Overview

A Phase I/II Trial for Intravitreous Treatment of Severe Ocular Von Hippel-Lindau Disease Using a Combination of the PDGF Antagonist E10030 and the VEGF Antagonist Ranibizumab

Status:
Completed
Trial end date:
2019-07-09
Target enrollment:
Participant gender:
Summary
Background: People with Von-Hippel-Lindau (VHL) disease may experience significant vision loss as a result of retinal capillary hemangiomas (RCH), the most common and often earliest manifestation of VHL. Objective: To investigate the safety and possible efficacy of combination investigational treatment with serial intravitreal injections of E10030, a PDGF-B antagonist, and ranibizumab, a VEGF-A antagonist, in participants with severe ocular VHL disease. Design: Three participants with severe ocular VHL disease will receive the combination investigational treatment in one eye and will be followed for 104 weeks. Primary Outcome: The safety of the combination investigational treatment, assessed by tabulation of adverse events reported through Week 52.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
National Eye Institute (NEI)
Treatments:
Ranibizumab