Pseudoxanthoma elasticum (PXE) is an inherited systemic disease characterized by changes in the elastic tissue. Pseudoxanthoma elasticum mainly affects the skin, eyes, heart, and GI system. The cutaneous and ocular findings of PXE are referred to as Grönblad-Strandberg syndrome. Pseudoxanthoma elasticum may be autosomal dominant or autosomal recessive. In Pseudoxanthoma elasticum, choroidal nevoscualrization (CNV) is a frequently occuring complication in late stage disease MedDRA version: 8.1 L
Conditions
Interventions
Trade Name: Lucentis
Product Name: Lucentis
Product Code: RFB002
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ranibizumab
Current Sponsor code: CRFB002ADE03
Other descriptive name:
Sponsors
Department of Ophthalmology, Bonn University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • 18 – 65 yrs. of age. • PXE-diagnosis ascertained by genetic analysis. • Angiographically diagnosed CNV in the study eye. • Written informed consent. • Best corrected visual acuity: 20/200 – 20/32 in the study eye. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • Patients with other retinal diseases including diseases of the retinal vessels or diabetic retinopathy. • Ocular surgery mess than three months prior to the commencment of the trial. • History of a not well meneged glaucoma. • Active intraocular inflammation or inflammation of the ocular adnexa. • Subfoveal fibrosis in the study eye. • Inability, to comply with the study protocol. • Major surgical interventions less than 1 month prior to the commencment of the trial. • Known allergy to humanized antibodies. • History of clinically significant cardiovascular or cerebrovascular disease less than 6 months before commencment of the trial
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To investigate the efficacy of Ranibizumab (0.5 mg) in improving or maintaining visual acuity in choroidal neovascularization due to Pseudoxanthoma elasticum;Secondary Objective: To investigate the effect of ranibizumab on • Vascular leakage as shown by fluorescein and indocyanin green angiography, • Retinal thickness as shown by optical coherence tomography, • Funduscopical/morphological aspects of the disease as documented by dilated fundus exam, auto-fluorescence and photography, • Quality of life of the investigated sample. ;Primary end point(s): • Visual acuity (as measured by the ETDRS chart at 4 meters or 1 meter distance one month after the 12th injection of Lucentis compared to the baseline visual acuity). | — |
Countries
Germany
Outcome results
None listed