Intravitreal Aflibercept (Eylea) for Therapy of Choroidal Neovascularization (CNV) and Fibrovascular Proliferation (FVP) in Patients With Pseudoxanthoma Elasticum (PXE)

University Hospital, Bonn1 site in 1 country15 target enrollmentSeptember 2015

ConditionsPseudoxanthoma Elasticum

InterventionsAflibercept

DrugsAflibercept

Overview

Phase: Phase 2
Intervention: Aflibercept
Conditions: Pseudoxanthoma Elasticum
Sponsor: University Hospital, Bonn
Enrollment: 15
Locations: 1
Primary Endpoint: Change in distance best corrected visual acuity between end-of study visit and screening visit
Status: Completed
Last Updated: 6 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The purpose of this study is to determine whether Aflibercept (Eylea) is effective in the treatment of choroidal neovascularization and fibrovascular proliferation in patients with pseudoxanthoma elasticum (PXE) in terms of preservation or improvement of visual acuity.

Registry

clinicaltrials.gov

Start Date

September 2015

End Date

July 2, 2018

Last Updated

6 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

University Hospital, Bonn

Responsible Party

Principal Investigator

Frank G. Holz

Prof. Dr. med. Frank G. Holz

University Hospital, Bonn

Eligibility Criteria

Inclusion Criteria

•Diagnosis of PXE by moleculargenetic diagnosis and/or skin biopsy
•Diagnosed CNV or FVP
•Age 18-65 years
•Voluntary participation in this study as proven by written informed consent
•Ability to follow study instructions and likely to attend and complete all required visits
•Best corrected visual acuity between 20/400 and 20/20 at treated eye
•Male and female patients with childbearing potential must use an approved contraceptive method (Pearl Index \< 1) before and during the trial
•Pre-menopausal female patients with childbearing potential: a negative pregnancy test must be obtained

Exclusion Criteria

•Subject is unable to understand the nature, scope, significance and consequences of this clinical trial
•Patients with known allergy or hypersensitivity to Eylea or preparations with similar chemical structure
•Treatment in another clinical trial with therapeutic intervention or use of any other investigational medicinal product (IMP) during the trial of within 30 days before enrolment
•Known or persistent abuse of medication, drugs or alcohol
•Women who are pregnant or breast feeding
•Lack of eligibility at discretion of the investigator
•Ocular operations within a month prior to enrolment
•Non-controlled glaucoma
•Active intraocular inflammation or inflammation of ocular adnexa
•Other diseases resulting in distinct visual constraint

Arms & Interventions

Aflibercept

2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use

Intervention: Aflibercept

Outcomes

Primary Outcomes

Change in distance best corrected visual acuity between end-of study visit and screening visit

Time Frame: Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Secondary Outcomes

Change in light increment sensitivity of central visual field between end-of study visit and screening visit measured by microperimetry(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))
Change in chorioretinal neovascularization and leakage measured by angiography between end-of study visit and screening visit(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))
Change in sub- and intraretinal und subpigmentepithelial fluids assessed using optical coherence tomography (OCT)(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))
Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))
Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))
Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)(12 months)
Change in the health status of the total population measured by quality of life questionnaire Visual Function Questionnaire (VFQ)-25(Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7))