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

This study has been completed.
Sponsor:
Collaborator:
Novartis
Information provided by:
University Hospital, Bonn
ClinicalTrials.gov Identifier:
NCT00510965
First received: August 1, 2007
Last updated: June 14, 2012
Last verified: August 2011
  Purpose

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.


Condition Intervention Phase
Choroidal Neovascularization
Drug: Intravitreal injection ranibizumab
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome)

Resource links provided by NLM:


Further study details as provided by University Hospital, Bonn:

Primary Outcome Measures:
  • Best corrected visual acuity [ Time Frame: one year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Reading ability [ Time Frame: one year ] [ Designated as safety issue: No ]
  • Changes in retinal thickness assessed by optical coherence tomography [ Time Frame: one year ] [ Designated as safety issue: No ]
  • Changes in parafoveal leakage assessed by fluorescein angiography [ Time Frame: one year ] [ Designated as safety issue: No ]
  • Quality of life [ Time Frame: one year ] [ Designated as safety issue: No ]

Enrollment: 10
Study Start Date: August 2007
Study Completion Date: April 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: A Drug: Intravitreal injection ranibizumab
Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year
Other Name: Lucentis

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.

  Eligibility

Ages Eligible for Study:   18 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00510965

Locations
Germany
University of Bonn
Bonn, Germany, 53127
Sponsors and Collaborators
University Hospital, Bonn
Novartis
Investigators
Principal Investigator: Frank G. Holz, MD University of Bonn, Department of Ophthalmology
Principal Investigator: Hendrik PN Scholl, MD, MA University of Bonn
  More Information

Additional Information:
No publications provided by University Hospital, Bonn

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: University of Bonn
ClinicalTrials.gov Identifier: NCT00510965     History of Changes
Other Study ID Numbers: CRFB002ADE03, EudraCT number 2006-006233-49
Study First Received: August 1, 2007
Last Updated: June 14, 2012
Health Authority: Germany: Paul-Ehrlich-Institut

Keywords provided by University Hospital, Bonn:
choroidal neovascularization
angioid streaks
pseudoxanthoma elasticum
Groenblad-Strandberg-Syndrome
therapy
ranibizumab
lucentis
Choroidal neovascularization in patients with PXE

Additional relevant MeSH terms:
Neovascularization, Pathologic
Pseudoxanthoma Elasticum
Choroidal Neovascularization
Metaplasia
Pathologic Processes
Hemostatic Disorders
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Hematologic Diseases
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Connective Tissue Diseases
Skin Diseases
Choroid Diseases
Uveal Diseases
Eye Diseases

ClinicalTrials.gov processed this record on July 20, 2014