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

Primary Purpose

Pseudoxanthoma Elasticum

Status

Completed

Phase

Phase 2

Locations

Germany

Study Type

Interventional

Intervention

Aflibercept

About this trial

This is an interventional treatment trial for Pseudoxanthoma Elasticum focused on measuring Choroidal neovascularization, Angioid streaks, Pseudoxanthoma Elasticum, vascular endothelial growth factor, aflibercept

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Department of Ophthalmology, Universtiy of Bonn

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Aflibercept

Arm Description

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change in light increment sensitivity of central visual field between end-of study visit and screening visit measured by microperimetry

Change in chorioretinal neovascularization and leakage measured by angiography between end-of study visit and screening visit

Change in sub- and intraretinal und subpigmentepithelial fluids assessed using optical coherence tomography (OCT)

Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging

Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)

Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Change in the health status of the total population measured by quality of life questionnaire Visual Function Questionnaire (VFQ)-25

Full Information

NCT ID

NCT02537054

First Posted

August 21, 2015

Last Updated

May 5, 2019

Sponsor

University Hospital, Bonn

1. Study Identification

Unique Protocol Identification Number

NCT02537054

Brief Title

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

Acronym

EyNeP

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2019

Overall Recruitment Status

Completed

Study Start Date

September 2015 (undefined)

Primary Completion Date

July 2, 2018 (Actual)

Study Completion Date

July 2, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

University Hospital, Bonn

4. Oversight

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Pseudoxanthoma Elasticum

Keywords

Choroidal neovascularization, Angioid streaks, Pseudoxanthoma Elasticum, vascular endothelial growth factor, aflibercept

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Aflibercept

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Aflibercept

Other Intervention Name(s)

Eylea

Intervention Description

Intravitreal injection

Primary Outcome Measure Information:

Title

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 Outcome Measure Information:

Title

Change in light increment sensitivity of central visual field between end-of study visit and screening visit measured by microperimetry

Time Frame

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

Title

Change in chorioretinal neovascularization and leakage measured by angiography between end-of study visit and screening visit

Time Frame

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

Title

Change in sub- and intraretinal und subpigmentepithelial fluids assessed using optical coherence tomography (OCT)

Time Frame

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

Title

Extent of fibrovascular proliferation on optical coherence tomography (OCT) imaging

Time Frame

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

Title

Changes fundus autofluorescence images using a confocal scanning laser ophthalmoscope (cSLO)

Time Frame

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

Title

Occurence of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Time Frame

12 months

Title

Change in the health status of the total population measured by quality of life questionnaire Visual Function Questionnaire (VFQ)-25

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

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 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Frank G. Holz, MD, DPhil

Organizational Affiliation

University Clinic Bonn

Official's Role

Principal Investigator

Facility Information:

Facility Name

Department of Ophthalmology, Universtiy of Bonn

City

Bonn

ZIP/Postal Code

53127

Country

Germany

12. IPD Sharing Statement

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

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