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

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ClinicalTrials.gov Identifier: NCT00510965
Recruitment Status : Completed
First Posted : August 3, 2007
Last Update Posted : June 15, 2012
Information provided by:
University Hospital, Bonn

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.

Condition or disease Intervention/treatment Phase
Choroidal Neovascularization Drug: Intravitreal injection ranibizumab Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Ranibizumab in Choroidal Neovascularization (CNV) Due to Pseudoxanthoma Elasticum (PXE, Groenblad-Strandberg-Syndrome)
Study Start Date : August 2007
Actual Primary Completion Date : April 2010
Actual Study Completion Date : April 2010

Arm Intervention/treatment
Experimental: A Drug: Intravitreal injection ranibizumab
Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year
Other Name: Lucentis

Primary Outcome Measures :
  1. Best corrected visual acuity [ Time Frame: one year ]

Secondary Outcome Measures :
  1. Reading ability [ Time Frame: one year ]
  2. Changes in retinal thickness assessed by optical coherence tomography [ Time Frame: one year ]
  3. Changes in parafoveal leakage assessed by fluorescein angiography [ Time Frame: one year ]
  4. Quality of life [ Time Frame: one year ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00510965

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University of Bonn
Bonn, Germany, 53127
Sponsors and Collaborators
University Hospital, Bonn
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Principal Investigator: Frank G. Holz, MD University of Bonn, Department of Ophthalmology
Principal Investigator: Hendrik PN Scholl, MD, MA University of Bonn
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: University of Bonn
ClinicalTrials.gov Identifier: NCT00510965    
Other Study ID Numbers: CRFB002ADE03
EudraCT number 2006-006233-49
First Posted: August 3, 2007    Key Record Dates
Last Update Posted: June 15, 2012
Last Verified: August 2011
Keywords provided by University Hospital, Bonn:
choroidal neovascularization
angioid streaks
pseudoxanthoma elasticum
Choroidal neovascularization in patients with PXE
Additional relevant MeSH terms:
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Choroidal Neovascularization
Pseudoxanthoma Elasticum
Neovascularization, Pathologic
Pathologic Processes
Choroid Diseases
Uveal Diseases
Eye Diseases
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
Angiogenesis Inhibitors
Angiogenesis Modulating Agents
Growth Substances
Physiological Effects of Drugs
Growth Inhibitors
Antineoplastic Agents