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

June 14, 2012 updated by: University Hospital, Bonn

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Germany
      • Bonn, Germany, 53127
        • University of Bonn

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 63 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: A
Drug: Intravitreal injection ranibizumab
Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year
Other Names:
  • Lucentis

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Best corrected visual acuity
Time Frame: one year
one year

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Bonn

Collaborators

Novartis

Investigators

  • Principal Investigator: Hendrik PN Scholl, MD, MA, University of Bonn
  • Principal Investigator: Frank G. Holz, MD, University of Bonn, Department of Ophthalmology

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

August 1, 2007

Primary Completion (Actual)

April 1, 2010

Study Completion (Actual)

April 1, 2010

Study Registration Dates

First Submitted

August 1, 2007

First Submitted That Met QC Criteria

August 2, 2007

First Posted (Estimate)

August 3, 2007

Study Record Updates

Last Update Posted (Estimate)

June 15, 2012

Last Update Submitted That Met QC Criteria

June 14, 2012

Last Verified

August 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRFB002ADE03
  • EudraCT number 2006-006233-49

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Choroidal Neovascularization

Clinical Trials on Intravitreal injection ranibizumab

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Belgium

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe