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

Primary Purpose

Choroidal Neovascularization

Status
Completed
Phase
Phase 2
Locations
Germany
Study Type
Interventional
Intervention
Intravitreal injection ranibizumab
Sponsored by
University Hospital, Bonn
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Choroidal Neovascularization focused on measuring choroidal neovascularization, angioid streaks, pseudoxanthoma elasticum, Groenblad-Strandberg-Syndrome, therapy, ranibizumab, lucentis, Choroidal neovascularization in patients with PXE

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • University of Bonn

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

A

Arm Description

Outcomes

Primary Outcome Measures

Best corrected visual acuity

Secondary Outcome Measures

Reading ability
Changes in retinal thickness assessed by optical coherence tomography
Changes in parafoveal leakage assessed by fluorescein angiography
Quality of life

Full Information

First Posted
August 1, 2007
Last Updated
June 14, 2012
Sponsor
University Hospital, Bonn
Collaborators
Novartis
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1. Study Identification

Unique Protocol Identification Number
NCT00510965
Brief Title
Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE)
Official Title
Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome)
Study Type
Interventional

2. Study Status

Record Verification Date
August 2011
Overall Recruitment Status
Completed
Study Start Date
August 2007 (undefined)
Primary Completion Date
April 2010 (Actual)
Study Completion Date
April 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
University Hospital, Bonn
Collaborators
Novartis

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Choroidal Neovascularization
Keywords
choroidal neovascularization, angioid streaks, pseudoxanthoma elasticum, Groenblad-Strandberg-Syndrome, therapy, ranibizumab, lucentis, Choroidal neovascularization in patients with PXE

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
A
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Intravitreal injection ranibizumab
Other Intervention Name(s)
Lucentis
Intervention Description
Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year
Primary Outcome Measure Information:
Title
Best corrected visual acuity
Time Frame
one year
Secondary Outcome Measure Information:
Title
Reading ability
Time Frame
one year
Title
Changes in retinal thickness assessed by optical coherence tomography
Time Frame
one year
Title
Changes in parafoveal leakage assessed by fluorescein angiography
Time Frame
one year
Title
Quality of life
Time Frame
one year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Frank G. Holz, MD
Organizational Affiliation
University of Bonn, Department of Ophthalmology
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Hendrik PN Scholl, MD, MA
Organizational Affiliation
University of Bonn
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany

12. IPD Sharing Statement

Citations:
PubMed Identifier
21704964
Citation
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.
Results Reference
derived
Links:
URL
http://www.augenklinik.uni-bonn.de
Description
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Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE)

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