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

NCT ID: NCT00510965

Last Updated: 2012-06-15

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-08-31
• Study Completion Date: 2010-04-30

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.

Conditions

Choroidal Neovascularization

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

A

Group Type: EXPERIMENTAL

Intravitreal injection ranibizumab

Intervention Type: DRUG

Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year

Interventions

Intravitreal injection ranibizumab

Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year

Intervention Type: DRUG

Other Intervention Names

Lucentis

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

• 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis

INDUSTRY

Sponsor Role: collaborator

University Hospital, Bonn

OTHER

Sponsor Role: lead

Responsible Party

University of Bonn

Principal Investigators

Frank G. Holz, MD

Role: PRINCIPAL_INVESTIGATOR

University of Bonn, Department of Ophthalmology

Hendrik PN Scholl, MD, MA

Role: PRINCIPAL_INVESTIGATOR

University of Bonn

Locations

University of Bonn

Bonn, , Germany

Countries

Germany

References

Finger RP, Charbel Issa P, Hendig D, Scholl HP, Holz FG. Monthly ranibizumab for choroidal neovascularizations secondary to angioid streaks in pseudoxanthoma elasticum: a one-year prospective study. Am J Ophthalmol. 2011 Oct;152(4):695-703. doi: 10.1016/j.ajo.2011.03.022. Epub 2011 Jun 25.

Reference Type: DERIVED

PMID: 21704964 (View on PubMed)

Related Links

http://www.augenklinik.uni-bonn.de

Related Info

Other Identifiers

EudraCT number 2006-006233-49

Identifier Type: -

Identifier Source: secondary_id

CRFB002ADE03

Identifier Type: -

Identifier Source: org_study_id