and all men who are sexually active are required to use two forms of birth control during the course of the study.

Exclusion criteria

What they're measuring

Change in Best Corrected Visual Acuity (BCVA) From Baseline to Week 36

Timeframe: Baseline and 36 Weeks

Trial details

NCT IDNCT00673816

SponsorNational Eye Institute (NEI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2011-02

Results submitted2011-09-27

View on ClinicalTrials.gov More Von Hippel-Lindau Syndrome trials