Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome)
Overview
- Phase
- Phase 2
- Intervention
- Intravitreal injection ranibizumab
- Conditions
- Choroidal Neovascularization
- Sponsor
- University Hospital, Bonn
- Enrollment
- 10
- Locations
- 1
- Primary Endpoint
- Best corrected visual acuity
- Status
- Completed
- Last Updated
- 13 years ago
Overview
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 Description
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of choroidal neovascularization in patients with pseudoxanthoma elasticum
- •Age between 18-65 years
- •Patient must be able to follow protocol
- •Written informed consent
- •Genetic diagnosis of pseudoxanthoma elasticum
- •Best corrected visual acuity between 20/200 - 20/32 in the treated eye
Exclusion Criteria
- •Patients who do not fulfill the inclusion criteria
- •Patients with other retinal vascular diseases such as diabetic retinopathy or venous occlusive disease
- •Ocular surgery 3 months before study enrollment
- •History of uncontrolled glaucoma
- •Active intraocular inflammation or inflammation of the ocular adnexa
- •Subfoveal fibrosis in the study eye
- •Inability to follow study protocol
- •Major surgery one month before study enrollment
- •History of severe cardiovascular disease or history of stroke 6 months before study enrollment
- •Allergies against the substances or components of the study medication
Arms & Interventions
A
Intervention: Intravitreal injection ranibizumab
Outcomes
Primary Outcomes
Best corrected visual acuity
Time Frame: one year
Secondary Outcomes
- Quality of life(one year)
- Reading ability(one year)
- Changes in retinal thickness assessed by optical coherence tomography(one year)
- Changes in parafoveal leakage assessed by fluorescein angiography(one year)