Intravitreal Aflibercept for Therapy of Patients With Pseudoxanthoma Elasticum (PXE)

Phase 2

Completed

• Conditions: Pseudoxanthoma Elasticum
• Interventions: Drug: Aflibercept

• Registration Number: NCT02537054
• Lead Sponsor: University Hospital, Bonn

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-08-21
• Study First Submitted QC: 2015-08-27
• Study Start: 2015-09
• Study First Posted: 2015-09-01
• Primary Completion: 2018-07-02
• Study Completion: 2018-07-02
• Status Verified: 2019-04
• Last Update Submitted: 2019-05-05
• Last Update Posted: 2019-05-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

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
• Distinct opacification of optical media
• Distinct subretinal fibrosis and /or atrophy that prevents a relevant treatment effect by Aflibercept at discretion of investigator
• Serious cardiovascular problems or stroke within 6 months before enrolment
• Simultaneous use of other Vascular Endothelial Growth Factor (VEGF)-inhibiting medication (systemic or ocular) within a month prior to enrolment

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Aflibercept	Aflibercept	2 mg/ dose (pro re nata, maximum 1 dose/ month), intravitreal use

Primary Outcome Measures

Name	Time	Method
Change in distance best corrected visual acuity between end-of study visit and screening visit	Screening visit (day -28 - 0) and end-of-study visit (day 360 +/- 7)

Secondary Outcome Measures

Name	Time	Method
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)

Trial Locations

Locations (1)

Department of Ophthalmology, Universtiy of Bonn; 🇩🇪 Bonn, Germany