Clinical Trial: Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE)

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome)

Brief Summary: The purpose of the study is to investigate the efficacy of a new drug called ranibizumab in the treatment of choroidal neovascularization in underlying angioid streaks due to Pseudoxanthoma elasticum. 10 patients will receive monthly injections of the drug in one eye over a period of one year.

Detailed Summary:

Pseudoxanthoma elasticum (PXE) is a rare hereditary systemic disease affecting mainly the skin, eyes and the cardiovascular system. Commonly, complicating choroidal neovascularization (CNV) of the central retina lead to a severely decreased visual acuity in the course of the disease. Onset of the symptoms varies with the extend of the PXE-associated findings.

In the past there has been no effective treatment for the disease's ocular complications. Recent studies in a limited number of patients with CNV treated with intravitreal injections of bevacizumab, an antagonist targeting vascular endothelial growth factor (VEGF), have shown a preservation of function and regression of CNV. An increase of visual acuity was reported in a subset of patients. Bevacizumab is a humanized antibody of which ranibizumab is a fragment.

This trial is initiated in order to investigate the effect of ranibizumab on functional outcome measures such as preservation of visual acuity as well as morphological outcome measures such as regression of CNV in angiography.

The safety and tolerability of ranibizumab will be investigated as well. As it has been tested on large numbers of patients suffering from age-related macular degeneration with only rare significant side effects or adverse events being reported, a good safety profile is assumed.

The study is conducted in a non-randomized, uncontrolled prospective setting at one center.

Patients will receive monthly injections over a period of one year.


Sponsor: University Hospital, Bonn

Current Primary Outcome: Best corrected visual acuity [ Time Frame: one year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Reading ability [ Time Frame: one year ]
  • Changes in retinal thickness assessed by optical coherence tomography [ Time Frame: one year ]
  • Changes in parafoveal leakage assessed by fluorescein angiography [ Time Frame: one year ]
  • Quality of life [ Time Frame: one year ]


Original Secondary Outcome:

  • Reading ability [ Time Frame: one year ]
  • Changes in retinal thickness assessed by optival coherence tomography [ Time Frame: one year ]
  • Changes in parafoveal leakage assessed by fluorescein angiography [ Time Frame: one year ]
  • Quality of life [ Time Frame: one year ]


Information By: University Hospital, Bonn

Dates:
Date Received: August 1, 2007
Date Started: August 2007
Date Completion:
Last Updated: June 14, 2012
Last Verified: August 2011