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

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.

Timeline

Start

2017-01-23

Primary completion

2019-07-09

Completion

2019-07-09

Publications

• Results Hwang CK, Chew EY, Cukras CA, Keenan TDL, Wong WT, Linehan WM, Chittiboina P, Pacak K, Wiley HE. Intravitreous treatment of severe ocular von Hippel-Lindau disease using a combination of the VEGF inhibitor, ranibizumab and PDGF inhibitor, E10030: Results from a phase 1/2 clinical trial. Clin Exp Ophthalmol. 2021 Dec;49(9):1048-1059. doi: 10.1111/ceo.14001. Epub 2021 Oct 26.

Drugs

Indications