Ranibizumab in choroidal neovascularization (CNV) due to Pseudoxanthoma elasticum (PXE, Groenblad-Strandberg Syndrome) - PXE-CNV 06

Phase 1

• Conditions: 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: 8.1Level: LLTClassification code 10037150Term: Pseudoxanthoma elasticum

• Registration Number: EUCTR2006-006231-49-DE
• Lead Sponsor: Department of Ophthalmology, Bonn University

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-03-06
• Last Update Posted: 5 April 2021

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 10

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

•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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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<br>•Vascular leakage as shown by fluorescein and indocyanin green angiography,<br>•Retinal thickness as shown by optical coherence tomography,<br>•Funduscopical/morphological aspects of the disease as documented by dilated fundus exam, auto-fluorescence and photography,<br>•Quality of life of the investigated sample.<br>;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).