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

University Hospital Bonn (UKB)

Status and phase

Completed

Phase 2

Conditions

Choroidal Neovascularization

Treatments

Drug: Intravitreal injection ranibizumab

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT00510965

EudraCT number 2006-006233-49

CRFB002ADE03

Details and patient eligibility

About

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.

Full 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.

Enrollment

10 patients

Sex

All

Ages

18 to 65 years old

Volunteers

No Healthy Volunteers

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