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

August 1, 2007
June 14, 2012
August 2007
April 2010   (final data collection date for primary outcome measure)
Best corrected visual acuity [ Time Frame: one year ] [ Designated as safety issue: No ]
Best corrected visual acuity [ Time Frame: one year ]
Complete list of historical versions of study NCT00510965 on ClinicalTrials.gov Archive Site
  • 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 ]
  • Reading ability [ Time Frame: one year ]
  • Changes in retinal thickness assessed by optival coherence tomography [ Time Frame: one year ]
  • Changes in parafoveal leakage assessed by fluorescein angiography [ Time Frame: one year ]
  • Quality of life [ Time Frame: one year ]
Not Provided
Not Provided
 
Ranibizumab to Treat Choroidal Neovascularization (CNV) in Patients With Pseudoxanthoma Elasticum (PXE)
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.

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.

Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Choroidal Neovascularization
Drug: Intravitreal injection ranibizumab
Monthly intravitreal injection of 0.5mg ranibizumab in one eye over one year
Other Name: Lucentis
Experimental: A
Intervention: Drug: Intravitreal injection ranibizumab
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. Epub 2011 Jun 25.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
April 2010
April 2010   (final data collection date for primary outcome measure)

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
Both
18 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
Germany
 
NCT00510965
CRFB002ADE03, EudraCT number 2006-006233-49
No
University of Bonn
University Hospital, Bonn
Novartis
Principal Investigator: Frank G. Holz, MD University of Bonn, Department of Ophthalmology
Principal Investigator: Hendrik PN Scholl, MD, MA University of Bonn
University Hospital, Bonn
August 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP