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

Study 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.

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

Conditions
-----------------
Choroidal Neovascularization

Intervention / Treatment
-----------------
* Drug: Intravitreal injection ranibizumab


Participation Criteria
=================
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 Low anticipated compliance Patients who participated in clinical trials simultaneously or within the last 60 days Pregnancy, lactation, women that may become pregnant and don't use safe contraception Chronic alcohol- or drug abuse within the last year Lacking legal competence or language ability Neurologic diseases such as multiple sclerosis Need of concomitant medication that is not allowed in combination with ranibizumab Previous intravitreal therapy with anti-angiogenic substances in the study eye within the last 6 months

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: Intravitreal injection ranibizumab<br>* Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year<br>* Other names: Lucentis;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Best corrected visual acuity |  | one year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| Quality of life |  | one year | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
choroidal neovascularization, angioid streaks, pseudoxanthoma elasticum, Groenblad-Strandberg-Syndrome, therapy, ranibizumab, lucentis, Choroidal neovascularization in patients with PXE